Yesterday and today we scanned - MIBG and MRI. Since I was off work as of yesterday, I went with Max to the MIBG on Thursday. He did well, except towards the end when he had a little meltdown on the full-body scan. However, that's nothing that a cheap plastic dinosaur the wonderful techs (ie, Melissa) keep around for just such a moment.
So, for the news everyone is waiting for.... the damn spot is still there on the T11 vertebrae. This is my read during scan, but it was clearly still there. I couldn't tell if the spot was larger or smaller than before. As well, no new spots either, which would be expected. We're meeting with the head of radiology to get a better idea of the interpretation of the data than the written report can provide.
Still, not the ultimate outcome we were hoping for, but at the worst we're stable with minimal, refractory disease.. But then, the outcome we'd most like to hear would be, "cancer gone and looks like its never coming back," but those are not words a rational NB parent ever expects to hear. But in this holiday season (since we just watched 'The Polar Express'), ya gotta believe!
Merry Christmas, Happy New Year and all the blessings of the winter holidays to everyone!
12/21/2007
12/17/2007
Fab photos
A friend of Neil Hutchison's is going to partipate in the RAAM (Race Across America) again this year. As part of their fundraising efforts, they wanted a picture of Sam and Max, in order to put some faces to their efforts.
Neil and Margot, Sam's parents, are friends with this amazing photographer. We met her on Sunday afternoon just east of La Jolla in a county park. You'd never know there was a 4-lane highway 100 yards away. It's quite a lovely place to be in the winter, with large, stately oak trees and lots of mud and leaves to get dirty in.
Deb Schwedhelm Photography [deb@debsphotographs.com] is her name, should you want to contact her. I'll be posting some additional pics taken last weekend by our family photographer Shannon Boyd, who is equally talented at capturing our family in a most favorable manner.
12/15/2007
Lab Rat?
The WSJ article on Sam Hutchison ran in today's (Saturday) Wall Street Journal. It's a Page One article so hopefully it will bring some good attention to our cause! You can click here to read the story for free.
12/04/2007
Persimmon season
Please read today's (December 4th) post from Lucas Tran's website. His mom Thy wrote it. It's sadly beautiful, and puts into perspective what important this time of year.
Lucas became an angel on October 16th.
Lucas became an angel on October 16th.
11/28/2007
Max featured during NBC tree lighting coverage
Max's story on NBC 7/39, which aired this past Wednesday, is now available to view online. Click here to go to the page. Please rate it (5 stars of course) to help make it more visible by driving it up in the "most viewed/rated/shared" lists.
11/23/2007
Guest speakers
Melissa and I were invited to speak at Rady Children's Hospital Auxilary "Holiday on Broadway" fundraiser two weeks ago. We thought it was a good idea for two reasons: One, the fundraiser was supporting pediatric cancer research at RCHSD. Secondly, we could tell Max's story and hopefully by doing so, create more awareness about neuroblastoma and our personal efforts to fund promising research that can be used by Max and his fellow NB buddies soon, if not now!
Our role was simple - to soften up the crowd with our (Max's) story prior to an auction, the proceeds of which would benefit pediatric cancer research. So, all in all a worthy cause and one in which we didn't mind participating.
As I mentioned to one of the women at our table, realizing it was perhaps a little gouche but altogether accurate thing to say, we were "the parents no one want to be," ie, the parents of a kid with cancer.
As most of you know, we are not afraid to play the cancer card and above all are willing to use Max as a hook to draw attention and funds as long as the people and the cause are something we personally trust and believe in. To that end, the night was a success and I believe they raised close to $50K. We also had the pleasure of sitting next to RCHSD president and CEO Kathleen Sellick and her husband Phil. A delightful couple, they were surprised when we told them Max has NB, because Phil had neuroblastoma as a young adult 20-some years ago, so its great to know that the head of our hospital has some insight to our plight with NB.
We were the only speakers for the evening. I spoke for about two minutes about how money follows the market and most research funding therefore goes the way of breast, lung and prostate cancers. Pediatric cancers make up such a small market... I let Melissa tell Max's story, and here's what she said:
Our role was simple - to soften up the crowd with our (Max's) story prior to an auction, the proceeds of which would benefit pediatric cancer research. So, all in all a worthy cause and one in which we didn't mind participating.
As I mentioned to one of the women at our table, realizing it was perhaps a little gouche but altogether accurate thing to say, we were "the parents no one want to be," ie, the parents of a kid with cancer.
As most of you know, we are not afraid to play the cancer card and above all are willing to use Max as a hook to draw attention and funds as long as the people and the cause are something we personally trust and believe in. To that end, the night was a success and I believe they raised close to $50K. We also had the pleasure of sitting next to RCHSD president and CEO Kathleen Sellick and her husband Phil. A delightful couple, they were surprised when we told them Max has NB, because Phil had neuroblastoma as a young adult 20-some years ago, so its great to know that the head of our hospital has some insight to our plight with NB.
We were the only speakers for the evening. I spoke for about two minutes about how money follows the market and most research funding therefore goes the way of breast, lung and prostate cancers. Pediatric cancers make up such a small market... I let Melissa tell Max's story, and here's what she said:
Max was 3 ½ years old when he was diagnosed with stage 4, high-risk neuroblastoma – a very aggressive cancer of the sympathetic nervous system that quickly spreads to other organs including the bones.
I remember him lying in the hospital bed shortly after we knew his diagnosis. His little body was frail, anemic, and he was in such pain that he was on a constant morphine drip. He couldn’t move and it hurt to be touched – which was awful for all of us because he;s a snuggler. He looked at me and said in a tiny voice, “Mommy, the doctors are never going to make me better.”
He had a shock of blonde hair, long eyelashes framing his blue eyes, and already had a great sense of humor. His hair and eyelashes soon fell out following his first chemotherapy treatment, but his good spirit remained.
He would tell the nurses, “leave me alone” when they came into his hospital room because he knew they were going to have to do something to him. It wasn’t long before he found that he could be in control - of the television and of how he was touched – and soon the nurses could do whatever they needed to do as long as they didn’t block his view and followed his particular instructions for certain procedures – which the nurses did with smiles on their faces.
Max underwent the standard treatment protocol for his diagnosis which included 6 high-dose chemo treatments, radiation, and a stem cell transplant – all in six months. His treatment went off without a hitch and he was NED – no evidence of disease - shortly after he turned 4.
One year later, right after his 5th birthday, a spot appeared on his follow-up scans. We soon found out that his cancer had come back. We also found out that even though 80% of kids who have had his diagnosis relapse – there was no treatment plan that was proven to make them well again. Just a number of ideas to choose from.
With the guidance of Max’s oncologist, Dr. Roberts, and another cancer parent, we agreed to sign Max up for a clinical trial based in Vermont. Max was only the second child on this trial, but it is a great trial, for a promising drug, and we believe it has done wonders in helping Max combat his cancer.
In the past year, Max has received 10 high-dose IV chemo treatments, 2 rounds of radiation on his spine, and has taken low-dose daily chemo for the past four months – this in addition to the trial drug that he was eligible to stop taking in March, but we decided it was truly a help and have kept him on it.
My husband and I have learned through this process the deficit of treatment possibilities for children with cancer. Lab research and clinical trials for pediatric cancers are grossly underfunded across America. We’ve learned that every 16 hours a child dies from neuroblastoma alone. We’ve learned that the best way to make a difference in Max’s life is to tell people about his battle.Mother Theresa said, “If I think of the many, I’ll never act. If I think of the one, I will.”
We hope that you will think of Max right now. Think of his beautiful blonde head and fun blue eyes. Think of Max teaching his two year old brother how to play Legos and dressing up like a super hero with his favorite blanket as a cape. Think of how much Max loves sharks and all of his friends in Mrs. Sturt’s 1st grade class. Think of Max riding his scooter and doing taekwondo, and running in and out of the waves at the beach with his big sister. Think of Max lying in a hospital bed getting poked through his port so he can get his IV chemo. Think of Max laying in a hospital bed one week after chemo too tired to move waiting for a blood transfusion. Think of Max being fed through a button in his tummy everyday because the chemo he’s received has ruined his taste. Think of all the fun Max misses at school when he has to be at the hospital for all his treatments and scans.
Think how nice it would be if Max didn’t have cancer.
11/18/2007
Doing well.
This afternoon as I was transferring all my data from this years' organizer to my new one, I stumbled upon a note I had written:
April 3, 2007Well, that was 8+ monthes ago. He's doing so well right now. It's very frightening when these kinds of thoughts pass through my head. It hurts all the way to very core of my body.
1:00pm
Almost cried just now. Max is in clinic watching a TV show where they're talking about how things change, like the seasons or how people change when they grow up. "How will you change?" they ask. Max looks at me and says, "My hair will grow back!" I'm suddenly struck by the thought of him not growing up and not having any hair ever again.
11/13/2007
Not Allowed at School
Last week Max had one of his physical therapy appointments during school hours. I picked him up right after his lunch-recess and he looked so sad. So, as I'm driving away from school I look in the rearview mirror and ask him what's the matter.
He looks back and says, "I've been holding my poo all day and I really have to go."
I respond, "Why didn't you go when you were at school, honey?"
Max says, "They don't allow poo at school."
(Needlesstosay, we talked about the obvious on the way to his appointment... where he took care of business and had a much better afternoon because of it.)
He looks back and says, "I've been holding my poo all day and I really have to go."
I respond, "Why didn't you go when you were at school, honey?"
Max says, "They don't allow poo at school."
(Needlesstosay, we talked about the obvious on the way to his appointment... where he took care of business and had a much better afternoon because of it.)
11/05/2007
Radiation Complete
Last Friday Max went in for his radiation appointment - first appointment of the day 6:45am. Luckily, Andy decided to take him so I didn't have to "do my hair" that early.
Max received 16 grays to the cancer spot on T11 in his spine. He was given anesthesia to allow the doctor and technicians full control over arranging his body in the best possible position to get the radiation exactly where it was needed (aka radiosurgery).
So now we wait. The radiation works over a few weeks and then Max will have a set of scans to see what's going on in there. Andy & I are hoping for the best... NED, of course.
In the meantime, Max is feeling great, enjoying school, and hating homework. His weight is up to 45.2 pounds! and he looks great. No more knobby, malnutritioned-looking knees, his face and body have filled in, AND I've had to let out the waists in all his pants! Hoo-yeah! Wednesday this week, he starts an after school Taekwondo class right at his school. We're hoping this class along with his weight gain and the physical therapy he gets will improve his strength, balance, agility, and self-confidence in taking part in sports/active play. He's been so weakened by the chemotherapy and cancer over the past three years that he's really missed out on a lot. It's no fun to be last in every race you run; not be able to catch the other kids when your IT; to get so tired that you have to sit down after only a short time of playing.
Max received 16 grays to the cancer spot on T11 in his spine. He was given anesthesia to allow the doctor and technicians full control over arranging his body in the best possible position to get the radiation exactly where it was needed (aka radiosurgery).
So now we wait. The radiation works over a few weeks and then Max will have a set of scans to see what's going on in there. Andy & I are hoping for the best... NED, of course.
In the meantime, Max is feeling great, enjoying school, and hating homework. His weight is up to 45.2 pounds! and he looks great. No more knobby, malnutritioned-looking knees, his face and body have filled in, AND I've had to let out the waists in all his pants! Hoo-yeah! Wednesday this week, he starts an after school Taekwondo class right at his school. We're hoping this class along with his weight gain and the physical therapy he gets will improve his strength, balance, agility, and self-confidence in taking part in sports/active play. He's been so weakened by the chemotherapy and cancer over the past three years that he's really missed out on a lot. It's no fun to be last in every race you run; not be able to catch the other kids when your IT; to get so tired that you have to sit down after only a short time of playing.
10/28/2007
Children in the back seat
A good article below, nothing you haven't heard before but reinforcing nonetheless.
Quick update: still waiting to get Max's radiation appointment, so we're somewhat treading water on our push to get to NED. It's frustrating- its been more than 10 days since our consultation appointment, and you know how pushy we can be! Granted, Max's situation isn't life-threatening, but we still want to get him zapped as soon as possible.
Once we know something definitive, we'll update. Until then, it's business as usual.
Sunday, Oct 28, 2007
As I See It: Research into child diseases takes a back seat on funding
http://www.kansascity.com/273/story/335066.html
By Cheryl Mozer
Why do people with rare diseases not seem to have any voice in America? If you have a rare disease in America, you cannot count on having a treatment for it because there is no profit to be made. I have no problem with companies making profits, but why don’t they “give back” a percentage of that money to help others whose diseases do not have a large enough market to warrant research for a cure?
This year, 12,500 children in the United States will be diagnosed with some type of childhood cancer. Our son survived neuroblastoma cancer, a disease affecting 1 in 10 million children. Half of the children with the late-stage disease that Jacob had do not survive.
None of the treatments Jacob received were approved in children and he was saved only by an experimental antibody therapy found in New York City. Although childhood cancer is the No. 1 cause of death by disease in children (more deaths than all other childhood diseases combined), funding remains consistently low.
A group of seven fathers recently bicycled across America from California to New York (through Kansas City) because there is no funding for the estimated $3 million to $5 million it would take to develop a promising proposed treatment.
These fathers were anxiously trying to get the funding for doctors to develop the treatment that could potentially save their children’s lives. On top of the normal stress of having a critically ill child, one should not have to bicycle 3,700 miles to raise money for a treatment that could be developed if only the money was there (see www. loneliestroad.org). Private foundations such as Alex’s Lemonade Stand Foundation are struggling to make up for the lack of funding.
There are other options that could pay for critical research. Please send e-mail to your state’s senators and representatives to urge passage of the bipartisan Conquer Childhood Cancer Act, which would provide funding that could save lives. Twenty clinical trials are in danger of closing because of a lack of funding, affecting 400 children.
None of the senators in Kansas or Missouri have yet co-sponsored the Conquer Childhood Cancer Act. Reps. Dennis Moore, Nancy Boyda and Jerry Moran of Kansas and Sam Graves and William Lacy Clay of Missouri are co-sponsors, and for this we thank them.
Although the large cancer organizations do great things for adults with cancer, only a tiny percentage of funding goes to research for treating childhood cancer. September was Childhood Cancer Awareness Month, which was largely ignored. If it was your child, neighbor, grandchild, niece or nephew, would this be acceptable?
© 2007 Kansas City Star and wire service sources. All Rights Reserved. http://www.kansascity.com/
Quick update: still waiting to get Max's radiation appointment, so we're somewhat treading water on our push to get to NED. It's frustrating- its been more than 10 days since our consultation appointment, and you know how pushy we can be! Granted, Max's situation isn't life-threatening, but we still want to get him zapped as soon as possible.
Once we know something definitive, we'll update. Until then, it's business as usual.
Sunday, Oct 28, 2007
As I See It: Research into child diseases takes a back seat on funding
http://www.kansascity.com/273/story/335066.html
By Cheryl Mozer
Why do people with rare diseases not seem to have any voice in America? If you have a rare disease in America, you cannot count on having a treatment for it because there is no profit to be made. I have no problem with companies making profits, but why don’t they “give back” a percentage of that money to help others whose diseases do not have a large enough market to warrant research for a cure?
This year, 12,500 children in the United States will be diagnosed with some type of childhood cancer. Our son survived neuroblastoma cancer, a disease affecting 1 in 10 million children. Half of the children with the late-stage disease that Jacob had do not survive.
None of the treatments Jacob received were approved in children and he was saved only by an experimental antibody therapy found in New York City. Although childhood cancer is the No. 1 cause of death by disease in children (more deaths than all other childhood diseases combined), funding remains consistently low.
A group of seven fathers recently bicycled across America from California to New York (through Kansas City) because there is no funding for the estimated $3 million to $5 million it would take to develop a promising proposed treatment.
These fathers were anxiously trying to get the funding for doctors to develop the treatment that could potentially save their children’s lives. On top of the normal stress of having a critically ill child, one should not have to bicycle 3,700 miles to raise money for a treatment that could be developed if only the money was there (see www. loneliestroad.org). Private foundations such as Alex’s Lemonade Stand Foundation are struggling to make up for the lack of funding.
There are other options that could pay for critical research. Please send e-mail to your state’s senators and representatives to urge passage of the bipartisan Conquer Childhood Cancer Act, which would provide funding that could save lives. Twenty clinical trials are in danger of closing because of a lack of funding, affecting 400 children.
None of the senators in Kansas or Missouri have yet co-sponsored the Conquer Childhood Cancer Act. Reps. Dennis Moore, Nancy Boyda and Jerry Moran of Kansas and Sam Graves and William Lacy Clay of Missouri are co-sponsors, and for this we thank them.
Although the large cancer organizations do great things for adults with cancer, only a tiny percentage of funding goes to research for treating childhood cancer. September was Childhood Cancer Awareness Month, which was largely ignored. If it was your child, neighbor, grandchild, niece or nephew, would this be acceptable?
© 2007 Kansas City Star and wire service sources. All Rights Reserved. http://www.kansascity.com/
10/25/2007
Fires update
We've been home since yesterday at noon. Kids are glad to be home. Lots of ash and debris from the winds Sunday, but otherwise - home sweet home!
Three years ago tomorrow, Max checked into Children's San Diego. Later the next day, he was diagnosed with stage IV neuroblastoma. Our family will never be the same, but we cherish and are blessed with every day of his precious life.
Three years ago tomorrow, Max checked into Children's San Diego. Later the next day, he was diagnosed with stage IV neuroblastoma. Our family will never be the same, but we cherish and are blessed with every day of his precious life.
10/22/2007
San Diego Fires
We're getting a lot calls and emails wondering if we're okay, and, Yes, we are. Carmel Valley (located on the east side of the I-5 from Del Mar) was asked to voluntarily evacuate early this morning, so we did. Packed up the kids and few items and went down to mom and dad's house in North Park. Max had a clinic appointment this morning, too.
The fires are still far east of our area. But the smoke and soot have been so bad since Sunday noon at our house that we would've left anyway. You couldn't even go outside and the inside of the house became smokey even with the house shut tight. School was closed today and will be again Tuesday due to air quality.
Should anything come up we will post. Thanks for caring!
The fires are still far east of our area. But the smoke and soot have been so bad since Sunday noon at our house that we would've left anyway. You couldn't even go outside and the inside of the house became smokey even with the house shut tight. School was closed today and will be again Tuesday due to air quality.
Should anything come up we will post. Thanks for caring!
10/18/2007
Wear Your Care Final!
Ok, folks, the numbers are in: Over $11,000 was raised and close to 1700 wristbands ordered! WOW!
For those of you who ordered a band and didn't receive it (due to our short-sightedness on how well this campaign would go!), the new bands should be arriving by Halloween. We're so sorry for the long wait! We'll have your bands to you as soon as we get them. Thank you for your patience!
As for ordering a band if you would like more or simply don't have yours yet, please make a donation of any amount directly to Magic Water then send me an email with your mailing address, band size preference (child, medium or large) and I'll send your bracelet to you. (I think Mrs. Sturt needs a break!)
Mrs. Sturt worked so hard on this project and the money is still rolling in. Thank you all so much for your support and for Wearing Your Care. I'd have to say that this fundraiser was a huge success, wouldn't you?
The proceeds from this fundraiser went to the Magic Water Project which moves lab research into clinical trials for our kids by providing the much needed funds to make it happen. We believe that there is a cure for NB - it just hasn't been tried yet.
For those of you who ordered a band and didn't receive it (due to our short-sightedness on how well this campaign would go!), the new bands should be arriving by Halloween. We're so sorry for the long wait! We'll have your bands to you as soon as we get them. Thank you for your patience!
As for ordering a band if you would like more or simply don't have yours yet, please make a donation of any amount directly to Magic Water then send me an email with your mailing address, band size preference (child, medium or large) and I'll send your bracelet to you. (I think Mrs. Sturt needs a break!)
Mrs. Sturt worked so hard on this project and the money is still rolling in. Thank you all so much for your support and for Wearing Your Care. I'd have to say that this fundraiser was a huge success, wouldn't you?
The proceeds from this fundraiser went to the Magic Water Project which moves lab research into clinical trials for our kids by providing the much needed funds to make it happen. We believe that there is a cure for NB - it just hasn't been tried yet.
10/16/2007
Lucas Tran
Another neuroblastoma (NB) child, one who by all counts shouldn't have died, passed away in the Bay Area this morning. Lucas Tran's parents did all that they could and should, and yet he still went from stable to progressive to hospice care in a little over 2 months.
Aaarrrgh! It makes me so mad! Lucas lives in the technology capitol of the world, and yet the majority of intellectual and financial support is directed at figuring out how to share homemade videos on the Internet, not at trying to find a cure for diseases that rob us of our future teachers, scientists and artists.
That's why there's no resting, no relaxing - once your child has NB, its like they've been kidnapped. And until the kidnapper is DEAD, you can't ever stop. We've got to kill NB.
Aaarrrgh! It makes me so mad! Lucas lives in the technology capitol of the world, and yet the majority of intellectual and financial support is directed at figuring out how to share homemade videos on the Internet, not at trying to find a cure for diseases that rob us of our future teachers, scientists and artists.
That's why there's no resting, no relaxing - once your child has NB, its like they've been kidnapped. And until the kidnapper is DEAD, you can't ever stop. We've got to kill NB.
10/14/2007
Reality Check
NED - what does this really mean to us?
#1 - it doesn't mean we can relax with Max's treatment
#2 - it doesn't mean his cancer is "cured"
#3 - it doesn't mean his cancer will not come back
Too many times, in reading other families blogs, I read the cancer has suddenly, horribly, taken over their childs body. Out of nowhere... it just comes back full steam.
Don't get me wrong - I'm happy Max is headed in the NED direction (he has a radiation set-up appointment on Tuesday to zap that bothersome spot on his spine) - but the nature of this disease is unforgiving, unrelenting. And so must we be in return.
#1 - it doesn't mean we can relax with Max's treatment
#2 - it doesn't mean his cancer is "cured"
#3 - it doesn't mean his cancer will not come back
Too many times, in reading other families blogs, I read the cancer has suddenly, horribly, taken over their childs body. Out of nowhere... it just comes back full steam.
Don't get me wrong - I'm happy Max is headed in the NED direction (he has a radiation set-up appointment on Tuesday to zap that bothersome spot on his spine) - but the nature of this disease is unforgiving, unrelenting. And so must we be in return.
10/13/2007
Article from Carmel Valley News
Students, community rally behind Solana Highlands firstgrader and encourage everyone to‘LIVE LIFE TO THE MAX’
By Janice Coy
By Janice Coy
Students at Solana Highlands Elementary School who are learning about caring as part of the Six Pillars of Character program have had the opportunity to participate in a hands on manner by showing their support for a fellow student with cancer.First grader Max Mikulak is battling a recurrence of neuroblastoma (NB). The six year old was first diagnosed with the cancer, which attacks the bones and their marrow, three years ago. His initial treatment killed all evidence of the disease, but last fall, a bone scan showed new spots of it on his spine.
Max is currently taking an oral form of chemotherapy and is able to regularly attend his first grade class where he is taught by Lisa Sturt.
Max’s mother, Melissa Mikulak, said the boy’s teachers have been very supportive of him and the family. This fall, Sturt asked the family if she could spearhead a fundraiser for the Magic Water Project, a non-profit organization that funds clinical research trials for patients with NB.
As part of the fundraiser, the school sold orange reminder bracelets embossed with “LIVE LIFE TO THE MAX” for a suggested donation of $5 each. Sturt said $10,707 was raised, and 1,379 bracelets were sold. Orders for the bracelets were closed out last Thursday, but Sturt said donations could still be made to the Magic Water Project at http://www.magicwater.org/.
“With September being Childhood Cancer Awareness Month,” Sturt said, “we thought this would be a tremendous opportunity to show Max and the Mikulaks how much we care.
Sturt said classes at other schools are participating in the fundraiser as well, and Solana Highland families are “spreading the care beyond our community” by sending bracelets around the country and abroad.
Sturt said classes at other schools are participating in the fundraiser as well, and Solana Highland families are “spreading the care beyond our community” by sending bracelets around the country and abroad.
“The outpouring of generosity and kindness has been overwhelming and there’s not a child or family that deserves it more,” she said. “The Mikulaks are always smiling and positive. They find the good in each and every situation.
“They have learned that each moment is precious.”
Andy and Melissa Mikulak have lived in Carmel Valley for five years with Max, their 8-year-old daughter Hannah, who is in third grade, and their 2-year-old son Nicolas.
Mikulak describes the caring as incredible and heartwarming. Last year, she said, Max’s kindergarten teacher, Ms. Wietz, also spearheaded a fundraiser for the Magic Water Project and raised $10,000.
Mikulak describes the caring as incredible and heartwarming. Last year, she said, Max’s kindergarten teacher, Ms. Wietz, also spearheaded a fundraiser for the Magic Water Project and raised $10,000.
“It’s a really, really great feeling to have so much support,” she said. She stays positive, she says, because “it is simply the only way to get through a crisis, especially with children involved.”
Mikulak, said finding out about the cancer’s recurrence was “even more shocking” than the initial diagnosis.
“What you learn is that NED (no evidence of disease) doesn’t mean your child is cured,” she said.
Mikulak said it was Max’s pediatrician who first suspected that he had NB which was later confirmed by a oncologist at Rady’s Children’s’ Hospital. His symptoms included a low-grade fever that lasted for about a month, constipation and pain in his legs that caused him to limp.
“When we would go to pick up Hannah in kindergarten, he would ask me to carry him, “ she said. “Eventually, he would lie in bed and couldn’t even get up to go pee.“It was horrifying.”
“When we would go to pick up Hannah in kindergarten, he would ask me to carry him, “ she said. “Eventually, he would lie in bed and couldn’t even get up to go pee.“It was horrifying.”
Mikulak said it is not known what causes NB, but research has shown that, while it is not genetic, it could be triggered while the fetus is growing nerve cells.
After his initial diagnosis, Max underwent chemotherapy and a stem cell transplant.There was no evidence of the disease afterwards.
“There is no treatment protocol for recurrent NB,” Mikulak said. “There is no cure for it, so there’s no real protocol.”
When the spots on Max’s spine multiplied, the Mikulaks decided, with the oncologist, to follow a treatment of 11 rounds of high-dose chemotherapy. Max lost his hair and had to miss 40-50 days of kindergarten last year because the chemotherapy suppressed his immune system. At the beginning of the summer, a bone scan showed that the cancer had shrunk.
“Last year he was completely bald, and sometimes he would have to wear a mask to school,” Mikulak said.
Despite his absences, his kindergarten teacher kept him a part of the classroom by visiting his kid-friendly blog with the other students. Max’s Web site is http://www.mikulak.org/. Mikulak said the teacher also came to the clinic where Max was to do homework with him.
“With the oral chemotherapy, his blood doesn’t get blasted, so he is able to participate in activities,” she said. “There’s actually fuzz on his head which is exciting.
“He goes to class every day. His mental health is just as important in helping him get through this so we allow him to do whatever activities he can.”
10/09/2007
Cloud 6 on the Horizon?
Gee, we have all these posts as of late, but nothing about Max and his battle. Let me get you up to date.
Max is feeling great. Home chemo is way easier on a kid than IV blasts. But you have to do what works. He’s been having a lot of nausea/vomiting since the Etoposide chemo was added to his daily meds. Of course, we didn’t figure this out for about three weeks. Kids don’t seem to have the capacity to recognize when they feel nauseous all the time. Dr. Roberts prescribed an additional anti-nausea medicine last week and Max seems to be feeling better because of it. He ate a hotdog Friday night and 4 pieces of sourdough at the Fish Market on Sunday (he hasn’t taken any food by mouth in 2-3 weeks).
Thank goodness for his mickey-button or the kid would have wasted away by now. We continue to give him 1500ml of his formula along with 400-600ml of veggie-brew daily. Yesterday his weight was up again to 19.0kgs. 41 pounds. Can I hear a “WOO-HOO!” Alright!
Today is Tuesday and it’s 7:30am - I’m at the hospital right now with Max. He’s in for an MRI. Max loves MRI day. Kooky-kid. I think he enjoys riding on the gurney down the halls and waking up to popsicles, graham crackers and a Disney movie afterward. He had an MIBG bone scan last week. Both are to track the spot on his spine that has remained unchanged over the past 5-6 months. It appeared on last weeks’ MIBG, which I can see while he’s having the scan, that the spot is still unchanged. This is good news – no growth.
Can anything be done to further diminish the spot? It’s bugging us a little that it’s just sitting there seemingly taunting us month after month.
Luckily, Max was on the nifurtimox trial so we have the added benefit of another oncologist – and NB specialist – to review his scans. Dr. Sholler suggested zapping the spot with another round of radiation could very well take him to NED status again (NED = No Evidence of Disease). I spoke with Max’s main oncologist (the extremely helpful, knowledgeable, and patient-with-neurotic-parents-who-email-him-daily Dr. Roberts) who spoke with Max’s radiation-oncologist about the possibility, and it looks like it is definitely doable. We’re now just waiting for an appointment time.
So, yeah, NED. What awesome news. We’re saying prayers, crossing our fingers and toes, wishing on stars, shaking chicken feet over Max while he sleeps… looking for good vibes!
Max is feeling great. Home chemo is way easier on a kid than IV blasts. But you have to do what works. He’s been having a lot of nausea/vomiting since the Etoposide chemo was added to his daily meds. Of course, we didn’t figure this out for about three weeks. Kids don’t seem to have the capacity to recognize when they feel nauseous all the time. Dr. Roberts prescribed an additional anti-nausea medicine last week and Max seems to be feeling better because of it. He ate a hotdog Friday night and 4 pieces of sourdough at the Fish Market on Sunday (he hasn’t taken any food by mouth in 2-3 weeks).
Thank goodness for his mickey-button or the kid would have wasted away by now. We continue to give him 1500ml of his formula along with 400-600ml of veggie-brew daily. Yesterday his weight was up again to 19.0kgs. 41 pounds. Can I hear a “WOO-HOO!” Alright!
Today is Tuesday and it’s 7:30am - I’m at the hospital right now with Max. He’s in for an MRI. Max loves MRI day. Kooky-kid. I think he enjoys riding on the gurney down the halls and waking up to popsicles, graham crackers and a Disney movie afterward. He had an MIBG bone scan last week. Both are to track the spot on his spine that has remained unchanged over the past 5-6 months. It appeared on last weeks’ MIBG, which I can see while he’s having the scan, that the spot is still unchanged. This is good news – no growth.
Can anything be done to further diminish the spot? It’s bugging us a little that it’s just sitting there seemingly taunting us month after month.
Luckily, Max was on the nifurtimox trial so we have the added benefit of another oncologist – and NB specialist – to review his scans. Dr. Sholler suggested zapping the spot with another round of radiation could very well take him to NED status again (NED = No Evidence of Disease). I spoke with Max’s main oncologist (the extremely helpful, knowledgeable, and patient-with-neurotic-parents-who-email-him-daily Dr. Roberts) who spoke with Max’s radiation-oncologist about the possibility, and it looks like it is definitely doable. We’re now just waiting for an appointment time.
So, yeah, NED. What awesome news. We’re saying prayers, crossing our fingers and toes, wishing on stars, shaking chicken feet over Max while he sleeps… looking for good vibes!
10/05/2007
Wear Your Care Update 3
This morning closed Mrs. Sturt's Wear Your Care Fundraiser for Max and the Magic Water Project (this doesn't mean you still can't get a band!). I betcha wanna know how many bracelets were sold and how much money was raised... Before I tell you, let me share some other gems with you:
Max and Hannah unfurled the total: 1379 bands were ordered and $10,707 was raised! But there's more! Mrs. Sturt is still receiving orders for the orange bands - as of this morning those orders pushed the total to almost $10,900.
- There was an all school assembly to celebrate the caring attitude demonstrated by all the students at Solana Highlands - not just for this incredible fundraiser but for the caring they showed each other all month long.
- Most of the kids and parents were wearing orange, as well as their Live Life to the Max! bands.
- A mom-friend of mine brought a bag full of orange hairspray to decorate the kids with.
- People from 20 other states in these United States of America ordered bracelets through this fundraiser.
- Bracelets were also sent to about 10 countries far and wide including Iraq, Sweden and Asia.
And the numbers are:
Max and Hannah unfurled the total: 1379 bands were ordered and $10,707 was raised! But there's more! Mrs. Sturt is still receiving orders for the orange bands - as of this morning those orders pushed the total to almost $10,900.Mrs. Sturt, still flush from the amazing outpouring of love from our community, surrounding schools and world in general, revealed her next fundraiser for Magic Water (as modeled by Hannah):
Magic Coins for Magic Water: a little change can do magic!
Magic Coins is a way for kids to keep on giving - even if just a little bit. Every classroom in our school will have a jar with the Magic Coins logo on it and they drop coins into it whenever they can. I think any school could do this fundraiser - don't you?
If you are interested in ordering a Live Life to the Max! band, please send a donation of $5 per band (or more!) along with a double-stampled SASE to: Mrs. Lisa Sturt c/o Solana Highlands Elementary School, 3520 Long Run Drive, San Diego, CA 92130
Thank you everyone. Today was a teary day, but for good reasons!
Ms. Weitz * Mrs. Sturt * Ms. Mulvaney -- sporting their Wear Your Care!
10/04/2007
Max Deux
Here's a post from another mom on the east coast who has her own little Max battling NB (Max Palmer). It feels so good to see so many NB parents liking what the Magic Water Project is doing for our kids.
September 24, 2007 at 01:28 PM EDT
Hi everyone- This isn't really a medical update though Max seems to be continuing on the bumpy road with some great days and not so great days. Today was a good day so we'll take it. Keep those good vibes coming!We just wanted to let you know about a cool kid named Max Mikulak who lives in sunny CA and is also fighting neuroblastoma. His 1st grade teacher is selling orange “Live Life to the MAX!” wristbands to raise money for neuroblastoma research. (I think they look like the livestrong bracelets). He has such a great teacher and school – every kid in his school bought one to support him.
The proceeds go to a really amazing organization called the Magic Water Project (link) which helps move ideas for treatment from the labs out into clinical trials that aren't getting funded for children to participate in. Its run by a smart group of parents and doctors like a venture capital- they research the ideas and selectively fund the ones that have the most potential.
Anyway, since WE have an inspiring little Max in our lives too- I talked to Max Mikulak's mom and am jumping on their idea. If you are interested in a bracelet to support the Maxs and a great cause, send $5 per wristband along with a double-stampled SASE to: Mrs. Lisa Sturt c/o Solana Highlands Elementary School 3520 Long Run Drive San Diego, CA 92130
10/02/2007
Vermont
It's Andy. I thought I'd post a quick update on my weekend trip to Vermont. The purpose was to get together parents - Neil (Sam's dad), myself, Pat (Will's dad), Terrill (Paul's dad), Ranan (cancer dad from NYC), and of course Meryl (Andrew's mom) - together with some researchers and clinical oncs - the incredible Dr. Giselle Sholler and Nick Heinz, PhD from UVM, Laurent Brard, MD/PhD from Brown, Jim Jameson, PhD from Kent State, Dr. Dan Durden from Aflac/Atlanta Children's Hospital and Dr. Sholler's lab staff including the wonderful Genevieve Johnson who always gets us our nifurtimox pills the next day after we ask for them :)
The purpose was to put together a MagicWater Project roadmap for 2008. Here are the outcomes (I'm sorry its so brief but I'm tired, I'm now in NYC and I have lots more work to do tonight and meetings bright and early):
1. Agree to meet every 3 months (may be obvious but not so when you consider work, treatment, etc)!
2. Begin to test 4 different "oxidative stress" compounds in mice that would 'turbocharge' chemotherapy (like Nifurtimox does) as well as be used in a maintenance treatment once the child got to NED, in order to see if there are additional agents that we could add to or substitute in case our current treatments stop working or a child relapses again. Those agents are:
- Vit C/K3
- Rapamyacin
- Artemisinin
- Omega-3's (yeah, fish oil)
- We may also try tetrathiomobylate
3. We also want to investigate the timing or chronotherapy component of administering agents. Some studies show that up to a 40% improvement can be made just by optimizing the time of day when an agent is administered.
4. Lastly, we agreed on a Phase II Nifurtimox trial, as well as a Phase II Nifurtimox trial for NED kids, so that we can see if giving Nifurtimox will help kids stay NED.
Obviously all of this is going to cost money. The Phase II Nifurtimox trial alone will probably cost at least $400,000. But, its worth it, and I'd dare say the readers of this blog would agree.
The best, I've saved for last. Please check out these two websites. Both of these amazing kids Neil and I met this weekend.
The first, Eden Brunskow, lives in Tustin Hills, CA but for most of the last two years has been at MSKCC. She is the second to last child to go on the nifurtimox trial. Neil and I, after taking the red-eye to Vermont Saturday night, dropped by the RMH in Burlington to meet Eden and her dad Paul. Neil knows Paul already but neither of us have met Eden, nor where we prepared to fall in love so quickly with her. She is absolutely darling and one of the cutest kids we've ever seen. She was so talkative and smart, entertaining us with stories of gophers and halloween (her fav holiday).
http://www.caringbridge.org/visit/edenbrunskow
The second child we visited right before leaving the campus of UVM on Monday. Dustin Cobb is the last child to get on the nifurtimox study (15th). He's had a tough time as of late and was in-patient with an infection when we stopped by to see him. Dustin's a cool dude that loves to play shoot-em-up video games. He also seems to enjoy getting tickled by the nurses and Dr. Sholler. Neil asked if the nurses and Dr. Sholler would tickle him but they declined.
http://www.caringbridge.org/ga/dustincobb/
Please visit both of these children's sites and let them know you're pulling for them both. I'm sure they'd love to know you've read their story.
More on Vermont later later (like maybe this weekend).
The purpose was to put together a MagicWater Project roadmap for 2008. Here are the outcomes (I'm sorry its so brief but I'm tired, I'm now in NYC and I have lots more work to do tonight and meetings bright and early):
1. Agree to meet every 3 months (may be obvious but not so when you consider work, treatment, etc)!
2. Begin to test 4 different "oxidative stress" compounds in mice that would 'turbocharge' chemotherapy (like Nifurtimox does) as well as be used in a maintenance treatment once the child got to NED, in order to see if there are additional agents that we could add to or substitute in case our current treatments stop working or a child relapses again. Those agents are:
- Vit C/K3
- Rapamyacin
- Artemisinin
- Omega-3's (yeah, fish oil)
- We may also try tetrathiomobylate
3. We also want to investigate the timing or chronotherapy component of administering agents. Some studies show that up to a 40% improvement can be made just by optimizing the time of day when an agent is administered.
4. Lastly, we agreed on a Phase II Nifurtimox trial, as well as a Phase II Nifurtimox trial for NED kids, so that we can see if giving Nifurtimox will help kids stay NED.
Obviously all of this is going to cost money. The Phase II Nifurtimox trial alone will probably cost at least $400,000. But, its worth it, and I'd dare say the readers of this blog would agree.
The best, I've saved for last. Please check out these two websites. Both of these amazing kids Neil and I met this weekend.
The first, Eden Brunskow, lives in Tustin Hills, CA but for most of the last two years has been at MSKCC. She is the second to last child to go on the nifurtimox trial. Neil and I, after taking the red-eye to Vermont Saturday night, dropped by the RMH in Burlington to meet Eden and her dad Paul. Neil knows Paul already but neither of us have met Eden, nor where we prepared to fall in love so quickly with her. She is absolutely darling and one of the cutest kids we've ever seen. She was so talkative and smart, entertaining us with stories of gophers and halloween (her fav holiday).
http://www.caringbridge.org/visit/edenbrunskow
The second child we visited right before leaving the campus of UVM on Monday. Dustin Cobb is the last child to get on the nifurtimox study (15th). He's had a tough time as of late and was in-patient with an infection when we stopped by to see him. Dustin's a cool dude that loves to play shoot-em-up video games. He also seems to enjoy getting tickled by the nurses and Dr. Sholler. Neil asked if the nurses and Dr. Sholler would tickle him but they declined.
http://www.caringbridge.org/ga/dustincobb/
Please visit both of these children's sites and let them know you're pulling for them both. I'm sure they'd love to know you've read their story.
More on Vermont later later (like maybe this weekend).
9/29/2007
Cancer IS Fun & Skateboarders Rock
We're back to the Cancer is Fun thing again. Let me explain: In The James Emails by Syd Birrell, he wrote that when his youngest son was asked what it's like to have a brother with cancer he answered, "Having cancer is fun!" Here's just another reason why:
Friday, Max and Sam (of TeamSam) were invited to a private skateboard demo with Tony Hawk, world famous pro-skateboarder, and a few other up-and-comers in the pro-skateboarder and snowboarding world. The invite was made possible by Sean Mortimer, whom, if I'm not mistaken, is the editor of Skateboarder magazine and is the husband of a teacher at Max's school, who, of course, must've bullied her husband into getting this set-up ;-). Forgive me if I'm completely or partially wrong on my facts about who set this up - but know that we are super stoked and you ROCK!
Friday, Max and Sam (of TeamSam) were invited to a private skateboard demo with Tony Hawk, world famous pro-skateboarder, and a few other up-and-comers in the pro-skateboarder and snowboarding world. The invite was made possible by Sean Mortimer, whom, if I'm not mistaken, is the editor of Skateboarder magazine and is the husband of a teacher at Max's school, who, of course, must've bullied her husband into getting this set-up ;-). Forgive me if I'm completely or partially wrong on my facts about who set this up - but know that we are super stoked and you ROCK!
Max hangin' with Shawn White (you know, the snowboarder from the Olympics?) and the man, Tony Hawk, ultra-world-famous pro-skateboarder and founder of the Tony Hawk Foundation. Everyday occurance for Max to bask in the glow of famous people.
Max and Sam waiting for their turn in the pipe with Bucky Lasek getting some big air (upper left corner of shot). The top of the ramp is 13.5 feet by the way and he's still going up... Bucky may be doing an Ollie - but I'm really not too sure of my skateboarding terminology, man.
Max & Sam had an awesome time. Max has never seen anything like this (even on TV) and had no idea what to expect. The most he's seen for skateboarding are some kids trying to flip their decks and stick the landing - which he was quite impressed with at the time. We sat down at one end of the pipe (I guess that's what it's called) and then the guys started riding. I thought Max's eyes were really going to pop out of his head. He was astonished. And how couldn't you be? What was really interesting to me was that the guys skating this day ranged in age from 19 to 42. And I tell ya what: you certainly couldn't tell one from the other when they're riding. They were all flippin' awesome!
A huge thanks to Tony Hawk for extending this invitation to Max & Sam. This was a private affair that a family had bid on at a Tony Hawk Foundation auction. Max and Sam have crashed their first party! Way to go, boys. Nice choice.
Cancer has it's fun moments and we will take advantage of all of them. When offers like this come up we're so thankful. To introduce Max to more of the world is essential to us because we don't know what the future has in store for him.
Look for more pics, video clips and a list of the pro-skateboarders that were performing on Max's Kid Site soon!
9/27/2007
Wear Your Care Update 2
"I am freaking out!"
Just received this email from the wonderful Mrs. Sturt tonight. She has 1000 orange wristbands. She's received 800 orders and is expecting way more. Her marketing of the Live Life to the Max campaign has surpassed even her goals (which is hard to believe, really, as she's hitting-up anyone who looks at her sideways for donations at Starbucks every morning. Why did I only order 1000 bands? What was I thinking?!)
I think it's funny that she's freaking out. We'll order more! So, if you don't go to our school and ordered a wristband, it may be a little delayed in getting to you - but it will get there!
BTW - she's also raised almost $6500 for Magic Water. Hoo-Yeah!
Thanks everyone.
Just received this email from the wonderful Mrs. Sturt tonight. She has 1000 orange wristbands. She's received 800 orders and is expecting way more. Her marketing of the Live Life to the Max campaign has surpassed even her goals (which is hard to believe, really, as she's hitting-up anyone who looks at her sideways for donations at Starbucks every morning. Why did I only order 1000 bands? What was I thinking?!)
I think it's funny that she's freaking out. We'll order more! So, if you don't go to our school and ordered a wristband, it may be a little delayed in getting to you - but it will get there!
BTW - she's also raised almost $6500 for Magic Water. Hoo-Yeah!
Thanks everyone.
9/24/2007
$17,422
What's with the numbers as titles to the blog entries today.
Well, I'm reading a book called "Made to Stick" which is, surprisingly, about how to make ideas "stick" in people's minds. One of the examples that is talked about in the book, is that people tend to give more to charity if they are not presented with numbers or 'rational' reasons for giving.
In other words, tell a story - don't cite statistics.
It seems us humans switch over, unconsciously, into analytical mode when presented with numbers and as a result, feel less emotionally affected by the cause for which our wallets are being sought.
However, this number - $17,422 - is a special number that will make your heart smile. You can read about it via the wonderful Pat of Will Lacey fame.
Well, I'm reading a book called "Made to Stick" which is, surprisingly, about how to make ideas "stick" in people's minds. One of the examples that is talked about in the book, is that people tend to give more to charity if they are not presented with numbers or 'rational' reasons for giving.
In other words, tell a story - don't cite statistics.
It seems us humans switch over, unconsciously, into analytical mode when presented with numbers and as a result, feel less emotionally affected by the cause for which our wallets are being sought.
However, this number - $17,422 - is a special number that will make your heart smile. You can read about it via the wonderful Pat of Will Lacey fame.
40+!
Melissa just emailed me from clinic with a great number: 18.9. What is 18.9 you ask? That's Max's weight in kilograms, so for the first time in his life, the boy is over 40 lbs! While that may not seem like a big deal, take a look at his weight over the past 6 months - I've pasted below since March '07. At the lowest he was under 35 lbs which is pretty darn skinny for a kid his age and height. While we're not resting on our laurels, it does feel good to be making measurable progress in the weight department.
15.8 - 3/19/07
17.1 - 4/16/07
17.3 - 5/11/07
16.9 - 5/29/07
17.4 - 6/11/07
17.2 - 7/9/07
16.9 - 7/30/07
17.4 - 8/6/07
16.7 - 8/17/07
16.8 - 8/30/07
17.6 - 9/6/07
18 - 9/13/07
18.1 - 9/17/07
18.9 - 9/24/07
15.8 - 3/19/07
17.1 - 4/16/07
17.3 - 5/11/07
16.9 - 5/29/07
17.4 - 6/11/07
17.2 - 7/9/07
16.9 - 7/30/07
17.4 - 8/6/07
16.7 - 8/17/07
16.8 - 8/30/07
17.6 - 9/6/07
18 - 9/13/07
18.1 - 9/17/07
18.9 - 9/24/07
9/21/2007
Wear Your Care - Update 1
I know we'll be writing several updates on this subject over the next two weeks, so I thought I'd better title this appropriately.
The Wear Your Care flyer went home in the school Wednesday envelopes (two days ago). I just received an update from Mrs. Sturt. She said 329 bracelets have been ordered and $3602 have been donated thus far! Wow!
She said she also received a call from one of our old neighbors in Chula Vista, The Luna Family, who wanted a few bracelets. (Thank you, Carmen! We miss all you guys down there.) A boy from another class brought Mrs. Sturt a bag of change that he earned and wanted to donate. And one teacher's class is doing a group recycling effort to make a class donation.
Can you feel the LOVE?
The Wear Your Care flyer went home in the school Wednesday envelopes (two days ago). I just received an update from Mrs. Sturt. She said 329 bracelets have been ordered and $3602 have been donated thus far! Wow!
She said she also received a call from one of our old neighbors in Chula Vista, The Luna Family, who wanted a few bracelets. (Thank you, Carmen! We miss all you guys down there.) A boy from another class brought Mrs. Sturt a bag of change that he earned and wanted to donate. And one teacher's class is doing a group recycling effort to make a class donation.
Can you feel the LOVE?
9/20/2007
Wear Your Care
At Max's school where he attends first grade - Solana Highlands - Max's teacher Lisa Sturt has put together a fundraiser that ties in with the school's 6 Pillars of Caring program and September's Childhood Cancer Awareness Month.
Orange reminder bracelets embossed with LIVE LIFE TO THE MAX! are being made available for a donation of $5 - the proceeds of which are being donated to Magic Water. The goal is that every single student at Solana Highlands will wear their care, and that they would share this Pillar of Caring with everyone they know.
Obviously this is a tremendously humbling experience. Our school community has opened up in ways that are unimaginable. Lisa Sturt, Max's teacher, is just INCREDIBLE. Along with his kindergarden teacher Debbie Weitz (who recommended Lisa for Max's 1st grade teacher), we feel extraordinarily lucky to have two such caring, energetic and empathetic teachers for Max.
Orange reminder bracelets embossed with LIVE LIFE TO THE MAX! are being made available for a donation of $5 - the proceeds of which are being donated to Magic Water. The goal is that every single student at Solana Highlands will wear their care, and that they would share this Pillar of Caring with everyone they know.
Obviously this is a tremendously humbling experience. Our school community has opened up in ways that are unimaginable. Lisa Sturt, Max's teacher, is just INCREDIBLE. Along with his kindergarden teacher Debbie Weitz (who recommended Lisa for Max's 1st grade teacher), we feel extraordinarily lucky to have two such caring, energetic and empathetic teachers for Max.
9/19/2007
Dr Sholler on Vermont Public Radio
The good doc behind the nifurtimox trial was on Vermont Public Radio today it seems. Here's a link if you'd like to listen by downloading an MP3 of the interview: http://www.publicbroadcasting.net/vpr/news.newsmain?action=article&ARTICLE_ID=1150519
Two important articles to read
Yesterday, Tuesday, was a good day to read the WSJ if you are interested in the state of healthcare in the US. I've risked the wrath of Rupert and posted them both below in their entirety. The first article below by Geeta Anand is best summed up as "if you are going to get a life-threatening illness, make sure it's a mainstream life-threatening illness, as the newest, most effective medicines may not be paid for by your insurance - evidence of efficacy to the contrary, as its too expensive and there's no 'competitors' with which the insurance co's can negotiate lower prices." This illustrates my arguement that insurance co's are not in the business of making us healthy (as their PR says), but in the business of evaluating and pricing risk. duh.
The second article is best summed up as "medical research exists in silos and most, not all medical researchers are driven by the desire to publish their own idea, not to collaborate and advance a cure and save lives if at the expense of their research and the resulting professional meaning the system assigns to such achievement." In other words, a common business practice - partnering - does not exist really in the medical research world. Shame.
September 18, 2007
BURDEN OF PROOF
As Costs Rise,
New Medicines
Face Pushback
Insurers Limit Coverage
To FDA-Approved Uses;
$300,000 Drug Denied
By GEETA ANAND
May Chin-Louis, 44 years old, has a ballooning brain tumor. Her doctors wanted to attack it with a colon-cancer drug that has shown promise in treating brain tumors.
But for four months, Ms. Chin-Louis's insurer, WellPoint Inc., refused to pay for the drug, called Avastin. It costs about $8,000 a dose, every other week. A WellPoint spokesman says it denied coverage initially because there isn't sufficient medical evidence proving Avastin is effective against brain tumors.
NARROWING SPECIALTIES
Some patients are being denied potentially effective therapies."She's a young patient with a family who has run out of options -- she's just looking for a chance," says her oncologist, Susan Chang. Patients with Ms. Chin-Louis's condition typically live for only about four months.
Doctors, particularly oncologists, rely on medicines approved for other diseases to try to save patients for whom all other treatments have failed. But as new medicines come to market at ever-higher prices, insurers are pushing back, limiting coverage of these drugs to only the disease for which they are specifically approved by the Food and Drug Administration -- or for which there is extensive evidence of efficacy in clinical trials.
"A lot of patients are being denied potentially effective therapies," says James Vredenburgh, an oncologist at Duke University Medical Center. "What's happening is totally arbitrary and it's 100% correlated to when the prices went up. Ten years ago, we never got questioned on our medical decision to prescribe the medicine with the best chance of helping our patients."
Insurers say they must limit use of the most expensive drugs to control health-care costs, which are surging at a 7% to 8% annual rate and continue to outpace inflation. It makes sense, they say, to require proof that a drug works in a patient's particular disease before doling out tens or hundreds of thousands of dollars.
"We're trying to bring new drugs to consumers, but trying to do it with employers getting the best value of every health-care dollar spent in the system," says Mohit Ghose, spokesman for America's Health Insurance Plans, an industry trade group.
High-priced medicines used to treat relatively small groups of patients -- categorized as "specialty pharmaceuticals" -- comprise the fastest-growing part of health spending, insurance officials say. By pushing generics, insurers have clamped down on spending on other types of medicines, which rose 6% in 2006, according to Express Scripts, Inc., one of the largest pharmacy-benefit managers.
By contrast, spending on specialty drugs soared 21% last year. They accounted for nearly a quarter of total drug spending in the U.S., according to Health Strategies Group, a New Jersey consulting firm.
Insurers have little leverage in negotiating the prices of many specialty drugs because they often extend lives and lack competition. Drug companies have the freedom to price these medicines almost as high as they like. The companies say the prices are needed to fund research of new medicines and to compensate shareholders.
Genentech Inc. charges $4,400 a month, or $47,000 for the average treatment course of Avastin for colon-cancer patients. Higher doses are used in treating brain tumors, raising the cost.
Once a unique, life-saving drug is approved by the FDA for a disease, insurers almost always are required to pay for it, regardless of price. But insurers have leeway when a medicine isn't specifically approved for a particular patient's disease. Coverage varies among companies.
"As an insurer, you go where the money is to try to cut costs," says Abbie Leibowitz, former chief medical officer of Aetna, Inc., who runs Health Advocate, which advises employers and employees on health insurance. Spending on specialty drugs is "where there is the greatest opportunity to affect cost."
Wall Street is seeing the effects of the pushback in lower-than-expected revenue from some of the hottest new cancer drugs. Genentech's Avastin is FDA-approved for certain kinds of colorectal and lung cancers. But the drug should also be a big-seller in breast cancer based on a large study reported two years ago showing efficacy in that disease, says Steven Harr, a Morgan Stanley analyst. His research shows less than 10% of breast-cancer patients are using the treatment, for which Genentech charges $7,700 per month for the average patient.
With prices so high, insurers "won't pay until it's FDA-approved," Dr. Harr says. "For the first time ever, there's elasticity in demand."
Karen Kacures, who owns a lawn-care company in St. Francis, Minn., found that out the hard way. Ms. Kacures was diagnosed in January with Pompe disease, a rare genetic disorder. It is caused by a deficiency in an enzyme that leads to progressive muscle-weakening and an early death.
Along with the devastating diagnosis, her doctors brought some hope: Genzyme Corp., a Cambridge, Mass., biotech company, recently brought to market the first treatment for the disease. The drug, called Myozyme, replaces the enzyme deficiency that causes muscle degeneration.
Dosed by weight, the drug is priced at an average of $300,000 a year, for a patient's entire life.
In March, Ms. Kacures's insurer, PreferredOne Community Health Plan, a Golden Valley, Minn., nonprofit with 400,000 members, refused to pay for the drug.
"We consider that for adults, the evidence is not there to support its use," says John Frederick, chief medical officer of PreferredOne. He noted that Genzyme conducted its clinical trial to win approval of the drug only in infants. In approving the drug, the FDA required Genzyme to conduct another trial, which is under way, to prove safety and efficacy in adults.
Dr. Frederick acknowledges that the FDA didn't restrict approval of the drug to infants, but he says it was "a little fuzzy."
Drugs that are high priced and treat smaller populations have "just kind of taken over" the pharmacy budget, Dr. Frederick says. In the most recent fiscal year, his company's spending on specialty drugs rose 23% over the previous year; spending on other pharmaceuticals was almost flat, he says. Generics are helping "stem the tide" of spending, he says, but specialty medicines are "a concern to us from a cost perspective."
Calling Ms. Kacures's case "wrenching," Dr. Frederick says he wishes there were "a big systemic cure. I dream of a big super-insurance plan to cover these tragic cases."
Ms. Kacures, 47, who has appealed the denial, says she doesn't have time to wait until Genzyme finishes its study in adults. "I want to get treated while I can still walk and breathe on my own during the day," she says. She uses a cane to walk and a breathing machine at night.
Some adults do receive the drug. Mark Lingenfelter, 45, of Eau Claire, Wis., started taking Myozyme in November, after Medicaid, the government's health-insurance program for the disabled and poor, agreed on appeal to cover the cost of the drug.
For the first time in three years, Mr. Lingenfelter says, he's been able to stand up out of his wheelchair. His breathing has improved enough to be able to talk on the phone for a few minutes, sometimes to offer encouragement to Ms. Kacures. "It's made a complete difference in my life," he says.
Gideon Sofer, a freshman at the University of California, Berkeley, was turned down last year when he tried to get approval from his mother's New Jersey insurance plan to pay for a new medicine. The drug, Revlimid, was approved by the FDA to treat a rare blood disorder and not yet tested in Mr. Sofer's condition, Crohn's disease, an inflammatory disorder that causes severe digestive problems.
Mr. Sofer had been using thalidomide, the only medicine that had helped control the mouth and intestinal sores caused by his disease. But thalidomide caused such drowsiness that he says he couldn't take morning classes, and he experienced nerve damage causing him to lose sensation in his feet.
Revlimid, made by Celgene Corp., is billed as an improved thalidomide, minus the drowsiness and nerve damage.
At first, Horizon Blue Cross Blue Shield of New Jersey covered it for him. "I felt so much less fatigue, my anemia improved, it was just great," Mr. Sofer says. His doctor in New York, Maria Abreu, director of the inflammatory bowel disease center at Mount Sinai School of Medicine in New York City, says Mr. Sofer's weekly blood reports showed gains in his iron levels.
But in August 2006, Mr. Sofer received a letter saying "it was determined that you do not meet the Horizon Blue Cross Blue Shield of New Jersey established medical necessity criteria for this drug." It noted Revlimid was approved for patients with a blood disorder and not Mr. Sofer's disease.
"Although benefits are denied, you may elect to receive the medication at your own expense," the letter said.
Neither Mr. Sofer nor his mother, a social worker, could afford the drug, which retails at about $8,800 for a month's supply. Mr. Sofer called Celgene and asked if the company would donate the drug. He was connected to a Celgene program, called Patient Support Solutions, which provides free Revlimid to patients who meet certain guidelines.
In a letter, Mr. Sofer was told he didn't qualify for free drugs; he says an employee told him that Crohn's disease patients weren't eligible.
Last year, not long after his prescription ran out, Mr. Sofer was hospitalized, severely dehydrated. He withdrew for the semester, feeling too sick to continue. "This has depleted me physically and spiritually," he said at the time.
Celgene's Patient Support Solutions says that this year, the free-drug program was expanded to include patients regardless of diagnosis, as long as they meet certain financial criteria, which the company declines to publicly detail.
In April, Mr. Sofer began receiving free Revlimid. His iron levels rebounded, though he continues to struggle with infections related to his disease. "For people like me, for whom nothing has worked, access to new treatments is absolutely critical," says the 23-year-old Mr. Sofer. "It's the only thing that keeps me hopeful, that keeps me living."
Ms. Chin-Louis was diagnosed with a brain tumor in late 2001. She had been working as an administrator at an accounting firm and raising two boys in Danville, Calif., when her speech periodically became jumbled. A surgeon removed the tumor, which initially grew slowly.
Last year, the tumor returned. She underwent radiation and took a chemotherapy drug approved by the FDA to treat brain cancer.
Early this year, a magnetic resonance imaging, or MRI, showed a bigger spot on her brain, suggesting the drug she was using no longer was working.
At the same time, researchers at Duke University Medical Center were reporting a promising study of Avastin in combination with chemotherapy for brain tumors. In a small trial of 32 patients, 72% on treatment were alive at six months, 38% of them with no progression of their tumors -- about double what would be expected for patients with Ms. Chin-Louis's kind of recurring brain tumor.
"It was early data, but it was very exciting," says Dr. Chang, director of the division of neuro-oncology at the University of California, San Francisco, Medical Center. In April, Dr. Chang asked Blue Cross of California to cover Avastin for Ms. Chin-Louis.
Blue Cross, owned by WellPoint, an Indianapolis company with more than 34 million members, denied coverage on May 21, saying in a letter: "Studies are ongoing to determine the role of this agent in the treatment of brain tumors."
Ms. Chin-Louis and her husband, a supervisor in sales at United Parcel Service Inc., say they couldn't afford the treatment. Her sister, Linda Tong, says she and her brother offered to cover the cost, but Ms. Chin-Louis initially resisted.
"I really felt that I had been paying for insurance for years for a reason, and when I needed medical care, my insurance company should pay," Ms. Chin-Louis says. "I didn't want to put that burden on my family."
In June, the doctor says she put Ms. Chin-Louis on a chemotherapy drug and appealed the denial for Avastin -- telling insurance officials she wanted to use the drugs in combination. On July 20, a brain scan showed the chemotherapy wasn't working: Ms. Chin-Louis's tumor had grown by 30%. She immediately agreed to allow her siblings to pay for Avastin, and she underwent her first treatment early last month.
In early August, asked by The Wall Street Journal about the Avastin denial, James Kappel, a spokesman for WellPoint, said: "While this patient's condition is very unfortunate, there is no medical evidence that supports the use of the drug" in brain tumors.
But Mr. Kappel called back the next day, saying WellPoint had approved Avastin for Ms. Chin-Louis. He said the company denied coverage at first because Dr. Chang sought to use the drug alone. Because Dr. Chang subsequently requested to treat Ms. Chin-Louis in combination with chemotherapy, as in the Duke study, WellPoint approved it, Mr. Kappel said.
Dr. Chang says the UCSF center has participated in a trial comparing Avastin on its own for brain tumors and in use with chemotherapy. Avastin alone appeared effective in some patients in the study, which has yet to be published, she says.
Dr. Chang says Ms. Chin-Louis has responded well so far to the Avastin treatments. "She's awake and feeling good and able to engage with her family," the doctor says. Ms. Chin-Louis is due for a brain scan later this week, which she hopes will show the tumor's growth in check.
"I know none of these treatments works forever," Ms. Chin-Louis says. "But I definitely want a little more time."
She'd like to celebrate her 45th birthday on Sept. 30, she said. She's eager to see her sister Linda's baby girl, due to arrive by Cesarean section on Nov. 2. And a small part of her is even daring to hope she will be in the audience when her eldest son Andrew graduates from high school this June.
Write to Geeta Anand at geeta.anand@wsj.com1
September 18, 2007
Will Sharing Ideas
Advance Cancer Research?
Hedge-Fund Managers Offer
$1 Million Prize to Combat
Scientists' Culture of Secrecy
By AMY DOCKSER MARCUS
After Hope Goldstein was diagnosed with advanced ovarian cancer in 2001, her family wanted to help. Her husband and two sons started researching the disease and quickly realized that even with surgery and chemotherapy, the prognosis wasn't good.
So they went in search of the one thing they believed still might help Mrs. Goldstein: new ideas.
They started calling cancer researchers, doctors at leading academic centers, specialists in ovarian cancer. In their conversations, they ran into an unexpected obstacle. Many people did have new ideas. But frequently, they weren't willing to share them.
Friends who worked in medical research tried to explain: Companies often will not pursue an idea for commercialization unless it is patented, requiring secrecy in the early stages. In addition, the grant process is competitive, and no one wants to get scooped. Perhaps most notably, professional advancement depends on publishing ideas in scientific journals.
In 2004, Mrs. Goldstein died. But her son Robert Goldstein, 41, continued to think about the issue. It seemed to him that what was holding back cancer research wasn't only a lack of money but a culture that discouraged the sharing of promising leads.
"If you have a great idea but someone else publishes it first, you get no credit, professionally or financially," says Gary Curhan of Harvard Medical School, one of the researchers who spoke with Robert Goldstein about the issue of sharing. "Ideas," says Dr. Curhan, "are our currency."
A managing partner at the hedge fund Gotham Capital in New York, Mr. Goldstein recognized similarities with his own profession. Money managers also were reluctant to share investment ideas. A few years earlier, Mr. Goldstein's business partner and friend, Joel Greenblatt, the 49-year-old founder of Gotham Capital, had created an online, selective group called the Value Investors Club, to spur idea sharing. Members shared investing strategies and commented on each other's research. A cash prize was awarded for the best idea of the week.
The two men thought that perhaps a similar model would work in cancer research. So this year they agreed to put up $1 million of their own money every year to fund the Gotham Prize for Cancer Research. Modeled on the Value Investors Club, the annual prize will go to the person who posts the best new cancer-research idea, judged by a board of respected scientists, at the prize's Web site by the end of December.
The winner of the Gotham Prize doesn't have to present a shred of evidence that the premise will work. To attract ideas from people outside the field of cancer research, there is no requirement that the winner be capable of seeing the idea through. And the prize money is earmarked for personal use, to be spent on anything the winner wants, even a fancy car or a bigger house.
This concept has been met with some skepticism, even by the scientific advisory board that will judge the ideas. Board member Meir Stampfer of Harvard Medical School said that when he was invited to join he asked, "Is this really a good use of their money? Maybe there is something better they can fund."
FUNDING A CURE
But the unusual nature of the prize illustrates the lengths to which patients and patient advocates are increasingly willing to go to boost research into their disease, especially rare illnesses for which there is little funding. Patients have hired their own experts to conduct research in particular diseases, and even helped to fund their own clinical trials. Efforts are also focused on overcoming the reluctance to share ideas.
Avichai Kremer, a 32-year-old who has ALS, the neurodegenerative illness also known as Lou Gehrig's disease, is co-founder of the Prize4Life, which is awarding a $1 million prize for ALS biomarker discovery. The National Institutes of Health announced this year a program called the New Innovator Award offering grants of up to $1.5 million over five years for innovative research projects that don't have extensive preliminary data.
In addition, the National Cancer Institute is helping companies find a way to collaborate in drug testing without worrying about intellectual-property issues. The medical journal the Oncologist is encouraging the publication of failed medical trials in order to bring the ideas behind these trials -- which otherwise would never see the light of day -- to a broader audience. And Mr. Goldstein's father, Alfred, with help from the family's other son, Mark, also developed a venture that aims to improve idea-sharing: Through "Project Hope," named for his late wife, Alfred Goldstein guarantees certain funding for specific projects and requires the researchers share results with each other on a regular basis.
The Gotham Prize is a particularly ambitious project that is attracting attention. The foundation of the Ira Sohn Investment Research Conference, which runs the conference as a benefit to raise money for cancer, will fund an additional $250,000 prize for the best pediatric-cancer idea submitted to the Gotham site. Ephraim Gildor, founder of Axiom Investment Advisors, is also providing financial support for the prize.
In setting up the prize, Mr. Greenblatt and Mr. Goldstein knew that they couldn't evaluate the new ideas themselves, so they set up a board of pre-eminent scientists and researchers. They include people to whom they have a personal connection, like Dr. Curhan, who is also Mr. Greenblatt's brother-in-law. Others such as Michael Kastan, director of the cancer center at St. Jude Children's Research Hospital in Memphis, Tenn., said they had received letters "out of the blue" inviting them to participate.
Dr. Curhan, the head of the advisory board, says all ideas submitted are vetted by at least two members of the board before being posted. As a result, although there are over 1,030 registered users, only 54 ideas have been posted. About 75% of the submitted ideas have been rejected so far, Dr. Curhan says. The tension is always how to eliminate ideas "not because they are new or different, but because there is no scientific basis for them," says Mr. Greenblatt. "It can't become a free-for-all. We want quality ideas or the site won't work."
To submit an idea, applicants need supply only a 500-to-1,000-word explanation of their concept and the reasoning behind it. They don't have to supply any data or evidence that the concept has been tested.
Some of the ideas accepted so far are very unusual. Someone who described himself as a businessman and former guitar player in a rock band proposed searching for the resonant frequency of cancer cells and then applying subsonic sound waves to cancer patients in order to trigger the body's immune response. Dr. Curhan says that idea sparked debate on the board. But it was posted in the end because some members argued that it couldn't totally be ruled out and was novel and outside-the-box.
There was also a proposal for a sort of Pap smear for early detection of breast cancer by a scientist who runs a major university lab but says it would be very difficult to get funding for such an early-stage project. Another comes from an AIDS researcher, who discovered an idea about brain cancer and thought as an outsider in cancer research he was unlikely to get funding from traditional sources. A survivor of testicular cancer posted an idea about an algorithm he created that he believes could lead to better early detection of recurrence.
Dr. Kastan of St. Jude says that even after reading all the ideas, "I am still waiting to see if it works." He says that most important scientific discoveries are made by "someone who was doing an experiment that had an unusual result that took them in a different direction. It is data that shows us the way, not a wild idea that comes out of nowhere." But he says he agreed to get involved because he recognized there weren't many places for scientists to share an early idea.
Bert Vogelstein of Johns Hopkins University School of Medicine, another advisory-board member, says he isn't sure he totally embraces the "anyone can have a great idea" approach that seemed to drive the initial concept. But he says that getting grants from government agencies like the NIH sometimes "forces people to do somewhat mundane experiments that follow up on other experiments rather than thinking creatively. In cancer in particular, we need more things that have never been done before because I think that's the only way we're going to beat the disease."
Similar thinking was behind the efforts at the Oncologist, a journal for cancer specialists, to publish online the results of so-called negative trials -- those that don't end up supporting the initial hypothesis. These trials don't result in the drug getting approved and aren't typically published. "A font of information" is being lost that could be helpful if looked at a new way, says Martin J. Murphy Jr., founding executive editor of the Oncologist.
At the NCI, scientists found that investigators at different companies wanted to share ideas and test different drugs in new combinations. But often they "couldn't do so because their own legal departments wouldn't let them because of intellectual property issues," says James Zwiebel of the NCI's Cancer Therapy Evaluation program. The NCI stepped in and crafted an agreement that each company signs that lets the NCI test a drug in combination with other companies' agents; the companies share in any information and other ideas that arise from the experiments.
Not everyone supports the premise that there's some great idea out there today waiting to be heard. "I don't sense a lot of holding back of exciting information," says Robert Ozols, senior vice president of medical science at Fox Chase Cancer Center and one of the oncologists the Goldsteins felt was willing to share ideas. "If we knew something worked, we'd be doing it."
But the prize applicants believe otherwise. Mark Kantrowitz, the publisher of FinAid.org, a Web site about student financial aid, and director of advanced projects for FastWeb.com, came across a posting about the prize while scouring the Web. In May 2003, at age 36, he was diagnosed with advanced testicular cancer. After completing treatment, he was put on a surveillance schedule of regular CT scans to pick up any possible recurrence. "The schedule seemed totally arbitrary," says Mr. Kantrowitz, who has a background in mathematics. He wrote an algorithm for a computer program that creates a surveillance schedule based on the greatest likelihood of catching a relapse as early as possible.
"I know it needs a clinical trial, but I come from outside the field and wouldn't know how to start it," he says. Even if he doesn't win the $1 million, he says he hopes an oncologist sees the idea online and likes it enough to test it. All of the ideas posted for the prize can be seen at www.gothamprize.org3, by users who register as guests.
David Rimm, a tenured professor of pathology at Yale University School of Medicine, runs a large, grant-funded research lab, where he says he can get money for his mainstream research. But things that are very early or "more out on the limb, are very difficult to get funded," he says. So he posted an idea to develop a kind of pap smear for early breast-cancer detection, using spectral spatial imaging technology on breast-tissue samples, to detect cellular changes beyond what the pathologist's eye can see.
Dr. Rimm says a colleague told him about the prize, but that he was surprised when he learned that the $1 million was for personal use. At the site, the founders had explained that one of the goals of the prize is to fill in current research gaps. "If they're just giving us money to buy a boat, it won't lead to filling the gaps," he says.
Mr. Goldstein and Mr. Greenblatt recognize that there is no guarantee that the winner will spend the money on shepherding the concept into a clinic. What they hope is that the prize site can serve as a kind of marketplace of ideas, where additional investors might see something interesting and decide to invest in some of the other projects as well. The prize's true value, Mr. Greenblatt said, will come from jump-starting that process. In the end, says Dr. Curhan, "if you get even three or four really good ideas the first year, that would be a huge success."
Write to Amy Dockser Marcus at amy.marcus@wsj.com
The second article is best summed up as "medical research exists in silos and most, not all medical researchers are driven by the desire to publish their own idea, not to collaborate and advance a cure and save lives if at the expense of their research and the resulting professional meaning the system assigns to such achievement." In other words, a common business practice - partnering - does not exist really in the medical research world. Shame.
September 18, 2007
BURDEN OF PROOF
As Costs Rise,
New Medicines
Face Pushback
Insurers Limit Coverage
To FDA-Approved Uses;
$300,000 Drug Denied
By GEETA ANAND
May Chin-Louis, 44 years old, has a ballooning brain tumor. Her doctors wanted to attack it with a colon-cancer drug that has shown promise in treating brain tumors.
But for four months, Ms. Chin-Louis's insurer, WellPoint Inc., refused to pay for the drug, called Avastin. It costs about $8,000 a dose, every other week. A WellPoint spokesman says it denied coverage initially because there isn't sufficient medical evidence proving Avastin is effective against brain tumors.
NARROWING SPECIALTIES
Some patients are being denied potentially effective therapies."She's a young patient with a family who has run out of options -- she's just looking for a chance," says her oncologist, Susan Chang. Patients with Ms. Chin-Louis's condition typically live for only about four months.
Doctors, particularly oncologists, rely on medicines approved for other diseases to try to save patients for whom all other treatments have failed. But as new medicines come to market at ever-higher prices, insurers are pushing back, limiting coverage of these drugs to only the disease for which they are specifically approved by the Food and Drug Administration -- or for which there is extensive evidence of efficacy in clinical trials.
"A lot of patients are being denied potentially effective therapies," says James Vredenburgh, an oncologist at Duke University Medical Center. "What's happening is totally arbitrary and it's 100% correlated to when the prices went up. Ten years ago, we never got questioned on our medical decision to prescribe the medicine with the best chance of helping our patients."
Insurers say they must limit use of the most expensive drugs to control health-care costs, which are surging at a 7% to 8% annual rate and continue to outpace inflation. It makes sense, they say, to require proof that a drug works in a patient's particular disease before doling out tens or hundreds of thousands of dollars.
"We're trying to bring new drugs to consumers, but trying to do it with employers getting the best value of every health-care dollar spent in the system," says Mohit Ghose, spokesman for America's Health Insurance Plans, an industry trade group.
High-priced medicines used to treat relatively small groups of patients -- categorized as "specialty pharmaceuticals" -- comprise the fastest-growing part of health spending, insurance officials say. By pushing generics, insurers have clamped down on spending on other types of medicines, which rose 6% in 2006, according to Express Scripts, Inc., one of the largest pharmacy-benefit managers.
By contrast, spending on specialty drugs soared 21% last year. They accounted for nearly a quarter of total drug spending in the U.S., according to Health Strategies Group, a New Jersey consulting firm.
Insurers have little leverage in negotiating the prices of many specialty drugs because they often extend lives and lack competition. Drug companies have the freedom to price these medicines almost as high as they like. The companies say the prices are needed to fund research of new medicines and to compensate shareholders.
Genentech Inc. charges $4,400 a month, or $47,000 for the average treatment course of Avastin for colon-cancer patients. Higher doses are used in treating brain tumors, raising the cost.
Once a unique, life-saving drug is approved by the FDA for a disease, insurers almost always are required to pay for it, regardless of price. But insurers have leeway when a medicine isn't specifically approved for a particular patient's disease. Coverage varies among companies.
"As an insurer, you go where the money is to try to cut costs," says Abbie Leibowitz, former chief medical officer of Aetna, Inc., who runs Health Advocate, which advises employers and employees on health insurance. Spending on specialty drugs is "where there is the greatest opportunity to affect cost."
Wall Street is seeing the effects of the pushback in lower-than-expected revenue from some of the hottest new cancer drugs. Genentech's Avastin is FDA-approved for certain kinds of colorectal and lung cancers. But the drug should also be a big-seller in breast cancer based on a large study reported two years ago showing efficacy in that disease, says Steven Harr, a Morgan Stanley analyst. His research shows less than 10% of breast-cancer patients are using the treatment, for which Genentech charges $7,700 per month for the average patient.
With prices so high, insurers "won't pay until it's FDA-approved," Dr. Harr says. "For the first time ever, there's elasticity in demand."
Karen Kacures, who owns a lawn-care company in St. Francis, Minn., found that out the hard way. Ms. Kacures was diagnosed in January with Pompe disease, a rare genetic disorder. It is caused by a deficiency in an enzyme that leads to progressive muscle-weakening and an early death.
Along with the devastating diagnosis, her doctors brought some hope: Genzyme Corp., a Cambridge, Mass., biotech company, recently brought to market the first treatment for the disease. The drug, called Myozyme, replaces the enzyme deficiency that causes muscle degeneration.
Dosed by weight, the drug is priced at an average of $300,000 a year, for a patient's entire life.
In March, Ms. Kacures's insurer, PreferredOne Community Health Plan, a Golden Valley, Minn., nonprofit with 400,000 members, refused to pay for the drug.
"We consider that for adults, the evidence is not there to support its use," says John Frederick, chief medical officer of PreferredOne. He noted that Genzyme conducted its clinical trial to win approval of the drug only in infants. In approving the drug, the FDA required Genzyme to conduct another trial, which is under way, to prove safety and efficacy in adults.
Dr. Frederick acknowledges that the FDA didn't restrict approval of the drug to infants, but he says it was "a little fuzzy."
Drugs that are high priced and treat smaller populations have "just kind of taken over" the pharmacy budget, Dr. Frederick says. In the most recent fiscal year, his company's spending on specialty drugs rose 23% over the previous year; spending on other pharmaceuticals was almost flat, he says. Generics are helping "stem the tide" of spending, he says, but specialty medicines are "a concern to us from a cost perspective."
Calling Ms. Kacures's case "wrenching," Dr. Frederick says he wishes there were "a big systemic cure. I dream of a big super-insurance plan to cover these tragic cases."
Ms. Kacures, 47, who has appealed the denial, says she doesn't have time to wait until Genzyme finishes its study in adults. "I want to get treated while I can still walk and breathe on my own during the day," she says. She uses a cane to walk and a breathing machine at night.
Some adults do receive the drug. Mark Lingenfelter, 45, of Eau Claire, Wis., started taking Myozyme in November, after Medicaid, the government's health-insurance program for the disabled and poor, agreed on appeal to cover the cost of the drug.
For the first time in three years, Mr. Lingenfelter says, he's been able to stand up out of his wheelchair. His breathing has improved enough to be able to talk on the phone for a few minutes, sometimes to offer encouragement to Ms. Kacures. "It's made a complete difference in my life," he says.
Gideon Sofer, a freshman at the University of California, Berkeley, was turned down last year when he tried to get approval from his mother's New Jersey insurance plan to pay for a new medicine. The drug, Revlimid, was approved by the FDA to treat a rare blood disorder and not yet tested in Mr. Sofer's condition, Crohn's disease, an inflammatory disorder that causes severe digestive problems.
Mr. Sofer had been using thalidomide, the only medicine that had helped control the mouth and intestinal sores caused by his disease. But thalidomide caused such drowsiness that he says he couldn't take morning classes, and he experienced nerve damage causing him to lose sensation in his feet.
Revlimid, made by Celgene Corp., is billed as an improved thalidomide, minus the drowsiness and nerve damage.
At first, Horizon Blue Cross Blue Shield of New Jersey covered it for him. "I felt so much less fatigue, my anemia improved, it was just great," Mr. Sofer says. His doctor in New York, Maria Abreu, director of the inflammatory bowel disease center at Mount Sinai School of Medicine in New York City, says Mr. Sofer's weekly blood reports showed gains in his iron levels.
But in August 2006, Mr. Sofer received a letter saying "it was determined that you do not meet the Horizon Blue Cross Blue Shield of New Jersey established medical necessity criteria for this drug." It noted Revlimid was approved for patients with a blood disorder and not Mr. Sofer's disease.
"Although benefits are denied, you may elect to receive the medication at your own expense," the letter said.
Neither Mr. Sofer nor his mother, a social worker, could afford the drug, which retails at about $8,800 for a month's supply. Mr. Sofer called Celgene and asked if the company would donate the drug. He was connected to a Celgene program, called Patient Support Solutions, which provides free Revlimid to patients who meet certain guidelines.
In a letter, Mr. Sofer was told he didn't qualify for free drugs; he says an employee told him that Crohn's disease patients weren't eligible.
Last year, not long after his prescription ran out, Mr. Sofer was hospitalized, severely dehydrated. He withdrew for the semester, feeling too sick to continue. "This has depleted me physically and spiritually," he said at the time.
Celgene's Patient Support Solutions says that this year, the free-drug program was expanded to include patients regardless of diagnosis, as long as they meet certain financial criteria, which the company declines to publicly detail.
In April, Mr. Sofer began receiving free Revlimid. His iron levels rebounded, though he continues to struggle with infections related to his disease. "For people like me, for whom nothing has worked, access to new treatments is absolutely critical," says the 23-year-old Mr. Sofer. "It's the only thing that keeps me hopeful, that keeps me living."
Ms. Chin-Louis was diagnosed with a brain tumor in late 2001. She had been working as an administrator at an accounting firm and raising two boys in Danville, Calif., when her speech periodically became jumbled. A surgeon removed the tumor, which initially grew slowly.
Last year, the tumor returned. She underwent radiation and took a chemotherapy drug approved by the FDA to treat brain cancer.
Early this year, a magnetic resonance imaging, or MRI, showed a bigger spot on her brain, suggesting the drug she was using no longer was working.
At the same time, researchers at Duke University Medical Center were reporting a promising study of Avastin in combination with chemotherapy for brain tumors. In a small trial of 32 patients, 72% on treatment were alive at six months, 38% of them with no progression of their tumors -- about double what would be expected for patients with Ms. Chin-Louis's kind of recurring brain tumor.
"It was early data, but it was very exciting," says Dr. Chang, director of the division of neuro-oncology at the University of California, San Francisco, Medical Center. In April, Dr. Chang asked Blue Cross of California to cover Avastin for Ms. Chin-Louis.
Blue Cross, owned by WellPoint, an Indianapolis company with more than 34 million members, denied coverage on May 21, saying in a letter: "Studies are ongoing to determine the role of this agent in the treatment of brain tumors."
Ms. Chin-Louis and her husband, a supervisor in sales at United Parcel Service Inc., say they couldn't afford the treatment. Her sister, Linda Tong, says she and her brother offered to cover the cost, but Ms. Chin-Louis initially resisted.
"I really felt that I had been paying for insurance for years for a reason, and when I needed medical care, my insurance company should pay," Ms. Chin-Louis says. "I didn't want to put that burden on my family."
In June, the doctor says she put Ms. Chin-Louis on a chemotherapy drug and appealed the denial for Avastin -- telling insurance officials she wanted to use the drugs in combination. On July 20, a brain scan showed the chemotherapy wasn't working: Ms. Chin-Louis's tumor had grown by 30%. She immediately agreed to allow her siblings to pay for Avastin, and she underwent her first treatment early last month.
In early August, asked by The Wall Street Journal about the Avastin denial, James Kappel, a spokesman for WellPoint, said: "While this patient's condition is very unfortunate, there is no medical evidence that supports the use of the drug" in brain tumors.
But Mr. Kappel called back the next day, saying WellPoint had approved Avastin for Ms. Chin-Louis. He said the company denied coverage at first because Dr. Chang sought to use the drug alone. Because Dr. Chang subsequently requested to treat Ms. Chin-Louis in combination with chemotherapy, as in the Duke study, WellPoint approved it, Mr. Kappel said.
Dr. Chang says the UCSF center has participated in a trial comparing Avastin on its own for brain tumors and in use with chemotherapy. Avastin alone appeared effective in some patients in the study, which has yet to be published, she says.
Dr. Chang says Ms. Chin-Louis has responded well so far to the Avastin treatments. "She's awake and feeling good and able to engage with her family," the doctor says. Ms. Chin-Louis is due for a brain scan later this week, which she hopes will show the tumor's growth in check.
"I know none of these treatments works forever," Ms. Chin-Louis says. "But I definitely want a little more time."
She'd like to celebrate her 45th birthday on Sept. 30, she said. She's eager to see her sister Linda's baby girl, due to arrive by Cesarean section on Nov. 2. And a small part of her is even daring to hope she will be in the audience when her eldest son Andrew graduates from high school this June.
Write to Geeta Anand at geeta.anand@wsj.com1
September 18, 2007
Will Sharing Ideas
Advance Cancer Research?
Hedge-Fund Managers Offer
$1 Million Prize to Combat
Scientists' Culture of Secrecy
By AMY DOCKSER MARCUS
After Hope Goldstein was diagnosed with advanced ovarian cancer in 2001, her family wanted to help. Her husband and two sons started researching the disease and quickly realized that even with surgery and chemotherapy, the prognosis wasn't good.
So they went in search of the one thing they believed still might help Mrs. Goldstein: new ideas.
They started calling cancer researchers, doctors at leading academic centers, specialists in ovarian cancer. In their conversations, they ran into an unexpected obstacle. Many people did have new ideas. But frequently, they weren't willing to share them.
Friends who worked in medical research tried to explain: Companies often will not pursue an idea for commercialization unless it is patented, requiring secrecy in the early stages. In addition, the grant process is competitive, and no one wants to get scooped. Perhaps most notably, professional advancement depends on publishing ideas in scientific journals.
In 2004, Mrs. Goldstein died. But her son Robert Goldstein, 41, continued to think about the issue. It seemed to him that what was holding back cancer research wasn't only a lack of money but a culture that discouraged the sharing of promising leads.
"If you have a great idea but someone else publishes it first, you get no credit, professionally or financially," says Gary Curhan of Harvard Medical School, one of the researchers who spoke with Robert Goldstein about the issue of sharing. "Ideas," says Dr. Curhan, "are our currency."
A managing partner at the hedge fund Gotham Capital in New York, Mr. Goldstein recognized similarities with his own profession. Money managers also were reluctant to share investment ideas. A few years earlier, Mr. Goldstein's business partner and friend, Joel Greenblatt, the 49-year-old founder of Gotham Capital, had created an online, selective group called the Value Investors Club, to spur idea sharing. Members shared investing strategies and commented on each other's research. A cash prize was awarded for the best idea of the week.
The two men thought that perhaps a similar model would work in cancer research. So this year they agreed to put up $1 million of their own money every year to fund the Gotham Prize for Cancer Research. Modeled on the Value Investors Club, the annual prize will go to the person who posts the best new cancer-research idea, judged by a board of respected scientists, at the prize's Web site by the end of December.
The winner of the Gotham Prize doesn't have to present a shred of evidence that the premise will work. To attract ideas from people outside the field of cancer research, there is no requirement that the winner be capable of seeing the idea through. And the prize money is earmarked for personal use, to be spent on anything the winner wants, even a fancy car or a bigger house.
This concept has been met with some skepticism, even by the scientific advisory board that will judge the ideas. Board member Meir Stampfer of Harvard Medical School said that when he was invited to join he asked, "Is this really a good use of their money? Maybe there is something better they can fund."
FUNDING A CURE
But the unusual nature of the prize illustrates the lengths to which patients and patient advocates are increasingly willing to go to boost research into their disease, especially rare illnesses for which there is little funding. Patients have hired their own experts to conduct research in particular diseases, and even helped to fund their own clinical trials. Efforts are also focused on overcoming the reluctance to share ideas.
Avichai Kremer, a 32-year-old who has ALS, the neurodegenerative illness also known as Lou Gehrig's disease, is co-founder of the Prize4Life, which is awarding a $1 million prize for ALS biomarker discovery. The National Institutes of Health announced this year a program called the New Innovator Award offering grants of up to $1.5 million over five years for innovative research projects that don't have extensive preliminary data.
In addition, the National Cancer Institute is helping companies find a way to collaborate in drug testing without worrying about intellectual-property issues. The medical journal the Oncologist is encouraging the publication of failed medical trials in order to bring the ideas behind these trials -- which otherwise would never see the light of day -- to a broader audience. And Mr. Goldstein's father, Alfred, with help from the family's other son, Mark, also developed a venture that aims to improve idea-sharing: Through "Project Hope," named for his late wife, Alfred Goldstein guarantees certain funding for specific projects and requires the researchers share results with each other on a regular basis.
The Gotham Prize is a particularly ambitious project that is attracting attention. The foundation of the Ira Sohn Investment Research Conference, which runs the conference as a benefit to raise money for cancer, will fund an additional $250,000 prize for the best pediatric-cancer idea submitted to the Gotham site. Ephraim Gildor, founder of Axiom Investment Advisors, is also providing financial support for the prize.
In setting up the prize, Mr. Greenblatt and Mr. Goldstein knew that they couldn't evaluate the new ideas themselves, so they set up a board of pre-eminent scientists and researchers. They include people to whom they have a personal connection, like Dr. Curhan, who is also Mr. Greenblatt's brother-in-law. Others such as Michael Kastan, director of the cancer center at St. Jude Children's Research Hospital in Memphis, Tenn., said they had received letters "out of the blue" inviting them to participate.
Dr. Curhan, the head of the advisory board, says all ideas submitted are vetted by at least two members of the board before being posted. As a result, although there are over 1,030 registered users, only 54 ideas have been posted. About 75% of the submitted ideas have been rejected so far, Dr. Curhan says. The tension is always how to eliminate ideas "not because they are new or different, but because there is no scientific basis for them," says Mr. Greenblatt. "It can't become a free-for-all. We want quality ideas or the site won't work."
To submit an idea, applicants need supply only a 500-to-1,000-word explanation of their concept and the reasoning behind it. They don't have to supply any data or evidence that the concept has been tested.
Some of the ideas accepted so far are very unusual. Someone who described himself as a businessman and former guitar player in a rock band proposed searching for the resonant frequency of cancer cells and then applying subsonic sound waves to cancer patients in order to trigger the body's immune response. Dr. Curhan says that idea sparked debate on the board. But it was posted in the end because some members argued that it couldn't totally be ruled out and was novel and outside-the-box.
There was also a proposal for a sort of Pap smear for early detection of breast cancer by a scientist who runs a major university lab but says it would be very difficult to get funding for such an early-stage project. Another comes from an AIDS researcher, who discovered an idea about brain cancer and thought as an outsider in cancer research he was unlikely to get funding from traditional sources. A survivor of testicular cancer posted an idea about an algorithm he created that he believes could lead to better early detection of recurrence.
Dr. Kastan of St. Jude says that even after reading all the ideas, "I am still waiting to see if it works." He says that most important scientific discoveries are made by "someone who was doing an experiment that had an unusual result that took them in a different direction. It is data that shows us the way, not a wild idea that comes out of nowhere." But he says he agreed to get involved because he recognized there weren't many places for scientists to share an early idea.
Bert Vogelstein of Johns Hopkins University School of Medicine, another advisory-board member, says he isn't sure he totally embraces the "anyone can have a great idea" approach that seemed to drive the initial concept. But he says that getting grants from government agencies like the NIH sometimes "forces people to do somewhat mundane experiments that follow up on other experiments rather than thinking creatively. In cancer in particular, we need more things that have never been done before because I think that's the only way we're going to beat the disease."
Similar thinking was behind the efforts at the Oncologist, a journal for cancer specialists, to publish online the results of so-called negative trials -- those that don't end up supporting the initial hypothesis. These trials don't result in the drug getting approved and aren't typically published. "A font of information" is being lost that could be helpful if looked at a new way, says Martin J. Murphy Jr., founding executive editor of the Oncologist.
At the NCI, scientists found that investigators at different companies wanted to share ideas and test different drugs in new combinations. But often they "couldn't do so because their own legal departments wouldn't let them because of intellectual property issues," says James Zwiebel of the NCI's Cancer Therapy Evaluation program. The NCI stepped in and crafted an agreement that each company signs that lets the NCI test a drug in combination with other companies' agents; the companies share in any information and other ideas that arise from the experiments.
Not everyone supports the premise that there's some great idea out there today waiting to be heard. "I don't sense a lot of holding back of exciting information," says Robert Ozols, senior vice president of medical science at Fox Chase Cancer Center and one of the oncologists the Goldsteins felt was willing to share ideas. "If we knew something worked, we'd be doing it."
But the prize applicants believe otherwise. Mark Kantrowitz, the publisher of FinAid.org, a Web site about student financial aid, and director of advanced projects for FastWeb.com, came across a posting about the prize while scouring the Web. In May 2003, at age 36, he was diagnosed with advanced testicular cancer. After completing treatment, he was put on a surveillance schedule of regular CT scans to pick up any possible recurrence. "The schedule seemed totally arbitrary," says Mr. Kantrowitz, who has a background in mathematics. He wrote an algorithm for a computer program that creates a surveillance schedule based on the greatest likelihood of catching a relapse as early as possible.
"I know it needs a clinical trial, but I come from outside the field and wouldn't know how to start it," he says. Even if he doesn't win the $1 million, he says he hopes an oncologist sees the idea online and likes it enough to test it. All of the ideas posted for the prize can be seen at www.gothamprize.org3, by users who register as guests.
David Rimm, a tenured professor of pathology at Yale University School of Medicine, runs a large, grant-funded research lab, where he says he can get money for his mainstream research. But things that are very early or "more out on the limb, are very difficult to get funded," he says. So he posted an idea to develop a kind of pap smear for early breast-cancer detection, using spectral spatial imaging technology on breast-tissue samples, to detect cellular changes beyond what the pathologist's eye can see.
Dr. Rimm says a colleague told him about the prize, but that he was surprised when he learned that the $1 million was for personal use. At the site, the founders had explained that one of the goals of the prize is to fill in current research gaps. "If they're just giving us money to buy a boat, it won't lead to filling the gaps," he says.
Mr. Goldstein and Mr. Greenblatt recognize that there is no guarantee that the winner will spend the money on shepherding the concept into a clinic. What they hope is that the prize site can serve as a kind of marketplace of ideas, where additional investors might see something interesting and decide to invest in some of the other projects as well. The prize's true value, Mr. Greenblatt said, will come from jump-starting that process. In the end, says Dr. Curhan, "if you get even three or four really good ideas the first year, that would be a huge success."
Write to Amy Dockser Marcus at amy.marcus@wsj.com
9/18/2007
Vacation pics
2007_08 175
Originally uploaded by amikulak
I've been meaning for some time to post some pictures from our August vacation. We basically stayed at home for two weeks with a side trip to Disneyland for four days, staying at the Grand Californian hotel and generally having a good time with ourselves. Hannah went on the California Screamin' roller coaster which is pretty darn scary even for an adult. She did awesome though she did freak out a little bit in the beginning (negative g's do that to me too!). Max rode the Toontown rollercoaster for the 2nd time in his life and this time LOVED it which is great!
9/17/2007
This Sunday in Braintree
Pat Lacey - and his entire extended family and friends network it seems at times - has been an extraordinary supporter of the MagicWater Project. Besides being a good guy, Pat's a fantastic writer and most importantly his 3-year old son Will is one of the coolest kids I've ever seen. This sunday in Braintree, MA, there is a fundraiser on behalf of Will. I thought I'd post the flyer here so that you can see how incredibly heartwarming it is to see Will's community come together to try to do good on behalf of others. I will post tomorrow about what's being done in our community, specifically what Lisa Sturt, Max's first grade teacher, is doing to raise money and teach the entire student body at Solana Highlands elementary about caring and compassion for others.
Starting chemo again
We've been given the green light to start Max back on his chemo. Since September 6th he's been off of it, having counts too low to keep taking even just the cyclophosphamide. But today, his ANC was 830, platelets 90, hemoglobin 9.6 and WBC 1.7, so back on the juice he goes. We've also gone back to Etoposide (he'll take his first dose tomorrow AM) since the experiment with oral Irinotecan. Not that we know definitively that one is more efficacious than the other. Max seems to tolerate the Etoposide better. We'll see. Cancer is fun, as this clearly illustrates.
Below, some pics from this weekend.
Hannah and Max (and Rufus, Hannah's classroom mascot that got to go home with her for the weekend) in front of 'Uncle' Brian's aggressor F5 Tiger (he gets to humiliate the Tom Cruises of the world during Top Gun training). Note the Russian star on the tail. Weird seeing that in the middle of North Island NAS.
Max is growing some hair, as you can kind of see in this photo. This has me totally freaked out as in my irrational state of mind hair=ineffective chemo and no hair=effective chemo. This obviously isn't the case but who says that cancer is rational? Nevertheless, it looks good on him and Max seems to be liking it.
Below, some pics from this weekend.
Hannah and Max (and Rufus, Hannah's classroom mascot that got to go home with her for the weekend) in front of 'Uncle' Brian's aggressor F5 Tiger (he gets to humiliate the Tom Cruises of the world during Top Gun training). Note the Russian star on the tail. Weird seeing that in the middle of North Island NAS.
Max is growing some hair, as you can kind of see in this photo. This has me totally freaked out as in my irrational state of mind hair=ineffective chemo and no hair=effective chemo. This obviously isn't the case but who says that cancer is rational? Nevertheless, it looks good on him and Max seems to be liking it.
9/14/2007
Max's Meds
So I'm preparing Max's nightly meds and supplements and I think, "Sheesh, I've gotta get a photo of this!"
Starting from the left: The first six bottles are the pills Max takes by mouth morning and night (bottle one is the nifurtimox, bottle two is his chemo -cyclophosphamide- pills). The four taller bottles in the rear-center are the liquid meds we give him through his mickey-button. The last six bottles are all the supplements we give him at night. We open all the capsules, mix them with yogurt and milk, then draw them into the big syringes and give them through his mickey-button, too. Standing up in the back in the clipboard we use to make sure we've given him everything at the right time everyday. The sheet gives us a 2-week look.
Fun!
Starting from the left: The first six bottles are the pills Max takes by mouth morning and night (bottle one is the nifurtimox, bottle two is his chemo -cyclophosphamide- pills). The four taller bottles in the rear-center are the liquid meds we give him through his mickey-button. The last six bottles are all the supplements we give him at night. We open all the capsules, mix them with yogurt and milk, then draw them into the big syringes and give them through his mickey-button, too. Standing up in the back in the clipboard we use to make sure we've given him everything at the right time everyday. The sheet gives us a 2-week look.Fun!
9/13/2007
Happy Birthday Nic!
Happy birthday to you little Nicky - you're two years old today but already have the spirit and energy of a much older boy. You've been the best little brother to your brother Max and sister Hannah. Mommy and Daddy know you're going to have a special life and are so happy to see you growing into such a sweet little boy.
9/10/2007
First grade, treatment change
It's been a while since our last post. Chalk it up to August blahs, back-to-school schedule, changes in Max's treatment and general lack of energy and will to do as little as possible cancer-related other than what was necessary (necessary is getting up a 6am to give Max his glutamine and zophran - unnecessary is blogging about it)!
Notice I slipped in a 'change of treatment' above. Just to check to see if you were reading, and yes, we made a slight change to Max's treatment regimen but we haven't been able to fully execute but I'll get to that in a minute.
We decided that we liked the oral chemo regimen that we were on, and are sticking with that until we see data that its not working. We switched from irinotecan back to etoposide/VP-16 which Max took a few times in July. We didn't like the side-effects of irino (nausea) and so hopefully etoposide will be better tolerated by his stomach.
As I mentioned above, we haven't had a chance to really execute this new treatment regimen because Max's counts have been low and not recovering. It could be the oral cytoxan every day is just too much, so we took him off as of last Thursday and will see this Thursday if his counts are high enough to start on the cytoxan again, adding the Etoposide in as soon as possible. Max is still taking the nifurtimox daily and he's tolerating that well, though we've switched to 1 pill in the AM and 2 in the PM. We think he still has some 'irritability' issues related to the nifurtimox but given how efficacious we think it is, its a small price to pay.
On the supplement front, Max continues to take an artimisinin compound containing artemisinin, artemether and artesunate. It's relatively expensive ($2/pill/day) but worth it. He's also taking glutamine, skullcap, vitamin D, acidopholus/bifidus, melatonin, tumeric, calcium, bromelain, papaya enzyme, fish oil, and olive oil. We give him zantac 2x a day to help protect his stomach from the chemo and the celebrex, zophran when he's nauseaus, and immodium when he's got the runs. Most of this we can give him through his g-tube but some he has to take orally and in that area Max has shown improvement (though not as good as Sam who shows off by taking 3-4 pills at a time).
Also, we discovered we weren't giving him enough calories a day to gain weight, so its back to the Nestle Peptamen for Max for the time being until we can figure something out. We 're still giving him the vegetable concoction that Melissa makes up from beets, kale, red cabbage and garlic, but that's during the day. At night and once during the day he gets the high-calorie Peptamen and it seems to have had its desired effect already, Max gaining a kilo in one week as of last Thursday. He's also starting to grow hair because of the move away from IV chemo, which has me personally terrified as I now have an justifiably irrational fear that if his hair's growing, so is his cancer.
Max started school two weeks ago. His teacher this year is Mrs Sturt and she came highly recommended and is a match made in heaven. She's very, very dedicated and super interested in Max's situation and how to contribute to his betterment. She's already planning two fundraisers at the school. Mrs. Sturt has a web page and blog up for Max's class and you can see it here.
Hannah continues to do well in everything at school, and her new teacher Ms Janis seems perfect for her and vice-versa. As usual, Hannah was hugging her new teacher on the first day like they were long-lost best friends.
This past weekend Melissa and I got away for two nights (sans kids) thanks to her family/my dad springing for a couple of nights at the Marriott in downtown. We had a fabulous marina and bay-view suite thanks to Blanche and thoroughly enjoyed ourselves. Thanks Matt, Jenny, Randee, Mike, Mom/Dad Porter, Greg, Blanche, Jarod, Bree, and 'ol man Mikulak!
Notice I slipped in a 'change of treatment' above. Just to check to see if you were reading, and yes, we made a slight change to Max's treatment regimen but we haven't been able to fully execute but I'll get to that in a minute.
We decided that we liked the oral chemo regimen that we were on, and are sticking with that until we see data that its not working. We switched from irinotecan back to etoposide/VP-16 which Max took a few times in July. We didn't like the side-effects of irino (nausea) and so hopefully etoposide will be better tolerated by his stomach.
As I mentioned above, we haven't had a chance to really execute this new treatment regimen because Max's counts have been low and not recovering. It could be the oral cytoxan every day is just too much, so we took him off as of last Thursday and will see this Thursday if his counts are high enough to start on the cytoxan again, adding the Etoposide in as soon as possible. Max is still taking the nifurtimox daily and he's tolerating that well, though we've switched to 1 pill in the AM and 2 in the PM. We think he still has some 'irritability' issues related to the nifurtimox but given how efficacious we think it is, its a small price to pay.
On the supplement front, Max continues to take an artimisinin compound containing artemisinin, artemether and artesunate. It's relatively expensive ($2/pill/day) but worth it. He's also taking glutamine, skullcap, vitamin D, acidopholus/bifidus, melatonin, tumeric, calcium, bromelain, papaya enzyme, fish oil, and olive oil. We give him zantac 2x a day to help protect his stomach from the chemo and the celebrex, zophran when he's nauseaus, and immodium when he's got the runs. Most of this we can give him through his g-tube but some he has to take orally and in that area Max has shown improvement (though not as good as Sam who shows off by taking 3-4 pills at a time).
Also, we discovered we weren't giving him enough calories a day to gain weight, so its back to the Nestle Peptamen for Max for the time being until we can figure something out. We 're still giving him the vegetable concoction that Melissa makes up from beets, kale, red cabbage and garlic, but that's during the day. At night and once during the day he gets the high-calorie Peptamen and it seems to have had its desired effect already, Max gaining a kilo in one week as of last Thursday. He's also starting to grow hair because of the move away from IV chemo, which has me personally terrified as I now have an justifiably irrational fear that if his hair's growing, so is his cancer.
Max started school two weeks ago. His teacher this year is Mrs Sturt and she came highly recommended and is a match made in heaven. She's very, very dedicated and super interested in Max's situation and how to contribute to his betterment. She's already planning two fundraisers at the school. Mrs. Sturt has a web page and blog up for Max's class and you can see it here.
Hannah continues to do well in everything at school, and her new teacher Ms Janis seems perfect for her and vice-versa. As usual, Hannah was hugging her new teacher on the first day like they were long-lost best friends.
This past weekend Melissa and I got away for two nights (sans kids) thanks to her family/my dad springing for a couple of nights at the Marriott in downtown. We had a fabulous marina and bay-view suite thanks to Blanche and thoroughly enjoyed ourselves. Thanks Matt, Jenny, Randee, Mike, Mom/Dad Porter, Greg, Blanche, Jarod, Bree, and 'ol man Mikulak!
8/20/2007
Back to the Grind
Andy's back to work today after a great two weeks off.
As Andy said in an earlier post, we hit the beach week-one just about everyday. Hannah's totally boogie boarding now, and Max runs around in the shallow waves and catches sand crabs. We dig a hole down by the water for Nic that serves as a pool and he plays there. Living this close to the beach is great!
This week we did Disneyland! All three kids loved it. Hands down favorite for Nic was the Buzz Lightyear ride (if you've been on it, you know why) with Small World holding a close second. Max graduated to Star Tours this year and just about came out of his skin - Star Wars fan that he is. Hannah moved up to the big-kid rides and did two rollercoasters: California Screamin' and Space Mountain (white knuckled on both but loved them just the same!). It is so fun to relive DLand through the eyes of your own kids. It really brings the magic alive.
I'll post photos over on Max's site of all the fun we had and you can read about how High School Musical 2 World Premier almost ruined our vacation on my blog.
As Andy said in an earlier post, we hit the beach week-one just about everyday. Hannah's totally boogie boarding now, and Max runs around in the shallow waves and catches sand crabs. We dig a hole down by the water for Nic that serves as a pool and he plays there. Living this close to the beach is great!
This week we did Disneyland! All three kids loved it. Hands down favorite for Nic was the Buzz Lightyear ride (if you've been on it, you know why) with Small World holding a close second. Max graduated to Star Tours this year and just about came out of his skin - Star Wars fan that he is. Hannah moved up to the big-kid rides and did two rollercoasters: California Screamin' and Space Mountain (white knuckled on both but loved them just the same!). It is so fun to relive DLand through the eyes of your own kids. It really brings the magic alive.
I'll post photos over on Max's site of all the fun we had and you can read about how High School Musical 2 World Premier almost ruined our vacation on my blog.
8/12/2007
Article on Will Lacey
Patrick Lacey, father of Will, has become more and more involved in the MagicWater Project. A friend of his has organized a fundraising event to take place in September and the local newspaper wrote an article about the event. The link is below. Since the money raised is going to the MagicWater Project, the reporter wanted to speak to someone from the project as well, and so I spoke with the reporter on Thursday.
http://ledger.southofboston.com/articles/2007/08/11/news/news09.txt
http://ledger.southofboston.com/articles/2007/08/11/news/news09.txt
8/09/2007
Quick update
Just a quick update here since I know its been awhile since our last post. We're 'on vacation' this week and next and have been having a blast. This week, we decided to stay at home and close by the hospital in case Max needed blood, etc, but as it turns out his counts have been great. On Monday he went to clinic and his #'s were:
ANC-1700
PLT-173K
WBC-2.4
Weight-17.4kg (up 1/2 kg in one week!)
His weight and energy levels are up. So is his LDH (one of the NB markers we follow), but his LDH has been up and down so much lately that I'm not concerned (yet). Waiting for his catecholamines from 7/30 and 8/6 (VMA/HVA) which were very good on 7/23 (VMA 7.1, lowest in months). Friday, Max goes to clinic once again for a final check before Disneyland next Mon-Thu (more on this later). Max has been tolerating his oral chemo very well. The irinotecan seems to upset his tummy a bit as he's NOT been throwing up on just the oral cytoxan.
This week, we've been enjoying being tourists in our own town. We hit the beach Sunday, Tuesday and are going again today and tomorrow with friends. Monday night we went to the Wild Animal Park for 'Park at Dark' which was fantastic. It was about 78 degrees when we left at 8pm and we had a wonderful time. The Wild Animal Park is in Escondido about 20 miles east of us in Del Mar and was originally built to allow for the reproduction of white rhinos (about 20 left in the world). It's now a full-fledged park and the best part is that it doesn't experience the crowds that the San Diego Zoo gets so its a nice relaxed time. The lions were especially active and were roaring as the sun went down which was incredibly chilling to hear. As we got off the tour that takes you around the park (the only way to see some of the animals), we stopped in front of the lions again and I swear the two females were looking for a way to jump the (too small) gap between them and us. I admit I was looking around as to where I might grab the kids and run to if one of them 'made it' ;). Last night we went to the San Diego Zoo. Tonight, tomorrow and Saturday night the kids will be rotating staying over at Melissa's parents house, giving us a kid-free night on Friday night to do as we please (go to dinner, have an adult conversation, sleep in).
Monday, we take off for four busy days at the Magical Kingdom. We're staying at the very nice Grand Californian hotel and are planning on 3 1/2 days at the park, taking off most of day 3 to kick it by the pool and in Downtown Disney. Max and Hannah are very excited and we will see how Nic handles the overstimulation. Since he's had to keep up with his older sibs since birth we think he'll do fine and will have loads of fun - much more than M or H would have had at his age.
We'll post pics soon. That's all folks!
ANC-1700
PLT-173K
WBC-2.4
Weight-17.4kg (up 1/2 kg in one week!)
His weight and energy levels are up. So is his LDH (one of the NB markers we follow), but his LDH has been up and down so much lately that I'm not concerned (yet). Waiting for his catecholamines from 7/30 and 8/6 (VMA/HVA) which were very good on 7/23 (VMA 7.1, lowest in months). Friday, Max goes to clinic once again for a final check before Disneyland next Mon-Thu (more on this later). Max has been tolerating his oral chemo very well. The irinotecan seems to upset his tummy a bit as he's NOT been throwing up on just the oral cytoxan.
This week, we've been enjoying being tourists in our own town. We hit the beach Sunday, Tuesday and are going again today and tomorrow with friends. Monday night we went to the Wild Animal Park for 'Park at Dark' which was fantastic. It was about 78 degrees when we left at 8pm and we had a wonderful time. The Wild Animal Park is in Escondido about 20 miles east of us in Del Mar and was originally built to allow for the reproduction of white rhinos (about 20 left in the world). It's now a full-fledged park and the best part is that it doesn't experience the crowds that the San Diego Zoo gets so its a nice relaxed time. The lions were especially active and were roaring as the sun went down which was incredibly chilling to hear. As we got off the tour that takes you around the park (the only way to see some of the animals), we stopped in front of the lions again and I swear the two females were looking for a way to jump the (too small) gap between them and us. I admit I was looking around as to where I might grab the kids and run to if one of them 'made it' ;). Last night we went to the San Diego Zoo. Tonight, tomorrow and Saturday night the kids will be rotating staying over at Melissa's parents house, giving us a kid-free night on Friday night to do as we please (go to dinner, have an adult conversation, sleep in).
Monday, we take off for four busy days at the Magical Kingdom. We're staying at the very nice Grand Californian hotel and are planning on 3 1/2 days at the park, taking off most of day 3 to kick it by the pool and in Downtown Disney. Max and Hannah are very excited and we will see how Nic handles the overstimulation. Since he's had to keep up with his older sibs since birth we think he'll do fine and will have loads of fun - much more than M or H would have had at his age.
We'll post pics soon. That's all folks!
7/31/2007
Nathan Gentry
Nathan became an angel on Sunday. Rest in peace Nathan, and may the Gentry family find peace in spite of their horrible loss.
7/30/2007
CT biopsy today
Max will be having a CT-guided needle biospy of his T11 vertebrae today. Hopefully, we'll get a good sample and can find out the state of maturity of those pesky cells that continue to light up on MIBG but which don't grow (or shrink) in size. Will update the blog later and let everyone know what happened.
7/25/2007
Nathan Gentry
From Will Lacey's blog today...
The boy and family who have given me so much strength and inspiration to do a better job, to try harder, to dig deeper, and who helped to make me a better dad because of their heroic efforts are coming to the end of Nathan's battle.
Nathan became transfusion dependent because his marrow has stopped producing platelets. Since he is in so much pain they decided to stop giving platelets since extending his life with platelets would only lead to more intense pain and suffering. Since you need platelets to clot your blood this leaves open the very real possibility for a catastrophic bleed.
I ask that if you are someone who prays to please ask your God to spare Nathan and his family that horror, to spare him any more pain, and to give his family the strength and peace that they need right now.
This disease sucks.
7/24/2007
A fine young man
Once again I get to be lazy and let better wordsmiths do my work for me. John Silva is an 11 year old whom I have never met and who has never met (to my knowledge) anyone with neuroblastoma. Nevertheless, John (did I say he just turned 11?), decided that instead of presents, he'd rather have his friends donate money to the MagicWater Project fund. Read about it here on Will Lacey's blog. Thank you John! Thank you John's friends and their parents!
7/23/2007
Latest #'s
It's scan week(s) for Max. He had MIBG scan last Wed (Melis reports stable on T11 but we are still waiting for the official word), MRI today. We skipped BMA (bone marrow aspiration) as he just had one, they've been negative since January and the last time he had one in June we landed in the hospital with an infection. No thanks, we'll take a pass on that this month! His LDH #'s are down (good): 632 today versus 722 on 7/2 but yo-yo on him so much that I'm beginning to discount the 100pt swings as just inherent variability. Still, as an emotional human its good to see them down.
We're about 75% through collecting other blood markers for the naturopath we're using to help us more acutely monitor various factors that are related to NB tumor growth and metastasis. I won't go into detail here because the tests are incomplete at this point, but I will say that Max's results are in the high-normal range, even with all the stuff we've been giving him that should theoretically address these factors (angiogenesis, inflammation). Still, there are some promising tools (tetrathiomobylate) that we haven't used yet, waiting for these tests to be done so we can baseline then measure progress. His Vit D levels are about mid-range on the 25(OH)D test which measure vit D. We're going to give him 400-800 IU extra daily to boost that a bit from the mid-range of 43 to hopefully the upper range of 70-80.
Max's counts were good today. We hope that the combination of oral chemo + continued supplemenation (added bromelain which may help stimulate platelet formation) + our new vegetable cocktail (kale, beets, red cabbage and garlic) that Max gets ~500ml of daily is helping to boost his immune system, blood cell production and overall health and energy. We're slowly transitioning away from the Nestle Peptamen, replacing it with the vege combo and more "natural" forms of nutrition, ie from real food you can buy and liquify in the cuisinart in order to push through Max's gtube.
We're about 75% through collecting other blood markers for the naturopath we're using to help us more acutely monitor various factors that are related to NB tumor growth and metastasis. I won't go into detail here because the tests are incomplete at this point, but I will say that Max's results are in the high-normal range, even with all the stuff we've been giving him that should theoretically address these factors (angiogenesis, inflammation). Still, there are some promising tools (tetrathiomobylate) that we haven't used yet, waiting for these tests to be done so we can baseline then measure progress. His Vit D levels are about mid-range on the 25(OH)D test which measure vit D. We're going to give him 400-800 IU extra daily to boost that a bit from the mid-range of 43 to hopefully the upper range of 70-80.
Max's counts were good today. We hope that the combination of oral chemo + continued supplemenation (added bromelain which may help stimulate platelet formation) + our new vegetable cocktail (kale, beets, red cabbage and garlic) that Max gets ~500ml of daily is helping to boost his immune system, blood cell production and overall health and energy. We're slowly transitioning away from the Nestle Peptamen, replacing it with the vege combo and more "natural" forms of nutrition, ie from real food you can buy and liquify in the cuisinart in order to push through Max's gtube.