Read original articleCV father’s MagicWater Project dedicated to working with cancer researchers and oncologists to speed up discoveries of potentially life-saving new drugs•Fundraising coordinator neededBy Catherine KolonkoAbout three years ago, a little Carmel Valley boy, Max Mikulak, was diagnosed with a type of children's cancer that is difficult to cure. When the cancer returned after traditional treatment, his parents began an odyssey through the world of science and medicine in search of new drugs that might keep him alive.
Max was displaying symptoms of anemia, including a lack of energy when he was diagnosed in 2004 with high-risk Stage 4 neuroblastoma, an aggressive pediatric cancer that is diagnosed in about 600 children per year in the United States, said his father Andy Mikulak. The cancer is more prevalent among babies under the age of 1 but much deadlier in older children. After the age of 2 the survival rate dips drastically
“If you wait another year and it sticks around, it becomes a deadly killer,” said Andy.
Neuroblastoma is a disease in which malignant cancer cells form in nerve tissue of the adrenal gland, neck, chest, or spinal cord. By the time it is diagnosed, the cancer has usually metastasized, most often to the lymph nodes, bones, bone marrow, liver and skin, according to the National Cancer Institute. Usual treatment options for severe cases like Max's involve high doses of chemotherapy, radiation and stem cell transplant.
For about a year after his initial treatment, the cancer disappeared but Max, now 6 years old, had a relapse in 2006 and has been battling the return of the cancer for 16 months. He's had most of the traditional treatments for his disease and there are only a few options left, none of which are very effective, Andy said. That dearth of treatment possibilities led Max's determined father to reach out to other parents in the same situation, not only as a support system but also in a hunt for new life-saving drugs.
“We were kind of thrust into this new world where you're on your own as far as treatments,” Andy said.
“All of a sudden you start to become an activist and you start to think 'How are you going to save our kids' lives.'”
Almost 80 percent of kids with cancer are essentially cured and become long-term survivors but then there are the 20 percent that don't, said Dr. William Roberts, an oncologist at Rady Children's Hospital in San Diego who oversees Max's treatment. For children with relapsed neuroblastoma “The outlook is not good,” Roberts said. “The number of kids surviving is about 30 percent, which is pretty dismal.”
For that reason, Roberts said he understands the mindset of parents like the Mikulaks who are open to trying something innovative with the idea of “What can we do differently to try to gain some ground?”
There are “tons and tons of drugs out there that might work” but have no visibility because there is no business reason to pursue them, said a frustrated Andy Mikulak. It is unfair that approved treatment options are so limited just because of the relatively small number of children affected by the deadly cancer, he said.
Last year Andy and Neil Hutchison, a San Diego father whose son also has relapsed neuroblastoma, started the MagicWater Project (www.magicwater.org), a foundation dedicated to working with cancer researchers and oncologists to speed up discoveries of potentially life-saving new drugs for children with relapsed neuroblastoma and medulloblastoma. Their goal is to find innovative, low toxicity, new treatments through funding of clinical trials and other research.
The foundation name was derived from the explanation by the parents of an 18-month old girl about why she needed chemotherapy. “He just called it magic water,” Andy said.
“We all have our own mechanisms for dealing with this with our kids,” Andy said, referring to the challenge for parents who must explain cancer treatment to their very young children. Calling the foundation magic water seemed appropriate for the foundation because it symbolizes hope and pays tribute to that little girl who eventually lost her battle to cancer, he said. “It's kind of the inspiration for what we're all working for,” he said.
Funding for MagicWater is done mainly through networking and from friends and family. Since its inception, a couple of people have written checks for as much as $100,000 and another $25,000 was acquired through a fundraiser at Max's school, Andy said. He compares the MagicWater project mindset to that of a venture capitalist who seeks investments that are high risk and high return, and notes that it is much different from a traditional cancer foundation that is more conservative, like a bank, he said.
“It's meant to speed up the process by which an agent can get tested in neuroblastoma and medulloblastoma,” Andy said.
One course of treatment now showing promising results initially caught the attention of researcher Giselle Sholler, a Vermont doctor. She had heard about a case reported by physicians from Brown University who treated a child for a parasitic disease known as Chagas who also happened to have neuroblastoma. The administration of the antibiotic nifurtimox, long used in South America to treat tropical illness, seemed to have an unexpected benefit on the cancer which went into remission.
“She started looking at it and thought it might have some interesting anti-tumor properties,” Andy said.
Nifurtimox was not approved for marketing in the states but had been available for years in other countries. In further lab testing of the drug, Sholler found that it appeared to shrink or kill neuroblastoma tumor cells. Her findings eventually led to a phase 1 clinical trial that included Max as a patient. The trial was designed to test the safety and toxicity of the drug in combination with a regimen of chemotherapy.“Sometimes you just have to go on these Phase 1 projects and hope for the best,” said Andy.
The results were “very encouraging” and that trial has opened the door to getting the drug available for other children, said Dr. William Roberts, Max's doctor at Rady Children’s Hospital in San Diego. Although the trial's objective was to determine the drug's safety profile, “Clearly there's a whole world of information, so you don't want to ignore it,” he said.
A phase 2 clinical trial headed by Sholler is underway in Vermont to further investigate the use of nifurtimox alone or in combination with other drugs as treatment of relapsed or refractory neuroblastoma and medulloblastoma. Children’s Hospitals in San Diego, St. Louis, and Atlanta are also expected to participate. The MagicWater project donated $150,000 to facilitate the study.
Another project that MagicWater recently funded with a $100,000 grant is research conducted by Dr. Donald Durden, scientific director at the Aflac Cancer Center in Atlanta and a professor of pediatrics at Emory University School of Medicine. He is conducting research in in vitro and animal models to discover novel combinations of new drug agents that could slow or stop the progress of neuroblastoma and medulloblastoma. The goal is to find the right combinations of agents that could then be tested in clinical trials with pediatric cancer patients.
One compound resulting from Durden's work may soon be tested in a clinical trial, he said. The low toxicity drug, which appears to be well tolerated, will soon be submitted to an investigational review board that monitors U.S. drug trials and ensures the safety of participating patients.
Durden said he understands the sense of urgency felt by parents who want to find new treatments that might save their children.
“You might say we're not waiting around,” he said. “We're trying to push the envelope a little bit. It's not like they have a few years to be thinking about this because the kids are going to die.”
Meanwhile, Andy and others involved with the MagicWater project continue to search the Internet and network with other parents, researchers, and doctors to find possible drug candidates worthy of funding while Max and other children like him fight their courageous battles with cancer. Max recently started a new round of chemotherapy and his daily medications are adjusted regularly based on their toxicity and efficacy.
Andy Mikulak and Neil Hutchison, MagicWater cofounders, are working to build the organization and they are currently seeking someone who can coordinate fundraising for the project. More information is available on the Internet site, including blogs, with updates on Max and other children can be found at www.magicwater.org.
