Developments in Neuroblastoma Symposium

 

On Thursday (21st), I attended the Developments in Neuroblastoma Symposium at the University of Vermont. Present were about 120 parents, researchers, doctors and pharmaceutical industry representatives, as well as some medical students and nurses. It was beautiful weather in Burlington - 88 degrees was the high that day.

After the dean of the medical college opened the Symposium, Dr. Giselle Sholler welcomed everyone and then I spoke briefly for a few minutes. I spoke about Max, about the need for greater collaboration using the example in an email from a friend, Alessandra Blassina, who is an oncology research scientist at Pfizer in La Jolla.

Some highlights of the day’s presentations:

• David Krag, MD, did a great presentation on phage display for neuroblastoma. Phage display is a method of creating patient tumor-specific antibodies (phage are viral particles that infect bacteria - bacteriophage - which then make antibodies for you who might know what that means) which can then be attached to another molecule like a neurosphere (white blood cell). The patient tumor-specific antibody then easily finds and attaches itself only to tumor cells, and the proximity of the neurosphere to the tumor cell enables the neurosphere to attack and kill the tumor cell. Nice. Simple. Elegant. Approximate time to clinical application: 2 years. Approximate cost to develop phage display in neuroblastoma to phase 1 clinical trial readiness: $250K. (Note that time and cost estimates for this research are my own based on my understanding of the issues involved and have not been reviewed by Dr. Krag. But I do believe them to be directionally accurate).
• Giselle Sholler, MD, presented a lot of information:
• Preclinical work on genomic analysis of patient samples for the development of the personalized clinical trial.
• Identifying BTK as an important target of neuroblastoma tumor initiating cells which has led to preclinical work testing a BTK inhibitor.
• TPI-287 trial that is currently open in Vermont – they recently just filled their first study cohort and should begin accruing for the 2nd cohort very soon. What is great about the TPI-287 trial design is that even though it’s a Phase I trial, she has designed it to be a multi-agent trial, but the first two rounds are single agent only, so the pharmaceutical company gets good toxicity and pharmacokinetics data, and then the additional adjuvant agent is added (Temodar). And though it’s a Phase I trial, like all smartly-designed trials for a rare disease like neuroblastoma, they collect efficacy data (ie, does it work?) in addition to the study’s main purpose which is to measure toxicity and establish an MTD (max. tolerated dose).

• Craig Webb, PhD, showed how he is developing predictive models from tumor-derived molecular data that can systematically identify targeted treatments from existing pharmacopeia for metastatic and/or refractory neuroblastoma. The methodology and processes that he created form the basic underpinnings of a possible predictive or “personalized” clinical trial for 2nd-relapse/progression after relapse and refractory neuroblastoma patients.
• Nai-Kong Cheung, MD, PhD, the keynote speaker from Memorial-Sloan Kettering Cancer Center in NYC and a noted neuroblastoma expert, presented a history of immunology and immunotherapy in neuroblastoma. While Cheung had no new data to present, it was good to hear his perspective and point-of-view on how MSK treats kids with its 3F8 immunotherapy treatment. In retrospect, I think it might have been good to give 3F8 a try on Max – I know some people reading this will cringe that I’m playing “coulda-shoulda-woulda” and that I should feel confident that we did all that we could and that we made all the right decisions regarding Max’s treatment. But until you’ve experienced what we have experienced, fighting neuroblastoma for the four years that Max did, you don’t get to make that call ;)
  

Afterwards, the parents present gathered for a few minutes alone with each other. Gilles Frydman, (founder of ACOR.org) whom I got to moderate the last panel of the day on Therapeutic Decision Making in Neuroblastoma, provided us with some helpful perspective on what other cancer groups have done to successfully move research forward quickly.

Overall, it was very good to see some of the newest ideas for how we might be able to find therapies that improve chances of survival for these kids while being less toxic to their little bodies. At the same time, while energized from being around "my people", I also came away depressed that my fight continues on behalf of kids I know, but whom aren't mine. Still, continuing is the only option for me. I can't think of a greater purpose than doing all that I can to defeat neuroblastoma.

I returned to San Diego on Friday. On Sunday, some of the medical students at UVM as well as Friends of Will ran teams in the Burlington Marathon to raise money for Dr. Sholler's program. In all, $30,000 was raised!