I’m on a plane as I write this heading down to Florida to visit the winning school from our Great Lemonade War contest (more on that in another blog), but can’t ignore the 
The research team, led by Yaël Mossé, MD from the Children’s Hospital of Philly (CHOP), explains that crizotinib appears to be effective in patients that test positive for the cancer causing gene, anaplastic lymphoma kinase (ALK). Crizotinib specifically looks to disrupt this gene and voilà – if all goes as planned, the cancer disappears. It’s all part of the personalized therapy approach that so many researchers are exploring these days and for good reason. Finding treatments that are effective in patients whose cancer has certain characteristics helps to create customized treatment plans that not only work, but also prevents kids from being subjected to other therapies or procedures that they know won’t work.
Crizotinib also showed promise in kids with specific forms of neuroblastoma that harbor the ALKgene. Neuroblastoma was the type of cancer that Alex had and although the tests for ALK were not available when she was in treatment, you better believe we would have looked into it if it was.
One more neat piece of info – Alex's Lemonade Stand Foundation (ALSF) had a role in this breakthrough! Although ALSF was not responsible for funding this particular clinical trial, we did provide previous support to the research team that allowed them to develop the trial. Dr. John Maris, one of the doctors who treated Alex at CHOP states it a bit more eloquently than me:
"Funding through ALSF was absolutely critical for the translation of our basic research findings to a clinical trial. It is a highly complex process (and often difficult to fund) to take a novel discovery and develop a safe and effective therapeutic strategy. ALSF funding allowed us to be extremely facile in getting the work done so that we could get a clinical trial open as soon as was humanly possible. While the actual trial was funded by the National Cancer Institute through the Children's Oncology Group, ALSF funding to CHOP to support core research infrastructure also enhanced our ability to bring children to our center from far and away for enrollment on the trial, and to receive the medication in a safe and very efficient manner."
- John Maris, MD, Chief, Division of Oncology, and Director, Center for Childhood Cancer Research at The Children’s Hospital of Philadelphia
- John Maris, MD, Chief, Division of Oncology, and Director, Center for Childhood Cancer Research at The Children’s Hospital of Philadelphia
So what happens now? Well, doctors are working hard to get crizotinib moved up to a front line treatment for kids with ALCL who present with the ALK gene, and the researchers are planning a larger scale trial for these patients as well as trials to further investigate the drug’s efficacy in kids with neuroblastoma.
It’s news like this that energizes me and the work that we do every day at ALSF – I’m sure the amazing researchers who work tirelessly to make this their life’s work feel similarly. Progress is being made, we are making a difference (thanks to your support) and kids’ lives are being saved. Awesome.
-Jay Scott (Alex’s Dad)
May 17, 2012
