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My friend, Beth is a pediatric oncologist who is also the recipient of a Baldrick's grant towards here research on Medulloblastoma. I asked her to summarize for us what she is doing to develop effective treatments for this disease, the same one that took Henry Scheck's life, and she wrote this:
Brain tumors are the leading cause of pediatric cancer deaths, and medulloblastoma is the most common malignant brain tumor. Current treatments for medulloblastoma, when effective, often have lifelong side effects. A potential new drug called IPI-926 is currently in Phase 1 trials for adults with cancer, and is being evaluated preclinically in medulloblastoma. IPI-926 is active and well tolerated in preclinical models of medulloblastoma. It works by interfering with the Hedgehog pathway, a cellular signaling network, which is activated in many medulloblastomas. IPI-926 is a semi-synthetic derivate of the plant-derived natural product, cyclopamine, and it is orally administered.It's a little jargon-y, which is understandable from one ensconced so deeply in her research, but the summary is this: they have a compound which shows some real potential for being an effective treatment for medulloblastoma! You go, Beth!
Ptc1-null mice develop early and aggressive medulloblastomas, which cause bulging skulls and difficulty walking within weeks of birth. All 11 of the mice treated with placebo died from their disease by two months of age. In contrast, all 12 mice that received daily IPI-926 showed dramatic improvement. After only 2 weeks of IPI-926 treatment, all skull deformities and neurologic deficits resolved. Furthermore, IPI-926 significantly improved survival rates (p < 0.0001). All of the IPI-926-treated mice were alive and well after six weeks of treatment, after their placebo-treated siblings had succumbed to their disease.
IPI-926 leads to dramatic clinical improvement and significantly prolongs life in a mouse model of aggressive medulloblastoma. This work will be presented at the 2009 American Association for Cancer Research national meeting, and a manuscript is in preparation for publication.
So, consider for a moment the potential that this research bears, and the possiblity that it might be scaled back, delayed, or shut down for lack of funding. If you can, please take a moment to donate to Baldrick's to support more research like this.