Part I: “Project Haystack” at Children’s Cancer Therapy Development Institute

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Dr. Charles Keller, Scientific Director of the Children’s Cancer Therapy Development Institute, describes for us in opening the show, the ongoing work at the Institute funded by parent-led foundations in its quest to make all forms of childhood cancer survivable, specifically Project Haystack, an international collaboration in rhabdomyosarcoma research.

In partnership with 10 family communities around the world, the Children’s Cancer Therapy Development Institute is embarking on a drug discovery project for rhabdomyosarcoma through an academic-pharma partnership with a major Swiss pharmaceutical company; 640,000-compounds have been screened across a range of cells-of-origin and mutational profiles for primary tumor cell cultures taken from murine sarcomas.

  • Four of the most potent hits share near-identical chemical structures.
  • All compounds have cell growth inhibition activity against alveolar RMS (ARMS) and embryonal RMS (ERMS), only mixed activity against UPS, but no activity against normal fibroblasts.
  • One of these compound hits is an FDA-approved cardiovascular medicine with a favorable long-term side effect profile, and completely unstudied with respect to rhabdomyosarcoma.

Ian Marci joins us from the Fight Like Mason Foundation, and Laura Roberts with the Elaine Roberts Foundation to share their experience, involvement in the research, and inspiration to keep working for a cure.

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