Part I: "Project Haystack" at Children's Cancer Therapy Development Institute

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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About Janet


Blessed with varied interests and an artistic and musical upbringing, Janet had health challenges throughout her young adult life. Despite these she graduated Cum Laude from Wellesley College with an award of distinction for acting, and had also been a champion equestrian. She began a family with her husband Barry later in life, and had finally found happiness with daughter Sophie-Marie (3/12/06) and then baby (Jack 8/30/08). Five weeks after his birth, the family escaped a wildfire in which all worldly possessions were lost. The family relocated in December of 2008 to Agua Dulce CA where they currently reside.

Jack began to have unsettling symptoms at the age of 3; he was taken to Children's Hospital Los Angeles and was diagnosed with DIPG, or diffuse intrinsic pontine glioma, on Friday Oct. 28, 2011, indisputably the darkest experience of Janet's life. The outrage of it made her determined to find the good in the situation, and she asked God to "Put me to work!" After Jack's death, she remained determined to start working to find solutions to DIPG and incorporated Jack's Angels at the end of 2012; the Foundation began its work in 2013. Despite the fact that DIPG is responsible for the majority of brain tumor deaths in children, she had been told there were no solutions for Jack because "the numbers aren't great enough for investors." This remains the primary motivation in her advocacy work, to prioritize children's lives in our medical system in the United States.