Funding was first jumpstarted by an anonymous donor
Recently, a teen developed an alveolar rhabdomyosarcoma (aRMS) with the typical Pax3:Foxo1 mutation found in this type of childhood cancer. And yet there was another gene variation found… a second mutation. maybe a cancer-causing mutation, maybe a bystander. A key question is whether this mutation makes the cancer unique, or common?
Where does this child’s situation fit in with most other cases? If this combination of mutations is unique, then are there other single-patient exceptional cases? or instead, do manageable subtypes exist across the continuum of alveolar rhabdomyosarcoma cases – subgroups for whom customized treatments can be assigned?
Can we define a new standard of care for every subgroup of children battling aRMS? This project is important because many children, teens and young adults with alveolar rhabdomyosarcoma need new treatment options. When metastatic, the five-year survival rate is only 8%. We want aRMS to be universally survivable — with the least side effects and highest quality of life after treatment. Children, teens and young adults with alveolar rhabdomyosarcoma will benefit from new drug treatments, matched to a child’s tumor’s genetics, being introduced into clinical trials. Extend the reach of this project to more and more children’s subtypes through crowdfunding contributions here.