Already funded by the Megan’s Mission Foundation
Rhabdomyosarcoma and soft tissue sarcomas are among the five greatest causes of death from childhood cancer and are among the most unaddressed causes of childhood cancer morbidity. Despite 49 years of cooperative group trials of intensified chemotherapy, the dismal survival for advanced disease remains unchanged. The goal of this project is to bring consumer (patient)-level participation and accountability to a new, strategic approach to developing, testing and prioritizing new rhabdomyosarcoma drugs for incorporation into cooperative group clinical trials. Unfortunately, many RMS patients do not have the luxury of time. Therefore, all studies will be focused on testing existing FDA-approved or already clinically-investigated drugs with the goal of repurposing them for quick transition to treat RMS. To further expedite this research project, this fundraising goal includes an allocation to support the employment of a full-time researcher dedicated to this project.
A major difference in this project from other grant -funded research is that patients will be embedded in our research team, and presentations of our progress will be given to these science-empowered patients and family members weekly. Want to be a citizen-scientist, too? Email email@example.com for more information.