By Rachele Rosati, MS | Lead Scientist and Team Member
December 15, 2019
Dear Project Neuroblastoma Supporters,
Our research to identify individually tailored treatments for children battling Neuroblastoma (NB) utilizing patient-derived tumor organoids has advanced tremendously in the last few months. Neuroblastoma is the second most common solid tumor in the pediatric population, where standard of care therapies often yield debilitating side effects and poor outcomes. With up to 40% of high-risk pediatric neuroblastoma patients succumbing to the disease, the time to act is now. Using leading edge laboratory technologies, we will identify personalized, targeted and less toxic treatments for pediatric cancers.
Earlier this spring, we met with our collaborator Dr. John Maris at Children’s Hospital in Philadelphia to evaluate three NB organoid models, each derived from a different patient with unique DNA alterations. We have been at work amplifying the neuroblastoma organoids in amounts sufficient for high-throughput drug combination testing and screen result analysis. Each model was exposed to hundreds of different drug combinations to find the best treatment for each patient.
Now that we have found the best drug combinations for each cancer model (see Figure attached), we will proceed to test them in avatar mice carrying neuroblastoma. This research will establish if these novel combinations will be effective to block tumor growth and assess their safety in a living organism. We are excited and hopeful because our research found potentially effective drugs for tumor samples that otherwise were resistant to standard of care therapies. Further research will evaluate these therapies for use in clinic for pediatric cancer care.
Our exciting progress in fighting this disease would not have been possible without the generous contributions of our donors. We have raised over $22,697 to date, yet it will take up to an additional $67,303 to complete this project. We appreciate your consideration in contributing toward our efforts to improve overall outcomes and quality of life for children battling high-risk neuroblastoma. Your enduring support will allow us to continue the fight against this tragic disease.
With thanks and warm wishes,
Rachele Rosati, MS, Lead scientist and Cure First Team
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