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Charles H. Hood Foundation | Yana Pikman, M.D. – January 2021
By identifying innovative pediatric advancements and providing funding in the critical phases of development, we are able to expedite high-impact breakthroughs that improve the health and lives of millions.
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Yana Pikman, M.D.

Assistant Professor of Pediatrics

Dana-Farber Cancer Institute

Precision Medicine for Pediatric Acute Leukemia


Key Words: Leukemia, Targeted therapy, Precision medicine

While deciphering the DNA code in cancer cells has become commonplace in the research laboratory, the integration of these technologies into the clinical care and treatment of children with leukemia has been lagging. We have conducted the first, multi-institutional national genomics-based precision medicine trial for children with relapsed/refractory/very high-risk leukemias to show that this approach can inform the diagnosis and treatment of these patients. Now, we will deploy these technologies to study the genomics of both newly diagnosed and relapsed/refractory pediatric patients across Northeast pediatric centers to study the feasibility and efficacy of this approach in a regional setting.  This study will also establish a multi-disciplinary group of leukemia experts, who will review the genomic data, discuss potential treatment implications and availability of a relevant targeted drug for children. Results from this project will define the types and frequency of specific mutations in children with newly diagnosed and relapsed/refractory leukemia in the Northeast region, and assess the impact of the genomic assessment on their clinical care. It will also assess the feasibility of this approach in a regional setting. Some leukemia may not have clear genomic changes that lead to sensitivity to a known targeted drug. We have tested the sensitivity of 200 pediatric leukemia patient samples using a large panel of drugs and will study if certain new genomic features of the leukemia can predict response to a particular compound. We will use data generated in this proposal to inform future targeted therapy trials for children with leukemia with the long-term goal of precise, safe, and more efficacious therapies for children with these disease. Development of novel, targeted therapies to treat pediatric patients with leukemia is essential to continue to improve survival and decrease toxicities of treatment.