Preventing Relapse After Bone Marrow Transplant in Pediatric Acute Lymphoblastic Leukemia (ALL) with a Personalized Treatment

Principal Investigator: Dr. Alix Seif

Disease: Acute Lymphoblastic Leukemia

Research Description: Pediatric acute lymphoblastic leukemia (ALL) affects approximately 3,000 children per year, and while rare, it is the sixth most common pediatric cancer. Children with ALL who need bone marrow transplants have a high risk of relapse after transplant, and these relapses are usually incurable. New ways to prevent relapse after transplant are urgently needed. Highly sensitive tests that use a fast method of identifying genetic fingerprints called “next generation sequencing” (NGS) can find very low levels of leukemia cells called “measurable residual disease” (MRD), and NGS MRD testing can find 1 leukemia cell in a million blood cells. The team will use NGS MRD testing after transplant to find those children with the highest chances of relapse. They will then match patients to personalized repurposed immune treatments based on features of each child’s leukemia. The treatments include daratumumab (approved for multiple myeloma) and blinatumomab (approved to treat already relapsed ALL). If this highly sensitive testing is successfully in providing pediatric ALL patients with personalized, repurposed treatment to prevent relapse in this pilot study, the results can be used as a basis for larger, follow-on studies.

Funding Partners: Multiple anonymous funders

CWR funding role: Primary funder