Dr. Pikman on Implications of a Matched Targeted Therapy Approach in Pediatric Leukemia

Yana Pikman, MD, a physician in the Cancer and Blood Disorders Center at Dana-Farber Cancer Institute and instructor of pediatrics at Harvard Medical School, discusses the implications of a matched targeted therapy approach in pediatric leukemia.

At the 2018 ASH Annual Meeting, preliminary data were presented on a study in which investigators sequenced samples of pediatric patients’ leukemia in an attempt to identify targetable mutations. If a target was found, investigators tried to pair it with a corresponding therapy. Pending further results, this may inform the integration of novel therapies into clinical practice for patients with pediatric leukemia, explains Pikman. This may fill a large unmet need, as new drugs are tested in adults before they are ever brought to pediatric patients, she adds.

Based on the initial results of the study, pediatric leukemia behaves differently than adult leukemia, says Pikman. As such, although novel drugs that are proven to be ineffective in adult patients are halted for further development, they may be effective in pediatric patients. Putting a genomic characterization of pediatric leukemia in place will help inform which drugs are needed as well as shape how these drugs are tested in trials for pediatric patients.