Dr. Mark W. Kieran on Dabrafenib in Pediatric Patients with BRAF V600-Mutant Disease

Mark W. Kieran, MD, PhD, director, pediatric medical neuro-oncology, institute physician associate professor of pediatrics, Harvard Medical School, discusses the first study of dabrafenib in pediatric patients with BRAF V600—mutant disease.

In the trial, dabrafenib was investigated in all pediatric patients with BRAF V600—mutations in both the body and the brain.

The study identified that the appropriate dose in children was similar to that given in adults, says Kieran. It then broke patients into cohorts of each different disease and treated them with dabrafenib. The goal of the study was to determine the exact dose, toxicities, and efficacy in each pediatric tumor type with the BRAF V600 mutation.

The results of the study were exciting, says Kieran. The drug proved to be very well tolerated, with less toxicities seen in kids than were previously seen in other trials with adults. Squamous-cell carcinoma can be a side effect of the drug for adults that have had a lot of sun exposure. Kids, who haven’t had as much lifetime sun exposure, did not experience this toxicity as much, says Kieran.

The drug also significantly reduced either the size or growth of tumors in kids with BRAF V600 mutation. This offers a lot of potential, especially in the most common type of pediatric brain tumor low-grade gliomas. In this subgroup over 80% of tumors disappeared, shrunk, or stopped growing completely.