FDA Approves First Combination Targeted Therapy for Most Common Pediatric Brain Cancer

The U.S. Food and Drug Administration has approved Novartis’ Tafinlar and Mekinist for the treatment of children with low-grade glioma (LGG) with a BRAF V600E mutation, offering new hope to patient families.

LGGs are the most common pediatric brain cancer, and children with the BRAF V600 mutation typically experience poor survival outcomes and a less favorable response to chemotherapy, the current standard of care. With the FDA’s approval, Tafinlar + Mekinist is the first targeted therapy since chemotherapy that is approved as a first-line option for pediatric patients with BRAF V600E LGG.

As a first-line treatment, children with BRAF V600E LGG will now be given the opportunity to take Tafinlar + Mekinist without having to first go through other treatments like chemotherapy and its toxic side effects. The FDA’s additional approval of the treatment’s liquid version means patients as young as one year of age will also be able to take it without having to swallow a pill. More information about the combination therapy approval can be found in Novartis’ announcement on their website.

“Pediatric cancer research is vital to uncover new treatment methods for a population,” says Dr. Eric Bouffet, the principal investigator of the clinical trial the FDA’s approval is based on and Associate Scientist Emeritus at The Hospital for Sick Children (SickKids). “Developing targeted therapies based on the unique genetic features of a patient’s tumor is the future of pediatric cancer care.”

This future where children can enjoy every minute of their life uninterrupted by brain cancer is possible. But developing targeted therapies takes time that pediatric brain tumor patients do not have — a process made even longer and more difficult because children with cancer don’t receive the same level of focus as adult patients from pharmaceutical companies, policymakers, and the public.

The Pediatric Brain Tumor Foundation’s research funding and legislative efforts are leading the way to accelerate the development and approval of new treatments. Our funding of MEK-related studies from basic science through clinical trials has contributed to the discoveries of Tafinlar + Mekinist and other MEK-targeting treatments for children with low-grade glioma. And our continued investments in research, resources, and advocacy are resulting in new discoveries for all pediatric brain tumor patients. Every dollar the Pediatric Brain Tumor Foundation invests in research attracts another 12 dollars in follow-on funding.

This impact is possible through the support of our donors, fundraisers, and volunteers – people like you. Don’t wait to give children with brain tumors the future they deserve. Donate and make a difference today.

If your family has questions or needs help navigating life after a child’s brain tumor diagnosis, the Pediatric Brain Tumor Foundation is here for you. Visit curethekids.org/family-resources to explore our resources and email [email protected] to connect directly with our Family Support team.