Spring 2025
By Kathy Clute
Photography by John Deputy
Gifts to The Dana-Farber Campaign accelerated efforts in our laboratories to find new measures to prevent and treat cancer, lessen the side effects of therapies, and stop every childhood cancer in its tracks. Dana-Farber research led to U.S. Food and Drug Administration approval in 2024 of a new medicine for children with the most common form of pediatric brain cancer.
The oral medication, tovorafenib, is for young patients with relapsed or progressive pediatric low-grade glioma, which is diagnosed in about 3,000 children in the U.S. each year. Standard treatments often leave these patients with lifelong neurological, hormonal, visual, or other problems, and newer treatments have been urgently needed to help those children survive with fewer long-term side effects.
“This is an example of what Dana-Farber is capable of,” said Karen Wright, MD. “An approval for a pediatric tumor doesn’t happen very often.”
The FDA action came after researchers in Dana-Farber’s Department of Cancer Biology spent more than a decade researching genetic mutations in gliomas. After their preclinical work proved that tovorafenib penetrated the blood-brain barrier and shut down a protein that was causing the cancer cells to grow unchecked, the FDA declared it a “breakthrough therapy” in 2020. This enabled clinical trials that led to the full approval four years later.