FDA Approves First Therapy for Children with NF1

On April 10, 2020, the US Food and Drug Administration (FDA) approved the drug selumentinib (Koselugo) for people with Neurofibromatosis Type 1 (NF1) who are 2 years of age and older. The first of its kind drug will be used to treat symptomatic, inoperable plexiform neurofibromas. These tumors are often detected in young children, and may involve the eye socket, face, arm, leg, back, chest, or abdomen.

The FDA approval came after a clinical trial conducted by the National Cancer Institute showed that nearly two thirds of patients had a positive response to the drug, with at least a 20% reduction in tumor volume. Of these patients, most had responses that lasted 12 months or longer. However, no patients experienced complete disappearance of their tumor.

“These are exciting times for families affected with NF1”, says NF Center Director, David H. Gutmann, MD, PhD. “This success is the result of decades of hard work by scientists, physicians, nurses, and patient advocacy groups.”

Give STL Day for NF is May 7th


On Thursday, May 7, 2020, the St. Louis Community Foundation will host its annual Give STL Day, a 24-hour online giving event designed to grow philanthropy in the St. Louis metropolitan area. The goal is to inspire the community to come together for 24 hours to contribute as many charitable dollars as possible to support the work of local nonprofit organizations. This is exceptionally important during the current COVID-19 pandemic.

The early donation period has already begun.  Please click here to help support the Washington University NF Center in our mission to provide exceptional care through groundbreaking research.