Clinical Trial Success Leads to FDA Approval for Selumetinib for Children with Inoperable Plexiform Neurofibromas
Published March 2020 | The New England Journal of Medicine
Children who develop NF1-related plexiform neurofibromas can experience disfigurement, weakness, loss of mobility and serious pain as these non-cancerous tumors grow. In some anatomical locations, these tumors can become life-threatening.
Now, years of work at Cincinnati Children’s, the National Cancer Institute and other medical centers to develop a medication to treat this rare disease have finally paid off.
The drug selumetinib was approved on April 10, 2020, by the US Food and Drug Administration for use in this population shortly after phase 2 clinical trial results were reported in March in The New England Journal of Medicine.
Brian Weiss, MD, director of the Neuroblastoma Program for the Cancer and Blood Diseases Institute at Cincinnati Children’s, co-authored the NCI-led study.
The researchers tracked 50 children who received two daily doses of selumetinib. Overall, 37 of the 50 children (74 percent) showed at least a partial response in tumor volume reduction, with 28 children (56 percent) showing a durable response. In addition, children reported less pain and improved quality of life.
“This drug is not a cure,” Weiss says. “However, the tumor volume reduction and associated pain relief have provided the first effective medical therapy for children with NF1. It was very exciting to be involved with this project.”
Clinical studies had been in the works since 2016, when the potential benefit of the drug was revealed through mouse-based studies led by Nancy Ratner, PhD, co-director of the Rasopathy Program at Cincinnati Children’s.
