A Cincinnati Children’s Hospital scientist, Nancy Ratner, PhD, has been recognized by NF Forward for her extensive contributions to neurofibromatosis (NF) research. The honor acknowledges her pivotal role in the development of selumetinib (KOSELUGO®), the first FDA-approved therapy for NF1 tumors.
Ratner, from the Division of Experimental Hematology and Cancer Biology, was celebrated at the beNeFit XII fundraiser in Detroit, hosted by the NF Forward foundation on November 23, 2024. NF Forward was established in 2017 by Dan and Jennifer Gilbert, following the loss of their son Nick to the disease.
Impact of NF1
Neurofibromatosis type 1 (NF1) is a genetic condition affecting approximately 3 million people worldwide. NF1 tumors, while typically non-cancerous, grow along nerve fibers and can cause pain, functional impairment, and life-threatening complications depending on their location.
Selumetinib Approval
In 2022, the U.S. Food and Drug Administration (FDA) approved selumetinib (KOSELUGO®) for pediatric patients with NF1 and symptomatic, inoperable plexiform neurofibromas. This approval marked a significant milestone, providing the first effective treatment option to directly address tumor size reduction in NF1. The approval was based on clinical trial data demonstrating the drug's ability to shrink NF1 tumors. Ratner's lab's research into MEK inhibitors was instrumental in demonstrating the potential of this class of agents for treating NF1.
NF Forward's Contribution
Since its inception, NF Forward has raised over $65 million to fund research initiatives and support individuals and families affected by NF. The foundation's commitment accelerates the development of new therapies and improves the quality of life for those living with NF.
