Nancy Ratner Honored for Contributions to Neurofibromatosis Type 1 (NF1) Research
Key Insights
Nancy Ratner, PhD, was honored by NF Forward (search) for her career contributions to neurofibromatosis (search) (NF) research, particularly NF1 (search).
Ratner's early work was crucial in the development of selumetinib (KOSELUGO®), the first FDA-approved treatment for NF1 (search) tumors in children.
The FDA approved selumetinib in 2022 based on research from Ratner's lab exploring MEK inhibitors (search)' potential to shrink tumors.
A Cincinnati Children’s Hospital scientist, Nancy Ratner, PhD, has been recognized by NF Forward (search) for her extensive contributions to neurofibromatosis (search) (NF) research. The honor acknowledges her pivotal role in the development of selumetinib (KOSELUGO®), the first FDA-approved therapy for NF1 (search) 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 (search) 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 (search) type 1 (NF1 (search)) 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 (search) 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 (search) was instrumental in demonstrating the potential of this class of agents for treating NF1.
NF Forward's Contribution
Since its inception, NF Forward (search) 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.