Nancy Ratner, Phd.

Nancy Ratner, PhD

A team led by cancer biologist Nancy Ratner, PhD, reported Dec. 29, 2016, in The New England Journal of Medicine that selumenitib shrinks tumors in children with plexiform neurofibromas caused by the genetic disorder neurofibromatosis type 1 (NF1).

The phase 1 clinical trial of 24 children showed that the patients tolerated the drug well and, in most cases, their tumors shrank. The trial was led by Brigitte Widemann, MD, of the National Cancer Institute’s (NCI) Pediatric Oncology Branch. Ratner was study co-investigator.

Ratner and her team in our Cancer and Blood Diseases Institute have studied NF1 for decades. They first reported in 2013 that blocking MEK—a critical protein in the NF1 molecular process—was effective at shrinking plexiform neurofibromas in mice. Selumetinib is an MEK inhibitor.

“Having a mouse model that predicts treatment response in humans is rare,” says Ratner, “and may identify even more effective therapies.”