In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1

Wednesday, March 18, 2020

Findings from a phase 2 clinical trial show that the drug selumetinib improves outcomes for children with the genetic disorder neurofibromatosis type 1 (NF1). In the trial, selumetinib shrank the inoperable tumors that develop with NF1 called plexiform neurofibromas, and children experienced reduced pain, improved function, and better overall quality of life after receiving the treatment.

The trial was led by intramural researchers in the Center for Cancer Research (CCR) at the National Cancer Institute (NCI), part of the National Institutes of Health. Results of the trial were published March 18, 2020, in the New England Journal of Medicine.

“Until now, no effective medical therapies have existed for children with NF1 and plexiform neurofibromas, and it’s been a long journey to find a drug that can help them,” said Brigitte Widemann, M.D., lead author of the study, and chief of CCR’s Pediatric Oncology Branch, which developed and coordinated the trial. “While this is not yet a cure, this treatment is shrinking tumors and it’s making children feel better and have a better quality of life.”

Eva Dombi, M.D., Trish Whitcomb, R.N., Brigitte Widemann, M.D., Andrea Gross, M.D., and Andrea Baldwin, C.R.N.P., of the Pediatric Oncology Branch at NCI.