Readers of this space know of the investment the Children’s Tumor Foundation made in the NF Preclinical Consortium, which demonstrated that MEK inhibitors have significant impact on tumor size in animal models. That work was instrumental in the development of a clinical trial led by Dr. Brigitte Widemann, who announced at the Foundation’s 2015 NF Conference that over half of the patients in the trial were seeing significant reduction in tumor size of their inoperable plexiform neurofibromas.
Since that time, the trial has entered its registration phase (the last step before submission to the FDA for approval), with continued positive results. One of those tremendous MEK success stories is of Philip Moss, whose tumor has shrunk to nearly half in size, and who you can read more about here: ctf.org/believe.
Today marks another significant step on the road to effective approved treatments for NF, with publication of the MEK trial success in the prestigious New England Journal of Medicine. Over 70% of trial participants have seen tumor reduction of at least 20% in size, a first in NF research.
To read the paper abstract at the New England Journal of Medicine: http://www.nejm.org/doi/full/10.1056/NEJMoa1605943
To read the National Institutes of Health press release: https://www.cancer.gov/news-events/press-releases/2016/selumetinib-shrinks-pediatric-tumors