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NF1 Clinical Trial of Selumetinib “Most Exciting” Says National Cancer Institute Director

By August 16, 2018February 27th, 2024NF1, Science & Research

Speaking before the House Energy and Commerce Committee of the U.S. House of Representatives on the implementation of the 21st Century Cures Act, Norman (Ned) Sharpless, M.D., Director of the National Cancer Institute (NCI) was asked by Missouri Congressman Billy Long, “Can you tell us about the most exciting thing that is being supported in the Cancer Moonshot?”

Sharpless focused his answer on the NCI’s Rare Tumor Initiative and the clinical trial presented in June at the ASCO Conference by Andrea Gross, MD of the NCI about the selumetinib (MEK inhibitor) clinical trial treating inoperable NF1 plexiform neurofibromas.

At the time, Dr. Sharpless had tweeted “tumors shrink, kids feel better, drug seems safe,” which he repeated at the Congressional briefing, additionally noting that several parents of children in the clinical trial had contacted him to share “before and after” photos showing dramatic reductions in tumor size.

One of those parents whose message was highlighted at this briefing was from Renie Moss, a member of the Board of Directors of the Children’s Tumor Foundation, and Chair of the Foundation’s Volunteer Leadership Council. Her son Philip has seen a 53% reduction in the size of his tumors while participating in this trial, and Dr. Sharpless showed the before and after photos of Philip to the congressional committee, noting that “his windpipe, his airway is not being compressed by the tumor anymore, and he has not the social stress of going to school with a big lesion like that.”

This clinical success is the result of an investment by the Children’s Tumor Foundation in the NF Preclinical Consortium, which showed the potential for clinical benefit in NF patients. That work was instrumental in the development of this selumetinib 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 reductions in tumor size of their inoperable plexiform neurofibromas.

Since that time, the trial has entered registration phase (the last step before submission to the FDA for approval), with continued positive results. Over 70% of trial participants have seen tumor reductions of at least 20% in size, a first in NF research. This study has also been reported in the prestigious New England Journal of Medicine, and selumetinib has received Orphan Drug Status from both the Food and Drug Administration (FDA) in the United States and the European Medicines Agency

Watch the Q&A with NCI Director Ned Sharpless here.

Read about the impact this trial is having on our NF Heroes, including the incredible before and after photos here.

Watch the Believe We Can End NF Videos of NF Hero Philip and NF Hero Jane, both of whom are participating in the clinical trial with tremendous results:

And read more about the Children’s Tumor Foundation strategy which is leading to these kinds of results for NF patients, here.

Additional Reading:

About 35% of people with NF1 in the NF Registry report having plexiform neurofibromas, which grow in and around nerves. Untreated, plexiforms can cause serious health problems by pressing against nearby nerves and organs. Surgical removal of plexiforms is sometimes done, but may not be feasible due to possible nerve damage and excessive bleeding. The tumors are difficult to remove completely and often re-grow.

The clinical trial Sharpless highlighted involved children ages 2-18 with inoperable plexiforms that were affecting their health.

So far, 72% of these patients responded to selumetinib with a reduction of at least 20% in tumor volume. The greatest reduction in tumor size reported so far was 53%. As an added benefit, the treatment reduced the need for pain medication while increasing muscle strength and range of motion. (Gross AM, et al., Neuro-Oncology, Vol. 20, Suppl. 2, June 2018).

These interim results are the best reported so far for any drug treatment for plexiforms.

Watch the Q&A with NCI Director Ned Sharpless here.