Rare Disease Report

Selumetinib for the Treatment of Neurofibromatosis Type 1

JANUARY 03, 2017
Andrew Black
Results from a Phase 1 study of selumetinib for the treatment of patients with neurofibromatosis and inoperable plexiform neurofibromas look encouraging. The results were published in the New England Journal of Medicine by Dombi and colleagues.
24 patients, ages 18 years old and younger who had a median tumor volume of 1205 ml were enrolled in the Phase 1 study. Patients were administered selumetinib every 12 hours at a dose of 20 to 30 mg per square meter of body-surface area on a continuous dosing schedule. The median number of cycles was 30.
The results of the trial showed a tumor volume decreases from baseline of ≥20% in 70% of the patients receiving selumetinib. 
A tumor volume decrease in this early-phase trial data shows some encouragement for a potential treatment down the line. 

About selumetinib

Selumetinib (AZD6244 or ARRY-142886) is an oral, highly selective MEK 1/2 inhibitor. MEK 1/2 are critical components of the RAS-ERK pathway, activation of which is implicated in driving cancer growth.
Earlier this year, the drug failed to show a significant effect in patients with either pancreatic or lung cancer. The drug is being developed by AstraZeneca.

About Neurofibromatosis Type 1

Neurofibromatosis type 1 (NF1), also called von Recklinghausen's disease, is a rare genetic disorder characterized by the development of multiple noncancerous (benign) tumors of nerves and skin (neurofibromas) and areas of abnormally decreased or increased coloration (hypo- or hyperpigmentation) of the skin.


Dombi E, Baldwin A, Marcus L, et al. Activity of Selumetinib in Neurofibromatosis Type 1–Related Plexiform Neurofibromas. New Engl J Med. 2016; 375:2550-2560. DOI: 10.1056/NEJMoa1605943

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