Pilot Study Suggests Selumetinib's Potential for Cutaneous Neurofibromas

A new nonrandomized pilot trial suggested the MEK inhibitor selumetinib was associated with a reduction in the size of cutaneous neurofibromas in patients with Neurofibromatosis Type 1 (NF1). 

These study, published as a brief report in JAMA Dermatology, included 11 adult patients with NF1 who received selumetinib over a set treatment period. The primary endpoint was the percentage reduction in the size of cutaneous neurofibromas from baseline to the end of treatment. Secondary endpoints included assessments of safety, tolerability, and symptom improvement.

There was a significant reduction (median, -28.5%) in tumor size among participants, and most patients tolerated the treatment well. Reported adverse events were consistent with the known safety profile of MEK inhibitors, which include gastrointestinal symptoms, rash, and fatigue. The median number of treatment cycles was 9 treatments, with only 4 patients completing the full 24 treatment cycles. The most common reported adverse events included dry skin and rash. 

The authors emphasized the need for larger randomized controlled trials to confirm the efficacy and safety of selumetinib in this patient population. 

"In this nonrandomized pilot trial, there was likely some shrinkage of cNF in response to treatment with systemic selumetinib, but it is not clear if the degree of shrinkage is clinically meaningful relative to the drug-related adverse events experienced by most participants, particularly the high frequency of cutaneous adverse events," they concluded.

Source: Gross A, et al. JAMA Dermatology. 2025. Doi:10.1001/jamadermatol.2024.6574