The MEK inhibitor selumetinib may reduce spinal neurofibroma burden in patients with NF1 and plexiform neurofibromas

Author: Sadhana Jackson, Eva H Baker, et al.

Published On: 10/26/2020

Neurofibromatosis Type 1 patients are affected by neurofibromas in a number of ways. Researchers sought to explore the treatment of spinal neurofibromas. Spinal neurofibromas (SNFs) in patients with NF1 can lead to progressive neurological deficits and may require multiple surgeries to relieve spinal cord compression. MEK inhibitors have shown recent success in the NF community, resulting in a sustained decrease of plexiform neurofibroma volumes in the majority of patients treated.

In this study, researchers demonstrate that selumetinib also reduces SNF burden and its effect on the spinal cord. "We propose that MEK inhibitors may have the potential to prevent cord compression and in select patients may obviate the need for surgery. Prospective standardized evaluations of selumetinib and its effect on SNF are needed to further define its role in the treatment of SNF." 

Read the full published article here.