Selumetinib Continues to Show Promise in Children with NF1

Author: National Cancer Institute
Published On: 06/08/2018

An investigational drug called selumetinib can shrink tumors in children and young adults with a genetic syndrome called neurofibromatosis type 1 (NF1) and may improve symptoms such as pain and reduced mobility that result from tumors called plexiform neurofibromas, which develop in many people with NF1, according to preliminary results from a clinical trial. 

The phase 2 trial, which involves 50 patients aged 2 to 18 with NF1 and plexiform neurofibromas, confirms the results of a smaller trial that in 2016demonstrated for the first time that the drug could shrink large tumors.

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