Children with neurofibromatosis type 1 (NF1) are at a greater risk of developing tumors of the nervous system including plexiform neurofibromas. Plexiform neurofibromas are benign, nerve sheath tumors that usually grow most rapidly in early childhood. These tumors can cause pain, affect normal organ function, and represent one of the most debilitating complications of NF1.
At present, there are no standard treatment options for plexiform neurofibromas except surgery. Surgery, however, is often difficult because the tumors frequently infiltrate and surround vital structures and organs.
Michael J. Fisher, MD, and researchers at the Center for Childhood Cancer Research are part of a consortium of hospitals conducting clinical trials to evaluate targeted cancer therapies as treatments for children with inoperable plexiform neurofibromas.
Results from a Phase I clinical trial demonstrated that selumetinib (a MEK enzyme inhibitor of the RAS/RAF/MEK/ERK cancer pathway) effectively shrank tumors in two-thirds of the children who participated in the study. Phase II clinical trials are underway to further evaluate selumetinib as a treatment for plexiform neurofibromas.
Additional clinical studies are being conducted at the Center for Childhood Cancer Research to evaluate other targeted and traditional cancer therapies, alone or in combination, as possible treatments for children with NF1-related plexiform neurofibromas.