Individuals with NF1 are at increased risk for developing tumors known as plexiform neurofibromas. These tumors are among the most debilitating features of NF1; however, there are currently no standard treatment options for reducing the size of these tumors other than surgery.
Brian Weiss, MD, of Cincinnati Children’s Hospital and his collaborators, including David H. Gutmann, MD PhD, found that a new treatment called sirolimus, while effective in keeping tumor size stable, does not cause reduction of tumor size. These findings indicate the continued need for NF1 clinical trials focused on finding effective treatments for the reduction of plexiform neurofibromas.
Studies are currently under way at Washington University to evaluate additional treatment strategies for these tumors as part of the Department of Defense NF Clinical Trials Consortium.
Weiss B, Widemann BC, Wolters P, Dombi E, Vinks AA, Cantor A, Korf B, Perentesis J, Gutmann DH, Schorry E, Packer R, Fisher MJ. Sirolimus for non-progressive NF1-associated plexiform neurofibromas: an NF clinical trials consortium phase II study. Pediatr Blood Cancer. 61: 982-6, 2014.