News Story

New Drug Shows Promise for NF1 Plexiform Neurofibromas

Weiss News
Plexiform neurofibromas are common benign nerve tumors seen in children and adults with neurofibromatosis type 1 (NF1). While these tumors are typically slow growing, some may require treatment.
In a study spearheaded by Dr. Brian Weiss at Cincinnati Children’s Hospital and conducted as part of the Department of Defense-funded NF Clinical Trials Consortium, Sirolimus was evaluated as a potential chemotherapy agent for plexiform neurofibromas. Sirolimus is a drug similar to rapamycin, a compound first shown to be effective for reducing the growth of mouse Nf1 optic gliomas in the laboratory of Dr. David Gutmann of the Washington University NF Center.
Dr. Weiss and colleagues found that Sirolimus decreased the time it took for a tumor to growth (“time to progression”) by almost four months. While this result is modest, it does suggest that some plexiform neurofibromas may respond favorably to this treatment.
Future work using related drugs as well as other classes of chemotherapy agents are currently being evaluated.
Weiss B, Widemann BC, Wolters P, Dombi E, Vinks A, Cantor A, Perentesis J, Schorry E, Ullrich N, Gutmann DH, Tonsgard J, Viskochil D, Korf B, Packer RJ, Fisher MJ. Sirolimus for progressive neurofibromatosis type 1-associated plexiform neurofibromas: a Neurofibromatosis Clinical Trials Consortium phase II study. Neuro Oncol. nou235, 2014, Epub ahead of print.