Researchers at Riley Children’s Health, led by Wade Clapp, MD, have decades of leadership in uncovering new treatments for neurofibromatosis (NF) type 1 and type 2.
In the past four years, published studies highlight the groundbreaking work of Riley NF researchers in the development of novel drug treatments cabozantinib and brigatinib. In another significant study, Riley physician-scientist Steven Rhodes, MD, PhD, and Dr. Clapp used genetically engineered mouse models to uncover the molecular origins of malignant peripheral nerve sheath tumor (MPNST), a rare and deadly form of cancer in people with NF1.
“What’s unique about Riley is that we’re not only doing the basic science research that leads to clinical trials in NF, we’re also leading the clinical trials,” said Brian D. Weiss, MD, who recently joined Riley Children’s as division chief of pediatric hematology/oncology/stem cell transplant. “Through a collaboration with adult neurologists at IU Health, we’re assembling a team with the potential to lead NF clinical trials in both children and adults, which will further strengthen our standing as a leader in this field.”
Dr. Weiss has directed multiple national clinical trials, particularly focusing on novel therapies for neuroblastoma and NF1-associated plexiform neurofibromas. He has served as the external advisory board chair for the Developmental and HyperActive Ras Treatment Spore grant, as well as the vice chair for the Neurofibromatosis Clinical Trials Consortium (NFCTC). He is currently the chair for the NFCTC Neurofibroma Committee.