Based on preclinical studies of an investigational drug to treat peripheral nerve tumors, researchers at Children’s Hospital of Philadelphia (CHOP) as part of the Neurofibromatosis Clinical Trials Consortium have shown that the drug, cabozantinib, reduces tumor volume and pain in patients with the genetic disorder neurofibromatosis type 1 (NF1). The results of the Phase 2 clinical trial, co-chaired by Michael J. Fisher, MD at CHOP, were published recently in Nature Medicine.
“This is the second class of drugs to demonstrate a very promising response rate for NF1 patients with these tumors,” said first author Fisher, Chief of the Section of Neuro-Oncology and Director of the Neurofibromatosis Program at CHOP, and Group Chair for the NF Clinical Trials Consortium, which includes 25 sites developing innovative biologically-based clinical trials for complications of NF. “Collectively, the data presented in this study illustrate a true bench-to-bedside approach, coordinating translational and clinical efforts to advance targeted therapies for a rare disease like NF1.”
Click here to read full article https://www.technologynetworks.com/neuroscience/news/drug-may-reduce-tumor-volume-in-neurofibromatosis-344622