From preclinical studies, researchers have found that an investigational drug called ‘cabozantinib’ can treat tumors form a genetic disorder known as neurofibromatosis type 1 (NF1).
"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."
NF1 is a rare disease affecting approximately 1 in 3000 people globally and is characterized by a proliferation of tumors throughout the nervous system.
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"It's incredibly exciting that we now have two classes of drugs that result in tumor responses, given that we had no promising agents only a few years ago," Dr. Fisher said. "However, despite this excitement, neither cabozantinib nor MEK inhibitors shrink all tumors or make them go away completely. Therefore, we are building on these results as well as ongoing laboratory studies and are planning future exploration of combination therapies, so that we can further improve outcomes for these patients with these debilitating and life-threatening tumors."
Source: Science Daily