A recent study spearheaded by Dr. Michael Fisher at Children’s Hospital of Philadelphia and Dr. D. Wade Clapp at Indiana University found that cabozantinib as an effective therapy for NF1-associated plexiform neurofibromas.
Using a preclinical mouse model of plexiform neurofibromas, this team showed cabozantinib was effective at reducing mouse tumors. Leveraging these exciting findings, they launched a Phase II study in patients older than 16 years of age with progressive or symptomatic plexiform neurofibromas. They found that no patient had continued tumor growth on cabozantinib, and many reported reduced pain symptoms.
This landmark study underscores the importance of preclinical models to the identification of promising therapies for patients living with NF1 tumors.
The report was recently published in Nature Medicine. PMID: 33442015