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.
Fisher MJ, Shih CS, Rhodes SD, Armstrong AE, Wolters PL, Dombi E, Zhang C, Angus SP, Johnson GL, Packer RJ, Allen JC, Ullrich NJ, Goldman S, Gutmann DH, Plotkin SR, Rosser T, Robertson KA, Widemann BC, Smith AE, Bessler WK, He Y, Park SJ, Mund JA, Jiang L, Bijangi-Vishehsaraei K, Robinson CT, Cutter GR, Korf BR; Neurofibromatosis Clinical Trials Consortium, Blakeley JO, Clapp DW. Cabozantinib for neurofibromatosis type 1–related plexiform neurofibromas: a phase 2 trial. Nat Med. 2021 Jan;27(1):165-173. doi: 10.1038/s41591-020-01193-6. Epub 2021 Jan 13. PMID: 33442015 Clinical Trial.