Additional exciting advances include the results of a new drug (pazopanib) to treat an inherited cancer condition called von Hippel-Lindau Disease (VHL), in which patients are predisposed to kidney cancers, pancreatic tumors, and hemangioblastomas (i.e., tumors involving the blood vessels). Study results showed that among 31 patients with VHL, overall response rate with the drug was 42%, with responses of 52% for renal cell carcinomas, 53% for pancreatic lesions, and 4% for CNS hemangioblastomas. Results are encouraging, and this may be a treatment option for individuals with VHL and growing or unresectable lesions, although safety and activity for these indications warrants further study. These advances serve to highlight some of the recent breakthroughs in the treatment of tumors among those with inherited cancer predisposition.
Jonasch E, et al. Lancet Oncol. 2018 Sept 17. doi 10.1016/S1470-2045(18)30487-X.