MK-6482 as a potential treatment for von Hippel-Lindau disease-associated clear cell renal cell carcinoma.

ABSTRACT:

Introduction

Von Hippel-Lindau (VHL) disease is an inherited autosomal dominant syndrome caused by a germline mutation and/or deletion of the VHL gene. Inappropriate hypoxia-inducible factor (HIF)-mediated transcription of proangiogenic and metabolic genes leads to the development of tumors and cysts in multiple organs. Surgery is a standard treatment for localized tumors with a risk of metastasis or organ dysfunction. Repeated surgeries cause substantial morbidity and have a major impact on quality of life. There is an urgent need to develop effective and safe systemic treatments for VHL disease manifestations. The small-molecule HIF 2 alpha inhibitor MK-6482 (belzutifan) has demonstrated significant efficacy in VHL disease related renal cell carcinomas, hemangioblastomas,

SUBMITTER: Hasanov E 

PROVIDER: S-EPMC8268068 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature