A new treatment for kidney cancers related to von Hippel-Lindau disease

2021

Challenge

Individuals with von Hippel-Lindau (VHL) disease develop many cancerous and non-cancerous tumors in the kidneys and other organs. Previously, there were no treatment options for VHL disease, and patients required repeated surgeries to remove the tumors. However, with each successive surgery, the risk of complications increases.

Advance

Building on decades of research to understand the genetics and biology of VHL disease, IRP researchers led by W. Marston Linehan, M.D., and Ramaprasad Srinivasan, M.D., Ph.D., conducted a clinical trial that showed significant reduction in the size of tumors in the kidneys and other organs when patients were treated with belzutifan, small-molecule drug developed by researchers at the University of Texas Southwestern Medical Center. Belzutifan works by blocking the activation of proteins called hypoxia-inducible factors, or HIFs, which accumulate in large numbers in the cells of patients with VHL disease and promote tumor growth.

Impact

Based on the results of this trial, belzutifan — now sold under the name Welireg^. — was approved by the U.S. Food and Drug Administration in 2021 as a treatment for VHL patients with renal cell carcinoma, pancreatic tumors, or central nervous system hemangioblastomas. The drug provides the first alternative to surgery for these patients, thereby avoiding the need for repeated surgeries to treat these patients and the risk of associated complications.

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