New clinical trial studies therapy for Von Hippel-Lindau disease-associated kidney cancer

patient chatting with his doctor

A new phase II study of PT2385 in patients with Von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC) is now recruiting patients at the NIH Clinical Center. VHL is a hereditary disease that can cause tumors to develop in many different organs. People with VHL also have an increased risk of developing ccRCC, a type of kidney cancer that develops in the network of tiny tubes that make up the kidney’s filtration system. Studies have found that sporadic ccRCC is particularly responsive to targeted therapy, but targeted therapy is not routinely used in VHL. PT2385, which is taken by mouth, targets a specific protein (HIF2-alpha) that activates genes that help cancer cells survive and multiply. The study, led by Ramaprasad Srinivasan, M.D., Ph.D., of the Urologic Oncology Branch, in partnership with Peloton Therapeutics, Inc., wants to see whether treatment with PT2385 can shrink or slow the growth of tumors in patients with VHL-associated ccRCC.

Dr. Srinivasan says, “This study aims to target a key protein that was shown by investigators at the NIH and elsewhere to be very important in causing kidney tumors in VHL patients. This study represents the first time this approach is being studied in VHL patients and is the culmination of years of research in this area. If successful, approaches such as this may help reduce the need for surgery in these patients.”

For more information about this trial and the Urologic Oncology Branch, led by Marston Linehan, please visit: To search all CCR clinicals trials, please visit:


Summary Posted: Thu, 06/01/2017