Amit Tirosh, MD, the recipient of an ICRF Clinical Research Career Development Award at Chaim Sheba Medical Center, has found that distinct changes in cellular metabolism characterize pancreatic tumors that develop in individuals with von Hippel-Lindau (VHL) syndrome, and he has identified a specific small molecule metabolite that may mediate the effects of the VHL mutation.

VHL syndrome is a rare disease that causes tumors and cysts to grow in the body, at sites including the brain and spinal cord, kidneys, pancreas, and adrenal glands. The tumors are usually benign (non-cancerous); but, some tumors, such as those in the pancreas and kidney, can become cancerous. von Hippel-Lindau disease is caused by mutations in the VHL gene, which normally functions to prevent cells from growing and dividing too rapidly or in an uncontrolled way.

Dr. Tirosh believes that this small molecule is a pro-tumor metabolite, which accelerates tumor proliferation and suppresses the immune response to the tumor in pancreatic cancer patients with VHL. His team will test this hypothesis and, if correct, this small molecule may become a potential target for treatment of pancreatic cancers (and possibly other cancers, including carcinoid tumors) in VHL patients.