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Clinical trial evaluates reduced dosing of cyclophosphamide to prevent severe acute graft-versus-host disease

Christopher G. Kanakry, M.D., Lasker Clinical Research Scholar in the Experimental Transplantation and Immunotherapy Branch, is leading a study that may help people with cancers that begin in blood-forming tissue who are at high risk for graft-versus-host disease (GVHD) following bone marrow transplant. This study will evaluate if reduced dosing of cyclophosphamide is effective in preventing severe acute GVHD.

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Blood test reveals when benign NF1 tumors turn cancerous

People with neurofibromatosis type 1 (NF1) often develop non-cancerous, or benign, tumors that grow along nerves. These tumors can sometimes become cancerous, but there hasn’t been a good way to determine whether this transformation to cancer has happened. In a new study, researchers co-led by Jack F. Shern, M.D., Lasker Clinical Research Scholar in the Pediatric Oncology Branch, have developed a blood test that could one day offer a highly sensitive and inexpensive approach to detect cancer early in people with NF1.

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FDA approves belzutifan, first drug for cancers associated with von Hippel-Lindau disease

On August 13, 2021, the Food and Drug Administration approved belzutifan, a new drug for adult patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC), central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors, not requiring immediate surgery.

Ramaprasad Srinivasan, M.D., Ph.D., Investigator in the Urologic Oncology Branch (UOB), designed the ongoing study and played a key leadership role as the principal investigator on the cooperative research and development agreement under which NCI served as a site in the study. Belzutifan is now the first and only approved systemic therapy for certain patients with VHL-associated RCC.

VHL disease is a rare, inherited disorder that causes tumors and cysts to grow in certain parts of the body. Patients with this disease have an increased risk of certain types of cancer, especially kidney cancer and pancreatic cancer. The VHL gene was originally identified by Marston Linehan, M.D., and colleagues in the UOB in the 1990s, and the group continues to define the methods for clinical management of VHL disease.

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