National Cancer Institute - Cancer.gov

Clinical Trials

Areas of the body affected by von Hippel-Lindau (VHL) disease. Credit: NCI Visuals

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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Micrograph of small cell carcinoma

Clinical trial investigates drug combination for relapsed small cell lung cancers and advanced neuroendocrine cancers

Anish Thomas, M.B.B.S., M.D., Lasker Clinical Research Scholar in the Developmental Therapeutics Branch (DTB), and Jaydira Del Rivero, M.D., Assistant Research Physician in DTB, are heading a trial testing the safety and efficacy of berzosertib, an ATR inhibitor, in combination with lurbinectedin to treat relapsed small cell lung cancer or high-grade neuroendocrine cancers.

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Microscopic image of oral chronic graft-versus-host disease

Collaborative study leads to FDA approval of belumosudil for chronic graft-versus-host disease

The Food and Drug Administration (FDA) approved belumosudil on July 16, 2021, for people 12 years and older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy. Chronic GVHD is a complex condition that can be life threatening and occurs when donated stem cells attack healthy tissues in a patient’s body. Steven Z. Pavletic, M.D., M.S., Senior Clinician in the Immune Deficiency Cellular Therapy Program, guided CCR’s involvement in the pivotal consortium study that led to FDA approval of belumosudil. Pavletic was part of the trial clinical leadership at CCR, one of the 28 centers that enrolled study patients. The study found belumosudil to be safe and well-tolerated, and it may have the potential to improve overall patient well-being.

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illustration of the drug, viralym-m

New study tests therapy in patients with hemorrhagic cystitis after bone marrow transplant

Jennifer A. Kanakry, M.D., Associate Research Physician in the Experimental Transplantation and Immunotherapy Branch, is leading CCR’s participation in a study of a drug, Viralym-M, that targets six viruses that can cause hemorrhagic cystitis (HC) in people who have had a hematopoietic cell transplant. Researchers are most interested in Viralym-M’s ability to clear the symptoms of HC faster than a placebo and evaluating Viralym-M’s effect on five other viruses.

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