Our Discoveries

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.

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.

Protein that drives aggressive liver cancer identified

Cancers with more protein diversity (heterogeneity) tend to be more aggressive and less responsive to treatment. CCR researchers led by Xin Wei Wang, Ph.D., Deputy Chief of the Laboratory of Human Carcinogenesis and co-Director of the CCR Liver Cancer Program, have identified a protein that may drive an evolution toward greater heterogeneity in two forms of liver cancer.

Immune cells promote breast cancer’s metastasis to the brain in young mice

Animal studies led by Patricia S. Steeg, Ph.D., Deputy Chief of the Women’s Malignancies Branch, suggest that younger immune systems promote breast cancer’s metastasis to the brain — but that the risk can be reduced by triggering immune changes that usually occur later in life.

International study of rhabdomyosarcoma finds genetic clues, potential for tailored therapy

New findings from the largest-ever international study on rhabdomyosarcoma – the most common type of soft tissue sarcoma in children – suggest that children with the disease could benefit from tumor genetic testing. Javed Khan, M.D., Deputy Chief of the Genetics Branch, led the study.

Schematic showing the transcription of DNA into RNA

Live-cell imaging reveals variability in RNA processing

Real-time imaging of messenger RNA synthesis from hundreds of human genes reveals surprising variability in the way cells’ splicing machinery processes these molecules.

Personalized approach to prostate cancer diagnosis could mean less invasive biopsies

Systematic and MRI-guided biopsies can each find cancers that the other technique can miss — but not everyone needs both. According to a new analysis, magnetic resonance imaging can be used to determine which biopsy approach is appropriate for an individual.

New fluorescent probes developed that can help clinicians pinpoint cancer metastases

New chemical compounds developed by CCR researchers and clinicians are safe and highly effective in fluorescently highlighting microscopic metastases. The new compounds could have great utility in complex living systems based on experiments done in mouse models of metastatic human ovarian cancer.

Stem cell transplant after CAR T-cell therapy effective for young leukemia patients

In a clinical trial, CCR physician-scientists found that long-term survival for young patients with B-cell acute lymphoblastic leukemia was significantly better for those who received CD19-CAR T-cell therapy followed by a stem cell transplant. As a result of these findings, use of stem cell transplant is now recommended following CAR T-cell therapy.

New tool predicts which treatments may work best in cancer patients

A new precision oncology software tool, called SELECT, analyzes tumor transcriptomics data to predict which therapies are most beneficial for an individual patient. When applied to data from over 30 different clinical trials, SELECT was predictive of patient responses to targeted and immune therapies in about 80 percent of the trial datasets.

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