Clinical Trials

Clinical trial tests targeted radioactive agent as therapy for metastatic prostate cancer

Prostate-specific membrane antigen (PSMA) is present on the surface of all prostate cancer cells but is highly expressed on cells of castration-resistant prostate cancer (mCRPC). Frank Lin, M.D., Investigator in the the Molecular Imaging Program, is leading NCI’s effort to study the effect of targeted treatment with a radioactive agent to test dosing and overall effect for these patients.

A Conversation with Christina Annunziata, M.D., Ph.D.

Christina Annunziata, M.D., Ph.D., is an Investigator in the Women’s Malignancies Branch at the Center for Cancer Research. She has spent most of her career studying the molecular underpinnings of ovarian cancer with the goal of discovering novel treatments. In our Q&A, Dr. Annunziata discusses her personal and professional milestones as well as new directions for her research.

Clinical trial studies CAR T-cell therapy for relapsed/refractory acute myeloid leukemia

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow, the soft inner part of long bones where new blood cells are made. With AML, bone marrow cells don't grow the way they're supposed to. Instead, immature bone marrow cells grow uncontrollably and build up in the body. This study is testing the effect of chimeric antigen receptor T cells (CAR T cells) for children and young adults.

New clinical trial evaluates chemotherapy delivery for stomach cancer

A clinical trial testing chemotherapy delivery for stomach cancer that has spread to the lining of abdominal cavity is underway at the NIH Clinical Center. Investigators are evaluating how this delivery may improve outcomes for patients with this rare condition.

Study confirms effective, less toxic alternative to standard treatment for adults with Burkitt lymphoma

In a new study, an alternative treatment regimen that is less toxic than standard dose-intensive chemotherapy was found to be highly effective for adults with Burkitt lymphoma across all age groups and independent of HIV status. In addition to being better tolerated, the regimen, called dose-adjusted (DA) EPOCH-R, is already an option for diffuse large B-cell lymphomas and can be administered in an outpatient setting. The study was led by researchers in the Center for Cancer Research, and the DA-EPOCH-R regimen was originally developed by researchers led by Wyndham Wilson, M.D., Ph.D., Senior Investigator in the Lymphoid Malignancies Branch.

FDA approves pomalidomide for AIDS-related Kaposi sarcoma

On May 14, 2020, the Food and Drug Administration expanded the indication of pomalidomide (POMALYST, Celgene Corporation) to include treating adult patients with AIDS-related Kaposi sarcoma after failure of highly active antiretroviral therapy and Kaposi sarcoma in adult patients who are HIV-negative. This oral therapy is the first new treatment option available for those with Kaposi sarcoma in more than 20 years.

NCI initiative aims to boost CAR T-cell therapy clinical trials

Researchers at the Center for Cancer Research are part of a new NCI initiative to manufacture CAR T-cell therapies for clinical trials being conducted at multiple hospitals. Nirali N. Shah, M.D., Lasker Clinical Research Scholar in the Pediatric Oncology Branch, is a co-lead investigator on the first trial of this initiative that is testing a CAR T-cell therapy designed to target a protein on cancer cells called CD33 in children and young adults with advanced forms of acute myeloid leukemia.

The discovery of selumetinib for children with NF1

When Brigitte Widemann, M.D., Chief of the Pediatric Oncology Branch (POB), saw selumetinib shrinking the first child’s tumor, she couldn’t believe her eyes. Then, when she saw the second patient’s tumor shrinking, “I thought, oh my gosh, it’s working!” she recalls. Over 30 years of NCI-led and NCI-supported research culminated in clinical trials of selumetinib for kids with neurofibromatosis type 1 (NF1) and associated tumors called plexiform neurofibromas (PNs). With Food and Drug Administration (FDA) approval, selumetinib is now the first effective treatment for kids with PNs that can't be removed by surgery.

FDA approves first therapy for children with neurofibromatosis type 1

The Food and Drug Administration (FDA) has approved selumetinib (Koselugo) to treat children with neurofibromatosis type 1 (NF1) and tumors called plexiform neurofibromas. The approval was based on results from a clinical trial, designed and led by Brigitte Widemann, M.D., Chief of the Pediatric Oncology Branch (POB), and Andrea M. Gross, M.D., Assistant Research Physician in POB, that showed the drug shrank neurofibromas in 70 percent of patients. For many children in the trial, treatment with selumetinib also had clinical benefit, improving their pain, function and quality of life.

CCR researchers show selumetinib shrinks tumors in children with NF1

Findings from a phase 2 clinical trial show that the drug selumetinib improves outcomes for children with the genetic disorder neurofibromatosis type 1 (NF1). In the trial, selumetinib shrank the inoperable tumors that develop with NF1 called plexiform neurofibromas, and children experienced reduced pain, improved function, and better overall quality of life after receiving the treatment. This trial was led by Brigitte Widemann, M.D., Chief of the Pediatric Oncology Branch (POB), and Andrea M. Gross, M.D., Assistant Research Physician in POB, and the results were published March 18, 2020, in the New England Journal of Medicine.

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