Treating a rare HIV-associated cancer without chemotherapy

2020

Challenge

Kaposi’s sarcoma is a rare cancer that grows on the skin as well as in the lymph nodes, lungs, and other regions of the body, and patients with the illness have limited treatment options. If the disease is associated with HIV infection, antiretroviral therapy is the first-line treatment, augmented with traditional chemotherapy. Therapies are less well-established for those without HIV infection, although these patients generally receive chemotherapy as well. Unfortunately, responses are limited by the toxic side effects of chemotherapy.

Advance

A breakthrough clinical trial led by IRP senior investigator Robert Yarchoan, M.D., in collaboration with Celgene Corporation, now Bristol-Myers Squibb, demonstrated the safety, pharmacologic activity, and efficacy of pomalidomide, an orally delivered drug that works through a different mechanism of action than traditional, toxic chemotherapeutics. Pomalidomide is a small molecule drug designed to modulate the immune system.

Impact

Based on the results from this clinical trial, the U.S. Food and Drug Administration (FDA) approved the use of pomalidomide (POMALYST^®) in 2020 to include treating both HIV-positive and HIV-negative patients with Kaposi’s sarcoma. Pomalidomide provides Kaposi’s sarcoma patients with a treatment option that is not only less toxic than chemotherapy, but also easier to administer. While other systemic drugs used to treat this cancer are delivered intravenously, pomalidomide is given orally, making it a more feasible option for people living in lower- and middle-income countries.

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