National Cancer Institute NCI Cancer Bulletin: A Trusted Source for Cancer Research News
November 17, 2009 • Volume 6 / Number 22

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FDA Update

New Treatment for Rare Skin Cancer Approved by FDA

The FDA has approved the drug romidepsin (Istodax) for the treatment of cutaneous T-cell lymphoma (CTCL), a rare cancer with about 1,500 new cases diagnosed each year. The approval covers the use of romidepsin in patients whose disease has recurred or worsened following chemotherapy.

In two phase II clinical trials involving 96 and 71 patients, respectively, approximately 35 percent of patients experienced some tumor shrinkage with romidepsin, lasting a median of 15 months in the larger trial and 11 months in the smaller trial. Six percent of patients had their tumors disappear altogether.

“CTCL is a devastating cancer in which many patients suffer from disfiguring tumors, horribly itchy and infected skin, and, in advanced stages, lesions in other organs,” said Dr. Youn Kim, director of the Multidisciplinary Cutaneous Lymphoma Group at Stanford Cancer Center, in a news release from Gloucester Pharmaceuticals, which manufactures romidepsin. “Current systemic therapies have proved inadequate,” she continued, so the drug will address “a significant unmet need.”

Romidepsin is only the second agent in a class of drugs known as histone deacetylase inhibitors to be approved by the FDA. These drugs are thought to work in part by activating genes that suppress tumor growth.

Although the compound was initially identified and pursued by another pharmaceutical company, much of the preclinical development and the first human trials involving romidepsin were conducted by Drs. Susan Bates and Richard Piekarz of NCI.

Early animal model studies showed that the drug caused significant cardiotoxicity, explained Dr. John Wright from NCI’s Cancer Therapy Evaluation Program. “The NCI Developmental Therapeutics Program identified in animals an administration strategy that minimized cardiac toxicity and also completed additional toxicology, pharmacology, and formulation studies that paved the way for the first human clinical trials, which were led by NCI,” he said.

Romidepsin is being studied in clinical trials for other indications, including peripheral T-cell lymphoma.