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New Drug Effective for Rare Genetic Skin Cancer: Studies
Date:6/6/2012

By Barbara Bronson Gray
HealthDay Reporter

WEDNESDAY, June 6 (HealthDay News) -- When a clinical trial is stopped abruptly just eight months after its start, it's either very good or very bad news.

In the case of a study on a skin cancer drug, the results were so impressive that the trial's independent data and safety monitoring board decided to offer the drug immediately to the study participants who were taking placebos.

The drug, vismodegib (Erivedge), was approved in January by the U.S. Food and Drug Administration for people with aggressive, large basal cell carcinoma that had spread to the lymph nodes or other body systems. The researchers wanted to test the oral medication against a disfiguring form of skin cancer called basal cell nevus syndrome, a rare genetic condition.

Researchers followed 41 patients with basal cell nevus syndrome and found that those taking vismodegib got an average of slightly more than two new cancers, while those not taking the drug developed 29.

The study was reported in the June 7 New England Journal of Medicine.

"This is one of the first clinical trials that show the drug can be used in prevention," said Dr. Jean Tang, a co-author of the study and assistant professor in the department of dermatology at Stanford University School of Medicine.

Tang said the positive effects of the drug are visible within a month after starting to take it. "This is a life-changing drug for these patients," she said.

Basal cell nevus syndrome typically starts at puberty. The condition can involve hundreds of basal cell lesions, often requiring many surgical and nonsurgical procedures to treat.

Basal cell carcinoma of the skin is the most common cancer worldwide, and its prevalence is increasing. There are about 2.1 million new cases of non-melanoma skin cancer treated in the United States every year, according to journal background information. Some 750
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