Studies show new drug to be effective in treating skin cancer, Stanford researchers say

Public release date: 6-Jun-2012 [ | E-mail | Share ]

Contact: Tracie White traciew@stanford.edu 650-723-7628 Stanford University Medical Center

STANFORD, Calif. A new drug has been shown to be effective in treating and preventing the most common cancer in the United States: basal cell carcinoma skin cancer, according to a new study in the New England Journal of Medicine to be published June 7.

The drug, vismodegib (trade name: Erivedge), was tested in a clinical trial in patients with Gorlin syndrome, a rare disease in which individuals have tens to hundreds of disfiguring basal cell carcinoma tumors. The findings are being released in conjunction with two other papers in the same journal issue that show the effectiveness of vismodegib in treating BCCs that are advanced or metastatic. All three articles include authors from the Stanford University School of Medicine.

"It is a landmark day for patients with basal cell carcinoma and all those involved in their care the greatest advance in therapy yet seen for this disease," writes John Lear, MD, in an accompanying editorial. Lear is a consultant dermatologist at Manchester Royal Infirmary and the Dermatology Centre of Hope Hospital in Manchester, UK.

The research is being published five months after vismodegib became the first drug in its class to gain approval from the U.S. Food and Drug Administration. Vismodegib, which was developed by the biotech firm Genentech/Roche, targets a molecular signaling pathway in cells called the Hedgehog signaling pathway. The drug works by inhibiting the Hedgehog pathway, which is inappropriately activated in basal cell carcinomas. While the pathway is important in early embryonic development, it is generally inactive in healthy adults. In January, the FDA approved the drug for use in treating advanced forms of basal cell carcinoma.

In the study of the drug's effect on patients with Gorlin syndrome (also known as Basal Cell Nevus Syndrome) the researchers showed that subjects taking vismodegib developed an average of two new tumors per year, compared with 29 new tumors in subjects taking placebo. The drug is taken daily in a pill form. This investigator-initiated, double-blind placebo trial involved 41 patients with Gorlin syndrome and was stopped early due to the overwhelming effectiveness of vismodegib, the article states. It was considered unethical not to offer the drug to those participants taking a placebo.

"How often in your life do you get to have worked within a field where you finally get to test to a drug that actually changes people's lives?" said Jean Tang, MD, PhD, assistant professor of dermatology at Stanford and first author of the study. "We were very excited about the results."

She added: "In most subjects, all the carcinomas clinically disappeared. No tumors progressed while the subjects took vismodegib."

Currently there is no good treatment for Gorlin syndrome, which afflicts one in 50,000 people; these individuals often develop their first BCC tumor in childhood. The constant growth of BCC tumors, while generally non-lethal, can be treated surgically but the persistent operations leave scarring, and some BCC tumors can eventually become inoperable.

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Studies show new drug to be effective in treating skin cancer, Stanford researchers say