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Studies show new drug may treat and prevent basal cell carcinoma

Each year approximately 2.8 million people in the United States are diagnosed with basal cell carcinoma (BCC), the country's most common form of cancer. New studies published today in the New England Journal of Medicine show the drug vismodegib (trade name: Erivedge) may help treat and prevent this type of skin cancer that is rarely fatal but potentially disfiguring.

Three papers, all with Stanford authors, demonstrate the effectiveness of vismodegib. The first U.S. Food and Drug Administration-approved drug in its class, vismodegib was shown in two of the studies to be effective in treating advanced or metastatic BCC tumors.

According to our release, one clinical trial, tested the use of vismodegib to treat patients who have Gorlin syndrome, which is characterized by tens to hundreds of BCC tumors. A second clinical trial tested the drug's effectiveness in treating advanced basal cell carcinomas. From the release:

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.


The second of the three papers presents the findings from the phase-2 Genentech-sponsored clinical trial that were the basis for the FDA's decision to allow vismodegib to be used to treat advanced forms of BCC in adults. The drug was successful 43 percent of the time in either complete or partial shrinkage of tumors in the 96 patients with advanced disease who participated in the trial, the study reports.

Researcher and author on two of the studies Anthony Oro, MD, PhD, professor of dermatology at Stanford, was most excited about having a treatment for many of his patients who are running out of options:

We now have a brand new class of drugs that can treat these cancers. As a dermatologist, this is exciting to see. There is nothing for these patients that works. Their cancers are often surgically inoperable.

Previously: Hope for basal cell carcinoma prevention? and Common drug might help prevent skin cancers

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