It's the most common form of skin cancer, but in its advanced stages, basal cell carcinoma has the potential to become disfiguring and life threatening. An international phase 2 study headed by Mayo Clinic led to the recent Food and Drug Administration approval of the first drug of its kind to help advanced basal cell carcinoma patients who have few treatment options.

The results appear in the June 7 edition of the New England Journal of Medicine.

The study found the drug Erivedge (vismodegib) shrank advanced basal cell carcinoma tumors in 43 percent of patients with locally advanced disease and in 30 percent of patients whose disease spread to other organs.

"This targeted therapy represents a new paradigm in cancer treatment," says lead researcher Aleksandar Sekulic, M.D., Ph.D., a dermatologist and cancer researcher at Mayo Clinic in Arizona.

More than 2 million cases of basal and squamous cell skin cancer are found in this country each year. Basal cell carcinoma accounts for approximately 80 percent of all diagnosed non-melanoma skin cancers, according to the American Cancer Society. It occurs when a basal cell develops a mutation in its DNA, causing it to multiply rapidly, with the potential of forming a cancerous tumor. In most cases, when basal cell carcinoma is diagnosed early it is treated effectively by surgery. When the cancer reaches an advanced state, surgery is not always an option or can be disfiguring. The disease can also be life threatening if left untreated or if it further advances into the skin, bone and tissue.

Erivedge can shrink a tumor by targeting a molecular signaling pathway that fuels the cancer cells and shut it down, Dr. Sekulic says.

"These findings are very exciting because we haven't had any therapies before that worked to this degree for advanced basal cell carcinoma," he says. Dr. Sekulic adds that more research is needed to determine if the drug has the potential to improve treatment for those in earlier stages of the disease, those with multiple basal cell carcinomas and those with a genetic predisposition to the disease.

The study included researchers from MD Anderson Cancer Center, Houston; Stanford University School of Medicine, Stanford, Calif.; Sint-Augustinus Hospital, Antwerp, Belgium; University of Colorado Cancer Center, Denver; Sarah Cannon Research Institute, Nashville, Tenn.; University of California, San Francisco; Dana-Farber Cancer Institute, Boston; Mount Sinai Medical Center, New York; John Hopkins University, Baltimore; Genentech, Inc., San Francisco; and the Universitatsklinikum Schleswig-Holstein, Kiel, Germany.