Vismodegib is an effective treatment for patients with basal cell carcinomas

A new drug is effective in preventing new basal cell carcinomas in patients with an inherited predisposition to the disease.

These patients with basal cell nevus syndrome develop large numbers of basal cells, which can become locally invasive or metastatic, according to a discussion presented by renowned oncologist Dr. Daniel D. Von Hoff at the 102nd annual meeting of the American Association for Cancer Research (AACR).

In an initial study, Dr. Von Hoff and his team at TGen Clinical Research Service at Scottsdale Healthcare (TCRS) found that the drug, vismodegib (GDC-0449), a hedgehog pathway inhibitor, was effective in shrinking advanced invasive or metastatic basal cell carcinomas. TCRS was the first to evaluate vismodegib, produced by Genentech. TCRS is a partnership of the Translational Genomics Research Institute (TGen) and the Virginia G. Piper Cancer Center at Scottsdale Healthcare in Scottsdale, Ariz.

At Sunday's conference plenary session, titled: "The Future of Cancer Research: Challenges and Opportunities," Dr. Von Hoff discussed a new prevention and treatment approach for patients who have basal cell nevus syndrome. Specifically, he discussed the effect of the drug on basal cell nevus syndrome, an advanced form of basal cell carcinoma that produces often-disfiguring tumors of the jaw, the sole of the foot, the brain and ribs.

A team of investigators from Children's Hospital Oakland Research Institute (CHORI) in Oakland, Calif., headed by Dr. Ervin H. Epstein Jr., presented dramatic results at the conference demonstrating that vismodegib entirely prevented the development of basal cell carcinomas in patients with basal cell nevus syndrome.

These findings are "a stunning result, which brings hope to patients who otherwise may need disfiguring surgery, especially for cancers that arise on the face and upper part of the body," said Dr. Von Hoff, a past president of AACR.

"We are so pleased that the results obtained by TCRS could be a part of the work that has made a difference for so many patients," said Dr. Von Hoff, who also is Physician-In-Chief and Distinguished Professor at TGen; Chief Scientific Officer at Scottsdale Healthcare and US Oncology; and Professor of Medicine at Mayo Clinic.

Basal cell nevus syndrome is an inherited genetic disease, which results in the development of multiple, sometimes hundreds of basal cell carcinomas. The sporadic (non-inherited) form of basal cell carcinoma is the most common skin cancer. While most cases are curable, in some patients there is a tendency for recurrent cancers and surgery may not be possible.

More than 15,000 physicians and researchers from across the globe attend the annual AACR conference, which runs April 2-6 at the Orange County Convention Center in Orlando, FL.