New York, NY (June 6, 2012) A clinical study has demonstrated that a new drug, a targeted molecular therapy called vismodegib (trade name Erivedge), can dramatically shrink basal cell skin cancers and prevent the formation of new ones, in patients with basal cell nevus syndrome (BCNS). This rare genetic condition causes dozens, and sometimes hundreds or thousands, of skin cancers on each patient's body. The primary treatment option is surgical removal. These study results are significant as they indicate the possibility of an alternative treatment with oral medication; although side effects remain a consideration.
The phase II clinical study, led by researchers at NewYork-Presbyterian Hospital/Columbia University Medical Center (NYPH/CUMC) and Children's Hospital of Oakland Research Institute (CHORI), was published today in the online edition of the New England Journal of Medicine.
"In its current formulation, vismodegib is appropriate only for BCNS patients with very large numbers of basal cell skin cancers. Still, this is a huge step forward, pointing to the day when we can offer every one of these patients an alternative to repeated surgery, which can be disfiguring and burdensome," said study co-leader David R. Bickers, MD, the Carl Truman Nelson Professor and chairman of dermatology at CUMC and director of dermatology at NewYork-Presbyterian Hospital/CUMC. The study was co-led by Ervin H. Epstein, Jr., MD, a senior scientist at CHORI.
The study is the first to evaluate vismodegib in patients with BCNS. Forty-two patients were randomized to receive either vismodegib (taken orally) or a placebo, for a maximum of 18 months. Overall, the study tracked more than 2,000 existing surgically eligible basal cell skin cancers (SEBs) and documented 694 new SEBs, on the 42 patients.
Patients taking vismodegib experienced an average of 2.3 new SEBs, compared with 29 for patients in the placebo group. Among patients taking t
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Columbia University Medical Center