Clinical trial to evaluate whether topical medication can prevent common skin cancer

Dr. Martin A. Weinstock, a professor of dermatology and epidemiology at Brown University, will lead a six-year clinical trial to evaluate the effectiveness of a topical medication as a way to prevent the most common type of cancer in the United States.

Backed by a $34 million award from the U.S. Department of Veterans Affairs Cooperative Studies Program, the study will investigate the potential of imiquimod, a topical medication with minimal side effects, as a preventive measure against basal cell carcinoma. Weinstock — who is the chief of dermatology research for the V.A. Providence Healthcare System — will lead the trial with co-chair Dr. Robert Dellavalle, chief of dermatology for the V.A. Eastern Colorado Health Care System and a University of Colorado School of Medicine professor.

Basal cell carcinoma usually occurs on the face and requires surgery to avoid serious complications. An effective preventive medication could help many patients avoid or at least postpone the risks of surgery, and decrease the need for medical visits and their resulting costs, Weinstock said.

“These lesions are typically treated with what I call a ‘cut and wait’ approach,” he said, noting that skin damage and scarring are undesirable side effects. “Unfortunately, we don’t have anything better right now.”

More than 1,600 participants, including U.S. military veterans at high risk for basal cell carcinoma, will be recruited from 17 V.A. medical centers for the trial.  They will apply the cream to their faces daily for up to 12 weeks and be followed actively for three years to see if their skin cancer risk is reduced, with an additional year of passive follow-up.  In addition to evaluating effectiveness of the treatment, researchers will collect genetic material from some participants to determine factors that may indicate greater risk reduction and better tolerance of imiquimod therapy. This will help target therapy to those who will benefit from it the most.

Weinstock said that developing ways to actively prevent basal and squamous cell carcinoma has been a goal since he joined the Brown faculty in 1988. He has been involved with two other national studies directed at skin cancer therapies — one of these clinical trials found that topical application of a cream containing 5-fluorouracil 5% reduced the risk of squamous cell carcinoma by 75% for a year.

“There’s good reason to believe that we’ll see in this upcoming trial that imiquimod has similar preventative effects on BCC,” Weinstock said. And if that turns out to be the case, he said, “it would fundamentally transform our approach to the disease – we need to proactively prevent this cancer that afflicts millions each year.”