By Liam Davenport
A new drug for a type of skin cancer caused by a rare genetic disease can not only substantially shrink the tumors, but can also prevent the growth of new cancers, US investigators have discovered.
Basal cell nevus syndrome (BCNS) is a genetic condition that can cause dozens, or even thousands, of skin cancers known as basal-cell carcinomas all over a patient's body. The current treatment is frequent and repetitive surgery, which can result in scarring and, consequently, a reduction in quality of life.
Ervin Epstein, from the Children's Hospital Oakland Research Institute in California, and colleagues gave the drug vismodegib or a dummy drug (placebo) to 42 patients with BCNS, who had at least 10 surgically treatable basal-cell carcinomas, for at least 18 months.
After 8 months, patients who received vismodegib had significantly fewer new tumors compared with those given placebo. In addition, patients who received vismodegib had a 65% reduction in the size of existing tumors, compared with just an 11% reduction in tumor size among those who received the dummy drug.
No tumors became more severe in patients given vismodegib. However, it was relatively common for patients given vismodegib to experience side effects, such as loss of taste, muscle cramps, hair loss, and weight loss, and 54% of these patients stopped using the drug due to such effects.
David Bickers, from Columbia University Medical Center in New York, USA, a member of the research team, commented: "The purpose of the study was to see whether… we could match the performance of a surgeon, and in many ways, we could."
He added: "Vismodegib is [currently] 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."
Dr Bickers continued: "The challenge now is to see if we can lessen the adverse effects while achieving the same therapeutic benefits- by modifying the dosing schedule or perhaps by alternating drug treatment with [for example] photodynamic therapy, which can be effective for smaller lesions."
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