A new study has found vismodegib to be effective in shrinking and preventing the formation of basal cell carcinoma (BCC) in patients with basal cell nevus syndrome (BCNS).
Oakland, Calif. - A new study has found vismodegib to be effective in shrinking and preventing the formation of basal cell carcinoma (BCC) in patients with basal cell nevus syndrome (BCNS).
This study and another, however, also report significant adverse effects.
Newswise.com reports that one study, led by researchers with Children’s Hospital Oakland Research Institute New York-Presbyterian Hospital/Columbia University Medical Center, was the first to evaluate the effectiveness of this drug in patients with BCNS. The randomized, double-blind study included 42 patients receiving either oral vismodegib or placebo for a maximum of 18 months.
Patients receiving vismodegib had an annual average of 2.3 new surgically eligible BCCs, compared to an average of 29 for those receiving placebo. In addition to the decreased frequency of tumors, the size of the control group’s tumors decreased by an average of 65 percent.
According to the study abstract, “Patients receiving vismodegib routinely had grade 1 or 2 adverse events of loss of taste, muscle cramps, hair loss and weight loss. Overall, 54 percent of patients (14 of 26) receiving vismodegib discontinued drug treatment owing to adverse events.”
According to the abstract of another vismodegib study, researchers found that in 33 patients with metastatic BCC, the independently assessed response rate was 30 percent, and that in 63 patients with locally advanced BCC, the independently assessed response rate was 43 percent, with complete responses in 13 patients (21 percent). The median duration of response was 7.6 months in both cohorts. Adverse events, occurring in more than 30 percent of patients, were muscle spasms, alopecia, dysgeusia, weight loss and fatigue. Serious adverse events were reported in 25 percent of patients, and seven deaths due to adverse events were noted.
The Food and Drug Administration in January approved vismodegib for the treatment of patients with metastatic BCC or with locally advanced BCC that has recurred following surgery or who are not candidates for surgery or radiation.
Both studies were funded by vismodegib manufacturer Genentech. The studies were published online in the New England Journal of Medicine.
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