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14-06-2012 | Genetics | Article

Vismodegib effective treatment for patients with basal-cell carcinoma


Free abstract

MedWire News: Study findings show that patients with locally advanced or metastatic basal-cell carcinoma respond to treatment with vismodegib, a small-molecule inhibitor of the hedgehog signaling pathway.

Vismodegib was found to dramatically shrink basal cell skin cancers and prevent the formation of new tumors in patients with basal cell nevus syndrome (BCNS), a rare genetic condition that causes multiple skin cancers.

The results offer patients the possibility of an alternative treatment with oral medication, but the side effects associated with the drug mean that it is not a suitable alternative to surgery for patients with sporadic basal cell cancers.

"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," said study co-author David Bickers (New York-Presbyterian Hospital, USA) in an associated press release.

Oral vismodegib 150 mg/day was given to 33 patients with metastatic basal-cell carcinoma and 63 patients with locally advanced basal-cell carcinoma who had inoperable disease or for whom surgery was inappropriate. Patients received vismodegib until disease progression, unacceptable toxic effects, or discontinuation of the dose.

The response rate for patients with locally advanced disease was higher than for patients with metastatic disease, at 43% versus 30%. A complete response was observed in 21% of patients with locally advanced basal-cell carcinoma.

The median duration of response in patients with metastatic and locally advanced basal-cell carcinoma was 7.6 months, with a median progression-free survival of 9.5 months.

Biopsy specimens from 54% of patients with advanced disease showed no residual basal-cell carcinoma in any target lesions. Visible reductions in tumor size and improvement in appearance were seen for the majority of these patients.

All patients experienced at least one adverse event, with 57% experiencing grade 1 or 2 adverse events. Adverse events included muscle spasms, alopecia, taste disturbance, weight loss, and fatigue. Serious adverse events were reported in 25% of patients, with seven deaths occurring due to adverse events.

"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 other modalities such as photodynamic therapy, which can be effective for smaller lesions," said Bickers.

The findings are published in TheNew England Journal of Medicine.

By Ingrid Grasmo

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