Roche Holding AG and Curis Inc.’s experimental cancer drug has been shown to prevent and shrink basal cell skin tumours in patients having basal cell nevus syndrome, a rare disease significantly increasing the risk of developing basal cell cancer.

Data from a mid-stage study involving vismodegib, a drug designed for inhibiting the hedgehog signaling pathway involved with proper embryo development, is scheduled for presentation Saturday at the annual meeting of American Association for Cancer Research. The pathway becomes less active in adults, with research suggesting malfunctioning of the hedgehog pathway malfunctions results in the development of basal cell skin cancer, the commonest cancer type, including other types of cancers.

There are several drugs for targeting the hedgehog pathway under development, however, Roche and Curis appear to be leading the development. After release of data from a Phase 2 study of vismodegib, a larger clinical study known as Phase 3 study will be completed before regulatory approval of the new drug is sought, though the US Food and Drug Administration allows companies temporary approval for promising life-saving treatments, while earlier stage studies are still being conducted.

The Phase 2 study of vismodegib has shown patients treated with it developed 0.07 new basal cell carcinomas per month compared with 1.74 new basal cell carcinomas per month for those given placebos. The vismodegib group also saw a significant decrease in the size of existing basal cell carcinomas, which was essentially unchanged in the placebo group.

As well, none of the patients receiving vismodegib required surgical removal of any basal cell carcinoma during the course of the study. Basal cell nevus syndrome can cause patients to develop lesions requiring surgical removal several times a month.