Hedgehog Pathway Inhibitors

There is a new class of oral agents which inhibit the pathway that is activated in basal cell carcinoma, the hedgehog pathway. (The hedgehog signalling pathway is one of the key regulators of development and gives cells information they need for development).

Agents have been used for individuals with locally advanced or metastatic basal cell carcinomas (spread to surrounding tissue). Two companies are developing these drugs at present. Roche - vismodegib (brand name Erivedge) and Novartis (LDE 225). One study also shows that vismodegib dramatically reduces the number of new basal cell carcinomas that develop in Gorlin syndrome. They also reduce the size of existing lesions, making this an exciting new development for individuals with Gorlin syndrome.

The side effects include muscle cramps, loss of taste, hair loss, nausea and weight loss so it is not to be used in every individual, just those with many basal cell carcinomas and lesions that are not amenable for sugery.  Currently the treatments are not licensed in the UK and are only available for the management of basal cell carcinomas in clinical trials but it is hoped they will be widely available towards the end of 2013.

Ref: Lear J. Oral Hedgehog Pathway Inhibitors for Basal Cell Carcinomas. New England Journal of Medicine: 366-2225-2226.


Next Review January 2016

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 Originally produced by Professor P. A. Farndon, Clinical Geneticist, Jim Costello (deceased) and Margaret Costello.  We are reliant on a team of medical advisors for the clinical content of the website. We are grateful for their continuing support. 
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