AACR 2011: New inhibitor prevents lesions and reduces tumour size in basal cell cancer

Dr Ervin Epstein - Children’s Hospital of Oakland Research Institute, California, USA.

A new hedgehog pathway inhibitor demonstrated efficacy in preventing and treating basal cell cancer among patients with basal cell nevus syndrome, a rare inheritable disease. It is known that basal cell nevus syndrome is caused by a mutation in the PTCH gene, which encodes a primary inhibitor of the hedgehog signalling pathway. Using this information, researchers developed the anti-hedgehog signalling pathway drug GDC-0449.

Dr Ervine Epstein discusses the extremely positive results of a Phase I trial which demonstrated that GDC-0449 significantly reduced the size of existing basal cell carcinomas and the development of new carcinomas. Although many skin cancers can be successfully treated with surgery, this therapy would be very useful for treating patients with many basal cell carcinomas.