The investigational targeted therapy vismodegib (GDC-0449) may provide a significant benefit to patients with locally advanced or metastatic basal cell skin cancers. The results of this Phase II clinical trial were presented at the 2011 European Multidisciplinary Cancer Congress.
Basal cell carcinoma is the most commonly diagnosed type of skin cancer. Most cases can be treated with surgery or other types of local treatment and are not life-threatening, but the condition often occurs on the face and can be disfiguring. In the most severe cases, the cancer may be very large, may invade structures other than the skin, or may spread to other parts of the body. In these advanced cases, it may not be possible to surgically remove the cancer, and treatment options are limited.
Vismodegib targets a specific biological pathway (the Hedgehog pathway) that is thought to play a role in more than 90% of cases of basal cell carcinoma. Taken orally, vismodegib inhibits the abnormal signaling in this pathway that contributes to cancer growth.
To evaluate vismodegib for the treatment of advanced basal cell carcinoma, researchers conducted a Phase II clinical trial among 104 patients with cancer that was locally advanced or had spread to other parts of the body (metastatic). None of the patients were candidates for surgical treatment of the cancer.
- A reduction or elimination of detectable cancer was observed in 43% of patients with locally advanced basal cell carcinoma and 30% of patients with metastatic basal cell carcinoma.
- Common side effects included muscle spasms, hair loss, taste disturbance, weight loss, and fatigue.
Vismodegib offers a promising approach to the treatment of advanced basal cell skin cancer, but is still investigational. Patients with advanced basal cell skin cancer may wish to talk with their physician about the risks and benefits of participating in a clinical trial that further evaluates this or other novel treatment approaches.
Reference: Dirix L, Migden MR, Oro AE et al. A pivotal multicenter trial evaluating efficacy and safety of the Hedgehog pathway inhibitor (HPI) vismodegib in patients with advanced basal cell carcinoma (BCC). Presented at the 2011 European Multidisciplinary Cancer Conference. Stockholm, Sweden. September 23-27, 2011. Abstract LBA1.