Morsani College of Medicine

Dermatology & Cutaneous Surgery

New Drug for the Treatment of Advanced Basal Cell Carcinoma

Genetech has submitted a New Drug Application for vismodegib to the U.S. Food and Drug Administration (FDA) for the treatment of people with advanced basal cell carcinoma (BCC) for whom surgery is considered inappropriate. BCC is the most common type of skin cancer which affects thousands of people each year. Most BCCs are easily treated through surgery. However, if left untreated, they can become very large and difficult to treat by standard means. Rarely, BCCs may even metastasize. Currently, there are limited treatment options once BCC reaches an advanced stage.
Research has shown that mutations in the Hedgehog pathway are implicated in most BCCs. These mutations activate the Hedgehog pathway and promote growth of cancer cells. Vismodegib is an inhibitor of the Hedgehog pathway.
In the ERIVANCE trial, 104 patients with locally advanced or metastatic BCC were treated with 150mg of vismodegib orally. A clinical benefit was noted in 75% of patients: tumors shrank or did not enlarge. The most common drug-related adverse events were muscle spasms, hair loss, altered taste sensation, weight loss, fatigue, nausea, decreased appetite and diarrhea. Four patients experience severe adverse events thought to be related to vismodegib.
It is important to note that vismodegib did not cure any patients but does offer some hope for treatment of these otherwise debilitating cancers. Early detection is key in preventing these cancers from growing to an advanced stage. Early detection can be facilitated by examining your skin monthly for any new or suspicious growths such as ones that change color, size, shape, bleed, or are symptomatic. Some BCCs can be difficult to detect or may not arouse your suspicion which is why it is important to be examined by a dermatologist at least once a year.
For more information, patients can contact the Genentech clinical trial call center at 888-662-6728.

by: Basil S. Cherpelis, M.D.