Oral drug in clinical trials shows promise in treating basal cell carcinoma

Key Points

New York - Investigators are studying vismodegib (previously known as GDC-0449, Genentech), a new oral inhibitor of the Sonic hedgehog signaling pathway, known to play an important role in basal cell carcinoma (BCC). The drug is showing promise in treating this type of cancer - the No. 1 skin cancer in the United States.

If vismodegib proves to be efficacious and safe, it would offer new treatment options for a segment of patients.

BCC research

In most cases, surgical excision successfully removes BCC, but not always.

In a phase 1 study investigating the safety and tolerability of the drug in 33 patients with locally advanced or metastatic BCCs (median duration of study treatment, 9.8 months), 18 responded to the drug - with two having a complete response and 16 a partial response (Von Hoff DD, et al. N Engl J Med. 2009; 361(12):1164-1172). Adverse events included fatigue, muscle spasms, atrial fibrillation and hyponatremia.

"The phase 1 study showed very promising results," Dr. Marmur says.

Dr. Marmur and her colleagues are preparing to submit results for publication from an ongoing phase 2 clinical trial of the drug that began in 2009, and was conducted to determine the efficacy and safety of vismodegib in patients with nonsurgical advanced or metastatic basal cell cancer. Adverse effects from the study include hair loss, dysgeusia and muscle cramps.

While the results have been promising, Dr. Marmur says based on published data, it won't be an option for every patient.

"I don't think it's ever going to replace Mohs surgery," she says. "It's not meant to be a replacement for standard surgical treatments for sure, and that's because ... even though it's been quite successful in the studies that have been published, the side effect profile is still significant."