Investigational Drug May Halt Gallbladder Cancer

CINCINNATI—A new drug shown effective in patients with renal (kidney) cancer may also improve the survival and quality of life of patients with advanced gallbladder cancer, a rare but quick-to-spread disease that is difficult to diagnose and treat.

Led locally by Malek Safa, MD, at the University of Cincinnati (UC), this national, phase-2 clinical trial will determine whether an oral medication recently approved by the FDA for the treatment of advanced renal cancer can also effectively slow gallbladder cancer growth in patients who would not benefit from surgery.

The gallbladder—located behind the liver—stores bile, a fluid made by the liver to help digest food in the stomach and intestines. Gallbladder cancer is difficult to diagnose because it starts deep in the organ and grows outward, so it often goes undetected until it’s very advanced.

“Gallbladder cancer is a sneaky disease because patients usually don’t show signs or symptoms until it has already spread to other parts of the body,” explains Dr. Safa, assistant professor of medicine and director of the gastrointestinal medical oncology program.

“The disease doesn’t appear as a mass like most cancers—it’s more like a tissue thickening, so it’s hard to detect and often misdiagnosed as gallstones,” he adds. “Those who are lucky enough to detect the cancer early usually do so when they go into surgery to have their gallbladder removed for other reasons.”

The investigational drug—known as BAY 43-9006 while in testing—works by inhibiting multiple proteins in the cancer cell that are linked to tumor development. By blocking these proteins, scientists believe they can slow the progression of the cancer and improve the patient’s quality of life and chances for survival.

Surgery is the only curative treatment option for gallbladder cancer. Because the gallbladder is close to the liver and other organs, many patients are ineligible for it because their cancer has already spread to surrounding tissue and cannot be eliminated with surgery. 

Average survival for patients with advanced gallbladder cancer that cannot be treated surgically is three to six months, Dr. Safa says.

“If this investigational drug proves effective,” he adds, “it will give patients who cannot have surgery new hope for prolonged survival.”

Study participants nationwide will receive the drug orally twice a day until the treatment is no longer effective. Researchers will track patient response to the drug by taking frequent blood samples and X-ray scans every eight weeks.

According to the American Cancer Society, more than 8,500 American men and women will be diagnosed with gallbladder cancer and about 3,260 will die from the disease in 2006.

This study is sponsored by the Southwest Oncology Group, a research division of the National Cancer Institute. To learn more, call the UC Cancer Center clinical trials office at (513) 584-7698.