Targeted agent shows promise in biliary cancer study

Apr 20, 2009

An experimental agent has shown promising results in people with advanced biliary cancer, according to a multi-institutional clinical trial led by cancer researchers at the Ohio State University.

The agent, known as AZD6244 (ARRY-142886), blocks certain enzymes that need to proliferate and survive.

About 100,000 patients are diagnosed with biliary cancer worldwide every year, representing 15-20 percent of all liver cancer cases. Most patients present at later stages of the disease, which has a universally poor outcome.

The findings of the 28-patient, phase II study will be reported April 20 at the annual meeting of the American Association for Cancer Research in Denver, Colorado.

"This is a malignancy for which there is no standard of care," says the study's principal investigator Dr. Tanios Bekaii-Saab, assistant professor of medicine and pharmacology, and a medical oncologist who specializes in at Ohio State's Comprehensive Cancer Center-James Cancer Hospital and Solove Research Institute.

"And while it is a small study, it provides a strong rational for developing this agent further in larger trials either alone or in combination with other drugs, with the hope that we can establish a new standard of care for biliary cancers in the near future."

Bekaii-Saab noted that the average progression-free survival achieved by patients in the study was one of the highest reported in the literature for this with many patients gaining weight in a disease that is typically associated with significant weight loss. The drug, which is administered orally, seems to be well tolerated with mild toxicities.

The agent belongs to a class of drugs called protein kinase inhibitors. This particular drug targets a protein kinase called MEK 1/2, which is part of a chemical pathway that is often damaged in many biliary cancer cases, Bekaii-Saab says.

In addition to Ohio State, study sites included the University of North Carolina, Vanderbilt University and Emory University.

The patients, with an average age of 56 years, had advanced (metastatic) biliary cancer.

By the trial's end, one patient had a complete response to the treatment (the tumor shrank until it was undetectable), two patients had partial tumor shrinkage and 17 patients showed no further growth in tumor size; that is, they had stable disease which often was durable.

A preliminary analysis of the study shows that patients experienced no cancer progression for 5.4 months on average, a time almost double what would typically be expected with therapy in biliary cancer. This is despite the fact that 40 percent of patients had one prior therapy before receiving AZD6244. A more final analysis of the study is underway.

Patients who lacked a target protein called pERK did not seem to respond to the drug, suggesting that the drug may not work if the protein is missing in the cancer cells. "This is an important finding, as it suggests that we may be able to identify patients who may not respond to the drug in the future," he adds.

Source: Ohio State University Medical Center

Explore further: Researchers develop web-based app to predict glioma mutations

add to favorites email to friend print save as pdf

Related Stories

Knocking Out Survival Protein Could Aid Leukemia Treatment

Apr 23, 2007

An effective way to fight leukemia might be to knock out a specific protein that protects cancer cells from dying, a new study shows. The findings suggest that a drug that can block this “survival protein” might on its ...

Older AML patients show promising response in drug study

Dec 08, 2008

Older patients with acute myloid leukemia (AML) who were once told that nothing could be done for them are finding new hope – and life – through a clinical trial at The Ohio State University Comprehensive Cancer Center ...

Recommended for you

Biomarker in aggressive breast cancer identified

12 hours ago

Two Northwestern University scientists have identified a biomarker strongly associated with basal-like breast cancer, a highly aggressive carcinoma that is resistant to many types of chemotherapy. The biomarker, ...

MRI better detects recurrent breast cancer

13 hours ago

(HealthDay)—Single-screening breast magnetic resonance imaging (MRI) detects 18.1 additional cancers after negative findings with mammography and ultrasonography (US) per 1,000 women with a history of breast ...

Natural (born) killer cells battle pediatric leukemia

Aug 19, 2014

Researchers at Children's Hospital Los Angeles have shown that a select team of immune-system cells from patients with leukemia can be multiplied in the lab, creating an army of natural killer cells that ...

User comments : 0