The experimental agent selumetinib 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.
Selumetinib, also known as AZD6244 (ARRY-142886), blocks a protein called MEK, which cancer cells need to proliferate and survive.
Biliary cancer is a malignancy of cells lining the bile ducts and gall bladder. About 100,000 patients are diagnosed annually worldwide with the disease, 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 this 28-patient, multi-institutional phase II study are reported online in the Journal of Clinical Oncology.
"This malignancy has no good standard of care," says principal investigator Dr. Tanios Bekaii-Saab, medical director of gastrointestinal oncology and a medical oncologist at Ohio State's Comprehensive Cancer Center-James Cancer Hospital and Solove Research Institute.
"Our study provides a strong rationale for developing this agent further in larger trials, probably in combination with other drugs, which we hope will enable us to establish a new standard of care for biliary cancers in the near future," adds Bekaii-Saab.
In addition to Ohio State, trial participants included the University of North Carolina, Vanderbilt University and Emory University.
Selumetinib belongs to a class of drugs called protein-kinase inhibitors. The agent selectively inhibits the protein kinases MEK1 and MEK2. It is part of a signaling pathway that is often damaged in biliary cancer cells. The trial showed the following:
- One patient responded completely – the tumor shrunk until it was undetectable – and two patients showed partial tumor shrinkage;
- In 17 patients, the tumor stopped growing. This condition of stable disease lasted up to 16 weeks in the majority of cases;
- Patients experienced no cancer progression for a promising 3.7 months on average, despite the fact that nearly 40 percent of patients had prior therapy before receiving selumetinib (such tumors tend to be more resistant to treatment);
- Patients taking the drug regained weight – about 9 pounds on average. This included patients with tumors that may not have responded to the drug, Bekaii-Saab notes.
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