Cholangiocarcinoma, also known as bile duct cancer, is steadily rising in incidence worldwide. Symptoms often go undetected until the disease is far advanced. Surgical resection of tumors is considered the best approach toward attempting a cure, but less than half of patients whose tumors are surgically resected survive past five years, and those whose tumors are not surgically removed face a median survival time of just nine months.

A new Hoosier Cancer Research Network study may help researchers learn whether an investigational combination of immunotherapy drugs might lead to improvements in tumor response in cholangiocarcinoma patients who have received prior therapy for their cancer.

The study, known as HCRN GI16-263, is now open to accrual at the Georgetown Lombardi Comprehensive Cancer Center in Washington, D.C. Additional sites will open the study in the near future.

Aiwu Ruth He, MD, PhD, of Georgetown Lombardi, is leading the study, along with co-investigators Iver Renuka, MD, of Roswell Park Cancer Institute, and Davendra P. S. Sohal, MD, MPH, of Cleveland Clinic’s Taussig Cancer Institute.

The study will help researchers determine how patients with cholangiocarcinoma will respond to treatment with pembrolizumab and Sylatron (peginterferon alfa-2b). The study will also determine whether this combination of drugs is safe and if there are unacceptable side effects.

Cancer cells often create proteins called PD-L1 that act as signals to turn off part of the immune system that recognizes cancer cells as foreign. This allows cancer cells to hide from the immune system. Pembrolizumab is a drug that blocks this signal and allows the immune system to recognize and attack these cancer cells. Researchers involved in this study believe that combining pembrolizumab with Sylatron will help the pembrolizumab to target the cancer cells.

Sylatron is approved by the U.S. Food and Drug Administration (FDA) in certain settings, but not for cholangiocarcinoma. Likewise, pembrolizumab is approved for other types of cancer, but not for cholangiocarcinoma. The use of these drugs should be considered “investigational.” This means using these drugs in this setting has not been approved by the FDA.

The study will enroll 44 subjects. All participants will receive the same treatment. Once a week for 12 weeks, participants will receive injections of Sylatron. Beginning the fourth week, they will also receive an intravenous infusion of pembrolizumab every three weeks. As long as a participant’s disease does not worsen, they may continue to receive pembrolizumab every three weeks.

To enroll in HCRN GI16-263, subjects must have cholangiocarcinoma and have received prior treatment for their cancer. For more information about this study, including full eligibility requirements, visit www.clinicaltrials.gov (study #NCT02982720).

This trial is supported by Merck Sharp & Dohme Corp.

About Hoosier Cancer Research Network:

Hoosier Cancer Research Network (formerly known as Hoosier Oncology Group) conducts innovative cancer research in collaboration with academic and community physicians and scientists across the United States. The organization provides comprehensive clinical trial management and support, from conception through publication. Created in 1984 as a program of the Walther Cancer Institute, Hoosier Cancer Research Network became an independent nonprofit clinical research organization in 2007. Since its founding, Hoosier Cancer Research Network has initiated more than 160 trials in a variety of cancer types and supportive care, resulting in more than 350 publications. More than 5,000 subjects have participated in Hoosier Cancer Research Network clinical trials.