Cholangiocarcinoma Patients with a Specific Genetic Biomarker Now have a New Treatment Option


FDA grants approval to Merk & Co. for Keytruda (pembrolizumab)

“The hope is that advanced genetic information will one day be able to identify which patients are most likely to benefit from a specific treatment.”

— Marion Schwartz

SALT LAKE CITY, UTAH, USA, June 14, 2017 / — The U.S. Food and Drug Administration has granted approval to Merck & Co. for the first cancer drug based on a patients’ individual genetic characteristics, regardless of where in the body the disease started. Keytruda (pembrolizumab) is for patients with solid tumors identified as having a biomarker called microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR). This genetic defect activates cells to search out and destroy the cancer.

“This is an important first for the cancer community,” Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence, said in a statement. “Until now, the FDA has approved cancer treatments based on where in the body the cancer started—for example, lung or breast cancers. We have now approved a drug based on a tumor’s biomarker without regard to the tumor’s original location.”

Keytruda has been shown to have certain effects that are likely to benefit cholangiocarcinoma patients especially in cases where their tumors continued to grow and spread after past treatment. Of the 149 patients who received Keytruda in clinical trials, 39.6% had a complete or partial response. The response lasted for six months or more for 78% of those patients. Merck is currently conducting further studies in patients with MSI-H or dMMR tumors to verify the treatment benefits of Keytruda.

According to the Cholangiocarcinoma Foundation (CCF) Chief Advocacy Officer, Marion Schwartz, “Molecular profiling provides detailed information about the genetic changes that are present in each patient’s tumor.” It is strongly recommended that all cholangiocarcinoma patients ask to be tested to determine if they have (MSI-H) or other biomarkers that may offer more treatment options.

Due to these exciting developments, CCF is gathering information from ALL cholangiocarcinoma patients about genetic testing. Help us make progress in cancer research by participating in this Brief Survey.