Cholangiocarcinoma Research

Cholangiocarcinoma is a rare cancer of the bile ducts with a dire prognosis that has been rising in incidence in the U.S. over the past 30 years.

Cholangiocarcinoma is a prime example of what makes a rare cancer so challenging: an absence of basic research tools, published information, and funding that results in a lack of treatment options. And without specific treatments, patients have little hope for survival.


Nabeel Bardeesy, PhD
, Gallagher Endowed Chair in Gastrointestinal Cancer Research, Massachusetts General Hospital Cancer Center;
Associate Professor, Harvard Medical School

A founding goal of TargetCancer Foundation was to change the research landscape for this disease through a long-term commitment to the Bardeesy Lab at the Massachusetts General Hospital Cancer Center. In 2015, that funding extended from the lab to the clinic for the first time, as TargetCancer Foundation initiatives led directly to a new cholangiocarcinoma clinical trial.

Through strategic seed funding to Dr. Nabeel Bardeesy’s lab, research has progressed faster than ever expected by focusing on key research elements necessary to build a comprehensive research program:

Understanding driver mutations. Among other studies, TargetCancer Foundation has supported research into the functions of the IDH1 and 2 genes, which are mutated in about 30% of intrahepatic cholangiocarcinoma cases. The Bardeesy lab published a groundbreaking paper in Nature (July 2014) on the effects of mutant IDH1/2 in causing cholangiocarcinoma. This research informs the design and interpretation of clinical trials using mutant IDH inhibiting drugs, and holds great promise for the identification of effective cholangiocarcinoma treatments.

In 2016, funding was extended to also support research into the role of FGFR2 gene alteration, which appears in about 10% of intrahepatic cholangiocarcinoma cases and hold great potential as a therapeutic target.


As published in Nature in July 2014.

Cell lines and patient-derived xenografts. The TargetCancer Foundation Cholangiocarcinoma Cell Line Bank was established in 2012 in response to the need for a collection of well-characterized cholangiocarcinoma cell lines and associated xenografts.

Community building. No single lab can take on the tremendous task of studying a rare cancer. Through programs like the TargetCancer Foundation Think Tank on Cholangiocarcinoma, we have helped build a collaborative community of researchers across disease areas.

Animal models. With our help, the Bardeesy Lab has successfully engineered a series of mouse models representing several different genetic mutations, allowing for a deeper study of the events driving the development of cholangiocarcinoma in humans.

These areas of focus have led to tremendous progress in a relatively short time, and the impact for patients is real. The first clinical trial informed by TargetCancer Foundation-funded research opened in 2014, and a second trial that opened in 2015 is currently enrolling patients. The potential for the Bardeesy Lab to continue driving research forward remains strong, as does TargetCancer Foundation’s support for these initiatives.

Grants awarded

$707,000 over six years


Nabeel Bardeesy, PhD
Andrew Zhu, M.D., PhD




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