Cholangiocarcinoma Clinical Trial

What if there is an existing, FDA approved drug that is effective against an untreatable cancer like cholangiocarcinoma, but we don’t know about it?

It is a question that Paul Poth asked himself when creating TargetCancer Foundation after his own cholangiocarcinoma diagnosis. Six years later, TargetCancer Foundation’s investment in cholangiocarcinoma research at the Bardeesy Lab has led to a new clinical trial that is testing the effectiveness of dasatinib- an existing, FDA-approved treatment, currently used to treat leukemia– against a specific sub-type of cholangiocarcinoma.

Click here to learn more about the Patient Assistance Fund for patients enrolled in this trial.

Before his death in 2009, Paul Poth provided TargetCancer Foundation’s first grant to the Center for Molecular Therapeutics (CMT) at Massachusetts General Hospital, a lab using innovative technology to test cancer cell lines against existing treatments. That grant set in motion a timeline of events that would rapidly lead to this new trial:

  • 2012: The TargetCancer Foundation Cholangiocarcinoma Cell Line Bank is created in an effort to create cell lines, which are traditionally lacking in rare cancers like cholangiocarcinoma.
  • 2013: The Cell Line Bank creates a robust repository of cholangiocarcinoma cell lines at the Massachusetts General Hospital Cancer Center.
  • Late 2013: Those cell lines are entered into the screening program at the CMT where they are tested against hundreds of different drugs in many different combinations.
  • Early 2014: Cholangiocarcinoma cell lines with a particular genetic alteration show a response to dasatinib- an existing, FDA-approved drug already being used to treat leukemia.
  • Spring 2015: These results are confirmed in the laboratory and fast-tracked to a phase two clinical trial, financially sponsored by TargetCancer Foundation.

TargetCancer Foundation’s support has been absolutely critical to the success of our project. With their seed funding, we were able conceive and launch our entire program seeking to identify the Achilles Heel of specific subtypes of cholangiocarcinoma. It is fitting that TargetCancer Foundation, who has been with us each step of the way, is now helping to enable us to launch a clinical trial which we hope will provide real options for patients with the terrible disease. – Dr. Nabeel Bardeesy

On average, it can take 10 years and cost $1 billion to bring a brand new drug to market. Skipping that process and repurposing an existing, FDA-approved drug eliminates the lengthy research, development, and approval process. Most importantly, it rapidly makes the treatment available to patients who need it. TargetCancer Foundation is proud to bring its founding goals full circle in such a short time to potentially help bring a new treatment to patients.

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