Targeting the EGFR and FGFR cellular pathways for bile duct cancer

February 14, 2014 by Steve Yozwiak

Researchers at the Translational Genomics Research Institute (TGen) and physicians at Mayo Clinic's Individualized Medicine Clinic have personalized drug treatments for patients with cholangiocarcinoma using genomic sequencing technologies.

Potential new treatment approaches are being validated to develop new tests that physicians can use to guide therapy for this aggressive of the bile ducts that progresses quickly and is difficult to treat.

Clinically important findings suggest that targeting the EGFR and FGFR cellular pathways may benefit thousands of patients with this disease, according to the study published today in the journal PLOS Genetics.

Half of the patients treated in this study responded to either ponatinib (typically used for certain types of leukemia) or pazopinib (a kidney cancer drug), depending on the identified through sequencing.

"In 3 out of the 6 patients we analyzed, we found compelling, treatable and unexpected genetic alterations that would never have been found by normal testing methods for cholangiocarcinoma," said Dr. Mitesh Borad, a Mayo Clinic oncologist and lead author of the paper. "We treated these three patients with drugs that attack these genetic alterations and saw tumor shrinkage. This gives us hope for better treatments for this aggressive, hard-to-treat cancer."

Because of these encouraging early results, the team of 49 doctors and researchers proposes large-scale clinical trials to test EFGR and FGFR inhibitors as possible treatments for biliary tract cancers that harbor mutations in these genes and pathways.

"Our results demonstrate that if we find the right molecular context, more appropriate therapies can be chosen that improve outcomes," said Dr. John Carpten, TGen Deputy Director of Basic Science and Director of TGen's Integrated Cancer Genomics Division, and the study's senior author. "We now hope to design larger clinical studies to treat patients' tumors harboring these novel genomic aberrations to further explore the precise extent of clinical benefit for patients with primary or advanced ."

Bile duct cancer is a rare cancer that occurs mostly in people older than age 50. Surgical approaches, such as resection and liver transplantation, represent the only curative treatment approaches, said Dr. Borad. Most , however, present with surgically unresectable or metastatic disease at the time of diagnosis. Standard-of-care chemotherapies are not curative and there is an unmet need for newer approaches, Dr. Borad added.

Explore further: Study uncovers possible genetic markers in breast cancer that spreads to the brain

Related Stories

Study uncovers possible genetic markers in breast cancer that spreads to the brain

February 10, 2014
The Translational Genomics Research Institute (TGen) has uncovered possible genetic origins of breast cancer that spreads to the brain, according to a first-of-its-kind study published in the scientific journal PLOS ONE.

First large-scale study of whole-genome testing helps identify best treatment for women with advanced breast cancer

February 6, 2014
The first large-scale study testing all the DNA—the entire genome—of tumor cells from more than 400 women with advanced breast cancer has identified individuals with a good chance of benefiting from specific treatments ...

Scientists propose a breast cancer drug for bladder cancer patients

January 22, 2014
Researchers at Mayo Clinic have found amplification of HER2, a known driver of some breast cancers, in a type of bladder cancer called micropapillary urothelial carcinoma (MPUC) and have shown that the presence of HER2 amplification ...

Whole genome sequencing of rare olfactory neuroblastoma

May 23, 2012
The Translational Genomics Research Institute (TGen) and the Virginia G. Piper Cancer Center at Scottsdale Healthcare have conducted whole genome sequencing (WGS) of a rare nasal tract cancer called olfactory neuroblastoma ...

Study shows ability to do next-generation sequencing for patients with advanced cancers

October 30, 2013
A pilot study led by the Translational Genomics Research Institute (TGen) and the Virginia G. Piper Cancer Center at Scottsdale Healthcare shows that, even for patients with advanced and rapidly transforming cancer, researchers ...

TGen-US Oncology data guides treatment of metastatic triple-negative breast cancer patients

December 6, 2012
Genomic sequencing has revealed therapeutic drug targets for difficult-to-treat, metastatic triple-negative breast cancer (TNBC), according to an unprecedented study by the Translational Genomic Research Institute (TGen) ...

Recommended for you

Gene variants identified that may influence sexual orientation in men and boys

December 8, 2017
(Medical Xpress)—A large team of researchers from several institutions in the U.S. and one each from Australia and the U.K. has found two gene variants that appear to be more prevalent in gay men than straight men, adding ...

Disease caused by reduction of most abundant cellular protein identified

December 8, 2017
An international team of scientists and doctors has identified a new disease that results in low levels of a common protein found inside our cells.

Study finds genetic mutation causes 'vicious cycle' in most common form of amyotrophic lateral sclerosis

December 8, 2017
University of Michigan-led research brings scientists one step closer to understanding the development of neurodegenerative disorders such as ALS.

Mutations in neurons accumulate as we age: The process may explain normal cognitive decline and neurodegeneration

December 7, 2017
Scientists have wondered whether somatic (non-inherited) mutations play a role in aging and brain degeneration, but until recently there was no good technology to test this idea. A study published online today in Science, ...

Researchers find genes may 'snowball' obesity

December 7, 2017
There are nine genes that make you gain more weight if you already have a high body mass index, McMaster University researchers have found.

Gene therapy shows promise against blood-clotting disease

December 7, 2017
Gene therapy has freed 10 men from nearly all symptoms of hemophilia for a year so far, in a study that fuels hopes that a one-time treatment can give long-lasting help and perhaps even cure the blood disease.

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.