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- Posted Thursday February 13, 2014
TGen and Mayo Clinic use genomic sequencing to help identify new therapies for bile duct cancer
Study targets EGFR and FGFR cellular pathways to treat rare disease
SCOTTSDALE, Ariz. - Feb. 13, 2014 - 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 cancer 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 genetic
alterations 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
cholangiocarcinoma."
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 patients, 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.
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About Mayo Clinic
Recognizing 150 years of serving humanity in 2014, Mayo Clinic is
a nonprofit worldwide leader in medical care, research and
education for people from all walks of life. For more information,
visit 150years.mayoclinic.org, www.mayoclinic.org and newsnetwork.mayoclinic.org.
About the Mayo Clinic Center for Individualized
Medicine
The Mayo Clinic Center for Individualized Medicine is home to the
Individualized Medicine Clinic, the world's first integrated,
multidisciplinary genomics clinic, serving patients with advanced
cancer and complex diagnoses. The center discovers and integrates
the latest in genomic, molecular and clinical sciences into
personalized care for each Mayo Clinic patient. Visit
http://mayoresearch.mayo.edu/center-for-individualized-medicine
for more information.
Media Contact:
Sam Smith
Mayo Clinic Public Affairs
507-284-5005
[email protected]
*
About TGen
Translational Genomics Research Institute (TGen) is a Phoenix,
Arizona-based non-profit organization dedicated to conducting
groundbreaking research with life changing results. TGen is focused
on helping patients with cancer, neurological disorders and
diabetes, through cutting edge translational research (the process
of rapidly moving research towards patient benefit).TGen
physicians and scientists work to unravel the genetic components of
both common and rare complex diseases in adults and children.
Working with collaborators in the scientific and medical
communities literally worldwide, TGen makes a substantial
contribution to help our patients through efficiency and
effectiveness of the translational process. For more information,
visit:www.tgen.org.
Press Contact:
Steve Yozwiak
TGen Senior Science Writer
602-343-8704
[email protected]