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- Posted Tuesday March 10, 2015
Brain tumor patients put on fast track in revolutionary clinical trial
TGen, Barrow, Ivy, Karmanos 'Phase 0' studies evaluate if drugs can reach cancer in the brain
PHOENIX, Ariz. - March 10, 2015 - Brain tumor experts at Barrow Neurological Institute at Dignity Health St. Joseph's Hospital and Medical Center have launched a revolutionary fast-track approach to cancer research, giving new hope to brain cancer patients. In partnership with The Ben & Catherine Ivy Foundation, the Translational Genomics Research Institute (TGen) and the Barbara Ann Karmanos Cancer Institute, the Barrow studies are the first of their kind.
Called "Phase 0 Trials," these early-phase clinical trials
shorten the evaluation of drug therapies from an average of five
years to only six months at a fraction of the cost.
During each single-patient trial, participants are administered an
experimental drug already proven to be safe in other types of
cancer.Within hours, Barrow neurosurgeons remove the brain
tumor and within weeks it is determined if the experimental drug
penetrated the tumor and whether it is having a positive effect on
the tumor.If the drug reaches the tumor and shows activity,
it moves into a Phase 2 clinical trial for patients. If it does not
prove to be effective, the patient has the option to enter another
study.
"Brain tumors are among the deadliest of all cancers and during
the last 30 years, little progress has been made on successful
treatments to increase life expectancy," says Dr. Nader Sanai,
Director of the Barrow Brain Tumor Research Center. "This Phase 0
approach for malignant brain tumors is a game changer in cancer
research and our program is the first of its kind in the
world."
These studies are called Phase 0 clinical trials because they
occur outside of the three lengthy phases that evaluate a drug's
safety, effectiveness, side effects and dosage.After years of
research and millions of dollars, the vast majority of experimental
drugs for brain tumors have proven ineffectual and never brought to
market.
One of the major difficulties in treating brain tumors is finding
drugs that can penetrate the blood-brain barrier, which buffers the
brain from the rest of the body's blood-circulatory system. Located
along capillaries, the blood-brain barrier protects the brain from
rapid changes in the body's metabolic conditions and minimizes
exposure to molecules that are toxic to neurons in the brain.
"The main question answered by these Phase 0 clinical trials is
this: Does the drug get to the tumor?" said Dr. Michael Berens,
TGen Deputy Director for Research Resources. "Next, we look at how
much of the drug is enough, and if there are any signs of patient
benefit."
TGen is using its world-class expertise in genomic sequencing to
analyze brain tumor samples and help determine which drugs might
work best.
"Because of what is known about how each drug works, we think
there are certain genetic mutations that would make certain
patients more responsive to the drug," said Dr. Berens, who also is
Director of TGen's Cancer and Cell Biology Division and TGen's
Glioma Research Lab.
Karmanos is analyzing samples of the resected brain tumors,
following surgery, to determine how much of the drug accumulated in
the tumor, and what effect, if any, the drugs have on the
cancer.
Phase 0 trials were recently introduced by the FDA and have never
before been widely used for brain tumor patients. This is the first
non-governmental study coalition to push forward with these trials,
an initiative that has drawn interest from some of the world's
largest cancer therapy developers. The first of these Phase 0
clinical trials was made possible largely through a generous grant
by The Ben & Catherine Ivy Foundation.
"The absence of truly effective drugs for brain tumors is one of
the medical community's most critical unmet needs," says Catherine
Ivy, President of The Ben & Catherine Ivy Foundation. "These
Phase 0 clinical trials are part of a much larger research strategy
funded by the Ivy Foundation to address this need and give new hope
to brain tumor patients worldwide."
Dr. Sanai believes the trials will provide encouragement to brain
tumor patients, who often feel abandoned.
"Because of the high cost of developing and testing drugs, there
is very little in the pharmaceutical pipeline that has reached
brain tumor patients," says Dr. Sanai. "It has just been too
expensive for drug companies to develop new therapies for the
relatively small number of brain cancer patients.These Phase
0 trials can be the answer.They are safe, cost effective and
quick to give us an answer. If a new drug doesn't work, we move to
the next one without the patient losing any time. For the first
time, I can tell every brain tumor patient, 'I have something new
for you'."
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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]
About The Ben & Catherine Ivy
Foundation
The Ben & Catherine Ivy Foundation, based in Scottsdale,
Ariz., was formed in 2005, when Ben Ivy lost his battle with
glioblastoma multiforme (GBM). Since then, the Foundation has
contributed more than $50 million to research in gliomas within the
United States and Canada, with the goal of better diagnostics and
treatments that offer long-term survival and a high quality of life
for patients with brain tumors. The Ben & Catherine Ivy
Foundation is the largest privately funded foundation of its kind
in the United States. For more information, visit www.ivyfoundation.org.
About Barrow Neurological Institute
Barrow Neurological Institute at Dignity Health St. Joseph's
Hospital and Medical Center is an internationally renowned medical
center that offers care for people from throughout the world with
brain and spine diseases, disorders and injuries. More brain
surgeries are performed at Barrow than any hospital in the United
States and the Institute trains more neurosurgeons than anywhere in
the world. Barrow is also the busiest operative brain tumor center
in the United States. St. Joseph's is consistently voted among the
top hospitals in the United States for neurology and neurosurgery.
For more information about Barrow, visit www.thebarrow.org.
About the Barbara Ann Karmanos Cancer
Institute
Located in mid-town Detroit, Michigan, the Barbara Ann Karmanos
Cancer Institute is one of 40 National Cancer Institute-designated
comprehensive cancer centers in the United States. Through the
commitment of 1,000 staff, including nearly 300 physicians and
researchers on faculty at the Wayne State University (WSU) School
of Medicine, and supported by thousands of volunteer and financial
donors, Karmanos strives to prevent, detect and eradicate all forms
of cancer. Its long-term partnership with the WSU School of
Medicine enhances the collaboration of critical research and
academics related to cancer care. For more information call visit
www.karmanos.org.