(Philadelphia, PA) - Researchers at the University
of Pennsylvania Health System are studying the effectiveness
of a novel drug-delivery approach that attacks only cancerous cells
in the brain to extend the lives of people with Glioblastoma Multiforme
(GBM), the most common and aggressive form of primary brain tumors.
A highly malignant tumor that can double in size in 30 days, GBM
infiltrates the normal brain tissue surrounding the tumor. Each
year 10,000 Americans are diagnosed with GBMs, and the median survival
time after detection is 12 months.
Currently there are very limited treatment options to prevent the
rapid recurrence or progression of the tumor. However, preliminary
results of the clinical trials at Penn, called the “PRECISE
Study” using the new experimental drug IL13-PE38QQR (IL13),
show great promise, says Kevin D. Judy, MD, Associate
Professor, Department of Neurosurgery at Penn, and the study’s
principal investigator. “The drug works like Pac Man,”
notes Judy. “It goes in and eats only the leftover tumors
and is harmless to healthy brain cells. Given this monstrous disease,
one of the challenges is to search for therapies selected to attack
only tumors and not the good tissue, which is what IL13 does.”
Through convection-enhanced delivery (CED), an experimental method
using positive pressure infusion, the IL13 is delivered directly
in the area at risk for remaining tumor cells after the tumor is
removed. “The way the drug is distributed is a very important
component of this study,” Judy says. “The placement
of the catheters is the key to get the drug distributed in the right
areas to kill those stray cells.”
There are about 50 participating hospitals, and the University
of Pennsylvania Health System is the only from the region in the
study. “It is one of the largest and most comprehensive clinical
trials ever conducted to identify new and more effective treatments
for recurrent GBM,” says Judy.
The median survival rate for 97 patients on IL13 almost doubled
from 26 weeks to 44 weeks. Several patients have survived for more
than three years, and the U.S. Food and Drug Administration (FDA)
has granted IL13 rare fast-track designation to accelerate its approval.
The Phase III PRECISE trial looks at the patient’s overall
survival, quality of life during treatment, and safety of IL13,
compared to patients treated with GLIADEL® Wafer in the treatment
of first recurrent GBM following removal of the tumor. Patients
have a 2 out of 3 chance to receive the IL13 and a 1 out of 3 chance
to receive GLIADEL. GLIADEL is a form of BCNU, a known cancer drug
that is currently approved by the FDA and sold for the treatment
of recurrent or newly diagnosed GBM. GLIADEL extends median survival
by eight weeks.
Patient Sonia Goldstein Participates in Penn Study
Knowing her family’s predisposition for dementia,
Sonia Goldstein decided to get an MRI, but did not expect to hear
that a brain tumor was detected during her exam. When they learned
the alarming news, friends directed her to Dr. Kevin Judy.
In August 2004, Judy surgically removed Goldstein’s primary
tumor, but despite radiation and chemotherapy, the tumor grew back
in Spring 2005. In May 2005, Judy performed surgery to remove the
recurrent tumor and four days later inserted four catheters into
the surrounding area of her brain. The IL13 was then delivered directly
in the area at risk for remaining tumor cells for the next 96 hours.
“I was so happy to have the option to participate in the
study and to be chosen to receive this new treatment,” Goldstein
For Goldstein and her husband of 53 years, Bennett, the outcome
has given them more treasured time together with their two children
and four grandchildren. MRIs given every three months confirm the
continued effectiveness of the treatment. Her husband says she is
a “pillar of the community,” and continues to enjoy
traveling and social events at her synagogue.
For additional information on the PRECISE trial at Penn, call (215)
662-6462. For general information, visit www.PreciseTrial.com.
PENN Medicine is a $2.7 billion enterprise
dedicated to the related missions of medical education, biomedical
research, and high-quality patient care. PENN Medicine consists
of the University of Pennsylvania School of Medicine (founded in
1765 as the nation's first medical school) and the University of
Pennsylvania Health System.
Penn’s School of Medicine is ranked #2 in the nation
for receipt of NIH research funds; and ranked #4 in the nation in
U.S. News & World Report’s most recent ranking of top
research-oriented medical schools. Supporting 1,400 fulltime faculty
and 700 students, the School of Medicine is recognized worldwide
for its superior education and training of the next generation of
physician-scientists and leaders of academic medicine.
The University of Pennsylvania Health System comprises: its
flagship hospital, the Hospital of the University of Pennsylvania,
consistently rated one of the nation’s “Honor Roll”
hospitals by U.S. News & World Report; Pennsylvania Hospital,
the nation's first hospital; Penn Presbyterian Medical Center; a
faculty practice plan; a primary-care provider network; two multispecialty
satellite facilities; and home health care and hospice.