WRITER: JENNIFER CRONIN
CONTACT: BECKY SOGLIN
2130 Medical Laboratories
Iowa City IA 52242
(319) 335-6660; fax (319) 335-8034
Release: Sept. 7, 1999
UI research makes possible gene therapy trial for MD
IOWA CITY, Iowa -- The efforts of a University of
Iowa Health Care researcher and members of his lab are making it possible
for researchers at another university to begin testing what could be a promising
treatment for limb girdle muscular dystrophy: gene therapy.
Kevin Campbell, Ph.D., UI professor of physiology
and biophysics, and neurology, and a Howard Hughes Medical Institute Investigator,
has spent much of his career focused on understanding and trying to find ways
to treat muscular dystrophy, which is a group of hereditary diseases characterized
by progressive muscle weakness and degeneration.
On Thursday, Sept. 2, Ohio State University researchers
started the first human study looking at gene therapy as a way to treat limb
girdle muscular dystrophy, thanks in part to Campbell's work. The limb girdle
type of muscular dystrophy involves initial deterioration of shoulder and
pelvic girdle muscles, with relative sparing of other muscle groups. It is
thought that limb girdle muscular dystrophy affects between one in 20,000
and one in 50,000 individuals.
Many researchers believe that gene therapy is one
of the most promising possibilities for treating muscular dystrophy, as well
as many other genetic diseases. Gene therapy involves using a vector, often
a disabled cold virus, as a vehicle to supply cells with the needed materials
to correct a defect and thus treat a disease and/or its symptoms. In the case
of limb girdle muscular dystrophy, researchers injected the muscles' cells
with an adenovirus carrying a protein particle called alpha sarcoglycan, which
is necessary to protect a muscle from becoming damaged.
Campbell and members of his lab have been instrumental
in many important, early advances in muscular dystrophy research. Campbell
and his team were the first researchers to explain the basic science of how
the defective sarcoglycan complex causes limb girdle muscular dystrophy.
The Campbell lab was also the first group to demonstrate
functional restoration of a muscle using gene transfer and to develop an animal
model on which to effectively test the gene therapy. In June, at the American
Society of Human Gene Therapy meeting in Washington, D. C., the Campbell
group presented findings that showed how the gene therapy
technique causes large regions of muscle to express the corrected defect.
"This work has now made it possible to begin human
trials," Campbell said. "The work at Ohio State is an initial study, but it
is an exciting start. The goal of this initial trial is to make sure that
the treatment is safe and to find out how the immune system responds to the
Ohio State researchers have begun testing the approach
on a small muscle in the foot. The trial involves six patients, one of whom
was an individual initially diagnosed by the Campbell lab.
"If initial studies are safe, then the investigators will
start to inject larger muscles," said Campbell, who will help analyze the
muscle tissue biopsies.
A second trial would involve multiple medical centers.
The UI would likely participate in the expanded investigation, which would
be designed to test the effectiveness of the gene therapy.
While the gene therapy strategy is promising, Campbell
indicated that there are still many issues to consider. He also cautioned
against thinking of gene therapy as a possible cure for muscular dystrophy
at this time.