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Item
#3
Gene
Therapy Seen Hopeful for Patients with Diabetes
New
study using a genetic growth factor to repair diabetes-damaged
nerves begins.
The
first patient has been enrolled in a groundbreaking gene therapy
study at Caritas St. Elizabeth's Medical Center that seeks to use
a genetic growth factor to repair diabetes-damaged nerves.
The
National Institutes of Health gave us the OK two weeks ago, and
we've enrolled our first patient,'' said Dr. Alan Ropper, chief of
neurology. ``It's exciting. This is the first trial using a gene
for a nerve disease of this sort.''
In
the $10.2 million federally funded study, which will enroll 196
patients over four years, doctors will inject vascular endothelial
growth factor, or VEGF, into the muscles near the damaged
peripheral nerves in patients' legs.
The
idea is that the DNA in the growth factor will be picked up by
cells in the nerves and produce tiny new blood vessels to nourish
and restore the damaged nerves.
“We
have strong evidence that it happens in experimental (animal)
models,'' Ropper said.
Diabetic
neuropathy is a common complication among diabetics. In the
disease, the peripheral nerves in the hands, legs and feet become
damaged, resulting in pain and numbness. The condition is caused
when the tiny blood vessels in the nerves are blocked.
There
is no cure at present. Many patients take anticonvulsive
medications or painkillers.
The
study follows the work of the late gene therapy pioneer Dr.
Jeffrey Isner, who used a similar growth factor to grow new blood
vessels for patients with heart and leg blockages.
In
the process of studies on the leg patients, researchers noticed
that many of them, including some diabetics, were seeing their
neuropathic pain improved, Ropper said.
The
new study specifically targets neuropathy.
While
gene therapy trials have been controversial, Ropper noted that
this trial involves injecting the DNA directly into patients'
muscles, rather than using a virus to deliver the material. Many
gene therapy trials that have run into problems used a virus as a
delivery agent.
Ropper
said gene therapy also may be able to help those with peripheral
nerve damage from other causes, such as chemotherapy.
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