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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.