Gene Therapy May Help With Diseased Limbs

A U.S. study suggests gene therapy is a safe method to explore in patients whose lower limbs are at risk for amputation because of poor circulation.

In a Phase I clinical trial almost half the patients receiving gene therapy reported complete resolution of chronic pain one year after treatment, and more than one-quarter of patients with chronic wounds experienced complete healing of those ulcers in the same timeframe.

The researchers are the first to report on testing of the effects of the hypoxia-inducible factor-1 alpha — HIF-1a — gene as the basis of treatment for limbs damaged by compromised blood flow, according to the report published in the March 13 issue of the journal Circulation.

Though the trial largely focused on the therapy’s safety, the bottom line is that 34 patients improved to varying degrees with this treatment, said first author Dr. Sanjay Rajagopalan of the Ohio State University Medical Center.

The treatment is currently being tested in a major Phase II clinical trial in the United States and Europe.

If this gene therapy approach were to prove safe and effective after exhaustive testing in Phase III studies, it would provide clinicians with an alternative approach to treating patients with serious blood flow problems in their lower limbs, Rajagopalan said.