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Published on 29 April 2009, 08:39


Categories: Diabetes Diabetic Polyneuropathy VEGF

Gene Therapy Shows Potential in Treating Diabetic Polyneuropathy

Boston, MA - Intramuscular injections of vascular endothelial growth factor (VEGF) gene show promise for improving neuropathic symptoms in individuals with diabetic polyneuropathy, a condition most commonly affecting diabetics who have had the disease for many years. These findings were presented in April at the American Academy of Neurology Annual Meeting in Seattle.

Symptoms associated with diabetic polyneuropathy include loss of sensation and pain in the legs and feet, weakness, and imbalance. Due to loss of sensation, the development of ulcerations on the feet often go undetected, potentially resulting in amputation, a procedure 15 times more prevalent in diabetics.

Study participants who received the VEGF gene injection had improvement in sensory symptoms and pain, though not in many other characteristics such as nerve conduction tests. “Most patients had fairly severe neuropathy and the expectation for improvement was therefore not high” said Allan Ropper, MD, executive vice chair of the Department of Neurology at Brigham and Women’s Hospital (BWH).

Preclinical studies using animal models have shown that injecting VEGF into the muscle adjacent to nerve trunks can improve clinical function, blood flow in the nerves, and electrophysiological function.

Thirty nine patients were randomly chosen to receive three sets of intramuscular injections of plasmid VEGF in one leg while 11 patients received a placebo in one leg. The injections were given at eight standardized sites adjacent to the sciatic, tibial, and peroneal nerves. Six months after treatment, participants receiving the VEGF injection had greater improvement in symptoms in the injected leg than those receiving the placebo.

In this study, the investigators used a form of the gene that is active without packaging it in a virus, offering a major advantage in safety. “The study shows that this form of gene transfer therapy can be performed relatively safely but further investigation using a larger study group is needed before it can be introduced as a mainstream therapy,” said Dr. Ropper, noting that this study was limited by its small size and rigid participation criteria.

Brigham and Women's Hospital (BWH) is a 777-bed nonprofit teaching affiliate of Harvard Medical School and a founding member of Partners HealthCare, an integrated health care delivery network. In July of 2008, the hospital opened the Carl J. and Ruth Shapiro Cardiovascular Center, the most advanced center of its kind. BWH is committed to excellence in patient care with expertise in virtually every specialty of medicine and surgery.  The BWH medical preeminence dates back to 1832, and today that rich history in clinical care is coupled with its national leadership in quality improvement and patient safety initiatives and its dedication to educating and training the next generation of health care professionals. Through investigation and discovery conducted at its Biomedical Research Institute (BRI), BWH is an international leader in basic, clinical and translational research on human diseases, involving more than 860 physician-investigators and renowned biomedical scientists and faculty supported by more than $416 M in funding. BWH is also home to major landmark epidemiologic population studies, including the Nurses' and Physicians' Health Studies and the Women's Health Initiative. For more information about BWH, please visit www.brighamandwomens.org

Contact: Lori Shanks, Senior Media Relations Specialist, 617-534-1604, Ljshanks@partners.org

Source: Brigham and Women's Hospital (BWH)


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Categories: Diabetes Diabetic Polyneuropathy VEGF