​Gene Therapy Could Be Useful Treatment For Diabetic Incontinence, Suggest Early Studies At University Of Pittsburgh

DALLAS, May 4, 1999 — Researchers at the University of Pittsburgh Medical Center (UPMC) have for the first time demonstrated the feasibility of using gene therapy to treat urinary incontinence in patients with diabetes. Their results, which were presented today at the American Urological Association 1999 annual meeting, have implications for the more than half of all diabetics who suffer from diabetic cystopathy, for which there is no treatment.

Diabetes causes a number of complications, some of which are associated with defects in the nervous system or damaged nerves. Such is the case with diabetic cystopathy, whereby the signals between the brain and the bladder are impaired, and it becomes impossible to control the urge to empty the bladder.

Because patients with diabetes do not have a sufficient amount of a substance called nerve growth factor (NGF), which in healthy people serves to heal or fortify weakened or injured nerve cells, the UPMC researchers surmised that gene therapy delivery of NGF could provide a potential treatment for incontinence. Results from their animal studies—the first to explore gene therapy for incontinence—suggest they are on the right track.

"We are encouraged that our study may soon translate into real clinical benefit for diabetic patients," said Michael B. Chancellor, M.D., associate professor of urologic surgery at the University of Pittsburgh School of Medicine.

In the study, rats with induced defects of the bladder—an animal model for diabetic cystopathy—received genes with NGF. Researchers inserted the gene into a herpes simplex virus type 1 vector and delivered it directly to the bladder and to nerves leading to the bladder.

"The gene was expressed and the animals had no side effects as a result of the procedure," reported Naoki Yokoyama, M.D., a member of Dr. Chancellor’s research team.