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A Single Injection May Mean a Permanent Cure

Reversing autoimmune type 1 diabetes without immunosuppression has proven to be extremely difficult, but Pittsburgh researchers have achieved that outcome in pre-clinical trials with an engineered, safe virus that does gene therapy.

Julie Grant, reporting for Pittsburgh’s local CBS affiliate, KDKA, recently visited with McGowan Institute for Regenerative Medicine affiliated faculty member George Gittes, MD (pictured), Director of the Richard King Mellon Foundation Institute for Pediatric Research and Co-Scientific Director at UPMC Children’s Hospital of Pittsburgh of UPMC. She learned that reversing autoimmune type 1 diabetes without immunosuppression has proven to be extremely difficult, but Dr. Gittes and researchers at Children’s Hospital and the University of Pittsburgh School of Medicine have achieved that outcome in pre-clinical trials with an engineered, safe virus that does gene therapy.

“We infuse the viruses into the pancreatic tissue. It permeates through the pancreas, but it finds the cells that are the ones that have the capability to turn into insulin cells and it makes that happen,” said Dr. Gittes.

The researchers have pioneered a novel procedure — an infusion process using an endoscope that delivers the virus directly to the pancreas, so other cells in the body are not affected.

“In a human, we could go through the mouth, down the stomach, to where the pancreas opening is, infuse the virus back up into the pancreas,” said Dr. Gittes.

One infusion could mean long-term results that would allow a diabetic patient to regulate their own blood sugar, replacing the need for insulin injections.

“The idea that you could do a single injection and see a permanent change is very exciting,” said Dr. Gittes.

A clinical trial for gene therapy to treat diabetes has never been done. Dr. Gittes’ team appears to be closer than ever, already having success in pre-clinical trials.

“Once we get a consistent result …, we will then go to the FDA and present them with the trial we want to do in diabetic patients,” said Dr. Gittes.

According to Dr. Gittes, they are pretty close to being ready for a clinical trial. This means closer, than perhaps ever before, to a cure.

In type 1 diabetes, the body mistakenly recognizes the insulin-producing ‘beta’ cells in the body as foreign and kills them, resulting in high blood sugar levels. Patients require lifelong insulin therapy either through injections or an insulin pump. The team’s current work however, represents a major advance in efforts to develop a long-term therapeutic approach by stimulating the body’s own pancreatic cells to produce insulin.

Illustration: McGowan Institute for Regenerative Medicine.

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KDKA/CBS Pittsburgh w/video (11/14/18)

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