BEER-SHEVA, Israel—After almost a century, is the end for diabetes near? “There have been no major advancements toward a cure for type 1 diabetes for nearly 100 years, but now we’re pulling the rug out from under it,” says Dr. Eli Lewis, director of the Clinical Islet Laboratory of the Department of Clinical Biochemistry and Pharmacology at Ben-Gurion University of the Negev (BGU). A clinical study on a promising treatment for type 1 diabetes (T1DM) developed by Dr. Lewis in collaboration with researchers at the University of Colorado had an “exceptional outcome” after an eight-week treatment of alpha1-antitrypsin (AAT). 
Lewis describes the clinical trial results as “an excellent beginning in our mission to determine the exciting possibilities of a safe therapy for autoimmune diabetes.” He believes that researchers “will see similar results in a number of U.S. patients who recently received this treatment outside the trials within several months of diagnosis and are still completely insulin-free.”
The researchers are now recruiting for three more extension trials. However, it will still be a minimum of two years for AAT to receive U.S. Food and Drug Administration (FDA) approval as an on-label treatment for T1DM. Because AAT was already approved by the FDA, it received fast-track approval into human clinical trials in the United States, Lewis says.
“Tissue damage actually plays a role in type 1 diabetes, which affects about 8.3 percent of the U.S. population and requires insulin treatment,” Lewis explains. “When the pancreatic tissue is damaged, it doesn’t produce insulin, which allows the body to remove glucose from the blood into storage locations such as liver and muscle. Tying to fix the damage can lead to more damage.”
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Read MoreIn 2003, Lewis began to study the role of inflammation in injured islets, the insulin-producing cells in the pancreas. His first paper on the subject was published in 2005. He discovered that AAT, an anti-inflammatory drug based on a natural protein the body produces each day, shows promise for reducing insulin dependence. AAT, which is generally used to treat emphysema, can actually cure a person of T1DM, he maintains.
The islets are the targets of an autoimmune response in T1DM. Healthy people produce systemic AAT in the liver to repair tissue and reduce inflammation. While AAT is present in patients with T1DM, it does not function in its glycated form.
According to the study published in the Journal of Clinical Endocrinology & Metabolism, the goal was to evaluate safety and effectiveness of a novel therapy using the anti-inflammatory serum protein AAT in T1DM patients in the first of three published Phase 1 open trials that took place at BGU, the Barbara Davis Center for Childhood Diabetes at the University of Colorado School of Medicine and the Joslin Diabetes Center (affiliated with Harvard Medical School).
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Read MoreRecently diagnosed patients received weekly injections of functioning AAT in the form of a liquid slow-drip infusion, then a yearlong follow-up with 12 patients.
“Patient compliance was maximal, an impressive achievement when one considers several ongoing trials in Type 1 Diabetes that offer poorly tolerated approaches,” Lewis says, adding: “Following treatment of eight to 12 weeks with AAT, in several patients, it allowed proper glucose levels to be controlled without the need for any insulin injections for more than two years. The patients, even youngsters and adolescents, showed no adverse affects and a remarkable safety profile. They basically regained the ability to fight inflammation and protect damaged cells from aberrant immune responses. After the AAT therapy was withdrawn, several patients showed that glucose levels were controlled without the need for insulin injections for more than two years.”
Lewis describes the clinical trial results as “an excellent beginning in our mission to determine the exciting possibilities of a safe therapy for autoimmune diabetes.” He believes that researchers “will see similar results in a number of U.S. patients who recently received this treatment outside the trials within several months of diagnosis and are still completely insulin-free.”
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Read MorePatients recently diagnosed with the disease demonstrated better results than patients who have had the disease for a long time. Different age groups behave differently as well.
The researchers are now recruiting for three more extension trials. However, it will still be a minimum of two years for AAT to receive U.S. Food and Drug Administration (FDA) approval as an on-label treatment for T1DM. Because AAT was already approved by the FDA, it received fast-track approval into human clinical trials in the United States, Lewis says.
The study was funded by Omni Bio Pharmaceutical Inc. Dr. Charles A. Dinarello of the University of Colorado has received research support from the National Institutes of Health, via grant AI 15614.
“This is a natural substance that the body produces to heal itself,” Lewis concludes. “We hope that other diseases can be addressed by it too.”
