Clinical Trial of Diabetes Regenerative Therapy Shows Positive Results

New York, NY, March 8, 2007 — One of JDRF's industry partners, Transition Therapeutics, Inc., recently announced interim data from an exploratory Phase IIa clinical trial of a diabetes regenerative product, E1-INT, a therapeutic combination product that may be effective at regenerating the insulin producing beta cells that are lost when people develop type 1 diabetes.  

In the small Phase IIa trial, both type 1 and type 2 diabetes patients showed improvements in important measures of blood glucose control.  Among type 1 patients receiving the drugs for four weeks, more than half decreased their average daily insulin usage by more than 20 percent, or reduced their HbA1c levels (a long-term measure of blood sugar control) by 1.2 to 2 percent in the months post-treatment.

"These early clinical results of a beta cell regenerative product are quite encouraging and underscore the possible potential of beta cell regenerative therapeutics in diabetes," noted Dr. Richard Insel, Executive Vice President of Research at JDRF.

The therapeutic combination used in the trial is based on preclinical studies funded by JDRF in the late 1980s.  With a JDRF grant, scientists identified that the hormone gastrin had beta cell regenerative therapeutic potential. Continued testing through the 1990s of gastrin in combination with growth factors demonstrated that the combination of the growth factor EGF with gastrin, had distinct regenerative potential in animals. In 2005, in a project funded partly by JDRF, scientists showed that the EGF-gastrin combination induced human islets to multiply in mice.

Transition Therapeutics took over testing of the EGF-gastrin therapy, using human analogues of these proteins and named the therapy E1-I.N.T.  If combined with therapies that block the autoimmune attack, a treatment that spurs beta cell regeneration could potentially lead to a cure for type 1 diabetes.

Regeneration: A Field With Great Promise
Although JDRF did not fund the E1-I.N.T. trials, it is currently partnering with Transition Therapeutics to conduct clinical trials of a similar regenerative therapeutic combination product, GLP1-I.N.T.  The GLP1-I.N.T. combination is a second-generation regenerative product, which could have even better effects than the E1-I.N.T. combination.  JDRF is providing up to $4 million to the project over two years, to push this diabetes regenerative product into Phase II clinical trials in type 1 diabetes patients. These trials are set to begin later this year.

This partnership between JDRF and Transition Therapeutics is facilitated through JDRF's Industry Discovery and Development Partnership program, which allows JDRF to partner with pharmaceutical, biotech, and medical device businesses that are looking to develop drugs, treatments, technologies, and other therapeutics leading to a cure, reversal or prevention of type 1 diabetes and its complications.

In addition to providing hope for restoring the ability to make insulin, beta cell regeneration could be an essential complement to other diabetes therapies, such as islet transplantation. For example, JDRF is funding a project at City of Hope National Medical Center in Los Angeles investigating whether giving EGF and gastrin to patients after an islet transplant will allow them to produce enough insulin even when receiving islets from a single donor and to have prolonged function of the transplanted islets. Currently, two or more donors are usually needed for a successful islet transplant outcome, limiting the number of patients who can be treated, and the transplanted islets lose function after several years.

For additional information about the Transition Therapeutics clinical study, please visit http://www.transitiontherapeutics.com/news/article.php