possible cure for type 1

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Let's hope they start human tests that turn ooutt so well.

Experimental Cure for Type 1 Diabetes Has Almost 80-percent Success Rate

6-Jun-2011

An experimental cure for Type 1 diabetes has a nearly 80 percent success

rate in curing diabetic mice. The results, presented at The Endocrine

Society's 93rd Annual Meeting in Boston, offer possible hope of curing a

disease that affects 3 million Americans.

" With just one injection of this gene therapy, the mice remain diabetes-free

long term and have a return of normal insulin levels in the body, " said

Vijay Yechoor, MD, the principal investigator and an assistant professor at

Baylor College of Medicine in Houston.

Yechoor and his co-workers used their new gene therapy in a no obese mouse

model of Type 1 diabetes. The therapy attempts to counter the two defects

that cause this autoimmune form of diabetes: autoimmune attack and

destruction of the insulin-producing beta cells by T cells. First, the

researchers genetically engineer the formation of new beta cells in the

liver using neurogenin3. This gene defines the development of pancreatic

islets, which are clusters of beta cells and other cells. Along with

neurogenin3, they give an islet growth factor gene called betacellulin to

stimulate growth of these new islets.

The second part of the therapy aims to prevent the mouse's immune system

from killing the newly formed islets and beta cells. Previously the research

team combined neurogenin3 with the gene for interleukin-10, which regulates

the immune system. However, with that gene, they achieved only a 50 percent

cure rate in diabetic mice, Yechoor said.

In the new study, the investigators added a gene called CD274 or PD-L1

(programmed cell death 1 ligand-1). It inhibits activity of the T cells only

around the new islets in the liver and not in the rest of the body, he

explained.

" We want the gene to inactivate T cells only when they come to the new islet

cells. Otherwise, the whole body would become immunocompromised, " Yechoor

said.

This treatment reversed diabetes in 17 of 22 mice, or 78 percent. Diabetic

mice that otherwise live only six to eight weeks were growing normally and

were free of diabetes as long as 18 weeks after injection of the gene

therapy, Yechoor said.

This treatment approach, he said, " has the potential to be a curative

therapy for Type 1 diabetes. "

The other mice reportedly responded to the gene therapy initially but then

became diabetic again. There are two possibilities, according to Yechoor,

why the therapy did not achieve a 100 percent cure rate.

" T cells are the predominant part of islet destruction, but other pathways,

including beta cells could also contribute, meaning we would need to target

those pathways as well, " Yechoor said. " Or maybe the efficiency of this new

protective gene is not sufficient, and we need to give a larger dose. "

Funding for this study came from the National Institutes of Health and the

Juvenile Diabetes Research Foundation.

Source: The Endocrine Society

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