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The Pharmaceutical Journal Vol 267 No 7163 p281-283
1 September 2001

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Compound stops autoimmune response in diabetes

A compound that might be useful for treating autoimmune diseases mediated by pathogenic T-helper type 1 (Th1) cells has been identified.

Type 1 diabetes is one of the diseases characterised by an uncontrolled Th1 response. The compound, a-galactosylceramide (a-GalCer), activates natural killer T cells which are known to have immunoregulatory effects.

Dr Seokmann Hong, Vanderbilt University School of Medicine, Tennessee, and colleagues say that a-GalCer potentiates the T-helper type 2 (Th2) adaptive immune response. This gives protection against the effects of Th1 cells thus preventing the development of the autoimmune disease. The researchers found that administration of a-GalCer prevented development of diabetes in mice. The researchers comment: “a-GalCer might be useful for the treatment of a variety of diseases that are mediated by pathogenic Th1 cells.” They add: “Our findings identify natural killer T cells as novel targets for immunotherapy.” (Nature Medicine 2001; 7:1052.)

A second group of researchers also has evidence to support the role of a-GalCer in preventing diabetes through activating natural killer T cells (ibid, p1057). They report: “a-GalCer treatment protects female non-obese diabetic mice from type I diabetes.”

In addition, they found that a-GalCer prolonged the survival of pancreatic islets transplanted into newly diabetic non-obese mice. Transplanted grafts survived for 22 weeks in mice treated with a-GalCer compared with 10 days in a control group.

They suggest that the treatment might be useful in preventing diabetes both at an early stage and later on, after the onset of insulitis. It could dampen the recurrent autoimmune response in patients with diabetes who receive islet cell transplants, they add.

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