From Mice to Man: Innovative Therapy Mitigates Type 1 Diabetes

  • Posted on November 13, 2014 by Pharma Models Blogging Team in Diabetes

Researchers have now developed an antibody therapy to treat Type 1 diabetes through the use of mouse models. A human clinical trial is now underway to see if the therapy rituximab is just as effective in people, according to the American Association for the Advancement of Science.

Type 1 diabetes is a serious disorder that takes place when the immune system destroys insulin-producing cells in the pancreas. Scientists spent many years targeting T cells because they destroy the tissues, but more recently, researchers have begun looking at B cells, which stimulate T cells by presenting antigens.

The drug rituximab, which also treats rheumatoid arthritis, was used to kill off the B cells. A network of scientists has fundraised for the inception of a clinical trial to test the drug in patients with Type 1 diabetes. So far, 82 people have enrolled and it will take at least one more year to finish the research.

However, some researchers have been concerned with the small amount of animal studies conducted on this drug. This is why immunologist Li Wen of Yale University and her colleagues conducted a study in which a mouse model was genetically engineered to test the therapy.

The mice were genetically predisposed to diabetes. The researchers engineered a molecule that rituximab targets on the external part of the mouse models’ B cells. The researchers designed a rituximab-like antibody to test on the animal models. The results show that, after 35 weeks, about 70 percent of the mice receiving the antibody were diabetic while nearly 100 percent in the placebo group had diabetes.

Wen explained that the mice had a significant delay in acquiring diabetes, which was equivalent to 10 or 15 years of a delay in human patients. The researchers found that the new B cells the mice grew were less likely to produce autoantibodies, which usually occurs with Type 1 diabetes. This shows that the drug was able to improve the functioning of the immune system as well as destroy the original B cells present in the mice.

A study published in the Diabetes Care journal explains further use of the monoclonal antibody rituximab for treating Type 1 diabetes. One case study is reviewed in which a young woman is diagnosed with the disease as well as a low platelet count. After administration of a course of rituximab, her platelet count became normal and her need for insulin dosages decreased significantly until it was no longer necessary.

Before clinical trials, animal studies are first needed to test rituximab and other medications. Clearly, animal models impact drug development for a variety of diseases including Type 1 diabetes.

The drug rituximab does have some side effects like rashes and infections. While not every drug that works in mice has the same results in humans, all medicines that are prescribed to human patients were once successful in animal models. The rituximab medication may have a bright future depending on future clinical trials.

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