A JDRF-funded clinical trial has shown that a drug used to treat certain types of cancer can protect beta cells, and therefore preserve insulin production, in people newly diagnosed with type 1 diabetes.
The trial, conducted in the US through the international Type 1 Diabetes TrialNet consortium and published in the New England Journal of Medicine, provides the first conclusive evidence that it is possible to target particular types of immune cells to reduce or prevent the onset of type 1 diabetes.
The compound, called rituximab, specifically targets a type of immune cell known as B cells. B cells organize the immune system and are responsible for telling other cells when and where to attack. Previous research has shown that reducing the number of B cells in mice predisposed to type 1 diabetes resulted in the disease being delayed and even prevented. The big question was whether it would also be the case in humans.
Excitingly, it was. Researchers reported that patients given rituximab treatment had a significantly slower disease progression, better blood glucose control and lower insulin requirements compared to the control group.
Some challenges were experienced, however. Patients on the rituximab treatment were given four doses of the drug over a one year period yet most experienced a decline in effect soon after treatment stopped. Some patients also experienced side effects that, though relatively mild, were not desirable for long term treatment.
Researchers will now conduct further trials to look at ways of prolonging the action of the treatment to ensure the autoimmune process that causes type 1 diabetes is safely prevented in the long term.
While work is yet to be done, this research is the first step towards the development of a safe and effective way to prevent the immune attack behind type 1 diabetes — an essential prerequisite for both disease prevention ongoing protection for insulin-producing replaced or regrown through other means.