Friedreich’s ataxia (FA) is caused by a reduction in the amount of a cellular protein called frataxin. The reduction in frataxin causes oxidative stress and dysfunction of a part of the cell called the mitochondria, which is responsible for producing cellular energy.

Exenatide is a drug that was developed for the treatment of type 2 diabetes. Drugs similar to exenatide are also known to have cardiovascular and neuroprotective effects.

In a study published in January 2020, researchers showed that exenatide is able to cause a small increase in frataxin in the cerebellum of mouse models. They then went on to test this in cells taken from people with FA. In these cells, exenatide increased frataxin, reduced oxidative stress, and improved mitochondrial function.

Based on these promising results in animal and cell models, they tested exenatide in people with FA in a small clinical trial in Belgium. Five week exenatide treatment increased frataxin expression in platelets (cells in the blood) of people with FA. This small study was not designed to see changes in symptoms associated with FA, but the results could support a larger trial which could measure these effects.

There is evidence that exenatide and drugs similar to exenatide are not able to reach the brain in high concentrations, due to problems crossing the blood-brain-barrier. This is a drawback of using these drugs for a neurological condition such as FA. However, if this turns out to be true, the evidence presented in this paper shows that exploring similar drugs that are able to cross over to the brain could be beneficial to people with FA.

Read the full paper here.

Posted on 26/03/2020