On March 19th, the pharmaceutical company Stealth BioTherapeutics presented data on their molecule called SBT-589 in preclinical models of Friedreich’s ataxia (FA) at the Wellcome Trust Conference on Mitochondrial Medicine-Therapeutic Development.
SBT-589 showed protective effects on the energy centres of cells known as the mitochondria, in both cells taken from people with FA and mitochondria taken from heart cells. Further to this, SBT-589 was shown to have protective effects in a mouse model of cardiomyopathy caused by FA (thickening of heart muscle, making it harder to pump blood).
Mice treated once per day with SBT-589 showed significantly reduced thickening of the heart muscle (known as hypertrophy) and delayed mortality compared to mice not given SBT-589.
The genetic mutation in the FXN gene underlying FA leads to a deficiency in a protein called frataxin. Frataxin is needed for synthesis of ATP molecules, which release energy when broken down. Heart cells are highly dependent on ATP as an energy source to continuously pump blood around the body. Mitigating heart disease in FA is a crucial aim of disease-modifying therapies for the condition, as heart failure and sudden cardiac events are among the leading causes of mortality in those with FA.
Read the official press release from Stealth BioTherapeutics here.
