Epicatechin is a naturally occurring compound which Cardero Therapeutics and the Mayo Clinic have identified as a potential treatment for Friedreich’s ataxia (FA).

The flavonols (+)-epicatechin and (-)-epicatechin are found in very small amounts in wine, dark chocolate, and green tea.

FA is a rare genetic disorder caused by mutations in the gene encoding for a protein called frataxin. This protein is important for the normal functioning of mitochondria, the energy factories of the cells. The lack of frataxin leads to a slow and progressive impairment of muscle coordination (known as ataxia), loss of muscle strength and sensation, and impaired speech, vision, and hearing.

There are currently no therapies available that can cure or effectively treat the condition. Medications can only treat symptoms of FA and address disease-associated problems that affect cardiovascular, neurological, or motor functions.

How epicatechin works

(-)-Epicatechin has been shown to improve mitochondrial function in different models of cardiac and neuromuscular diseases. A study reported that it reduces oxidative stress and helps restore the formation of new mitochondria in senile mice.

In a small Phase 1 study, (-)-epicatechin was found to help improve mitochondrial enzyme function and plasma levels of frataxin. These changes, in turn, may help reduce the risk of cardiovascular and neurodegenerative diseases and improve the structure of skeletal muscles and muscle strength, counteracting the gradual deterioration of muscular and neurological functions associated with Friedreich’s ataxia.

More recent research suggests that (+)-epicatechin may be more potent than (-)-epicatechin.

(+)-Epicatechin in clinical trials

A double-blind, randomized Phase 1 clinical trial (NCT02330276) testing (+)-epicatechin in healthy volunteers and in people with pre-diabetes was successfully completed in 2015. The trial demonstrated that (+)-epicatechin is safe and has a good tolerability in healthy people. The enrollment was small, with just 12 people.

On the basis of these findings, a new Phase 2 open-label study (NCT02660112) was started in 2016. The trial will test the safety and effectiveness of (+)-epicatechin in people with early FA (those with less than seven years of disease duration). The study is currently recruiting participants, and is expected to be completed in December 2018.


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