Resveratrol is a natural compound found in the skin of red grapes, peanuts, blueberries, and some other berries. It is a very powerful antioxidant that helps prevent cell damage caused by free radicals. Lately, resveratrol been under intense investigation as a potential treatment for Friedeich’s ataxia, as it has been shown to improve mitochondrial function by increasing frataxin levels in laboratory studies.

Frataxin is a protein that exists in the mitochondria, the energy-producing centers of cells. The energy-producing chain is broken when there is no frataxin, as in the case of FA where mutations in the FXN gene stop the production of frataxin protein.

How resveratrol works

The great antioxidant capacity of resveratrol helps prevent cell damage caused by free radicals. It also has been reported to have neuro-protective effects in cell and animal models.

Free radicals are molecular species that have an unpaired electron. The presence of this unpaired electron makes them unstable and highly reactive, attacking important molecules in human cells, such as lipids, nucleic acids, and proteins.

Resveratrol in Friedreich’s Ataxia research

The Friedreich’s Ataxia Research Alliance (FARA) funded an open-label, pilot study (NCT01339884), the primary objective of which was to evaluate the safety, tolerability, and effectiveness of two different doses of resveratrol (1 g and 5 g) in 27 participants with FA for 12 weeks. The treatment was completed by 24 participants.

The primary outcome measure was the levels of lymphocyte frataxin. Although no difference in either treatment group was seen, some secondary objectives, such as neurological rating scales and speech measures, showed improvement in the high dose group. But there was no placebo group with which to compare this result. The group that received the higher dose drug had gastrointestinal adverse events such as diarrhea, loose stools, and abdominal pain.

Other details

Jupiter Orphan Therapeutics is developing a new formulation of resveratrol called JOT101, which may be able to deliver resveratrol to the body more effectively so that the full potential of the compound is reached. Researchers at Jupiter Orphan think that a better absorption, distribution, metabolism, and excretion of this new formulation may lead to smaller amounts of drug being needed for effective treatment, alleviating side effects associated with its use.

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