Experimental Treatments for Friedreich's Ataxia


CTI-1601 is an investigational therapy designed to deliver the frataxin protein to the mitochondria of Friedreich’s ataxia patients. Dosing was completed in a Phase 1 trial in December 2020, and top-line data are expected in the second quarter of 2021.

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Gene Therapy

Gene therapy consists of the intracellular delivery of genetic material to generate a therapeutic effect by correcting an existing abnormality or providing the cells with a new function. This approach could be used to treat FA by either correcting the faulty frataxin gene or any damage caused by the disease on other systems in the body, such as the heart.

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LX2006 is an investigational gene therapy for heart disease in people with Friedreich’s ataxia (FA). The therapy, being developed by Lexeo Therapeutics, is intended for cardiomyopathy in FA. Cardiomyopathy is a disease of the heart muscle that can affect the heart’s size, shape, thickness, and function. Heart disease is the primary cause of death in FA patients.

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Leriglitazone, or MIN-102, is an oral medication being developed for treating several diseases of the brain and spinal cord, including Friedreich’s ataxia. It has shown potential to treat the disease in recent animal and cell studies, and positive top-line results from a Phase 2 trial were released in December 2020.

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Methylprednisolone is a steroid used to reduce inflammation, and may be able to help ease the symptoms of Friedreich’s ataxia. A Phase 1 trial was completed in 2019, but it did not meet its primary objective, with results showing no clear benefit for FA patients. No information is available yet on further studies.

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Nicotinamide, or vitamin B3, is being studied as a potential treatment for Friedreich’s ataxia. Initial study results show that it may improve frataxin production but that this may not translate into easing symptoms of the disease. A Phase 2 trial testing nicotinamide was recruiting FA patients in Europe as of December 2019.

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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. A Phase 2 trial studying micronized resveratrol is currently recruiting Friedreich’s ataxia patients.

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Vatiquinone (PTC-743), previously known as EPI-743, is an investigational oral treatment designed to limit neuroinflammation and nerve cell damage in people FA. Vatiquinone, a vitamin D derivative, is a small molecule that works by blocking the activity of 15-lipoxygenase, an enzyme that regulates signaling pathways that control neuroinflammation and oxidative stress. It is currently in Phase 2 trials.

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Visit the Friedreich’s Ataxia News forums to connect with others in the FA community. These forums are designed for FA patients, caregivers, and medical professionals to discuss issues, ask questions, and share experiences.

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