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FRDA news - MitoQ update

Here is what we have learned over the the past few months about Mitoquinone (MitoQ). MitoQ is a quinone similar to CoQ10 and Idebenone but NOT identical.

Promising new approach? Yes.

Oxidative stress resulting from free radicals is a major cause of the pathology of Friedreich's ataxia (FRDA). Oxidative stress in FRDA is brought about by the shortage of the frataxin protein in the mitochondria.

CoQ10 and Idebenone are antioxidants and serve to eliminate free radicals. CoQ10 and Idebenone are distributed widely throughout the cell. The drug, MitoQ, on the other hand, is an antioxidant intended to target mitochondria specifically. Researchers hope MitoQ can be made to target the mitochondria selectively by joining the quinone (like CoQ10) with an additional element that will concentrate the antioxidant in the mitochondria at much higher rates than the antioxidants that are widely distributed throughout the cell. In this way, they hope to combat oxidative stress where it originates and does much of its damage in Friedreich's ataxia -- in the mitochondria.

Like Idebenone, MitoQ is not a naturally occuring substance. Therefore, it is subject to the Food and Drug Administration (FDA) process (meaning dose escalation/toxicity tests in animals, then humans, and double blind placebo controlled clinical trials).

According to the article posted on 01/16/03, "It could be available to patients in about TWO YEARS -- IF it satisfies quality, safety and efficacy standards."

***MitoQ is not available to patients at this date--it is in the developmental phase.***

Good news-- FARA has recently awarded a research grant to Dr. Mike Murphy who is one of the 2 researchers who developed the compound, MitoQ, at Otago University.

This funding will allow Dr. Murphy to continue his work in pursuing this potential therapy. The testing at this stage of development is not on humans.

Dr. Murphy, along with about 100 other FRDA researchers from around the world, will participate in the upcoming international scientific conference on Friedreich's ataxia sponsored by FARA.

The overall objectives of the conference are to integrate the most up-to-date information from the various research disciplines relevant to FRDA, to identify promising new avenues for research, to foster collaborations among researchers in the field, to encourage new investigators to enter the field, and to coordinate approaches to clinical studies and clinical trials. Given all that has happened in FRDA research since our last conference in 1999, the 2003 conference should be very interesting and productive.



Friedreich's Ataxia Research Alliance
2001 Jefferson Davis Hwy., Suite 209
Arlington, Virginia USA 22202
Tel (703) 413-4468, Fax (703) 413-4467