Big news - Ascorbic acid treatment for CMT 1A - French research

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Abstract from Nat Med. 2004 Mar 21

Ascorbic acid treatment corrects the phenotype of a mouse model of

Charcot-Marie-Tooth disease.

Passage E, Norreel JC, Noack-Fraissignes P, Sanguedolce V, Pizant J,

Thirion X, Robaglia-Schlupp A, Pellissier JF, Fontes M.

[1] Institut National de la Sante et de la Recherche Medicale UMR491,

IPHM, Faculte de Medecine de la Timone, 27 Bd. J. Moulin, 13385

Marseille Cedex 5, France. [2] These authors contributed equally to this

work.

Charcot-Marie-Tooth disease (CMT) is the most common hereditary

peripheral neuropathy, affecting 1 in 2,500 people. The only treatment

currently available is rehabilitation or corrective surgery. The most

frequent form of the disease, CMT-1A, involves abnormal myelination of

the peripheral nerves. Here we used a mouse model of CMT-1A to test the

ability of ascorbic acid, a known promoter of myelination, to correct

the CMT-1A phenotype. Ascorbic acid treatment resulted in substantial

amelioration of the CMT-1A phenotype, and reduced the expression of

PMP22 to a level below what is necessary to induce the disease

phenotype. As ascorbic acid has already been approved by the FDA for

other clinical indications, it offers an immediate therapeutic

possibility for patients with the disease.