Effect of ascorbic acid in patients with Charcot–Marie–Tooth disease type 1A: a multicentre, randomised, double-blind, placebo-controlled trial

Micallef, Joëlle; Attarian, Shahram; Dubourg, Odile; Gonnaud, Pierre-Marie; Hogrel, Jean‐Yves; Stojkovic, Tanya; Bernard, R.; Jouve, Élisabeth; Pitel, Séverine; Vacherot, Francois; Remec, Jean-François; Jomir, Laurent; Azabou, Éric; Al-Moussawi, Mahmoud; Lefebvre, Marie-Noëlle; Attolini, Laurence; Yaici, Sadek; Tanesse, Daniel; Fontés, Michel; Pouget, Jean; Blin, Olivier

doi:10.1016/s1474-4422(09)70260-1

Cited by 117 publications

(84 citation statements)

References 34 publications

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“…For CMT disease, ascorbic acid, progesterone and HDAC6 inhibitors have been shown to be beneficial in animal models (20)(21)(22)(23) but not in clinical trials (21,(24)(25)(26). Such specific treatments according to the pathophysiology of each neuropathy are necessary.…”

Section: Discussionmentioning

confidence: 99%

Safety and Efficacy of Intravenous Ultra-high Dose Methylcobalamin Treatment for Peripheral Neuropathy: A Phase I/II Open Label Clinical Trial

et al. 2014

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Objective No clinically effective treatment for promoting peripheral axonal regeneration has yet been established. Several experimental studies in vitro and in vivo have shown that a high dose of methylcobalamin (MeCbl), an analogue of vitamin B12, promotes axonal growth in peripheral nerve injury. We herein assessed the safety and efficacy of an ultra-high dose MeCbl treatment for patients with peripheral neuropathy and chronic axonal degeneration. Methods Fourteen patients with immune-mediated or hereditary neuropathy in the chronic progressive or stable phase were enrolled. MeCbl, 25 mg/day for 10 days followed by monthly 25 mg for 5 months, was intravenously administered. The patients were evaluated before and 1 year following treatment. The primary endpoints were safety and improvement in the Medical Research Council (MRC) sum score in at least two muscles of the 20 muscles. This trial is registered with the University Hospital Medical Information Network (UMIN) Center in Japan under the ID: UMIN000009359. Results There were no adverse effects in twelve of the patients, whereas treatment was discontinued in two patients who had seborrheic dermatitis at 3 months and respiratory tract infection at 2 months, respectively. Therefore, twelve patients were evaluated for the primary outcomes; the MRC sum score was improved in seven of the patients and unchanged or worsened in the remaining five patients. Conclusion Intravenous ultra-high dose MeCbl treatment is a safe and potentially efficacious therapy for patients with peripheral neuropathy and chronic axonal degeneration.

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Section: Discussionmentioning

confidence: 99%

Safety and Efficacy of Intravenous Ultra-high Dose Methylcobalamin Treatment for Peripheral Neuropathy: A Phase I/II Open Label Clinical Trial

et al. 2014

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“…The CMT Neuropathy Score has been used as the primary outcome measure in several trials in adults with CMT (Micallef et al, 2009;Pareyson et al, 2011) and can detect increasing impairment over time (Shy et al, 2008;Burns et al, 2012). However, it has limited sensitivity in younger CMT-patients and those with only mild symptoms (Haberlova and Seeman, 2010).…”

Section: Translational Implicationsmentioning

confidence: 99%

Binaural speech processing in individuals with auditory neuropathy

Rance¹,

Ryan²,

Carew³

et al. 2012

Neuroscience

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“…Furthermore, in vitro and animal studies showed that AA improves the clinical and pathological phenotype of a mouse model of Chercot-Marie-tooth disease 1A (CMT1A) (Kaya et al, 2007;Passage et al, 2004), which led to various clinical trials examining AA administration in CMT1A. However, none of these trials showed a signifi cant benefi t of AA in the treatment of CMT1A patients (Burns et al, 2009;Micallef et al, 2009;Verhamme et al, 2009). The lack of eff ectiveness in the treatments was diffi cult to interpret because there were no studies assessing SVCT2 expression in Schwann cells and peripheral nerves, data that was recently obtained by (Gess et al, 2010).…”

Section: Vitamin C In the Central Nervous Systemmentioning

confidence: 99%

Typical and atypical stem cells in the brain, vitamin C effect and neuropathology

Nualart¹,

Salazar²,

Oyarce³

et al. 2012

Biol. Res.

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Stem cells are considered a valuable cellular resource for tissue replacement therapies in most brain disorders. Stem cells have the ability to self-replicate and diff erentiate into numerous cell types, including neurons, oligodendrocytes and astrocytes. As a result, stem cells have been considered the "holy grail" of modern medical neuroscience. Despite their tremendous therapeutic potential, little is known about the mechanisms that regulate their diff erentiation. In this review, we analyze stem cells in embryonic and adult brains, and illustrate the diff erentiation pathways that give origin to most brain cells. We also evaluate the emergent role of the well known anti-oxidant, vitamin C, in stem cell diff erentiation. We believe that a complete understanding of all molecular players, including vitamin C, in stem cell diff erentiation will positively impact on the use of stem cell transplantation for neurodegenerative diseases.

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Effect of ascorbic acid in patients with Charcot–Marie–Tooth disease type 1A: a multicentre, randomised, double-blind, placebo-controlled trial

Cited by 117 publications

References 34 publications

Safety and Efficacy of Intravenous Ultra-high Dose Methylcobalamin Treatment for Peripheral Neuropathy: A Phase I/II Open Label Clinical Trial

Safety and Efficacy of Intravenous Ultra-high Dose Methylcobalamin Treatment for Peripheral Neuropathy: A Phase I/II Open Label Clinical Trial

Binaural speech processing in individuals with auditory neuropathy

Typical and atypical stem cells in the brain, vitamin C effect and neuropathology

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