C Toth1. 1. Department of Clinical Neurosciences, University of Calgary, Calgary, AB, Canada. corytoth@shaw.ca
Abstract
BACKGROUND: Preclinical studies have suggested that ascorbic acid (AA) treatment in a mouse model of Charcot-Marie-Tooth type 1A (CMT1A) improves motor function and prolongs lifespan. AIMS: I sought to determine the safety and tolerability of AA in adult patients with CMT1A. METHODS: An open-label cohort-controlled 2-year pilot study was used to evaluate the tolerability of 5 g of AA daily. Secondary measurements consisted of clinical and electrophysiological measurements at 0, 12, and 24 months in CMT1A patients. RESULTS: Twelve CMT1A patients received AA and 10 CMT1A patients formed a cohort group followed in identical manner. Five (42%) patients tolerated this dose of AA for the entire 2-year span, with six patients (50%) developing intolerable gastrointestinal side effects. No significant differences in clinical, disability, or electrophysiological measurements occurred between baseline and final follow-up in patients receiving AA when compared with cohorts. CONCLUSIONS: High dose AA was not well tolerated in all adult CMT1A patients who may be susceptible to gastrointestinal adverse effects of AA. Studies with greater powers to detect efficacy will be required to test the validity of AA as a therapy in CMT1A patients. Doses lower than 5 g of AA daily may be required for maintenance of tolerability in the CMT1A population.
BACKGROUND: Preclinical studies have suggested that ascorbic acid (AA) treatment in a mouse model of Charcot-Marie-Tooth type 1A (CMT1A) improves motor function and prolongs lifespan. AIMS: I sought to determine the safety and tolerability of AA in adult patients with CMT1A. METHODS: An open-label cohort-controlled 2-year pilot study was used to evaluate the tolerability of 5 g of AA daily. Secondary measurements consisted of clinical and electrophysiological measurements at 0, 12, and 24 months in CMT1A patients. RESULTS: Twelve CMT1A patients received AA and 10 CMT1A patients formed a cohort group followed in identical manner. Five (42%) patients tolerated this dose of AA for the entire 2-year span, with six patients (50%) developing intolerable gastrointestinal side effects. No significant differences in clinical, disability, or electrophysiological measurements occurred between baseline and final follow-up in patients receiving AA when compared with cohorts. CONCLUSIONS: High dose AA was not well tolerated in all adult CMT1A patients who may be susceptible to gastrointestinal adverse effects of AA. Studies with greater powers to detect efficacy will be required to test the validity of AA as a therapy in CMT1A patients. Doses lower than 5 g of AA daily may be required for maintenance of tolerability in the CMT1A population.
Authors: Camiel Verhamme; Rob J de Haan; Marinus Vermeulen; Frank Baas; Marianne de Visser; Ivo N van Schaik Journal: BMC Med Date: 2009-11-12 Impact factor: 8.775
Authors: Davide Pareyson; Mary M Reilly; Angelo Schenone; Gian Maria Fabrizi; Tiziana Cavallaro; Lucio Santoro; Giuseppe Vita; Aldo Quattrone; Luca Padua; Franco Gemignani; Francesco Visioli; Matilde Laurà; Davide Radice; Daniela Calabrese; Richard A C Hughes; Alessandra Solari Journal: Lancet Neurol Date: 2011-04 Impact factor: 44.182
Authors: Burkhard Gess; Jonathan Baets; Peter De Jonghe; Mary M Reilly; Davide Pareyson; Peter Young Journal: Cochrane Database Syst Rev Date: 2015-12-11