title: Effect of oral curcumin on Déjérine-Sottas disease.
abstract: Curcumin is the newest therapeutic agent for ameliorating the clinical and neuropathologic phenotype of a mouse model of Déjérine-Sottas disease. We undertook a 12-month dose-escalation safety trial of oral curcumin in a 15-year-old Caucasian girl with Déjérine-Sottas disease (point mutation, Ser72Leu) complicated by severe weakness, scoliosis, and respiratory impairment. The patient received 50 mg/kg/day oral curcumin for the first 4 months and 75 mg/kg/day thereafter, to complete a 12-month trial. Outcome measures included muscle strength, pulmonary function, upper/lower extremity disability, neurophysiologic studies, and health-related quality of life. After 12 months, the patient experienced no adverse events, and reported good compliance. There was little improvement in objective outcome measures. Knee flexion and foot strength increased slightly, but hand and elbow strength decreased. Pulmonary function, hand function, and measures of upper/lower extremity disability were stable or reduced. Her neurophysiologic findings were unchanged. Parent-reported quality of life improved for most domains, especially self-esteem, during the 12 months of treatment. Child-reported quality of life, assessed at the final visit, mirrored these results, with overall feelings of happiness and contentment. Further studies are required to explore the efficacy and safety of curcumin for severe demyelinating neuropathies of infancy and early childhood.
authors.affiliation: Discipline of Paediatrics and Child Health, Sydney Medical School, University of Sydney and Institute for Neuromuscular Research, Children's Hospital at Westmead, Sydney, New South Wales 2145, Australia. joshuab2@chw.edu.au
authors.forename: Joshua
authors.initials: J
authors.lastname: Burns
authors.forename: Pathma D
authors.initials: PD
authors.lastname: Joseph
authors.forename: Kristy J
authors.initials: KJ
authors.lastname: Rose
authors.forename: Monique M
authors.initials: MM
authors.lastname: Ryan
authors.forename: Robert A
authors.initials: RA
authors.lastname: Ouvrier
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000008:administration & dosage / Q000627:therapeutic use*; D003474:Curcumin / Q000008:administration & dosage / Q000627:therapeutic use*; D064420:Drug-Related Side Effects and Adverse Reactions; D005260:Female; D015417:Hereditary Sensory and Motor Neuropathy / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology; D006801:Humans; D009185:Myelin Proteins / Q000235:genetics; D017354:Point Mutation; D011788:Quality of Life; D013497:Sural Nerve / Q000473:pathology; D016896:Treatment Outcome
chemical_list: D000894:Anti-Inflammatory Agents, Non-Steroidal; D009185:Myelin Proteins; C073687:PMP22 protein, human; D003474:Curcumin