Skip to main content
Top
Gepubliceerd in: Journal of Autism and Developmental Disorders 11/2010

01-11-2010 | Brief Report

Brief Report: Acamprosate in Fragile X Syndrome

Auteurs: Craig A. Erickson, Jennifer E. Mullett, Christopher J. McDougle

Gepubliceerd in: Journal of Autism and Developmental Disorders | Uitgave 11/2010

Log in om toegang te krijgen
share
DELEN

Deel dit onderdeel of sectie (kopieer de link)

  • Optie A:
    Klik op de rechtermuisknop op de link en selecteer de optie “linkadres kopiëren”
  • Optie B:
    Deel de link per e-mail

Abstract

Glutamatergic dysfunction is implicated in the pathophysiology of fragile X syndrome (FXS). We report on the first trial of acamprosate, a drug with putative mGluR5 antagonism, in three adults with FXS and autism. Medical records describing open-label treatment with acamprosate in 3 patients with FXS and a comorbid diagnosis of autistic disorder were reviewed. In all three patients, acamprosate was associated with improved linguistic communication. Three patients received acamprosate over a mean 21.3 weeks of treatment. All patients showed global clinical benefit as rated with the Clinical Global Impressions-Improvement scale. Marked communication improvement was unexpected and has potential implications for the treatment of FXS, as well as idiopathic autism.
Literatuur
go back to reference al Qatari, M., Khan, S., Harris, B., & Littleton, J. (2001). Acamprosate is neuroprotective against glutamate-induced excitotoxicity when enhanced by ethanol withdrawal in neocortical cultures of fetal rat brain. Alcoholism, Clinical and Experimental Research, 25(9), 1276–1283.CrossRefPubMed al Qatari, M., Khan, S., Harris, B., & Littleton, J. (2001). Acamprosate is neuroprotective against glutamate-induced excitotoxicity when enhanced by ethanol withdrawal in neocortical cultures of fetal rat brain. Alcoholism, Clinical and Experimental Research, 25(9), 1276–1283.CrossRefPubMed
go back to reference American Psychiatric Association. (2000). Diagnostic and statistical manual of mental disorders, Four Edition, Text Revision (4th ed.). Washington, DC: American Psychiatric Association.CrossRef American Psychiatric Association. (2000). Diagnostic and statistical manual of mental disorders, Four Edition, Text Revision (4th ed.). Washington, DC: American Psychiatric Association.CrossRef
go back to reference Bear, M. F., Huber, K. M., & Warren, S. T. (2004). The mGluR theory of fragile X mental retardation. Trends in Neurosciences, 27(7), 370–377.CrossRefPubMed Bear, M. F., Huber, K. M., & Warren, S. T. (2004). The mGluR theory of fragile X mental retardation. Trends in Neurosciences, 27(7), 370–377.CrossRefPubMed
go back to reference Berry-Kravis, E., Hessl, D., Coffey, S., Hervey, C., Schneider, A., Yuhas, J., et al. (2009). A pilot open label, single dose trial of fenobam in adults with fragile X syndrome. Journal of Medical Genetics, 46(4), 266–271.CrossRefPubMed Berry-Kravis, E., Hessl, D., Coffey, S., Hervey, C., Schneider, A., Yuhas, J., et al. (2009). A pilot open label, single dose trial of fenobam in adults with fragile X syndrome. Journal of Medical Genetics, 46(4), 266–271.CrossRefPubMed
go back to reference Berry-Kravis, E., Sumis, A., Hervey, C., Nelson, M., Porges, S. W., Weng, N., et al. (2008). Open-label treatment trial of lithium to target the underlying defect in fragile X syndrome. Journal of Developmental and Behavioral Pediatrics, 29(4), 293–302.CrossRefPubMed Berry-Kravis, E., Sumis, A., Hervey, C., Nelson, M., Porges, S. W., Weng, N., et al. (2008). Open-label treatment trial of lithium to target the underlying defect in fragile X syndrome. Journal of Developmental and Behavioral Pediatrics, 29(4), 293–302.CrossRefPubMed
go back to reference Blednov, Y. A., & Adron Harris, R. (2008). Metabotropic glutamate receptor 5 (mGluR5) regulation of ethanol sedation, dependence and consumption: Relationship to acamprosate actions. International Journal of Neuropsychopharmacology, 11(6), 775–793.PubMed Blednov, Y. A., & Adron Harris, R. (2008). Metabotropic glutamate receptor 5 (mGluR5) regulation of ethanol sedation, dependence and consumption: Relationship to acamprosate actions. International Journal of Neuropsychopharmacology, 11(6), 775–793.PubMed
go back to reference Chuang, S. C., Zhao, W., Bauchwitz, R., Yan, Q., Bianchi, R., & Wong, R. K. (2005). Prolonged epileptiform discharges induced by altered group I metabotropic glutamate receptor-mediated synaptic responses in hippocampal slices of a fragile X mouse model. Journal of Neuroscience, 25(35), 8048–8055.CrossRefPubMed Chuang, S. C., Zhao, W., Bauchwitz, R., Yan, Q., Bianchi, R., & Wong, R. K. (2005). Prolonged epileptiform discharges induced by altered group I metabotropic glutamate receptor-mediated synaptic responses in hippocampal slices of a fragile X mouse model. Journal of Neuroscience, 25(35), 8048–8055.CrossRefPubMed
go back to reference de Diego-Otero, Y., Romero-Zerbo, Y., el Bekay, R., Decara, J., Sanchez, L., Rodriguez-de Fonseca, F., et al. (2009). Alpha-tocopherol protects against oxidative stress in the fragile X knockout mouse: An experimental therapeutic approach for the Fmr1 deficiency. Neuropsychopharmacology, 34(4), 1011–1026.CrossRefPubMed de Diego-Otero, Y., Romero-Zerbo, Y., el Bekay, R., Decara, J., Sanchez, L., Rodriguez-de Fonseca, F., et al. (2009). Alpha-tocopherol protects against oxidative stress in the fragile X knockout mouse: An experimental therapeutic approach for the Fmr1 deficiency. Neuropsychopharmacology, 34(4), 1011–1026.CrossRefPubMed
go back to reference de Vrij, F. M., Levenga, J., van der Linde, H. C., Koekkoek, S. K., De Zeeuw, C. I., Nelson, D. L., et al. (2008). Rescue of behavioral phenotype and neuronal protrusion morphology in Fmr1 KO mice. Neurobiology of Diseases, 31(1), 127–132.CrossRef de Vrij, F. M., Levenga, J., van der Linde, H. C., Koekkoek, S. K., De Zeeuw, C. I., Nelson, D. L., et al. (2008). Rescue of behavioral phenotype and neuronal protrusion morphology in Fmr1 KO mice. Neurobiology of Diseases, 31(1), 127–132.CrossRef
go back to reference De Witte, P., Littleton, J., Parot, P., & Koob, G. (2005). Neuroprotective and abstinence-promoting effects of acamprosate: Elucidating the mechanism of action. CNS Drugs, 19(6), 517–537.CrossRefPubMed De Witte, P., Littleton, J., Parot, P., & Koob, G. (2005). Neuroprotective and abstinence-promoting effects of acamprosate: Elucidating the mechanism of action. CNS Drugs, 19(6), 517–537.CrossRefPubMed
go back to reference Devys, D., Lutz, Y., Rouyer, N., Bellocq, J. P., & Mandel, J. L. (1993). The FMR-1 protein is cytoplasmic, most abundant in neurons and appears normal in carriers of a fragile X premutation. Nature Genetics, 4(4), 335–340.CrossRefPubMed Devys, D., Lutz, Y., Rouyer, N., Bellocq, J. P., & Mandel, J. L. (1993). The FMR-1 protein is cytoplasmic, most abundant in neurons and appears normal in carriers of a fragile X premutation. Nature Genetics, 4(4), 335–340.CrossRefPubMed
go back to reference Dolen, G., & Bear, M. F. (2008). Role for metabotropic glutamate receptor 5 (mGluR5) in the pathogenesis of fragile X syndrome. Journal of Physiology, 586(6), 1503–1508.CrossRefPubMed Dolen, G., & Bear, M. F. (2008). Role for metabotropic glutamate receptor 5 (mGluR5) in the pathogenesis of fragile X syndrome. Journal of Physiology, 586(6), 1503–1508.CrossRefPubMed
go back to reference Dolen, G., Osterweil, E., Rao, B. S., Smith, G. B., Auerbach, B. D., Chattarji, S., et al. (2007). Correction of fragile X syndrome in mice. Neuron, 56(6), 955–962.CrossRefPubMed Dolen, G., Osterweil, E., Rao, B. S., Smith, G. B., Auerbach, B. D., Chattarji, S., et al. (2007). Correction of fragile X syndrome in mice. Neuron, 56(6), 955–962.CrossRefPubMed
go back to reference el Bekay, R., Romero-Zerbo, Y., Decara, J., Sanchez-Salido, L., Del Arco-Herrera, I., Rodriguez-de Fonseca, F., et al. (2007). Enhanced markers of oxidative stress, altered antioxidants and NADPH-oxidase activation in brains from Fragile X mental retardation 1-deficient mice, a pathological model for Fragile X syndrome. European Journal of Neuroscience, 26(11), 3169–3180.CrossRefPubMed el Bekay, R., Romero-Zerbo, Y., Decara, J., Sanchez-Salido, L., Del Arco-Herrera, I., Rodriguez-de Fonseca, F., et al. (2007). Enhanced markers of oxidative stress, altered antioxidants and NADPH-oxidase activation in brains from Fragile X mental retardation 1-deficient mice, a pathological model for Fragile X syndrome. European Journal of Neuroscience, 26(11), 3169–3180.CrossRefPubMed
go back to reference Erickson, C. A., Mullett, J. E., & McDougle, C. J. (2009). Open-label memantine in Fragile X Syndrome. J Autism Dev Disord. Erickson, C. A., Mullett, J. E., & McDougle, C. J. (2009). Open-label memantine in Fragile X Syndrome. J Autism Dev Disord.
go back to reference Greenough, W. T., Klintsova, A. Y., Irwin, S. A., Galvez, R., Bates, K. E., & Weiler, I. J. (2001). Synaptic regulation of protein synthesis and the fragile X protein. Proc Natl Acad Sci USA, 98(13), 7101–7106.CrossRefPubMed Greenough, W. T., Klintsova, A. Y., Irwin, S. A., Galvez, R., Bates, K. E., & Weiler, I. J. (2001). Synaptic regulation of protein synthesis and the fragile X protein. Proc Natl Acad Sci USA, 98(13), 7101–7106.CrossRefPubMed
go back to reference Gupta, T., Syed, Y. M., Revis, A. A., Miller, S. A., Martinez, M., Cohn, K. A., et al. (2008). Acute effects of acamprosate and MPEP on ethanol Drinking-in-the-Dark in male C57BL/6J mice. Alcoholism, Clinical and Experimental Research, 32(11), 1992–1998.PubMed Gupta, T., Syed, Y. M., Revis, A. A., Miller, S. A., Martinez, M., Cohn, K. A., et al. (2008). Acute effects of acamprosate and MPEP on ethanol Drinking-in-the-Dark in male C57BL/6J mice. Alcoholism, Clinical and Experimental Research, 32(11), 1992–1998.PubMed
go back to reference Guy, W. (1976). ECDEU assessment manual for psychopharmacology, Publication No. 76–338. Washington, DC: US DHEW, NIMH. Guy, W. (1976). ECDEU assessment manual for psychopharmacology, Publication No. 76–338. Washington, DC: US DHEW, NIMH.
go back to reference Hagerman, P. J. (2008). The fragile X prevalence paradox. Journal of Medical Genetics, 45(8), 498–499.CrossRefPubMed Hagerman, P. J. (2008). The fragile X prevalence paradox. Journal of Medical Genetics, 45(8), 498–499.CrossRefPubMed
go back to reference Harris, B. R., Prendergast, M. A., Gibson, D. A., Rogers, D. T., Blanchard, J. A., Holley, R. C., et al. (2002). Acamprosate inhibits the binding and neurotoxic effects of trans-ACPD, suggesting a novel site of action at metabotropic glutamate receptors. Alcoholism, Clinical and Experimental Research, 26(12), 1779–1793.PubMed Harris, B. R., Prendergast, M. A., Gibson, D. A., Rogers, D. T., Blanchard, J. A., Holley, R. C., et al. (2002). Acamprosate inhibits the binding and neurotoxic effects of trans-ACPD, suggesting a novel site of action at metabotropic glutamate receptors. Alcoholism, Clinical and Experimental Research, 26(12), 1779–1793.PubMed
go back to reference Jaeschke, G., Wettstein, J. G., Nordquist, R. E., & Spooren, W. (2008). mGlu5 receptor antagonists and their potential therapeutic potential. Expert Opinion on Therapeutic Patents, 18(2), 123–142.CrossRef Jaeschke, G., Wettstein, J. G., Nordquist, R. E., & Spooren, W. (2008). mGlu5 receptor antagonists and their potential therapeutic potential. Expert Opinion on Therapeutic Patents, 18(2), 123–142.CrossRef
go back to reference Lord, C., Rutter, M., & Le Couteur, A. (1994). Autism diagnostic interview-revised: A revised version of a diagnostic interview for caregivers of individuals with possible pervasive developmental disorders. Journal of Autism and Developmental Disorders, 24(5), 659–685.CrossRefPubMed Lord, C., Rutter, M., & Le Couteur, A. (1994). Autism diagnostic interview-revised: A revised version of a diagnostic interview for caregivers of individuals with possible pervasive developmental disorders. Journal of Autism and Developmental Disorders, 24(5), 659–685.CrossRefPubMed
go back to reference Mann, K., Kiefer, F., Spanagel, R., & Littleton, J. (2008). Acamprosate: Recent findings and future research directions. Alcoholism, Clinical and Experimental Research, 32(7), 1105–1110.CrossRefPubMed Mann, K., Kiefer, F., Spanagel, R., & Littleton, J. (2008). Acamprosate: Recent findings and future research directions. Alcoholism, Clinical and Experimental Research, 32(7), 1105–1110.CrossRefPubMed
go back to reference McBride, S. M., Choi, C. H., Wang, Y., Liebelt, D., Braunstein, E., Ferreiro, D., et al. (2005). Pharmacological rescue of synaptic plasticity, courtship behavior, and mushroom body defects in a Drosophila model of fragile X syndrome. Neuron, 45(5), 753–764.CrossRefPubMed McBride, S. M., Choi, C. H., Wang, Y., Liebelt, D., Braunstein, E., Ferreiro, D., et al. (2005). Pharmacological rescue of synaptic plasticity, courtship behavior, and mushroom body defects in a Drosophila model of fragile X syndrome. Neuron, 45(5), 753–764.CrossRefPubMed
go back to reference Pieretti, M., Zhang, F. P., Fu, Y. H., Warren, S. T., Oostra, B. A., Caskey, C. T., et al. (1991). Absence of expression of the FMR-1 gene in fragile X syndrome. Cell, 66(4), 817–822.CrossRefPubMed Pieretti, M., Zhang, F. P., Fu, Y. H., Warren, S. T., Oostra, B. A., Caskey, C. T., et al. (1991). Absence of expression of the FMR-1 gene in fragile X syndrome. Cell, 66(4), 817–822.CrossRefPubMed
go back to reference Reilly, M. T., Lobo, I. A., McCracken, L. M., Borghese, C. M., Gong, D., Horishita, T., et al. (2008). Effects of acamprosate on neuronal receptors and ion channels expressed in Xenopus oocytes. Alcoholism, Clinical and Experimental Research, 32(2), 188–196.CrossRefPubMed Reilly, M. T., Lobo, I. A., McCracken, L. M., Borghese, C. M., Gong, D., Horishita, T., et al. (2008). Effects of acamprosate on neuronal receptors and ion channels expressed in Xenopus oocytes. Alcoholism, Clinical and Experimental Research, 32(2), 188–196.CrossRefPubMed
go back to reference Romero-Zerbo, Y., Decara, J., el Bekay, R., Sanchez-Salido, L., Del Arco-Herrera, I., de Fonseca, F. R., et al. (2009). Protective effects of melatonin against oxidative stress in Fmr1 knockout mice: A therapeutic research model for the fragile X syndrome. Journal of Pineal Research, 46(2), 224–234.CrossRefPubMed Romero-Zerbo, Y., Decara, J., el Bekay, R., Sanchez-Salido, L., Del Arco-Herrera, I., de Fonseca, F. R., et al. (2009). Protective effects of melatonin against oxidative stress in Fmr1 knockout mice: A therapeutic research model for the fragile X syndrome. Journal of Pineal Research, 46(2), 224–234.CrossRefPubMed
go back to reference Ronesi, J. A., & Huber, K. M. (2008). Metabotropic glutamate receptors and fragile x mental retardation protein: Partners in translational regulation at the synapse. Sci Signal, 1(5), pe6. Ronesi, J. A., & Huber, K. M. (2008). Metabotropic glutamate receptors and fragile x mental retardation protein: Partners in translational regulation at the synapse. Sci Signal, 1(5), pe6.
go back to reference Yan, Q. J., Rammal, M., Tranfaglia, M., & Bauchwitz, R. P. (2005). Suppression of two major Fragile X Syndrome mouse model phenotypes by the mGluR5 antagonist MPEP. Neuropharmacology, 49(7), 1053–1066.CrossRefPubMed Yan, Q. J., Rammal, M., Tranfaglia, M., & Bauchwitz, R. P. (2005). Suppression of two major Fragile X Syndrome mouse model phenotypes by the mGluR5 antagonist MPEP. Neuropharmacology, 49(7), 1053–1066.CrossRefPubMed
Metagegevens
Titel
Brief Report: Acamprosate in Fragile X Syndrome
Auteurs
Craig A. Erickson
Jennifer E. Mullett
Christopher J. McDougle
Publicatiedatum
01-11-2010
Uitgeverij
Springer US
Gepubliceerd in
Journal of Autism and Developmental Disorders / Uitgave 11/2010
Print ISSN: 0162-3257
Elektronisch ISSN: 1573-3432
DOI
https://doi.org/10.1007/s10803-010-0988-9

Andere artikelen Uitgave 11/2010

Journal of Autism and Developmental Disorders 11/2010 Naar de uitgave