IGF-1 in Friedreich’s Ataxia – proof-of-concept trial

[Background] Friedreich’s ataxia is an autosomal recessive, severely incapacitating disorder. There is little objective evidence regarding FRDA management. Abnormalities in the insulin/insulin-like growth factor 1 (IGF-1) system (IIS) signalling pathway were thought to play a role in the physiopatho...

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Detalles Bibliográficos
Autores: Sanz-Gallego, Irene, Torres Alemán, Ignacio, Arpa, Javier
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2014
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/125781
Acceso en línea:http://hdl.handle.net/10261/125781
Access Level:acceso abierto
Palabra clave:Friedreich’s ataxia
IGF-1 therapy
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spelling IGF-1 in Friedreich’s Ataxia – proof-of-concept trialSanz-Gallego, IreneTorres Alemán, IgnacioArpa, JavierFriedreich’s ataxiaIGF-1 therapy[Background] Friedreich’s ataxia is an autosomal recessive, severely incapacitating disorder. There is little objective evidence regarding FRDA management. Abnormalities in the insulin/insulin-like growth factor 1 (IGF-1) system (IIS) signalling pathway were thought to play a role in the physiopathological processes of various neurodegenerative disorders, including spinocerebellar ataxias. The objective of the study was to test the safety, tolerability, and efficacy of therapy with IGF-1 in Friedreich’s ataxia (FRDA) patients in a clinical pilot study.[Results] A total of 4 females and 1 male were included in the study; 23 to 36 years of age (average 26.6 ± 5.4), diagnosed with FRDA with normal ventricular function. Patients were treated with IGF-1 therapy with 50 μg/kg twice a day subcutaneously for 12 months. The efficacy of this therapy was assessed by changes from baseline on the scale for the assessment and rating of ataxia, (SARA) and by changes from baseline in echocardiogram parameters. The annual worsening rate (AWR) was estimated in this series as a SARA score of -0.4 ± 0.83 (CI 95%: -1.28 to 0.48) SARA score, whereas the AWR for our FRDA cohort was estimated as a SARA score of 2.05 ± 1.23 (CI 95%: 1.58 to 2.52). Echocardiographic parameters remained normal and stable.[Conclusion] Our results seem to indicate a benefit of this IGF-1 therapy to neurological functions in FRDA.Dr. Arpa has received grant funding from the Spanish Ministry of Health.Peer reviewedBioMed CentralMinisterio de Sanidad, Servicios Sociales e Igualdad (España)Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]2015201520142015info:eu-repo/semantics/articlehttp://purl.org/coar/resource_type/c_6501Publisher's versioninfo:eu-repo/semantics/publishedVersionhttp://hdl.handle.net/10261/125781reponame:DIGITAL.CSIC. Repositorio Institucional del CSICinstname:Consejo Superior de Investigaciones Científicas (CSIC)Ingléshttp://dx.doi.org/10.1186/2053-8871-1-10Síinfo:eu-repo/semantics/openAccessoai:digital.csic.es:10261/1257812026-05-22T06:33:51Z
dc.title.none.fl_str_mv IGF-1 in Friedreich’s Ataxia – proof-of-concept trial
title IGF-1 in Friedreich’s Ataxia – proof-of-concept trial
spellingShingle IGF-1 in Friedreich’s Ataxia – proof-of-concept trial
Sanz-Gallego, Irene
Friedreich’s ataxia
IGF-1 therapy
title_short IGF-1 in Friedreich’s Ataxia – proof-of-concept trial
title_full IGF-1 in Friedreich’s Ataxia – proof-of-concept trial
title_fullStr IGF-1 in Friedreich’s Ataxia – proof-of-concept trial
title_full_unstemmed IGF-1 in Friedreich’s Ataxia – proof-of-concept trial
title_sort IGF-1 in Friedreich’s Ataxia – proof-of-concept trial
dc.creator.none.fl_str_mv Sanz-Gallego, Irene
Torres Alemán, Ignacio
Arpa, Javier
author Sanz-Gallego, Irene
author_facet Sanz-Gallego, Irene
Torres Alemán, Ignacio
Arpa, Javier
author_role author
author2 Torres Alemán, Ignacio
Arpa, Javier
author2_role author
author
dc.contributor.none.fl_str_mv Ministerio de Sanidad, Servicios Sociales e Igualdad (España)
Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]
dc.subject.none.fl_str_mv Friedreich’s ataxia
IGF-1 therapy
topic Friedreich’s ataxia
IGF-1 therapy
description [Background] Friedreich’s ataxia is an autosomal recessive, severely incapacitating disorder. There is little objective evidence regarding FRDA management. Abnormalities in the insulin/insulin-like growth factor 1 (IGF-1) system (IIS) signalling pathway were thought to play a role in the physiopathological processes of various neurodegenerative disorders, including spinocerebellar ataxias. The objective of the study was to test the safety, tolerability, and efficacy of therapy with IGF-1 in Friedreich’s ataxia (FRDA) patients in a clinical pilot study.
publishDate 2014
dc.date.none.fl_str_mv 2014
2015
2015
2015
dc.type.none.fl_str_mv info:eu-repo/semantics/article
http://purl.org/coar/resource_type/c_6501
Publisher's version
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/10261/125781
url http://hdl.handle.net/10261/125781
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv http://dx.doi.org/10.1186/2053-8871-1-10
Sí
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv BioMed Central
publisher.none.fl_str_mv BioMed Central
dc.source.none.fl_str_mv reponame:DIGITAL.CSIC. Repositorio Institucional del CSIC
instname:Consejo Superior de Investigaciones Científicas (CSIC)
instname_str Consejo Superior de Investigaciones Científicas (CSIC)
reponame_str DIGITAL.CSIC. Repositorio Institucional del CSIC
collection DIGITAL.CSIC. Repositorio Institucional del CSIC
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15.811543

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