Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study

de la Rubia Ortí, José Enrique; Drehmer Rieger, Eraci; Platero Armero, José Luis; Benlloch García, María; Caplliure Llopis, Jordi; Villarón Casales, Carlos Alberto; de Bernardo Tejedor, María Nieves; Alarcón Jimenez, Jorge; Fuente, Cristian; Sandra Carrera, Julia; Sancho Cantus, David; García Pardo, María Pilar; Pascual Toran, Raquel; Juárez, Marta; Cuerda Ballester, María; Forner, Alfonso; Barrios Pitarque, Carlos; Obrador, Elena; Marchio, Patricia; Salvador, Rosario; Holmes, Holly E.; Dellinger, Ryan W.; Guarente, Leonard; Estrela, José M.

Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study

Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease, characterized by progressive loss of spinal and cortical motor neurons, leading to muscular atrophy, respiratory failure, and ultimately death. There is no known cure, and the clinical benefit of the two drugs approved t...

Descripción completa

Detalles Bibliográficos
Autores:	de la Rubia Ortí, José Enrique, Drehmer Rieger, Eraci, Platero Armero, José Luis, Benlloch García, María, Caplliure Llopis, Jordi, Villarón Casales, Carlos Alberto, de Bernardo Tejedor, María Nieves, Alarcón Jimenez, Jorge, Fuente, Cristian, Sandra Carrera, Julia, Sancho Cantus, David, García Pardo, María Pilar, Pascual Toran, Raquel, Juárez, Marta, Cuerda Ballester, María, Forner, Alfonso, Barrios Pitarque, Carlos, Obrador, Elena, Marchio, Patricia, Salvador, Rosario, Holmes, Holly E., Dellinger, Ryan W., Guarente, Leonard, Estrela, José M.
Tipo de recurso:	artículo
Fecha de publicación:	2019
País:	España
Institución:	Universidad Católica de Valencia San Vicente Mártir
Repositorio:	RIUCV. Repositorio de la Universidad Católica de Valencia San Vicente Mártir
Idioma:	inglés
OAI Identifier:	oai:riucv.ucv.es:20.500.12466/3210
Acceso en línea:	http://hdl.handle.net/20.500.12466/3210
Access Level:	acceso abierto
Palabra clave:	Amyotrophic lateral sclerosis 1-(beta-D-Ribofuranosyl) nicotinamide chloride 3,5-Dimethoxy-4′-hydroxytrans-stilbene Randomized control study Human 3207.11 Neuropatología

id	ES_c823abf823d0920fd92fcefcf0de54fe
oai_identifier_str	oai:riucv.ucv.es:20.500.12466/3210
network_acronym_str	ES
network_name_str	España
repository_id_str
spelling	Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot studyde la Rubia Ortí, José EnriqueDrehmer Rieger, EraciPlatero Armero, José LuisBenlloch García, MaríaCaplliure Llopis, JordiVillarón Casales, Carlos Albertode Bernardo Tejedor, María NievesAlarcón Jimenez, JorgeFuente, CristianSandra Carrera, JuliaSancho Cantus, DavidGarcía Pardo, María PilarPascual Toran, RaquelJuárez, MartaCuerda Ballester, MaríaForner, AlfonsoBarrios Pitarque, CarlosObrador, ElenaMarchio, PatriciaSalvador, RosarioHolmes, Holly E.Dellinger, Ryan W.Guarente, LeonardEstrela, José M.Amyotrophic lateral sclerosis1-(beta-D-Ribofuranosyl) nicotinamide chloride3,5-Dimethoxy-4′-hydroxytrans-stilbeneRandomized control studyHuman3207.11 NeuropatologíaAmyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease, characterized by progressive loss of spinal and cortical motor neurons, leading to muscular atrophy, respiratory failure, and ultimately death. There is no known cure, and the clinical benefit of the two drugs approved to treat ALS remains unclear. Novel disease-modifying therapeutics that are able to modulate the disease course are desperately needed. Our objective was to evaluate the efficacy and tolerability of Elysium Health's candidate drug EH301 in people with ALS (PALS).This was a single-center, prospective, double-blind, randomized, placebo-controlled pilot study. Thirty-two PALS were recruited thanks to the collaboration of the Spanish Foundation for ALS Research (FUNDELA). Study participants were randomized to receive either EH301 or placebo and underwent evaluation for 4 months. Differences between EH301 and placebo-treated participants were evaluated based on standard clinical endpoints, including the revised ALS functional rating scale (ALSFRS-R), forced vital capacity (FVC), and the Medical Research Council (MRC) grading scale.Compared to placebo, participants treated with EH301 demonstrated significant improvements in the ALSFRS-R score, pulmonary function, muscular strength, and in skeletal muscle/fat weight ratio. EH301 was shown to significantly slow the progression of ALS relative to placebo, and even showed improvements in several key outcome measures compared with baseline.This study provides evidence in support of the disease-modifying effects of EH301 for the treatment of ALS.20232023-11-2920192019-01-2220192019-01-2220232023-11-28journal articlehttp://purl.org/coar/resource_type/c_6501info:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/20.500.12466/3210reponame:RIUCV. Repositorio de la Universidad Católica de Valencia San Vicente Mártirinstname:Universidad Católica de Valencia San Vicente MártirInglésengopen accesshttp://purl.org/coar/access_right/c_abf2Attribution-NonCommercial-NoDerivatives 4.0 Internacionalhttp://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:riucv.ucv.es:20.500.12466/32102026-06-19T08:32:07Z
dc.title.none.fl_str_mv	Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
title	Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
spellingShingle	Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study de la Rubia Ortí, José Enrique Amyotrophic lateral sclerosis 1-(beta-D-Ribofuranosyl) nicotinamide chloride 3,5-Dimethoxy-4′-hydroxytrans-stilbene Randomized control study Human 3207.11 Neuropatología
title_short	Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
title_full	Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
title_fullStr	Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
title_full_unstemmed	Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
title_sort	Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
dc.creator.none.fl_str_mv	de la Rubia Ortí, José Enrique Drehmer Rieger, Eraci Platero Armero, José Luis Benlloch García, María Caplliure Llopis, Jordi Villarón Casales, Carlos Alberto de Bernardo Tejedor, María Nieves Alarcón Jimenez, Jorge Fuente, Cristian Sandra Carrera, Julia Sancho Cantus, David García Pardo, María Pilar Pascual Toran, Raquel Juárez, Marta Cuerda Ballester, María Forner, Alfonso Barrios Pitarque, Carlos Obrador, Elena Marchio, Patricia Salvador, Rosario Holmes, Holly E. Dellinger, Ryan W. Guarente, Leonard Estrela, José M.
author	de la Rubia Ortí, José Enrique
author_facet	de la Rubia Ortí, José Enrique Drehmer Rieger, Eraci Platero Armero, José Luis Benlloch García, María Caplliure Llopis, Jordi Villarón Casales, Carlos Alberto de Bernardo Tejedor, María Nieves Alarcón Jimenez, Jorge Fuente, Cristian Sandra Carrera, Julia Sancho Cantus, David García Pardo, María Pilar Pascual Toran, Raquel Juárez, Marta Cuerda Ballester, María Forner, Alfonso Barrios Pitarque, Carlos Obrador, Elena Marchio, Patricia Salvador, Rosario Holmes, Holly E. Dellinger, Ryan W. Guarente, Leonard Estrela, José M.
author_role	author
author2	Drehmer Rieger, Eraci Platero Armero, José Luis Benlloch García, María Caplliure Llopis, Jordi Villarón Casales, Carlos Alberto de Bernardo Tejedor, María Nieves Alarcón Jimenez, Jorge Fuente, Cristian Sandra Carrera, Julia Sancho Cantus, David García Pardo, María Pilar Pascual Toran, Raquel Juárez, Marta Cuerda Ballester, María Forner, Alfonso Barrios Pitarque, Carlos Obrador, Elena Marchio, Patricia Salvador, Rosario Holmes, Holly E. Dellinger, Ryan W. Guarente, Leonard Estrela, José M.
author2_role	author author author author author author author author author author author author author author author author author author author author author author author
dc.contributor.none.fl_str_mv
dc.subject.none.fl_str_mv	Amyotrophic lateral sclerosis 1-(beta-D-Ribofuranosyl) nicotinamide chloride 3,5-Dimethoxy-4′-hydroxytrans-stilbene Randomized control study Human 3207.11 Neuropatología
topic	Amyotrophic lateral sclerosis 1-(beta-D-Ribofuranosyl) nicotinamide chloride 3,5-Dimethoxy-4′-hydroxytrans-stilbene Randomized control study Human 3207.11 Neuropatología
description	Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease, characterized by progressive loss of spinal and cortical motor neurons, leading to muscular atrophy, respiratory failure, and ultimately death. There is no known cure, and the clinical benefit of the two drugs approved to treat ALS remains unclear. Novel disease-modifying therapeutics that are able to modulate the disease course are desperately needed. Our objective was to evaluate the efficacy and tolerability of Elysium Health's candidate drug EH301 in people with ALS (PALS).This was a single-center, prospective, double-blind, randomized, placebo-controlled pilot study. Thirty-two PALS were recruited thanks to the collaboration of the Spanish Foundation for ALS Research (FUNDELA). Study participants were randomized to receive either EH301 or placebo and underwent evaluation for 4 months. Differences between EH301 and placebo-treated participants were evaluated based on standard clinical endpoints, including the revised ALS functional rating scale (ALSFRS-R), forced vital capacity (FVC), and the Medical Research Council (MRC) grading scale.Compared to placebo, participants treated with EH301 demonstrated significant improvements in the ALSFRS-R score, pulmonary function, muscular strength, and in skeletal muscle/fat weight ratio. EH301 was shown to significantly slow the progression of ALS relative to placebo, and even showed improvements in several key outcome measures compared with baseline.This study provides evidence in support of the disease-modifying effects of EH301 for the treatment of ALS.
publishDate	2019
dc.date.none.fl_str_mv	2019 2019-01-22 2019 2019-01-22 2023 2023-11-29 2023 2023-11-28
dc.type.none.fl_str_mv	journal article http://purl.org/coar/resource_type/c_6501
dc.type.openaire.fl_str_mv	info:eu-repo/semantics/article
format	article
dc.identifier.none.fl_str_mv	http://hdl.handle.net/20.500.12466/3210
url	http://hdl.handle.net/20.500.12466/3210
dc.language.none.fl_str_mv	Inglés eng
language_invalid_str_mv	Inglés
language	eng
dc.rights.none.fl_str_mv	open access http://purl.org/coar/access_right/c_abf2 Attribution-NonCommercial-NoDerivatives 4.0 Internacional http://creativecommons.org/licenses/by-nc-nd/4.0/
dc.rights.openaire.fl_str_mv	info:eu-repo/semantics/openAccess
rights_invalid_str_mv	open access http://purl.org/coar/access_right/c_abf2 Attribution-NonCommercial-NoDerivatives 4.0 Internacional http://creativecommons.org/licenses/by-nc-nd/4.0/
eu_rights_str_mv	openAccess
dc.format.none.fl_str_mv	application/pdf
dc.source.none.fl_str_mv	reponame:RIUCV. Repositorio de la Universidad Católica de Valencia San Vicente Mártir instname:Universidad Católica de Valencia San Vicente Mártir
instname_str	Universidad Católica de Valencia San Vicente Mártir
reponame_str	RIUCV. Repositorio de la Universidad Católica de Valencia San Vicente Mártir
collection	RIUCV. Repositorio de la Universidad Católica de Valencia San Vicente Mártir
repository.name.fl_str_mv
repository.mail.fl_str_mv
_version_	1869419260150808576
score	15,301603

Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study

Similares en LA Referencia