Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
Por:
de la Rubia JE, Drehmer E, Platero JL, Benlloch M, Caplliure-Llopis J, Villaron-Casales C, de Bernardo N, AlarcÓn J, Fuente C, Carrera S, Sancho D, GarcÍa-Pardo P, Pascual R, JuÁrez M, Cuerda-Ballester M, Forner A, Sancho-Castillo S, Barrios C, Obrador E, Marchio P, Salvador R, Holmes HE, Dellinger RW, Guarente L and Estrela JM
Publicada:
2 ene 2019
Resumen:
Background: 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). Methods: 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. Results: 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. Conclusions: This study provides evidence in support of the disease-modifying effects of EH301 for the treatment of ALS.
Filiaciones:
de la Rubia JE:
a Department of Nursing , Catholic University San Vicente Mártir , Valencia , Spain
Drehmer E:
b Department of Health and Functional Valorization , Catholic University San Vicente Martir , Valencia , Spain
Platero JL:
a Department of Nursing , Catholic University San Vicente Mártir , Valencia , Spain
Benlloch M:
a Department of Nursing , Catholic University San Vicente Mártir , Valencia , Spain
:
c Primary Care Service, University Hospital La Ribera , Alcira , Spain
Villaron-Casales C:
d Department of Physiotherapy , European University of Valencia , Valencia , Spain
de Bernardo N:
d Department of Physiotherapy , European University of Valencia , Valencia , Spain
AlarcÓn J:
d Department of Physiotherapy , European University of Valencia , Valencia , Spain
Fuente C:
a Department of Nursing , Catholic University San Vicente Mártir , Valencia , Spain
Carrera S:
e Department of Health Sciences , Catholic University San Vicente Martir , Valencia , Spain
Sancho D:
a Department of Nursing , Catholic University San Vicente Mártir , Valencia , Spain
GarcÍa-Pardo P:
f Department of Medicine , University Jaume I , Castellón , Spain
Pascual R:
g Rehabilitation Service, General University Hospital , Valencia , Spain
JuÁrez M:
h Clínica Artes & Timefit , Valencia , Spain
Cuerda-Ballester M:
a Department of Nursing , Catholic University San Vicente Mártir , Valencia , Spain
Forner A:
a Department of Nursing , Catholic University San Vicente Mártir , Valencia , Spain
Sancho-Castillo S:
a Department of Nursing , Catholic University San Vicente Mártir , Valencia , Spain
Barrios C:
i Institute for Research on Musculoskeletal Disorders , Catholic University San Vicente Mártir , Valencia , Spain
Obrador E:
j Department of Physiology , University of Valencia , Valencia , Spain
Marchio P:
k Virgen del Consuelo Hospital , Valencia , Spain
Salvador R:
j Department of Physiology , University of Valencia , Valencia , Spain
Holmes HE:
l Elysium Health, Inc. , New York , NY , USA and
Dellinger RW:
l Elysium Health, Inc. , New York , NY , USA and
Guarente L:
l Elysium Health, Inc. , New York , NY , USA and
m Department of Biology and Glenn Laboratories for the Science of Aging , MIT , Cambridge , MA , USA
Estrela JM:
j Department of Physiology , University of Valencia , Valencia , Spain
Green Published
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