Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
| Autor: | Marta JuÁrez, Sandra Carrera, Rosario Salvador, Leonard Guarente, Carlos Barrios, José M. Estrela, Jose Luis Platero, Patricia Marchio, María Cuerda-Ballester, Raquel Pascual, Ryan W. Dellinger, Eraci Drehmer, Pilar GarcÍa-Pardo, David Sancho, Nieves De Bernardo, Elena Obrador, Cristian Fuente, Alfonso Forner, Jose Enrique De La Rubia, Jordi Caplliure-Llopis, Jorge Alarcón, María Benlloch, Carlos Villaron-Casales, Sandra Sancho-Castillo, Holly E. Holmes |
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| Rok vydání: | 2019 |
| Předmět: |
Male
Vital Capacity Pilot Projects Gastroenterology law.invention Placebos 0302 clinical medicine Randomized controlled trial law Stilbenes Medicine Amyotrophic lateral sclerosis Medicamento 1-(beta-D-Ribofuranosyl)nicotinamide chloride Middle Aged Drug Combinations Treatment Outcome Neurology Tolerability Disease Progression Female 3 5-Dimethoxy-4′-hydroxy-trans-stilbene Niacinamide medicine.medical_specialty Investigación médica Placebo Double blind 03 medical and health sciences Atrophy Double-Blind Method Internal medicine randomized control study Humans Muscle Strength human Aged Electromyography business.industry Amyotrophic Lateral Sclerosis medicine.disease 5-Dimethoxy-4 '-hydroxy-trans-stilbene Método doble ciego 1-(beta-D-Ribofuranosyl) nicotinamide chloride Ribonucleosides Neurology (clinical) business 030217 neurology & neurosurgery Esclerosis amiotrófica lateral |
| Zdroj: | Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration r-FISABIO: Repositorio Institucional de Producción Científica Fundación para el Fomento de la Investigación Sanitaria y Biomédica de la Comunitat Valenciana (FISABIO) r-FISABIO. Repositorio Institucional de Producción Científica instname |
| ISSN: | 2167-9223 |
| Popis: | 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. Catholic University San Vicente Mártir (grant 2017-216-001). University of Valencia (grant OTR2017-18255INVES). 3.286 JCR (2019) Q2, 68/204 Clinical Neurology 1.249 SJR (2019) Q1, 40/169 Neurology No data IDR 2019 UEV |
| Databáze: | OpenAIRE |
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