6533b852fe1ef96bd12ab79b
RESEARCH PRODUCT
Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
Marta JuárezSandra CarreraRosario SalvadorLeonard GuarenteCarlos BarriosJosé M. EstrelaJose Luis PlateroPatricia MarchioMaría Cuerda BallesterRaquel PascualRyan W. DellingerEraci DrehmerPilar García-pardoDavid SanchoNieves De BernardoElena ObradorCristian FuenteAlfonso FornerJose Enrique De La RubiaJordi Caplliure-llopisJorge AlarcónMaría BenllochCarlos Villaron-casalesSandra Sancho CastilloHolly E. Holmessubject
MaleVital CapacityPilot ProjectsGastroenterologylaw.inventionPlacebos0302 clinical medicineRandomized controlled triallawStilbenesMedicineAmyotrophic lateral sclerosisMedicamento1-(beta-D-Ribofuranosyl)nicotinamide chlorideMiddle AgedDrug CombinationsTreatment OutcomeNeurologyTolerabilityDisease ProgressionFemale35-Dimethoxy-4′-hydroxy-trans-stilbeneNiacinamidemedicine.medical_specialty3Investigación médicaPlaceboDouble blind03 medical and health sciencesAtrophyDouble-Blind MethodInternal medicinerandomized control studyHumansMuscle StrengthhumanAgedElectromyographybusiness.industryAmyotrophic Lateral Sclerosismedicine.diseaseAmyotrophic lateral sclerosis5-Dimethoxy-4 '-hydroxy-trans-stilbeneMétodo doble ciego1-(beta-D-Ribofuranosyl) nicotinamide chlorideRibonucleosidesNeurology (clinical)business030217 neurology & neurosurgeryEsclerosis amiotrófica lateraldescription
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
| year | journal | country | edition | language |
|---|---|---|---|---|
| 2019-01-01 |