Edaravone: a new treatment for ALS on the horizon?

  title={Edaravone: a new treatment for ALS on the horizon?},
  author={Orla Hardiman and Leonard H. van den Berg},
  journal={The Lancet Neurology},

Emerging Therapies in Amyotrophic Lateral Sclerosis

The epidemiology, pathophysiology, and several current and emerging treatment options for ALS including stem cell therapy are highlighted.

Subpial delivery of adeno-associated virus 9-synapsin-caveolin-1 (AAV9-SynCav1) preserves motor neuron and neuromuscular junction morphology, motor function, delays disease onset, and extends survival in hSOD1G93A mice

Test whether neuron-targeted Cav-1 upregulation in the spinal cord using AAV9-SynCav1 could improve motor function and extend longevity in mutant humanized mouse and rat models of familial ALS without reducing the toxic monogenic component indicates the therapeutic applicability of SynC Cav1 to treat ALS.

Long-term survival analysis of masitinib in amyotrophic lateral sclerosis

Analysis of long-term OS indicates that oral masitinib (4.5 mg/kg/day) could prolong survival by over 2 years as compared with placebo, provided that treatment starts prior to severe impairment of functionality.

Innovating Clinical Trials for Amyotrophic Lateral Sclerosis

A flexible design framework is proposed which naturally adapts the trial duration when inaccurate assumptions are made at the design stage such as the enrollment or survival rate and may serve a blueprint for future clinical trials in this population of patients.

Pain Phenotypes in Rare Musculoskeletal and Neuromuscular Diseases

Targeting for Success: Demonstrating Proof-of-Concept with Mechanistic Early Phase Clinical Pharmacology Studies for Disease-Modification in Neurodegenerative Disorders

Increasing the rational use of mechanistic biomarkers in early phase trials for these (targeted) therapies can increase R&D productivity with a quick win/fast fail approach in an area that has seen a nearly 100% failure rate to date.

Progress in progestin-based therapies for neurological disorders

Oxidative Stress in Amyotrophic Lateral Sclerosis: Pathophysiology and Opportunities for Pharmacological Intervention

Although antioxidant strategies represent a very promising approach to slow the progression of the disease, it is of utmost need to invest on the characterization of OS profiles representative of each subtype of patient, in order to develop personalized therapies, allowing to understand the characteristics of antioxidants that have beneficial effects on different subtypes of patients.



What does the ALSFRS-R really measure? A longitudinal and survival analysis of functional dimension subscores in amyotrophic lateral sclerosis

Results provide robust evidence that the ALSFRS-R should not be reported as a single combined score, but rather as domain specific subscores.

Investigation of the therapeutic effects of edaravone, a free radical scavenger, on amyotrophic lateral sclerosis (Phase II study)

  • H. YoshinoA. Kimura
  • Medicine, Psychology
    Amyotrophic lateral sclerosis : official publication of the World Federation of Neurology Research Group on Motor Neuron Diseases
  • 2006
Data from the present study suggest that edaravone is safe and may delay the progression of functional motor disturbances by reducing oxidative stress in ALS patients.

The changing picture of amyotrophic lateral sclerosis: lessons from European registers

Going forward, lessons learned from the collective experience of operating population-based ALS registers in Europe for over two decades are offered, focusing on register design, maintenance, identification and management of bias and the value of cross-national harmonisation and integration.

Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients

The reduction of ALSFRS-R was smaller in the edaravone group than in the placebo group, but efficacy of edarvone for treatment of ALS was not demonstrated.