Dr Josep Gamez, head of the Peripheral Nervous System Research Group of the Vall d'Hebron Research Institute (VHIR) and Maria Salvadó, investigator from the same group, have participated in an international clinical trial (AB10015) to test both efficacy and safety of masitinib amyotrophic lateral sclerosis (ALS) treatment. The study was published in the journal https://www.tandfonline.com/doi/full/10.1080/21678421.2019.1632346" Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration.ALS is a neurodegenerative disease that results in a progressive loss of motor neurons, leading to muscle strength deterioration, paralysis, loss of speech and shortness of breath. Life expectancy of these patients is around four years. However, they may vary due to the heterogeneity of nerve degeneration and may be broadened if they choose to receive mechanical ventilation and enteral nutrition.The prevalence is estimated to be 235,000 people worldwide and it will suffer a drastic increase in the coming years. Currently the only available treatment is riluzole, a glutamate antagonist, and its effects are modest: it prolongs life slightly by delaying disease progression, but without improving quality of life or decreasing functional loss. Therefore, it is urgent to develop new effective treatments to stop ALS. In this sense, Dr. Gamez and Dr. Salvadó, together with other hospitals and research centres from 9 countries, participated in a multicentre, double-blind, randomized study with 394 patients to check the efficacy of masitinib (in doses of 4.5 or 3.0 mg / kg / d). versus riluzole (100mg / d) and placebo. The study duration was 48 weeks.Masitinib is an oral tyrosine kinase inhibitor that in the preclinical phase (animal model) has shown promising results for the treatment of ALS."The current study has concluded that masitinib at a dose of 4.5 mg / kg / d delays the functional impairment of patients by 27% compared to placebo", says Dr. Josep Gamez, who is also the coordinator of the CSUR Rare Neuromuscular Diseases, the CSUR Hereditary Ataxia and Paraplegias, and the European Reference Network for Rare Neuromuscular Diseases (EURO-NMD) at Vall d'Hebron Barcelona Hospital Campus.To evaluate the results, the ALA Functional Rating Scale (ALSFRS), a tool that monitors patients' functional changes, was considered. Improvements in secondary goals such as forced vital capacity (FVC) and ALSAQ-40 score (a questionnaire for the assessment of subjective health status) were also significant. In terms of adverse effects, they are similar to those already known for masitinib."In the specific case of the 23 patients recruited by the Vall d'Hebron Hospital's ELA Unit, five of them are still receiving compassionate treatment, after an average of 81 months since the onset of the disease. Survival has never been achieved in previous trials", says Dr. Gamez.In the opinion of Dr. Gamez, the drug has been shown to be better than other drugs in slowing progression in a subgroup of patients. "Patient stratification is a recognized criterion for future clinical trials due to the clinical heterogeneity of patients with ALS. For the same reason, the drug may be effective in some patients even when the overall analysis is negative", he says.As a result of this new clinical evidence, phase 3 of the study can be started to confirm the results and initiate the application for commercialization of the drug.