Authors' objectives: Masitinib is intended to be used as a treatment option for patients with amyotrophic lateral sclerosis (ALS) as an add-on therapy to riluzole. Masitinib is a highly selective, orally administered, tyrosine kinase inhibitor that targets the c-Kit, Lyn and Fyn signalling pathways. By combined targeting of c-Kit, Lyn and Fyn, masitinib is particularly efficient in controlling mast cell survival, differentiation, and degranulation. ALS, also known as Lou Gherig's disease, is a form of Motor Neurone Disease (MND); a condition that affects parts of the nervous system that control voluntary muscle movement. ALS constitutes 65-85% of MND; the incidence rises with age and at any one time, about 3,000 people in the UK have ALS. Based on the 1,028 newly diagnosed cases per year, the average annual crude incidence rate of ALS in Europe is fairly uniform at 2.16 per 100,000 per year. Most people with ALS die from respiratory failure, usually within 3 to 5 years from the onset of symptoms, however about 10% of those with ALS survive for 10 years or more. The only disease-modifying therapy licensed for use in MND is riluzole, other interventions aim to improve quality of life and manage the complications of the disease. Surgical intervention is also an option to enable feeding, and tracheostomy with or without ventilation support may be required to aid breathing as respiratory muscle weakness increases. Masitinib is currently in one phase III clinical trial comparing the efficacy and safety of masitinib with that of placebo in patients with ALS; results are anticipated in 2017.

Project Status: Completed

Year Published: 2015

URL for published report: http://www.hsric.nihr.ac.uk/topics/masitinib-for-amyotrophic-lateral-sclerosis-add-on-therapy-to-riluzole/

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Horizon Scanning Centre

Contact Address: The NIHR Horizon Scanning Centre, Department of Public Health, Epidemiology, and Biostatistics, School of Health and Population Sciences, University of Birmingham, 90 Vincent Drive, Edgbaston, Birmingham, B15 2SP. United Kingdom. Tel: +44 121 414 7831, Fax: +44 121 2269

Contact Name: c.packer@bham.ac.uk

Contact Email: c.packer@bham.ac.uk

Copyright: NIHR Horizon Scanning Research&Intelligence Centre (NIHR HSRIC)