Trophos announces conclusion of MitoTarget Consortium, achieving advanced understanding of neurodegenerative diseases

15-08-2012: Trophos SA announced the conclusion of the pan-European MitoTarget Project, the EU funded orphan disease project, with submission of the final report to the EC.

The results obtained in this project included additional confirmation that mitochondrial dysfunction plays an important role in neuron death, and therefore the progression of neurodegenerative diseases. The MitoTarget project contributed towards broader research required to discover which dysfunction/s is the leading cause of neurodegenerative diseases, key to finding effective drug targets in the future. It also further confirmed that Trophos’ lead drug candidate olesoxime, currently in an ongoing pivotal trial for the orphan neuromuscular disease Spinal Muscular Atrophy (SMA), targets mitochondria.

Various changes in mitochondrial function, distribution or number were found in all experimental models of neurodegenerative diseases including Alzheimer's Disease, Huntington's Disease, Hereditary Spastic Paraplegia (HSP), Amyotrophic Lateral Sclerosis (ALS), and even general aging. There were very promising results obtained in models of ALS, Huntington's Disease and Alzheimer's Disease.
 
Evidence produced by MitoTarget also proved mitochondrial targeted drugs should provide a part of the therapeutic strategy of neurodegenerative diseases. Combination therapeutics will be essential to attack multiple aspects of the disease including both the causes and the symptoms.

Trophos and the consortium have already published many of their results and further publications are planned describing results of the basic research as well as the clinical trial in ALS to aid all parties involved in future research to work towards eradicating and curing diseases that include SMA, Alzheimer's Disease, Huntington’s Disease as well as ALS.

The three-year project was initiated in 2007 with the creation of the consortium, commencing the main part of the activity in 2009, and was funded by the EU. Advocates included the British theoretical physicist, Stephen Hawking, CH, CBE, FRS, FRSA, and a patron of the Motor Neurone Disease Association.