Neurodegenerative Diseases: Complexity in need of a novel system-wide approach

Therapeutic discovery for neurodegenerative conditions has proven very challenging.  Progress in the discovery of disease-modifying drugs for diseases such as Parkinson's (PD), Alzheimer's (AD) and Amyotrophic Lateral Sclerosis (ALS/Motoneuron disease or Lou Gehrig's) based on targeted pharmacological approaches has been largely unsuccessful. This intractability to conventional single target approaches is now believed to be based on a mismatch with the complexity of disease biology. Advances in research into the molecular pathophysiology of these diseases has revealed a multiplicity of converging and interacting cellular systems that make pharmacological resolution at a single target relatively unworkable in the majority of disease conditions. However, the advent of informatics techniques that detect complex signatures in patterns emanating from both biological and chemical datasets, now enables the capturing of such complexity and its targeting with drugs in a way that is far more consistent with this complexity. Mitodys Therapeutics has developed a drug repositioning discovery workflow that combines the translation of complex gene expression patterns into predictive drug lists, and the screening and selection of disease condition-active drugs using human pluripotent stem cell-driven "disease in a dish" modelling that captures a disease molecular or cellular phenotype. The disease focus of the company is on select neurodegenerative disease conditions including a set of early involuntary movements (dyskinesia) suffered by PD patients that is induced by the administration of L-Dopa and a set of rare disorders such as ALS and Multiple System Atrophy (MSA).

MitoDys Therapeutics Ltd is a company registered in England and Wales. Registered number: 08227300.
Registered office: Cobwebs, Hixet Wood, Chipping Norton, OX7 3SB, United Kingdom