MICA: M102, A Combined NRF2 and HSF1 Activator for the Treatment of Motor Neuron Disease

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The aim of this project is to complete the key stages of drug development needed to enable a clinical trial for a promising new treatment for motor neuron disease (MND), a rapidly progressive, fatal neurodegenerative disorder. At the University of Sheffield we have identified a candidate drug called M102, which can get into the brain and spinal cord and activate a number of biological pathways which switch on the expression of a range of genes. These genes have a number of effects in cells which improve their ability to survive various stress factors which are known to play a role in the death of neurons. These stress factors include free radicals, inflammation and protein clumps or aggregates.We hope that by targeting multiple different causes of neuronal stress and death we will increase the probability that this drug will work in a wide range of patients and at all stages of the disease process. We have already shown in various animal models and in brain cells from patients with MND (derived from their skin cells) that we can slow down the disease process and protect neurons from death. The next stage is to manufacture the drug to accepted regulatory standards and test it for possible side effects in animals- a requirement before conducting studies in humans. These studies will be carried out by expert companies who specialise in this work. Another strand of work will be conducted at Sheffield to allow us to be able to identify using biological signals called biomarkers the MND patients who are most likely to benefit from taking the drug. These biomarkers increase the chance of the drug showing effects in large clinical studies.We have already done some preliminary studies to show that our plan is likely to be successful but we will conduct the work in stages to reduce the risks associated with problems arising. As a University it is unlikely that we would take the drug through all the stages of clinical testing and so we have an Industrial partner, Aclipse One, who will share the costs and the risks of the project but will then be able to take the drug through the clinical stages of development.