Project to test drug combinations for motor neuron disease

Neurons

A new project by researchers in Edinburgh aims to identify combinations of existing drugs that could be used together to treat motor neuron disease (MND).

Led by Prof Siddharthan Chandran, the £3.3 million project is funded by the medical research charity LifeArc, as part of an ongoing partnership between the charity and the UK DRI.

The partnership brings together the strengths of UK DRI’s research into discovery science with LifeArc’s translational expertise to take exciting lab discoveries forward and translate them into tangible benefits for patients.

There is currently only one drug approved to treat MND in the UK, riluzole, which has only a modest effect.

The new project seeks to drastically accelerate the development of new treatments by identifying existing drugs which target multiple disease mechanisms implicated in MND. Typically, new drugs can take up to 15 years to progress through development and clinical trial stages, but with this approach, treatments could be tested in the clinic within four years.

Prof Chandran, Group Leader at the UK Dementia Research Institute (UK DRI) and Director of the Euan MacDonald Centre for MND Research, the University of Edinburgh, said: “As has been shown for cancer therapy, using combinations of drugs that target different pathways might be our best chance of slowing or stopping the progression of MND. This innovative project is an important next step in identifying effective medicines for MND.”

The MND study

In the first stage of the study, the researchers will prioritise the top drug candidates, using both laboratory-based tests on motor neurons grown in the lab from patient donated stem cells, and a machine-learning, artificial intelligence approach to review published scientific studies of MND.

Next, the top candidate drugs will be tested in pairs in combination in the stem cell models of MND, against different biological pathways known to be implicated in MND.

The ultimate goal is to seek regulatory approval to test the most promising and effective combinations of drugs in the Euan MacDonald Centre’s MND-SMART (Motor Neuron Disease – Systematic Multi-arm Adaptive Randomised Trial) trial.

This pioneering trial across 20 sites in the UK is designed to shorten the time it takes to find medicines that can slow or stop MND by testing several treatments at the same time. It is also an adaptive trial which means that new drugs can be added, and those proven ineffective can be dropped.

The project complements another recently announced MND initiative, EXPERT-ALS, which aims to rapidly identify promising drug candidates in small scale trials, before definitive evaluation in Phase III platform trials such as MND-SMART.

Dr Paul Wright, MND Translational Challenge Lead at LifeArc said: “Our involvement in this research is part of an ambitious long-term £100m funding programme we have launched to help tackle neurodegenerative conditions and find treatments where none currently exist. By working with UK DRI we are uncovering promising life science research, like Professor Siddharthan’s, that we can support with funding or by offering our scientific resources and expertise in translational research.”

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