Researchers at the Euan MacDonald Centre for MND Research have recently published the findings of a research study that has provided a new focus for potential drug treatments for MND.

The research team created a model of MND in a dish using stem cells donated by people with the most common inherited form of MND (the C9orf72 mutation).

Using this model within their study, the research team, led by Dr Arpan Mehta, showed three things: Firstly that the motor nerve cables (axons) are damaged; secondly that the energy source, or batteries (mitochondria), within the motor nerve cables are also damaged; and thirdly that if the energy is boosted in the faulty batteries, the nerve cable damage can be repaired.

“This third finding is a new discovery and it opens up potentially new possibilities for treatments. This is an exciting discovery but the very first step in a journey.” Dr Arpan Mehta

The study was funded by the Medical Research Council, Motor Neurone Disease Association, Euan MacDonald Centre for MND Research, My Name’5 Doddie Foundation, UK Dementia Research Institute and Anne Rowling Regenerative Neurology Clinic.

What happens next?
Researchers in Edinburgh, along with many others across the international research community, will now work at pace to move forward with these finding. Using stem cells, donated by people with the commonest form of MND, we will screen hundreds and thousands of medicines, drugs, and compounds from nature, to see if they can reproduce the same effect of boosting the batteries of motor nerve cells.

To the best of our knowledge, there is no current licensed drug out there that specifically and safely targets the cellular batteries in motor nerve cells. But, that does not exclude the possibility that there may be medicines that can do this and researchers are now looking for them.

If a drug is found, the next step will be to ask the question: “are these medicines safe and effective?” This can only be done through properly regulated and conducted clinical trials.

How long will this take?
It is always difficult to predict how long this will take but given the rate of progress across the MND research community, we are cautiously hopeful that medicines that can begin to slow the disease will be identified in the coming decade.

The research community are now well placed, owing to advanced clinical trial platforms in place such as MND-SMART and TRICALS, to efficiently and swiftly take laboratory discoveries into clinical trials.