EXPERTS-ALS is a ground-breaking project that will provide a platform to test drugs rapidly for their potential to slow the progression of Amyotrophic Lateral Sclerosis (ALS, the commonest form of Motor Neuron Disease). This will be done at a pace and scale not previously possible using human signals suggesting clinical benefit

Although EXPERTS-ALS cannot prove that a drug does or does not have long-term benefit in MND, it will quickly identify the most promising candidate drugs to go forward into the larger clinical trials needed to test this.  


ALS (Amyotrophic Lateral Sclerosis) and MND (Motor Neuron Disease) are terms that are often used interchangeably. MND is a more general term referring to a group of neurological disorders that affect the motor neurons in the body. ALS is the most common type of MND, characterised by the progressive degeneration of both upper motor neurons (located in the brain) and lower motor neurons (located in the spinal cord and brainstem). This degeneration leads to muscle weakness, atrophy, and eventually paralysis. ALS affects 1 in 300 people and at present there is no effective therapy. 

What is ‘experimental medicine’? 

Experimental medicine refers to investigations undertaken in humans (rather than laboratory cells or animal models), to look for early evidence of benefit in terms of slowing disease and to understand more about the biochemical mechanisms underlying MND. When such studies are testing drugs, these will already have been through the important safety testing procedures.

Who is funding it? 

This is an £8m investment in MND research by the National Institute for Health & Care Research (NIHR) through the Department of Health and Social Care (DHSC). Patient charities MND Association, My Name’5 Doddie Foundation, MND Scotland, and medical research charity LifeArc intend to provide additional support to extend the study to five years and support additional lab research. 

Who is running it?

Professor Martin Turner (University of Oxford) and Professor Chris McDermott (University of Sheffield) are jointly leading this. They are consultant neurologists who have been working in MND care and research for 20 years. There is a large team of other people, which will extend the involvement to 11 MND centres spread across the UK. EXPERTS-ALS is a flagship project of the UK MND Research Institute (UK MNDRI) and brings together the scientific expertise from within the NIHR Biomedical Research Centres (BRCs) and the Dementia Research Institute (DRI) around the UK. 

You can read more about our project team here.

What are the problems that EXPERTS-ALS is aiming to solve? 

Is this a short-cut around standard drug trials? 

No. There is no substitute for randomised-controlled trials (RCTs), which also need to have a placebo (‘dummy pill’) arm to prove the benefit of any drug beyond doubt. Such Phase 3 studies are expensive and time-consuming. The EXPERTS-ALS platform can be used to prioritise the choice of drug according to signals of likely human benefit, which can be detected in small groups of patients in less than 6 months. EXPERTS-ALS will help to ensure the drugs with the highest chance of success go into the Phase 3 studies. 

How does the study work in broad terms? 

Read more about the EXPERTS-ALS workstreams here.

Will EXPERTS-ALS be open to everyone living with MND? 

No. Some people’s MND progresses at a significantly slower pace than average. Although good for those individuals, their blood NFL marker may not be high enough to be able to detect a significant change in response to the drug being tested. Some people may also be intolerant of some of the drugs being tested. 

When will it get started for patients? 

EXPERTS-ALS is the largest logistical undertaking of its kind in MND in the UK. Everyone involved is committed to getting it set up as soon as possible, with first dosing of patients hoped to be from Summer 2024. Because we will not start until next summer, we are not registering interest at this point but will begin this process next year. 

Follow the progress of the EXPERTS-ALS platform here. 

Learning from everyone and building capacity

Every person with ALS who takes part will also provide additional biological samples which will enable us to create a bank of samples. This will enable scientists around the UK to learn from every patient, understand why drugs are working and uncover new insights into ALS. These additional studies will take place in Universities across the UK in a coordinated manner and will increase the capacity to perform research into ALS and ultimately lead to effective treatments faster.


EXPERTS-ALS will mark one of the largest shifts in both ALS patient care expectation and prospects for more rapid drug testing. 

It is expected to address key unmet needs for:

EXPERTS-ALS will also increase the capacity for Experimental Medicine and clinical trial activity in ALS across the BRC sites, support early career researchers develop into independent ALS researchers of the future, and facilitate more efficient routes to ALS treatment development through the use of biomarkers.