Six people are diagnosed with motor neuron disease (MND) in the UK every day. Average survival after diagnosis is around three years, and for a third of people, it is less than one. That urgency is why the speed at which we can identify and test new treatments matters so much.

Finding effective treatments for MND is one of the hardest challenges in medicine. Traditional clinical trials are essential, but they can take many years to deliver results. The EXPERTS-ALS platform was created to complement these approaches: to move faster, learn more quickly, and focus resources on the treatments most likely to make a difference.

In this blog, we introduce EXPERTS-ALS, explain how it works, and look at the first three drugs now open to recruitment on the platform.

What is EXPERTS-ALS?

EXPERTS-ALS is a UK-wide research programme, developed through the UK MND Research Institute, designed to rapidly and efficiently test potential treatments for ALS. Rather than waiting years for a traditional trial to reach its primary endpoint, EXPERTS-ALS uses short-term studies, typically six to nine months, to look for early biological signs that a drug may be having a protective effect on motor neurons.

Central to this approach is measuring neurofilament light chain (NfL), a protein released into the bloodstream when nerve cells are damaged. By tracking NfL levels in participants over time, researchers can detect early biological signals indicating whether a treatment is slowing nerve cell damage. This makes NfL a powerful tool for identifying which treatments deserve further, larger-scale investigation.

This biomarker-led design is what makes EXPERTS-ALS genuinely innovative. It allows the team to make faster, more informed decisions about which therapies should progress, without asking participants to wait years for answers.

Repurposed and new drugs: what’s the difference?

EXPERTS-ALS is designed to evaluate two categories of treatment. Understanding the distinction helps explain why the platform can move quickly.

A repurposed drug is a medicine already approved for another condition that is now being tested for MND. Because these drugs have established safety profiles and are often widely available, they can enter clinical testing more quickly than entirely new compounds, and at lower cost. This enables EXPERTS-ALS to generate early evidence efficiently, particularly in the platform’s initial phase.

A new drug is a compound specifically developed to target a disease mechanism. These drugs must complete extensive safety testing before they can be given to people. Once that threshold is met, EXPERTS-ALS is designed to evaluate these compounds too.

The EXPERTS-ALS platform is designed to evaluate both approaches. While the current focus is on repurposed medicines, for which evidence already supports earlier testing, the platform will also test new drugs once they have completed the necessary safety studies. You can read more about our decision to prioritise repurposed drugs here.

The first three drugs

Using early biological readouts, EXPERTS-ALS can quickly identify the most promising treatments and prioritise them for larger, placebo-controlled trials. The first three drugs currently open to recruitment are:

  • Metformin
  • Nifedipine
  • Ropinirole

Why metformin?

Metformin is one of the most widely prescribed treatments for type 2 diabetes. Beyond its role in controlling blood sugar, research has shown that metformin can influence how cells produce energy and respond to stress, processes that are thought to be disrupted in ALS.

One theory of ALS suggests that motor neurons struggle to produce enough energy and are progressively damaged as a result. Metformin activates a pathway involved in energy regulation called AMPK, which may help to improve mitochondrial function (the cell’s energy-producing machinery), reduce oxidative stress (a process that causes cellular damage), and support autophagy (the cell’s natural recycling system). By restoring energy balance and reducing cellular stress, metformin could help motor neurons remain healthier for longer.

Why nifedipine?

Nifedipine is a well-established medication used to treat high blood pressure and certain heart conditions. It works by blocking specific channels that transport calcium into cells.

Calcium plays an essential role in nerve cell function, but too much calcium inside neurons can be harmful. In ALS, evidence suggests that abnormal calcium handling may contribute to nerve cell damage. By gently reducing calcium entry into cells, nifedipine may lower stress within motor neurons, reduce oxidative damage, and support healthier calcium balance in the brain and spinal cord.

Within EXPERTS-ALS, nifedipine is being tested over six months to build evidence in people with ALS and explore whether it shows signs of protecting motor neurons from excessive stress.

Why ropinirole?

Ropinirole is commonly used to treat Parkinson’s disease. While it primarily acts on dopamine receptors, it also has properties that may be relevant to ALS, including antioxidant effects and support for mitochondrial function.

Laboratory and early research suggest that ropinirole may reduce oxidative stress within neurons and support mitochondrial health and energy production. Although ALS and Parkinson’s disease are distinct conditions, they share some underlying cellular mechanisms, making ropinirole a logical candidate for drug repurposing.

Looking ahead

The biology of ALS is complex and still being uncovered. EXPERTS-ALS is designed to help researchers learn more about these processes while efficiently identifying which treatments show early promise. We’ve also recently announced the inclusion of smilagenin and neflamapimod in the platform, in partnership with industry, a further sign that EXPERTS-ALS is becoming a trusted mechanism for bringing academic and industry expertise together around the most promising therapies.

When strong biological signals are found, the EXPERTS-ALS team shares these findings openly, contributing to the national and international evidence base and helping to prioritise the right treatments for larger trials.

People living with MND cannot afford a slow research system. By shortening the time from laboratory discovery to patient benefit, EXPERTS-ALS is putting the speed and coordination of the UK MND Research Institute’s infrastructure to work, for the people who need it most.

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