Monday, 10 August 2026
Research
Metformin and Nifedipine will be removed from the EXPERTS-ALS trial, because results show they do not lower neurofilament light chain (NfL) levels in the blood.
EXPERTS-ALS is a type of trial designed to screen drugs rapidly for their potential to slow the progression of MND. This is carried out by looking at NfL changes in the blood.
Unlike phase 3 trials that require hundreds of participants and take years to run, EXPERTS-ALS requires relatively few participants (around 30 per arm), can be carried out more quickly, and at a fraction of the cost. If a promising signal is found, the drug can then be fast-tracked for a phase 3 trial.
When motor neurons are damaged, NfL is released into the bloodstream. If a drug could protect motor neurons, we’d therefore expect a drop in blood NfL levels. Interim analysis results from the trial found that Metformin and Nifedipine did not lower NfL levels in the blood, meaning they are unlikely to slow down disease progression.
Whilst the removal of Metformin and Nifedipine will be disappointing news for the MND community, these definitive results allow EXPERTS-ALS to quickly move on and test the next potentially effective treatments. Without high-quality research like this, we are unable to rule out drugs that don’t work or find new drugs that do work.
EXPERTS-ALS remains open and active and is continuing to recruit new participants for its other drug arms. Ropinirole, Doxycycline and Salbutamol will remain on the trial, and we look forward to seeing the results of those.
Finding effective drugs for MND is not easy. Whilst we're disappointed that the first two drugs tested in the EXPERTS-ALS platform did not show potential for treating MND, we are encouraged to see this data has been produced so quickly. People with MND don't have time to wait. EXPERTS-ALS is addressing this by accelerating the time it takes to get to a positive or negative signal. We will continue to support EXPERTS-ALS to ensure more promising drugs are tested, increasing the chances that we will find a positive signal in the future.
We are thankful to all the people living with MND that took part in EXPERTS-ALS, and to their families for making their participation possible. Without you, this research would not be possible.
For further information about this latest update, please visit the EXPERTS-ALS website here.
This study was funded by the UK’s National Institute for Health & Care Research (NIHR), alongside My Name’5 Doddie Foundation, the MND Association and LifeArc, and is a project of the MND Research Institute.
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