EXPERTS-ALS rules out Ropinirole as a potential treatment for MND

The latest results from the EXPERTS-ALS clinical trial have shown that Ropinirole is unlikely to be an effective treatment for motor neuron disease (MND), and the drug will now be removed from the study.

EXPERTS-ALS is an innovative trial designed to rapidly test candidate drugs for their potential to slow MND progression. Rather than relying on large, lengthy clinical trials, the study uses changes in levels of neurofilament light chain (NfL), a biomarker of nerve cell damage, to identify promising treatments more quickly.

When motor neurons are damaged, NfL is released into the bloodstream. If a treatment is having a protective effect on these nerve cells, researchers would expect to see a reduction in blood NfL levels over time. Results from the Ropinirole arm of the trial found no evidence of a reduction in NfL, indicating that the drug is unlikely to slow disease progression in people living with MND.

The findings add to previous interim results showing that Metformin and Nifedipine did not demonstrate the biological effects needed to justify further investigation in MND. While it is disappointing, these results are an important part of the drug discovery process. Identifying ineffective treatments early helps ensure that time, funding and research effort can be focused on the therapies with the greatest potential.

One of the key strengths of EXPERTS-ALS is its ability to make these decisions quickly. With around 30 participants per treatment arm, the platform can assess multiple drugs in parallel and generate robust evidence at a fraction of the time and cost required for a traditional Phase 3 trial. Treatments that show promising signals can then be prioritised for larger-scale testing.

EXPERTS-ALS remains open and active, with recruitment continuing into other treatment arms. Doxycycline and Salbutamol are still being evaluated, and researchers look forward to seeing whether either show signs of slowing disease progression.

Through studies such as EXPERTS-ALS, researchers are accelerating the search for effective MND treatments by quickly identifying which approaches should move forward, and which should not.

We are thankful to everyone living with MND taking part in EXPERTS-ALS, without you, this research would not be possible.

For further information about this latest update, please visit the EXPERTS-ALS website here.

EXPERTS-ALS is funded by a partnership between the UKRI Medical Research Council (MRC) and the National Institute for Health and Care Research (NIHR), with support from My Name’5 Doddie Foundation, the MND Association and LifeArc. It is a project of the UK MND Research Institute.