MND Clinical Trials update

Clinical Trials test new treatments, or existing treatments in a different condition, to evaluate their safety and effectiveness. Here, we have put together updates from some MND clinical trials within the last year. 

Clinical trials are a crucial step in any treatment being made available to people. Through clinical trials, vital information is gathered that can then be submitted to regulatory bodies, such as the Medicines and Healthcare products Regulatory Agency (MHRA) and Scottish Medicines Consortium (SMC). These organisations use this information to decide whether the treatment brings benefit and whether they will grant approval for use within their regions. There are different phases of clinical trials, each with different purposes. If you’d like more information, you can find it in this document we wrote in 2024.

Here we provide some updates from a selection of the many MND clinical trials which are ongoing worldwide. If you’d like more in depth information about Clinical Trials currently running in the UK, you can find it on the UK MND Clinical Studies Group website.

MND-SMART is the UK’s largest ever MND clinical trial and has allowed more people in Scotland to take part in an MND trial than ever before. MND SMART is a multi arm trial, meaning several drugs can be tested against one placebo group. This means that more drugs can be tested in a shorter amount of time and that there is less chance of a participant being placed on a placebo, compared to traditional trial methods. To date, four drugs have been tested in the MND SMART trial.

Updates from MND SMART in the last year include: In March 2026 the trial team made the decision to stop testing Amantadine. Their interim analysis of the data showed that it was not stopping the progression of MND. The trial continues to recruit people and Tacrolimus, which was added to the trial in early 2025, is still being tested. The trial team has indicated that they hope to add another drug arm in early 2027. During the last year MND SMART opened in a further two locations, bringing the total trial centres in the UK to 27 – including five in Scotland.

You can find more information about MND SMART on their website. 

EXPERTS-ALS is a UK based “pre-trial” rapid drug screening platform designed to accelerate progress towards drug therapies for MND. Participants on the trial have their levels of a biomarker, neurofilament light chain (NFL), measured. The changes in NFL levels can indicate if the treatment is potentially beneficial or not, and if it should be prioritised for testing in larger placebo-controlled trials. To date, five drugs have been tested by EXPERTS-ALS.

Updates from EXPERTS-ALS in the last year include: June 2026, Doxycycline and Salbutamol were added to the drugs being tested in the trial. August 2026, the trial committee decided, from interim analysis data, that Metformin and Nifedipine did not meaningfully slow MND progression and should not be tested further. In October 2026, the trial committee came to the same conclusion at interim analysis for Ropinirole. They opened two new UK sites, taking their total to 15, including one in Edinburgh.

More information can be found on their website.

PIONEER-ALS is a phase 2/3 trial run by VectorY Therapeutics investigating the safety and effects on clinical and biomarker factors in participants of a drug called Vtx-002. This experimental treatment is a gene therapy designed to target the misfolded TDP-43 clumps, seen in 95-97% of all MND cases, by helping the immune system to identify these clumps and remove them from cells.

Updates from PIONEER-ALS in the last year include: April 2026, VectorY announced that they had received EMA approval for PIONEER-ALS to run in the EU and UK, with sites located in the Netherlands, Belgium, and Sheffield (recruiting local participants only). September 2026, VectorY announced that they would be tripling their study size from 12 participants to 36 participants across the trial, which is scheduled to complete around October 2027.

More information can be found on VectorY’s website.

The FUSION trial, run by Ionis and Otsuka Pharmaceutical, tested a new, experimental treatment called Ulefnersen, for a rare inherited form of MND, FUS MND. The treatment was tested in participants across the world including the UK, USA, and several European countries. If you’d like to learn more about Ulefnersen, we previously published a news item about the trial and drug – Phase 3 trial for drug to treat a rare genetic form of MND shows encouraging results – MND Scotland.

Updates from FUSION in the last year include: September 2026, the companies announced positive topline results for Ulefnersen. They also announced that the drug had received Orphan status by both the Food and Drug Administration (FDA) in America and the European Medicines Agency (EMA) which can provide market protections and flexibility for companies making or testing treatments for groups of people with few or no alternative treatment options. There is an Early Access Programme (EAP) open for Ulefnersen. Otsuka confirmed to MND Scotland that the UK would be involved in the EAP but stated that no centres were currently selected. More information on the EAP can be found here.

More information on Ulefnersen and the FUSION trial can be found in the company’s press release – Otsuka Announces Transformative Phase 3 FUSION Results for Ulefnersen, Bringing the FUS-ALS Community Closer to a Potential Targeted Treatment | Otsuka US

The PARADIGM/PARAGON Trial, run by NeuroSense Therapeutics, was the name for the Phase 2b trial of a drug called PrimeC, which is a combination of two existing medications chosen to target known biological features of MND. In previous phases, PrimeC was shown to be safe for use in people. PARADIGM tested the drug in participants in Canada, Italy and Israel to further test safety as well as whether the drug showed beneficial effects for slowing MND progression. PARAGON is the name for the Phase 3 trial of PrimeC which will further test its effectiveness as a potential MND treatment and is expected to run in the US and EU.

Updates from PARADIGM/PARAGON in the last year include: November 2025, they announced that they had received FDA approval for their Phase 3 trial of PrimeC. In June 2026, the results from the PARADIGM trial were published as well as further information from the open label extension of the drug in a press release from NeuroSense. They stated a decrease in TDP-43 levels of those given PrimeC compared to the placebo group, although we don’t yet properly understand how this biomarker relates to progression of the disease. In October 2026 NeuroSense announced that they were awaiting a decision on funding for the PARAGON trial, which is expected to be made in the first few months of 2027.

More information can be found here –Paradigm – Neurosense.