Motor Neurone Disease Clinical Trials in the UK (2026)
26 May 202611 min readTrialConnect Research Team
Motor Neurone Disease (MND), also known as Amyotrophic Lateral Sclerosis (ALS), affects around 5,000 people in the UK at any one time, with about 1,500 new diagnoses per year. Average survival is 2-5 years from symptom onset, making clinical trials critically important. UK centres are at the forefront of gene therapy research, antisense oligonucleotide treatments, and novel neuroprotective approaches.
The UK is a world leader in MND research, coordinated through the MND Association, NIHR, and the MRC. Major trial centres include the UCL Queen Square MND Centre, the Oxford MND Centre, and centres in Sheffield, Birmingham, and Edinburgh. The UK was pivotal in trials of tofersen (SOD1 gene therapy) and continues to lead in genetic and disease-modifying research.
There are currently over 25 actively recruiting MND/ALS trials in the UK, including gene therapy, antisense, stem cell, and symptomatic treatment studies.
Types of MND/ALS Trials
Gene Therapy
Targeting genetic causes of MND — SOD1 gene silencing, C9orf72 antisense, and AAV-delivered gene therapy for familial ALS.
Antisense Oligonucleotides
ASOs that reduce production of toxic proteins like SOD1 and C9orf72 dipeptide repeat proteins.
Neuroprotection
Drugs that protect motor neurons from degeneration, including anti-inflammatory, antioxidant, and mitochondrial therapies.
Stem Cell Therapy
Investigational stem cell approaches to replace or support dying motor neurons and modify the disease environment.
Gene Therapy & Genetic Trials
Tofersen (SOD1-ALS) — antisense oligonucleotide targeting SOD1 mRNA to reduce toxic SOD1 protein. Now approved for SOD1-ALS. UK trials testing: earlier use in presymptomatic SOD1 carriers, long-term outcomes, and optimal dosing schedules
C9orf72 antisense therapy — ASOs targeting the most common genetic cause of familial ALS (C9orf72 hexanucleotide repeat expansion). UK Phase 1/2 trials at UCL Queen Square actively recruiting
AAV gene therapy — adeno-associated virus vectors delivering therapeutic genes to motor neurons, including anti-apoptotic and neurotrophic factors. UK early-phase trials ongoing
FUS-targeted therapy — for FUS-mutant ALS (rare but aggressive), with UK trials testing ASOs and small molecules targeting FUS protein
ATAXIN-2 targeting — ASO therapy targeting ataxin-2 (a modifier of ALS severity), in UK Phase 1/2 trials for both sporadic and familial ALS
Disease-Modifying & Symptomatic Trials
Riluzole optimisation — the only long-standing approved MND drug. UK trials testing: higher doses, combination with new agents, and biomarkers to predict response
Edaravone — free radical scavenger approved for ALS. UK trials evaluating: real-world effectiveness, optimal patient selection, and combination with riluzole
Anti-inflammatory approaches — targeting neuroinflammation with masitinib, azathioprine, and novel microglial modulators in UK Phase 2/3 trials
Mitochondrial therapies — drugs targeting mitochondrial dysfunction in ALS, including copper histidine and novel compounds in UK early-phase trials
Symptomatic management — trials for muscle cramps, sialorrhoea (excess saliva), respiratory support strategies, cognitive symptoms, and nutritional interventions
Stem Cell & Regenerative Approaches
Neural stem cell transplantation — direct injection of neural stem cells into the spinal cord to provide neurotrophic support. UK trials at Sheffield and UCL testing safety and efficacy
Mesenchymal stem cell therapy — MSCs engineered to secrete neurotrophic factors, delivered intrathecally. UK Phase 1/2 trials showing promising safety data
Induced pluripotent stem cell (iPSC) research — using patient-derived iPSCs to study disease mechanisms and screen drugs. UK biobank facilitating personalised medicine approaches
Exosome therapy — stem cell-derived exosomes carrying neuroprotective cargo as a less invasive alternative to cell transplantation, in UK preclinical and early-phase trials
Who Can Participate?
Gene therapy trials — confirmed SOD1, C9orf72, FUS, or other genetic mutation (for mutation-specific trials), may include presymptomatic gene carriers for some studies
Disease-modifying trials — probable or definite ALS (El Escorial criteria), disease duration <2-3 years, FVC >50% predicted, not on mechanical ventilation
Stem cell trials — early-stage ALS with preserved breathing function, willing to undergo intrathecal or intraspinal procedures
General criteria — age 18-80, able to give informed consent, no other significant neurological disease, adequate respiratory and nutritional function
UK MND/ALS Trial Locations
London — UCL Queen Square MND Centre, King's College Hospital
Oxford — Oxford MND Centre, John Radcliffe Hospital
Sheffield — Sheffield Teaching Hospitals (MND research hub)
Birmingham — Queen Elizabeth Hospital Birmingham
Edinburgh — Royal Infirmary of Edinburgh / Anne Rowling Clinic
Cambridge — Addenbrooke's Hospital
🧬 Tip: Get Genetic Testing Early
About 10% of MND is familial (inherited) and up to 15% of sporadic cases carry relevant gene mutations. Request genetic testing for SOD1, C9orf72, FUS, and TARDBP at diagnosis — this opens the door to gene-specific trials. The MND Association provides free genetic counselling. Also ask about neurofilament light chain (NfL) testing, which is becoming a key biomarker for trial eligibility and disease monitoring.
How to Find Your Match
Use our Smart Matcher to find motor neurone disease trials tailored to your specific situation. Whether you are newly diagnosed, exploring targeted therapy, or seeking advanced treatment options, we can match you to actively recruiting studies.