Last updated –
UK treatment trials
This page contains some information regarding UK in-human clinical studies. We do not attempt to duplicate nor be a prima facia source of such studies. We have provided links to the formal trial pages and/or the MND Association trial detail. We aim to provide some commentary on the studies from a patient perspective and give a visual eye-catching GREEN for studies that are open for recruitment, so you might inquire, and potentially take part.
First in Human studies (PH I)
ANQUR (QRL-201) Gene therapy for STATHMIN-2 sporadic MND Status – COLLECTING DATA
Ph II (mid stage)
EXPERTS-ALS Rapid Drug screening platform using NfL reduction as indicator Status – ONGOING RECRUITING PLATFORM
VHB937 (ASTRALS) Targeting micro glia immune reaction Status – COLLECTING DATA
MND SMART PII/PIII Platform Trial Status – RECRUITING
Ph III Studies
Lithium Carbonate (MAGNET Platform) UNC13A Carriers trial – FAILED with futility declared October 2025
ATLAS (Tofersen) asymptomatic SOD1 patients CLOSED Completing July 2027
ION363 (Jacifusen Ulefnersn) Gene therapy for FUS gene Completing March 2028
Selected Completed Studies
MIROCALS (Low dose IL2)
Phase 2 completed July 2022 – Peer review paper published May 2025. The huge delay in the results being published was the subject of much community frustration.
You can read the ongoing status and..
it’s history
VALOR (tofersen) Phase 3 SOD1 patients (2022) – The first truly effective treatment for any form of our disease.
Riluzole – The only marginally effective treatment for sporadic MND (1994/1996)
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