An experimental treatment for motor neurone disease is moving into patient testing in the UK through a new early-stage clinical trial.
The treatment, known as TRCN-1023, will be studied through the FUNCTION ALS trial at University College London Hospitals and Sheffield Teaching Hospitals.
It is an encouraging milestone in MND research, but researchers emphasise that the treatment remains experimental. The trial must establish its safety and biological effects before scientists can determine whether it may benefit people living with MND.
What is TRCN-1023?
TRCN-1023 is designed to restore the function of a protein called UNC13A, which helps nerve cells communicate with one another and with muscles.
In around 97% of people with MND, a protein called TDP-43 does not behave normally. This can interfere with the instructions used to produce UNC13A, resulting in lower levels of the working protein.
TRCN-1023 is an antisense oligonucleotide, commonly shortened to ASO. It is designed to correct the way the UNC13A instructions are processed at the RNA level, helping the body produce a functional version of the protein.
Although ASOs are sometimes described as genetic medicines, TRCN-1023 does not permanently change a person’s DNA.
What will the trial examine?
FUNCTION ALS is a Phase 1/2 trial expected to involve approximately 30 participants internationally.
Participants will receive either TRCN-1023 or a placebo through an injection into the fluid surrounding the spinal cord. Researchers will primarily examine:
- The treatment’s safety and tolerability
- How it behaves within the body
- Whether it produces the intended biological effects
- Changes in biomarkers, speech and movement
The initial study period will last 24 weeks. Because this is the first stage of testing in people, the trial cannot yet tell us whether TRCN-1023 is an effective treatment for MND.
UK recruitment is limited and subject to detailed eligibility criteria. Anyone interested in clinical research should speak to their specialist MND team rather than contacting the MND Foundation for access.
From laboratory research to patient testing
TRCN-1023 grew from research led by Professor Pietro Fratta at the UCL Queen Square Institute of Neurology and Professor Aaron Gitler at Stanford University.
Trace Neuroscience was established to develop the experimental treatment and is sponsoring the FUNCTION ALS trial.
Moving this work from the laboratory into a clinical study is an important step. It demonstrates how sustained research can identify promising biological targets and translate them into potential treatments for testing.
Why clinical capacity matters
The MND Foundation is not funding the FUNCTION ALS trial. However, its launch at Queen Square reinforces the importance of specialist clinicians and the clinical infrastructure needed to deliver increasingly complex MND research and treatments.
Separately, the Foundation is funding clinical capacity within the Queen Square neurology team to support access to Tofersen for eligible people with SOD1-related MND.
Scientific discovery is only one part of the journey. Progress must also be supported by the specialist people and services needed to deliver it safely and help suitable patients access it.
We will follow the FUNCTION ALS trial and share responsible updates when reliable findings become available.
