What is EXPERTS-ALS?
EXPERTS-ALS (EXPErimental medicine Route to Success in ALS) is a platform which aims to rapidly test drugs to assess their potential to slow MND progression. The platform will test three drugs at one time and, to start with, these will be repurposed drugs. These are drugs that have already been approved for use for other diseases and known to be safe, but in the laboratory have shown promise for MND. Everyone who takes part in the platform will receive a potential treatment - there is no placebo.
To assess the benefit of a potential treatment, EXPERTS-ALS will measure a marker of disease activity within the body, called neurofilament light chain (NfL). This is a protein that helps to maintain the structure of motor neurons and can be used as a marker of neuron damage, as levels of NfL in the blood rise as neurons are damaged and dying. Participant's levels of NfL will be measured throughout the study by taking blood samples. If the treatment is found to significantly reduce levels of NfL across the whole group of participants it suggests there are early signs of benefit and it should be priortised for further testing.
You can read more about EXPERTS-ALS in our blog post or on the EXPERTS-ALS website.
Which drugs are currently being tested?
Metformin
What is the drug thought to do in the body?
Metformin is an existing drug which is currently used in the treatment of diabetes to control blood sugar levels. It is now being investigated as a potential treatment for MND in those who have changes in the C9orf72 gene. In models of MND, metformin was able to reduce the number of toxic proteins that are made due to the C9orf72 gene change. Metformin works by blocking a key pathway (protein kinase R pathway) that is essential to the production of these toxic proteins.
Nifedipine
What is the drug thought to do in the body?
Nifedipine is an existing drug which is used to treat people with high blood pressure. It is now being investigated as a potential treatment of MND. Previous research has suggested that it could help to reduce cell damage from oxidative stress and helping cells survive longer. Nifedipine works by blocking the action of calcium channels in the body, which contribute to the oxidative stress response that damages cells.
Ropinirole
What is the drug thought to do in the body?
Ropinirole is an existing drug which is used to treat some symptoms of Parkinson's disease, such as muscle stiffness, spasms and tremors. A phase 1/2 clinical trial in people with MND found it to be safe and well tolerated but further trials are needed to see if it is an effective treatment. It has been suggested to help protect neurons from damage in models of MND. Ropinirole mimicks a chemical messenger in the brain called dopamine, which is involved in motor control and neuron signaling.
How can I take part in the trial?
Recruiting Sites
The trial is now recruiting in the UK at the following sites:
The following sites are still in set-up:
- Cambridge
- Edinburgh
- Exeter
- Liverpool
- University College London
- Newcastle
- Salford
Please contact the individual trial site, by clicking each site above, for more information on how to take part in the trial.
Trial Information
Phase: Phase 2
Placebo Controlled: No - this means that everyone joining the trial will receive a potential treatment.
Latest News
2024
November 2024- First sites are open for recruitment and screening people with MND.
2023
June 2023- EXPERTS-ALS platform was first announced.
Resources
Websites
Last updated: 29/11/2024