What is the drug thought to do in the body?
Small unstable molecules called free radicals are produced naturally by your body. Free radicals are removed by antioxidants. It is important to maintain a balance between free radicals and antioxidants. If there are too many free radicals, they can cause damage in the body. This damage is called ‘oxidative stress’. Oxidative stress is thought to be a contributor to MND. ALCAR is thought to reduce oxidative stress, and so reduce damage from free radicals in the body. ALCAR is produced naturally by the body, and is already known to be safe and well tolerated, so no phase 1 trial was needed.
Phase 2/3 - ALCALS
Current status: Not yet recruiting
Primary objective: To investigate whether ALCAR is effective in people with MND
The trial will assess the effect of ALCAR on disease progression, using the ALSFRS-R scale. It will also look at other clinical measures, and several disease biomarkers. These biomarkers will indicate whether the drug is having a biological effect. One biomarker which will be used is neurofilament light (NFL), a protein which is released into the blood and spinal fluid when neurons are damaged and die. People who take part will be randomly assigned to receive ALCAR (which will be tested in two doses) or placebo (dummy drug). They will receive the drug or placebo for 48 weeks. You can find out more about the trial here.
This trial will be recruiting in Italy and Australia.
Previous Clinical Trials
Phase 2
A small phase 2 trial was carried out to assess the effects of ALCAR on disability and survival in people with MND. The trial looked at how many participants were no longer self-sufficient at the end of the trial (able to swallow, handle utensils, and walk). Participants were randomly assigned to receive either ALCAR or a placebo, with 42 participants receiving ALCAR and 40 receiving placebo. The trial lasted 48 weeks. Less participants taking ALCAR became non-self-sufficient, suggesting that ALCAR may be effective in MND. ALCAR was found to be safe and well tolerated in people with MND.
Last updated: 23/05/25