What does the drug do?

RT001 is made from slightly modified linoleic acid (LA). Linoleic acid is typically part of a normal human diet and is found in most oils. RT001 can protect cells from lipid peroxidation – a process that is believed to cause damage in several neurodegenerative diseases, including MND. RT001 has previously been shown to be safe when administered to humans. However, RT001 can currently only be prescribed in research settings.

Trial Outcome

The results of the phase 2 clinical trial found that those who received RT001 had a small slowing in disease progression compared to those who took the placebo. However, this was found not to reach statistical significance due to the small size of the study. The results did show a potential that the treatment is more beneficial for those with severe disease, however an additional, larger, trial is needed to confirm these results. You can read more about the results here

Previous Clinical Trials

Phase 2

A Phase 2 trial, sponsored by Retrotope Inc, will test the safety and efficacy of RT001 in a randomised, double-blind, placebo-controlled study. Participants will be randomly assigned to receive RT001 or placebo (dummy drug looks exactly the same as the RT001, but does not contain any active medicine). Both the participant and the research team do not know who receives a placebo treatment or RT001. The entire study duration is 28 weeks. You can read more about the trial here.

Latest News


Sept 2022 - The results of the trial were announced, suggesting a small potential slowing in disease progression. You can read more here


April 2021 - The trial reached full enrollment of 40 participants within 6 weeks. You can read more here.


March 2021 - The trial opened for recruitment. 


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Last updated: 23/01/2023