Exploring new treatments through clinical trials 

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The clinical trials session is always the most anticipated and well attended session of the Symposium. This year was no different!  

Professor Merit Cudkowicz shared updates on a drug called Prime C. Prime C is a combination of two existing medications; an antibiotic called Ciprofloxacin and a non-steroidal anti-inflammatory agent called Celecoxib. Research has suggested Prime C may reduce inflammation and iron build-up in the brain, both of which are thought to contribute to motor neurone damage. Prime C was found to be safe and well tolerated in a phase 2 clinical trial, and showed promising signs of slowing disease progression over 6 months.  

Today, Professor Cudkowicz took a deeper dive into the results from the phase 2 trial and open-label extension. An open-label extension means everyone on the trial receives the drug after the initial trial. In the Prime C 12 month open label study, there was a 33% slowing in functional decline (as measured by the ALSFRS-R). Of particular note was an improvement in speech and swallowing. She also discussed how Prime C showed an effect on biological markers of MND after 18 months – it lowered levels of ferritin (a protein that stores iron in cells) and there was a small reduction in levels of a marker of neurone damage called Neurofilament Light chain. 

These results mean Prime C is now moving to a larger phase 3 trial, PARAGON in the US, Europe and Israel. This will be a 48-week study involving around 300 people with MND and aims to investigate whether treatment with Prime C can slow functional decline, measured by the ALSFRS-R. 

Read more Prime C

The MND Association’s vision is a world free from MND. Realising this vision means investing more in research, further developing partnerships with the research community, funding bodies and industry, while ensuring that advances in understanding and treating MND are communicated as quickly and effectively as possible.

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