The research, published in the British Journal of Pharmacology, also found PMX205 slowed disease progression and significantly increased the muscle strength of mice.
In human terms, the researchers hope these findings could mean people with MND retain their motor function for longer and also live longer, if treated with PMX205.
More studies are now underway to determine the safety of PMX205 before it can be tested in humans.
What is PMX205?
PMX205 is a novel drug that blocks a key component of the immune system called “complement C5a.” C5a is involved in inflammation, which is the body’s response to injury. C5a is increased in the brains and blood of people with MND, and is thought to speed up the death of motor neurones. PMX205 is an inhibitor of C5a and dampens down inflammation, preventing further damage to the body.
What does this new research mean?
PMX205 now has clearly shown to be beneficial in mouse and (previously) rat models of MND. Therefore, this helps the case to take PMX205 further towards clinical trials for MND.
Is it related to familial or sporadic MND?
To date, PMX205 has only been tested in the familial SOD1G93A model, however the inflammatory pathway is likely to be active in all forms of MND. Future studies are planned to test PMX205 in sporadic MND models.
Is this a new treatment?
This research is expected to lead to clinical trials of PMX205. Formal preclinical safety and toxicity studies need to be successfully completed first, and this will take about two years before human testing can occur. The drug has obtained FDA (Food and Drug Administration) and EMA (European Medicines Agency) ‘orphan drug’ approval, which allows for an accelerated progression to human trials. The first clinical trials in healthy volunteers with PMX205 are currently anticipated to commence in Australia in late 2018. If PMX205 is found to be safe in healthy volunteers, clinical trials in patients with MND may be possible during 2019, with Australia as one potential site of these trials.
This research was supported by the National Health and Medical Research Council and the MND Research Institute of Australia.