A world-first clinical trial investigating treatments for progressive multiple sclerosis (MS) will continue in Australia after alpha lipoic acid showed encouraging early results.
Interim findings from the UK-based Octopus trial found that alpha lipoic acid had shown enough promise to progress to the next stage of testing. A second treatment, metformin, will not continue after failing to demonstrate the early signs of effectiveness required for further investigation.
The Octopus trial uses a multi-arm, multi-stage adaptive design, allowing researchers to assess several potential treatments at the same time. Treatments that show sufficient promise can progress, while those that do not meet the required early measures can be discontinued and new treatment arms introduced.
The Australian extension of the trial, known as PLATYPUS, is joining Octopus at Stage 2 and is expected to expand recruitment across Australia.
MS Australia CEO Rohan Greenland said the early findings provided encouraging news for people living with progressive MS and represented an important stage in the international research effort.
“These are positive early results for people living with progressive MS and an important milestone in an international research effort that Australia is helping advance,” Mr Greenland said.
PLATYPUS is led in Australia by Griffith University neurologist Professor Simon Broadley, while Professor Jeremy Chataway of University College London leads the international Octopus trial.
Professor Broadley said the Stage 1 findings provided an encouraging indication that alpha lipoic acid could potentially help slow the progression of progressive MS, while stressing that the results do not yet prove the treatment is effective.
“The Octopus trial has shown that alpha lipoic acid was of benefit in Stage 1,” Professor Broadley said.
“While this is not proof that it works, it’s a positive sign that it may be a benefit, which we’re now going to test in Stage 2 over the next three years.”
Stage 1 assessed clinical measures and changes in brain volume over time. The next stage will examine whether alpha lipoic acid can slow the progression of disability among participants.
The decision to discontinue metformin demonstrates how the adaptive trial structure is intended to work. Rather than continuing to allocate research resources to a treatment that has not shown sufficient early promise, researchers can stop that treatment and focus on other options.
MSWA CEO Melanie Kiely said the initial findings demonstrated the potential value of the adaptive approach in allowing research to move between treatments more efficiently.
MSWA has been the leading Australian funder of PLATYPUS since its establishment, and the organisation said the trial’s expansion would allow more Australians to participate in the research.
For people living with progressive MS, the pace of research remains an important issue. Janine Watson, a Paralympian living with progressive MS and a member of MS Australia’s Lived Experience Expert Panel, said faster answers could provide greater hope for people facing the condition.
“It’s about giving back time to people with progressive MS and unfortunately with progressive MS, time is not our friend at all,” Ms Watson said.
PLATYPUS currently has participants enrolled through its lead site at Gold Coast University Hospital, which opened in October 2025. Further sites are expected to open in Perth and Hobart as recruitment expands across Australia.
Participants receiving alpha lipoic acid, along with those receiving their usual treatment, will remain enrolled in Stage 2. Researchers will monitor participants over the next three years to assess whether alpha lipoic acid can slow disability progression.
Additional treatment arms are expected to be introduced as other potential therapies are identified.
The Octopus trial is funded by the UK MS Society and sponsored by University College London’s Innovative Clinical Trials Unit. PLATYPUS is the Australian extension of the international trial, with Griffith University’s Professor Simon Broadley serving as Country Lead Investigator.
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