New adaptive clinical trial offers new hope for progressive multiple sclerosis

The first-ever adaptive clinical trial for Australians living with MS will seek to reverse neurological damage caused by progressive multiple sclerosis.

With an initial $4 million in funding, the trial, commencing in January 2024, will enable researchers to investigate the potential benefits of several medications simultaneously, giving hope to the cohort of people living with one of the most debilitating forms of MS and who currently have extremely limited treatment options.

Progressive MS is a clinical form of MS characterised by a progressive worsening of symptoms and disability without periods of recovery. This complex autoimmune and neurodegenerative condition manifests differently in individuals over time, but without treatment, disability steadily accumulates.

Working with a national and international group of clinicians and researchers, the multi-arm, multi-stage (MAMS) adaptive, innovative design, known as PLATYPUS (PLatform Adaptive Trial for remYelination and neuroProtection in mUltiple Sclerosis*), will test two repurposed drugs alongside each other, providing more timely results about whether the treatments are working.

MSWA is the leading funder of the trial having contributed $3 million to PLATYPUS. MSWA CEO Melanie Kiely said MSWA is proud to fund the ambitious research project which will transform the way we test treatments for progressive MS.

“PLATYPUS has the potential to deliver a significant breakthrough, as we aim to provide real-life outcomes which positively impact the lives of people living with progressive MS – which is always our focus.”

“By testing two repurposed drugs, we hope to find a treatment which can be quick to market for the people we support,” Ms Kiely said.

MS Australia CEO, Mr Rohan Greenland, said currently, despite a number of traditional clinical trials for progressive MS in Australia, there is no treatment that repairs damage in progressive MS.

“With an estimated 40% or around 13,000 people living with progressive MS, the launch of PLATYPUS today is a major milestone, the first adaptive clinical trial for MS in Australia. This will ensure a treatment opportunity for people with progressive MS, the greatest unmet need in the MS landscape,” Mr Greenland said.

PLATYPUS is an extension of the OCTOPUS (Optimal Clinical Trials Platform for Multiple Sclerosis) clinical trial, funded by the UK MS Society and launched April 2023.

Neurology Professor Simon Broadley from Griffith University’s School of Medicine and Dentistry and Chief Investigator of PLATYPUS, says the funding was critical and the new trials will allow the testing of multiple potential therapies simultaneously and bring results much faster than traditional clinical trial methods.

“Collaborating with our OCTOPUS partners in the UK, we’ll be trialling the drugs metformin, which is typically used to treat type 2 diabetes, and alpha-lipoic acid which is a health food supplement, these therapies have shown promise in promoting neuroprotection and/or myelin repair in MS,” Professor Broadley said.

The PLATYPUS trial will be rolled out across Australia through a collaboration of 20 academic and healthcare institutions and aims to recruit more than 250 participants in Australia.

“These multi-arm, multi-stage trials will require less time and fewer participants to assess the efficacy and cost-effectiveness of metformin and alpha-lipoic acid,” Professor Broadley said.

The PLATYPUS team includes leading, internationally recognised neurologists, neuroscientists, statisticians and health economists covering five states across Australia, as well as people with progressive MS.

Mr Nigel Caswell, diagnosed with MS in 1993 and MS Australia’s John Studdy Award winner in 2022, is excited for those people living with progressive MS in Australia.

“The launch of PLATYPUS today provides extraordinary hope for many people living with MS and is a major moment for MS research,” Mr Caswell said.

President of MS Australia, Associate Professor Des Graham, says he is personally and professionally delighted for Australia’s MS community that PLATYPUS can now be realised.

“With a $3 million grant from MSWA and a $1 million grant from MS Australia, this revolutionary adaptive trial provides tremendous promise for those living with progressive MS in Australia and MS Australia is extremely proud to lead this initiative,” Associate Professor Graham said.

MS Australia’s Head of Research, Dr Julia Morahan, says PLATYPUS will investigate whether these drugs reduce brain shrinkage and clinical progression of MS.

“We hope this will lead to positive trial outcomes that are readily translatable into practice, providing new hope for improved care for people with progressive MS in Australia and beyond,” Dr Morahan said.