Trial testing metformin, alpha-lipoic acid in progressive MS set to start

A new clinical trial in Australia, called PLATYPUS, will simultaneously test two medications that have already been approved for other conditions in people with progressive forms of multiple sclerosis (MS).

The study, expected to launch in January, will use an adaptive design, meaning data will be analyzed as it’s available rather than waiting till the end of the study as is usually done in clinical trials. This will let researchers drop medications that aren’t producing a meaningful effect and add new ones as the trial progresses.

“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,” Rohan Greenland, CEO of MS Australia, said in a press release.

Progressive forms of MS are marked by symptoms that continuously worsen over time, independent of relapses where symptoms suddenly worsen. These types of MS have historically proven more difficult to treat than relapsing types 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,” said Julia Morahan, PhD, head of research at MS Australia.

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What will the PLATYPUS study do?

The trial will be open to adults ages 25-70 with a diagnosis of either primary progressive MS (PPMS) or secondary progressive MS (SPMS). To be eligible, patients also must have an Expanded Disability Status Scale (EDSS) score of 4-8, meaning they must have substantial disability, but still be up and about most of the day. More information about participating will be made available pending regulatory approvals, according to MS Australia.

Participants in PLATYPUS, short for PLatform Adaptive Trial for remYelination and neuroProtection in mUltiple Sclerosis, will be randomly assigned to one of three groups.

One group will receive metformin, a drug that’s widely approved to treat diabetes and that may promote repair of myelin, the fatty covering around nerve fibers that’s damaged in MS. The second group will receive alpha-lipoic acid, a supplement thought to have nerve-protecting properties. The third group will be given a placebo.

“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,” said Simon Broadley, PhD, a professor at Griffith University and chief investigator for the PLATYPUS study.

“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,” said Melanie Kiely, CEO of MSWA, an MS-focused group in Western Australia helping to fund the trial with MS Australia.

“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,” said Des Graham, president of MS Australia.

The Australian study will follow the same protocol as the OCTOPUS study, a progressive MS clinical trial launched in the U.K. this year. Collectively, the two trials are expected to enroll some 1,600 people with progressive MS over the next five years.

The launch of the PLATYPUS trial “provides extraordinary hope for many people living with MS and is a major moment for MS research.” said Nigel Caswell, an MS patient for three decades who last year won MS Australia’s John Studdy Award.