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Progressive multiple sclerosi (MS) is a clinical form of MS characterized 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. 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.

Working with a national and international group of clinicians and researchers, the multi-arm, multi-stage 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. 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. 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 multiple sclerosis: “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 multiple sclerosis – 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 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: “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. With an estimated 40% or around 13,000 people living with progressive multiple sclerosis, 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.”

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 trialing 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. 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”.

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

  • Edited by Dr. Gianfrancesco COrmaci, PhD, specialist in Clinical Biochemistry.

Scientific references

Gilbert EAB, Livingston J et al. Brain Res. 2023; 1822:148648.

Narine M et al. Front Cell Neurosci. 2023 Oct 12; 17:1254303.

Burke T et al. Aust J Gen Pract. 2022 Apr; 51(4):221-224.

Thompson AJ, Carroll W et al. Mult Scler. 2022; 28(1):16-28.

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Dott. Gianfrancesco Cormaci
Dott. Gianfrancesco Cormaci
Laurea in Medicina e Chirurgia nel 1998; specialista in Biochimica Clinica dal 2002; dottorato in Neurobiologia nel 2006; Ex-ricercatore, ha trascorso 5 anni negli USA (2004-2008) alle dipendenze dell' NIH/NIDA e poi della Johns Hopkins University. Guardia medica presso la casa di Cura Sant'Agata a Catania. Medico penitenziario presso CC.SR. Cavadonna (SR) Si occupa di Medicina Preventiva personalizzata e intolleranze alimentari. Detentore di un brevetto per la fabbricazione di sfarinati gluten-free a partire da regolare farina di grano. Responsabile della sezione R&D della CoFood s.r.l. per la ricerca e sviluppo di nuovi prodotti alimentari, inclusi quelli a fini medici speciali.

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