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  • CORRESPONDENCE

The role of placebo control in clinical trials for neurodegenerative diseases

  1. Arpan R. Mehta

    1. ACORD at MRC Clinical Trials Unit, Institute of Clinical Trials and Methodology, University College London, London, UK; Euan MacDonald Centre for MND Research, Anne Rowling Regenerative Neurology Clinic, Centre for Clinical Brain Sciences, and UK Dementia Research Institute, University of Edinburgh, Edinburgh, UK; Oxford University Hospitals NHS Foundation Trust, Oxford, UK; National Hospital for Neurology and Neurosurgery, Queen Square, London, UK.

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  2. James R. Carpenter

    1. ACORD at MRC Clinical Trials Unit, Institute of Clinical Trials and Methodology, University College London, London, UK and Department of Medical Statistics, London School of Hygiene & Tropical Medicine, London, UK.

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  3. Jennifer M. Nicholas

    1. ACORD at MRC Clinical Trials Unit, Institute of Clinical Trials and Methodology, University College London, London, UK and Department of Medical Statistics, London School of Hygiene & Tropical Medicine, London, UK.

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  4. Jeremy Chataway

    1. ACORD at MRC Clinical Trials Unit, Institute of Clinical Trials and Methodology, University College London, London, UK; Queen Square Multiple Sclerosis Centre, Department of Neuroinflammation, UCL Queen Square Institute of Neurology, Faculty of Brain Sciences, University College London, London, UK; and National Institute for Health Research, University College London Hospitals, Biomedical Research Centre, London, UK.

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  5. Bruce Virgo

    1. MND-SMART Patient Advisory Group, University of Edinburgh, Edinburgh, UK.

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  6. Mahesh K. B. Parmar

    1. ACORD at MRC Clinical Trials Unit, Institute of Clinical Trials and Methodology, University College London, London, UK.

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  7. Siddharthan Chandran

    1. ACORD at MRC Clinical Trials Unit, Institute of Clinical Trials and Methodology, University College London, London, UK; Euan MacDonald Centre for MND Research, Anne Rowling Regenerative Neurology Clinic, Centre for Clinical Brain Sciences, and UK Dementia Research Institute, University of Edinburgh, Edinburgh, UK.

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  8. Suvankar Pal

    1. ACORD at MRC Clinical Trials Unit, Institute of Clinical Trials and Methodology, University College London, London, UK; Euan MacDonald Centre for MND Research, Anne Rowling Regenerative Neurology Clinic, and Centre for Clinical Brain Sciences, University of Edinburgh, Edinburgh, UK.

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Finding effective interventions for neurodegenerative diseases is arguably one of the final frontiers of medicine, in part because of a failure to study the complex nervous system using human experimental medicine systems1. Notwithstanding emergent promising medicines for the treatment of Alzheimer’s dementia2, there are no truly effective disease-modifying therapies for motor neuron disease (MND); riluzole, approved in 1995, extends survival by only 2–4 months. Biologically plausible candidate medicines are being tested in clinical trials, although most candidates (either repurposed or bespoke) fail in clinical trials3. This highlights the urgent need for a novel approach, apart from two-arm studies, that will deliver efficient (in terms of time, cost and patient resources) and definitive evaluation of multiple promising medicines in clinical trials4.

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Nature Medicine 29, 2682-2683 (2023)

doi: https://doi.org/10.1038/d41591-023-00080-0

Acknowledgements

MND-SMART and related activities are funded by the Euan MacDonald Centre for MND Research at the University of Edinburgh, MND Scotland, My Name’5 Doddie Foundation, the MND Association, the Alan Davidson Foundation, the Anne Rowling Regenerative Neurology Clinic at the University of Edinburgh, and the UK Dementia Research Institute, which receives its funding from UK DRI, funded by the Medical Research Council, the Alzheimer’s Society and Alzheimer’s Research UK. The views expressed in this publication are those of the authors and not necessarily those of the funders.

Competing interests

The authors declare no competing interests.

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