Evans, L.J. orcid.org/0000-0002-8317-2013, O’Brien, D. and Shaw, P.J. orcid.org/0000-0002-8925-2567 (2024) Current neuroprotective therapies and future prospects for motor neuron disease. In: Shaw, P.J. and Kirby, J., (eds.) International Review of Neurobiology. Elsevier , pp. 327-384.
Abstract
Four medications with neuroprotective disease-modifying effects are now in use for motor neuron disease (MND). With FDA approvals for tofersen, relyvrio and edaravone in just the past year, 2022 ended a quarter of a century when riluzole was the sole such drug to offer to patients. The acceleration of approvals may mean we are witnessing the beginning of a step-change in how MND can be treated. Improvements in understanding underlying disease biology has led to more therapies being developed to target specific and multiple disease mechanisms. Consideration for how the pipeline of new therapeutic agents coming through in clinical and preclinical development can be more effectively evaluated with biomarkers, advances in patient stratification and clinical trial design pave the way for more successful translation for this archetypal complex neurodegenerative disease. While it must be cautioned that only slowed rates of progression have so far been demonstrated, pre-empting rapid neurodegeneration by using neurofilament biomarkers to signal when to treat, as is currently being trialled with tofersen, may be more effective for patients with known genetic predisposition to MND. Early intervention with personalized medicines could mean that for some patients at least, in future we may be able to substantially treat what is considered by many to be one of the most distressing diseases in medicine.
Metadata
Item Type: | Book Section |
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Authors/Creators: |
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Editors: |
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Copyright, Publisher and Additional Information: | © 2024 Published by Elsevier Inc. |
Keywords: | Biomarkers; Genetics; Motor neuron disease; Neuroprotective therapies; New aspects of clinical trial design; Pathophysiology; Humans; Motor Neuron Disease; Neuroprotective Agents; Animals |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > School of Medicine and Population Health |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 02 Aug 2024 14:32 |
Last Modified: | 02 Aug 2024 14:32 |
Status: | Published |
Publisher: | Elsevier |
Refereed: | Yes |
Identification Number: | 10.1016/bs.irn.2024.04.003 |
Related URLs: | |
Sustainable Development Goals: | |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:215259 |