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A decade of optimizing drug development for rare neuromuscular disorders through TACT

This year marks the tenth anniversary of the TREAT-NMD Advisory Committee for Therapeutics (TACT), a group of multidisciplinary experts that evaluates drug development programmes for rare neuromuscular diseases and identifies pitfalls. Here, we discuss the experience with TACT based on its reviews of more than 50 applications and its potential as a model for other rare disorders.
  1. Kathryn R. Wagner

    1. Center for Genetic Muscle Disorders, Kennedy Krieger Institute and the Departments of Neurology and Neuroscience, Johns Hopkins School of Medicine, Baltimore, MD, USA.

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  2. Annamaria De Luca

    1. Unit of Pharmacology, Department of Pharmacy – Drug Sciences, University of Bari Aldo Moro, Bari, Italy.

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  3. Didier Caizergues

    1. Genethon, Evry, France.

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  4. James Dowling

    1. Division of Neurology and Program for Genetics and Genome Biology, Hospital for Sick Children, Toronto, Ontario, Canada.

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  5. Nathalie Goemans

    1. University Hospitals Leuven, Dept. of Child Neurology, Leuven, Belgium.

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  6. Heather Gordish-Dressman

    1. Division of Biostatistics, Children’s National Health System, Washington, DC, USA.

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  7. Miranda D. Grounds

    1. School of Human Sciences, the University of Western Australia, Perth, Western Australia, Australia.

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  8. Michael Kelly

    1. MyoTherix, Berkeley, CA, USA.

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  9. Anna Mayhew

    1. John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

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  10. Elizabeth M. McNally

    1. Center for Genetic Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.

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  11. Tracey Zoetis

    1. SciLucent, Herndon, VA, USA.

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  12. Joanne Lee

    1. John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

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  13. Cathy Turner

    1. John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

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  14. Dominic J. Wells

    1. Department of Comparative Biomedical Sciences, Royal Veterinary College, London, UK.

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  15. Cristina Csimma

    1. Csimma, Lincoln, MA, USA.

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  16. Volker Straub

    1. John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

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The TREAT-NMD Advisory Committee for Therapeutics (TACT) was initiated in 2009 to address the issue that most drug development programmes targeting neuromuscular disorders failed to bridge the gap between preclinical and clinical studies or failed in early-stage clinical trials1. This was due in part to a lack of knowledge of drug development processes in general and in part to specific challenges for conducting clinical trials in rare neuromuscular disorders, including the degree of specialization required and the extremely limited number of patients available for clinical trials.

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Nature Reviews Drug Discovery 19, 1-2 (2020)

doi: https://doi.org/10.1038/d41573-019-00199-1

Acknowledgements

TACT has received funding from Parent Project Muscular Dystrophy, Cure Duchenne, Muscular Dystrophy UK, MDA, Joining Jack, Duchenne UK, Duchenne Ireland, Myotubular Trust, Duchenne Now, Duchenne Children’s Trust and SMA Europe.

References

  1. Heslop, E. et al. The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development. Orphanet J. Rare Diseases 10, 49 (2015).

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  2. Willmann, R. et al. TREAT-NMD Neuromuscular Network. Enhancing translation: guidelines for standard pre-clinical experiments in mdx mice. Neuromuscul. Disord. 22, 43–49 (2012).

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Competing Interests

The authors declare no competing interests.

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