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‘N of 1’ therapies need a better model

Nature Medicine volume 27, page 939 (2021)Cite this article

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Oligonucleotides offer therapeutic potential for patients with genetic disorders carrying unique mutations, but developing individualized therapies is not supported by the current process for drug development.

Nusinersen for the treatment of spinal muscular atrophy is probably the best known antisense oligonucleotide (ASO) drug. This drug is administered into the central nervous system space via intrathecal injections, which allows exposure of motor neurons to large amounts of the drug at infrequent maintenance dosing (every 4 months). Depending on when treatment is initiated, nusinersen can prevent symptoms or slow disease progression. Nusinersen is marketed in over 40 countries.

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Acknowledgements

I thank R. Collin, Y. Elgersma, H. Graessner, W. van Roon-Mom, R. Schüle, M. Synofzik and T. Yu for useful discussions.

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  1. Leiden University Medical Center, Leiden, the Netherlands

    Annemieke Aartsma-Rus

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  1. Annemieke Aartsma-Rus
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Correspondence to Annemieke Aartsma-Rus.

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A.A.-R. has disclosures related to patents, consultancies, advisory-board memberships and honoraria. See http://www.exonskipping.nl/disclosures for details.

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Aartsma-Rus, A. ‘N of 1’ therapies need a better model. Nat Med 27, 939 (2021). https://doi.org/10.1038/s41591-021-01380-z

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