Exploring human biology with N-of-1 clinical trials
Studies on humans that exploit contemporary data-intensive, high-throughput ‘omic’ assay technologies, such as genomics, transcriptomics, proteomics and metabolomics, have unequivocally revealed that humans differ greatly at the molecular level. These differences, which are compounded by each indivi...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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Cambridge University Press
2023-01-01
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| Series: | Cambridge Prisms: Precision Medicine |
| Subjects: | |
| Online Access: | https://www.cambridge.org/core/product/identifier/S2752614322000151/type/journal_article |
| _version_ | 1797828651588255744 |
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| author | N. J. Schork B. Beaulieu-Jones W. S. Liang S. Smalley L. H. Goetz |
| author_facet | N. J. Schork B. Beaulieu-Jones W. S. Liang S. Smalley L. H. Goetz |
| author_sort | N. J. Schork |
| collection | DOAJ |
| description | Studies on humans that exploit contemporary data-intensive, high-throughput ‘omic’ assay technologies, such as genomics, transcriptomics, proteomics and metabolomics, have unequivocally revealed that humans differ greatly at the molecular level. These differences, which are compounded by each individual’s distinct behavioral and environmental exposures, impact individual responses to health interventions such as diet and drugs. Questions about the best way to tailor health interventions to individuals based on their nuanced genomic, physiologic, behavioral, etc. profiles have motivated the current emphasis on ‘precision’ medicine. This review’s purpose is to describe how the design and execution of N-of-1 (or personalized) multivariate clinical trials can advance the field. Such trials focus on individual responses to health interventions from a whole-person perspective, leverage emerging health monitoring technologies, and can be used to address the most relevant questions in the precision medicine era. This includes how to validate biomarkers that may indicate appropriate activity of an intervention as well as how to identify likely beneficial interventions for an individual. We also argue that multivariate N-of-1 and aggregated N-of-1 trials are ideal vehicles for advancing biomedical and translational science in the precision medicine era since the insights gained from them can not only shed light on how to treat or prevent diseases generally, but also provide insight into how to provide real-time care to the very individuals who are seeking attention for their health concerns in the first place. |
| first_indexed | 2024-04-09T13:07:47Z |
| format | Article |
| id | doaj.art-36692c152ebf4f0d8dd37d74fbe71080 |
| institution | Directory Open Access Journal |
| issn | 2752-6143 |
| language | English |
| last_indexed | 2024-04-09T13:07:47Z |
| publishDate | 2023-01-01 |
| publisher | Cambridge University Press |
| record_format | Article |
| series | Cambridge Prisms: Precision Medicine |
| spelling | doaj.art-36692c152ebf4f0d8dd37d74fbe710802023-05-12T11:18:17ZengCambridge University PressCambridge Prisms: Precision Medicine2752-61432023-01-01110.1017/pcm.2022.15Exploring human biology with N-of-1 clinical trialsN. J. Schork0https://orcid.org/0000-0003-0920-5013B. Beaulieu-Jones1W. S. Liang2S. Smalley3L. H. Goetz4https://orcid.org/0000-0003-0061-4769Department of Quantitative Medicine, The Translational Genomics Research Institute (TGen), Phoenix, AZ, USA Net.bio Inc., Los Angeles, CA, USANet.bio Inc., Los Angeles, CA, USA University of Chicago, Chicago, IL, USANet.bio Inc., Los Angeles, CA, USANet.bio Inc., Los Angeles, CA, USA The University of California Los Angeles, Los Angeles, CA, USADepartment of Quantitative Medicine, The Translational Genomics Research Institute (TGen), Phoenix, AZ, USA Net.bio Inc., Los Angeles, CA, USAStudies on humans that exploit contemporary data-intensive, high-throughput ‘omic’ assay technologies, such as genomics, transcriptomics, proteomics and metabolomics, have unequivocally revealed that humans differ greatly at the molecular level. These differences, which are compounded by each individual’s distinct behavioral and environmental exposures, impact individual responses to health interventions such as diet and drugs. Questions about the best way to tailor health interventions to individuals based on their nuanced genomic, physiologic, behavioral, etc. profiles have motivated the current emphasis on ‘precision’ medicine. This review’s purpose is to describe how the design and execution of N-of-1 (or personalized) multivariate clinical trials can advance the field. Such trials focus on individual responses to health interventions from a whole-person perspective, leverage emerging health monitoring technologies, and can be used to address the most relevant questions in the precision medicine era. This includes how to validate biomarkers that may indicate appropriate activity of an intervention as well as how to identify likely beneficial interventions for an individual. We also argue that multivariate N-of-1 and aggregated N-of-1 trials are ideal vehicles for advancing biomedical and translational science in the precision medicine era since the insights gained from them can not only shed light on how to treat or prevent diseases generally, but also provide insight into how to provide real-time care to the very individuals who are seeking attention for their health concerns in the first place.https://www.cambridge.org/core/product/identifier/S2752614322000151/type/journal_articleclinical trialsinterventionsintervention responselongitudinalprecision medicine |
| spellingShingle | N. J. Schork B. Beaulieu-Jones W. S. Liang S. Smalley L. H. Goetz Exploring human biology with N-of-1 clinical trials Cambridge Prisms: Precision Medicine clinical trials interventions intervention response longitudinal precision medicine |
| title | Exploring human biology with N-of-1 clinical trials |
| title_full | Exploring human biology with N-of-1 clinical trials |
| title_fullStr | Exploring human biology with N-of-1 clinical trials |
| title_full_unstemmed | Exploring human biology with N-of-1 clinical trials |
| title_short | Exploring human biology with N-of-1 clinical trials |
| title_sort | exploring human biology with n of 1 clinical trials |
| topic | clinical trials interventions intervention response longitudinal precision medicine |
| url | https://www.cambridge.org/core/product/identifier/S2752614322000151/type/journal_article |
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