Review: EXPLORING HUMAN BIOLOGY WITH N-OF-1 CLINICAL TRIALS — R0/PR2 DOI Creative Commons
Nicholas J. Schork, Brett K. Beaulieu‐Jones, Winnie S. Liang

et al.

Published: Nov. 27, 2022

Studies on humans that exploit contemporary data-intensive, high-throughput 'omic' assay technologies, such as genomics, transcriptomics, proteomics and metabolomics, have unequivocally revealed differ greatly at the molecular level. These differences, which are compounded by each individual's distinct behavioral environmental exposures, impact individual responses to health interventions diet drugs. Questions about best way tailor individuals based their nuanced genomic, physiologic, behavioral, etc. profiles motivated current emphasis 'precision' medicine. This review's purpose is describe how design execution of N-of-1 (or personalized) multivariate clinical trials can advance field. Such focus from a whole-person perspective, leverage emerging monitoring be used address most relevant questions in precision medicine era. includes validate biomarkers may indicate appropriate activity an intervention well identify likely beneficial for individual. We also argue aggregated ideal vehicles advancing biomedical translational science era since insights gained them not only shed light treat or prevent diseases generally, but provide insight into real-time care very who seeking attention concerns first place.

Language: Английский

Emerging therapeutic drug monitoring technologies: considerations and opportunities in precision medicine DOI Creative Commons
Winnie S. Liang, Brett K. Beaulieu‐Jones, Susan L. Smalley

et al.

Frontiers in Pharmacology, Journal Year: 2024, Volume and Issue: 15

Published: March 13, 2024

In recent years, the development of sensor and wearable technologies have led to their increased adoption in clinical health monitoring settings. One area that is early, but promising, stages use biosensors for therapeutic drug (TDM). Traditionally, TDM could only be performed certified laboratories was used specific scenarios optimize dosage based on measurement plasma/blood concentrations. Although has been typically pursued settings involving medications are challenging manage, basic approach useful characterizing activity. idea there likely a clear relationship between concentration (or other matrices) efficacy. However, these relationships may vary across individuals affected by genetic factors, comorbidities, lifestyle, diet. will valuable enabling precision medicine strategies determine efficacy drugs individuals, as well optimizing personalized dosing, especially since windows inter-individually. this mini-review, we discuss emerging applications, factors influence including interactions, polypharmacy, supplement use. We also how using within single subject (N-of-1) aggregated N-of-1 trial designs provides opportunities better capture response activity at individual level. Individualized solutions potential help treatment selection dosing regimens so right dose matched person context.

Language: Английский

Citations

10
Preclinical Development of Antisense Oligonucleotides to Rescue Aberrant Splicing Caused by an Ultrarare ABCA4 Variant in a Child with Early-Onset Stargardt Disease DOI Creative Commons
Nuria Suárez-Herrera, Catherina H. Z. Li, Nico Leijsten

et al.

Cells, Journal Year: 2024, Volume and Issue: 13(7), P. 601 - 601

Published: March 29, 2024

Precision medicine is rapidly gaining recognition in the field of (ultra)rare conditions, where only a few individuals world are affected. Clinical trial design for small number patients extremely challenging, and this reason, development N-of-1 strategies explored to accelerate customized therapy rare cases. A strong candidate approach Stargardt disease (STGD1), an autosomal recessive macular degeneration characterized by high genetic phenotypic heterogeneity. STGD1 caused pathogenic variants

Language: Английский

Citations

3
Towards Precision Sports Nutrition for Endurance Athletes: A Scoping Review of Application of Omics and Wearables Technologies DOI Open Access

Leon Bedrač,

Leon Deutsch,

Sanja Terzić

et al.

Nutrients, Journal Year: 2024, Volume and Issue: 16(22), P. 3943 - 3943

Published: Nov. 19, 2024

Endurance athletes require tailored nutrition strategies to optimize performance, recovery, and training adaptations. While traditional sports guidelines provide a foundational framework, individual variability in metabolic responses underscores the need for precision nutrition, informed by genetic, biological, environmental factors. This scoping review evaluates application of systems biology-driven endurance athletes, focusing on 'omics' wearable technologies.

Language: Английский

Citations

3
Brain Wave Oscillations as an Objective Neurophysiological Biomarker of Homeopathic Subjective Well-Being DOI Creative Commons
Marcus Zulian Teixeira

Homeopathy, Journal Year: 2024, Volume and Issue: unknown

Published: April 18, 2024

Homeopathy uses the "similitude principle" to arouse a therapeutic reaction in body against its own disorders. For this occur optimally, medicinal pathogenetic effects must present similarity with totality of individual's symptoms. To assess if has been successfully achieved, Hahnemann states that "improvement disposition and mind"-i.e., subjective well-being-is most important parameter consider.

Language: Английский

Citations

1
The Potential of Integrative Cancer Treatment Using Melatonin and the Challenge of Heterogeneity in Population-Based Studies: A Case Report of Colon Cancer and a Literature Review DOI Creative Commons
Smorodin Ep,

Valentin Chuzmarov,

Toomas Veidebaum

et al.

Current Oncology, Journal Year: 2024, Volume and Issue: 31(4), P. 1994 - 2023

Published: April 3, 2024

Melatonin is a multifunctional hormone regulator that maintains homeostasis through circadian rhythms, and desynchronization of these rhythms can lead to gastrointestinal disorders increase the risk cancer. Preliminary clinical studies have shown exogenous melatonin alleviates harmful effects anticancer therapy improves quality life, but results are still inconclusive due heterogeneity studies. A personalized approach testing parameters response integrative treatment with nontoxic bioavailable in patient-centered N-of-1 deserves greater attention. This case colon cancer analyzes discusses tumor pathology, adverse chemotherapy, dynamics markers inflammation (NLR, LMR, PLR ratios), tumors (CEA, CA 19-9, PSA), hemostasis (D-dimer activated partial thromboplastin time). The patient took during after nutrients (zinc, selenium, vitamin D, green tea, taxifolin), aspirin chemotherapy. patient’s PSA levels decreased CT combined (19 mg/day), normalized inflammatory alleviated symptoms polyneuropathy did not help thrombocytopenia. analyzed discussed context literature on oncostatic systemic effects, alleviating therapy-mediated association survival,

Language: Английский

Citations

0
Long-term treatment with insulin-like growth factor-1 in Phelan-McDermid syndrome: a case report DOI Open Access
Mara Parellada, Mónica Burdeus, David Fraguas

et al.

Rare Disease and Orphan Drugs Journal, Journal Year: 2024, Volume and Issue: 3(2)

Published: May 6, 2024

Phelan-McDermid syndrome (PMS) is a chromosomal microdeletion generally caused by loss-of-function variants or deletions affecting the SHANK3 gene. We report on case of 19-year-old woman with diagnosis PMS, autism, and developmental disability. She has been under clinical care since age 9 received treatment subcutaneous IGF-1 from 11 to 15 years age. The spanned 2 periods, totaling 35 months, interspersed 16-month off-treatment interval. Clinically significant improvement was evident during particularly in Social Responsiveness Scale, Aberrant Behavior Checklist, assessments, contrasted clear deterioration period. Sleep difficulties worsened first period, EKG repolarization abnormalities emerged second ultimately leading definitive discontinuation. In conclusion, an experimental long-term on-off-on adolescent PMS resulted mixed results, showcasing positive improvements alongside potentially severe adverse events long run.

Language: Английский

Citations

0
Review: EXPLORING HUMAN BIOLOGY WITH N-OF-1 CLINICAL TRIALS — R0/PR3 DOI Creative Commons
Nicholas J. Schork, Brett K. Beaulieu‐Jones, Winnie S. Liang

et al.

Published: Nov. 10, 2022

Studies on humans that exploit contemporary data-intensive, high-throughput 'omic' assay technologies, such as genomics, transcriptomics, proteomics and metabolomics, have unequivocally revealed differ greatly at the molecular level. These differences, which are compounded by each individual's distinct behavioral environmental exposures, impact individual responses to health interventions diet drugs. Questions about best way tailor individuals based their nuanced genomic, physiologic, behavioral, etc. profiles motivated current emphasis 'precision' medicine. This review's purpose is describe how design execution of N-of-1 (or personalized) multivariate clinical trials can advance field. Such focus from a whole-person perspective, leverage emerging monitoring be used address most relevant questions in precision medicine era. includes validate biomarkers may indicate appropriate activity an intervention well identify likely beneficial for individual. We also argue aggregated ideal vehicles advancing biomedical translational science era since insights gained them not only shed light treat or prevent diseases generally, but provide insight into real-time care very who seeking attention concerns first place.

Language: Английский

Citations

0
Review: EXPLORING HUMAN BIOLOGY WITH N-OF-1 CLINICAL TRIALS — R1/PR7 DOI Creative Commons
Nicholas J. Schork, Brett K. Beaulieu‐Jones, Winnie S. Liang

et al.

Published: Dec. 16, 2022

Studies on humans that exploit contemporary data-intensive, high-throughput 'omic' assay technologies, such as genomics, transcriptomics, proteomics and metabolomics, have unequivocally revealed differ greatly at the molecular level. These differences, which are compounded by each individual's distinct behavioral environmental exposures, impact individual responses to health interventions diet drugs. Questions about best way tailor individuals based their nuanced genomic, physiologic, behavioral, etc. profiles motivated current emphasis 'precision' medicine. This review's purpose is describe how design execution of N-of-1 (or personalized) multivariate clinical trials can advance field. Such focus from a whole-person perspective, leverage emerging monitoring be used address most relevant questions in precision medicine era. includes validate biomarkers may indicate appropriate activity an intervention well identify likely beneficial for individual. We also argue aggregated ideal vehicles advancing biomedical translational science era since insights gained them not only shed light treat or prevent diseases generally, but provide insight into real-time care very who seeking attention concerns first place.

Language: Английский

Citations

0
Decision: EXPLORING HUMAN BIOLOGY WITH N-OF-1 CLINICAL TRIALS — R0/PR5 DOI Creative Commons
Nicholas J. Schork, Brett K. Beaulieu‐Jones, Winnie S. Liang

et al.

Published: Dec. 5, 2022

Studies on humans that exploit contemporary data-intensive, high-throughput 'omic' assay technologies, such as genomics, transcriptomics, proteomics and metabolomics, have unequivocally revealed differ greatly at the molecular level. These differences, which are compounded by each individual's distinct behavioral environmental exposures, impact individual responses to health interventions diet drugs. Questions about best way tailor individuals based their nuanced genomic, physiologic, behavioral, etc. profiles motivated current emphasis 'precision' medicine. This review's purpose is describe how design execution of N-of-1 (or personalized) multivariate clinical trials can advance field. Such focus from a whole-person perspective, leverage emerging monitoring be used address most relevant questions in precision medicine era. includes validate biomarkers may indicate appropriate activity an intervention well identify likely beneficial for individual. We also argue aggregated ideal vehicles advancing biomedical translational science era since insights gained them not only shed light treat or prevent diseases generally, but provide insight into real-time care very who seeking attention concerns first place.

Language: Английский

Citations

0
Author comment: EXPLORING HUMAN BIOLOGY WITH N-OF-1 CLINICAL TRIALS — R0/PR1 DOI Creative Commons
Nicholas J. Schork

Published: Sept. 26, 2022

Studies on humans that exploit contemporary data-intensive, high-throughput 'omic' assay technologies, such as genomics, transcriptomics, proteomics and metabolomics, have unequivocally revealed differ greatly at the molecular level. These differences, which are compounded by each individual's distinct behavioral environmental exposures, impact individual responses to health interventions diet drugs. Questions about best way tailor individuals based their nuanced genomic, physiologic, behavioral, etc. profiles motivated current emphasis 'precision' medicine. This review's purpose is describe how design execution of N-of-1 (or personalized) multivariate clinical trials can advance field. Such focus from a whole-person perspective, leverage emerging monitoring be used address most relevant questions in precision medicine era. includes validate biomarkers may indicate appropriate activity an intervention well identify likely beneficial for individual. We also argue aggregated ideal vehicles advancing biomedical translational science era since insights gained them not only shed light treat or prevent diseases generally, but provide insight into real-time care very who seeking attention concerns first place.

Language: Английский

Citations

0