TY - JOUR
T1 - A Precision Medicine Initiative for Alzheimer’s disease
T2 - the road ahead to biomarker-guided integrative disease modeling
AU - for the Alzheimer Precision Medicine Initiative
AU - Hampel, H.
AU - O’Bryant, S. E.
AU - Durrleman, S.
AU - Younesi, E.
AU - Rojkova, K.
AU - Escott-Price, V.
AU - Corvol, J. C.
AU - Broich, K.
AU - Dubois, B.
AU - Lista, S.
N1 - Funding Information:
H. H. and J. C. C. are supported by the AXA Research Fund, the Fondation Université Pierre et Marie Curie and the Fondation pour la Recherche sur Alzheimer, Paris, France. The research leading to these results has received funding from the program ‘Investissements d’avenir’ ANR-10-IAIHU-06 (H. H., J. C. C.). S. E. O. is supported by the National Institute on Aging of the National Institutes of Health under Award Numbers R01AG051848 and R56AG054073. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. S. D. is funded by the European Research Council (ERC) under grant agreement No 678304, European Union’s Horizon 2020 research and innovation program under grant agreement No 666992, and the program ‘Investissements d’avenir’ ANR-10-IAIHU-06.
Publisher Copyright:
© 2017 International Menopause Society.
PY - 2017/3/4
Y1 - 2017/3/4
N2 - After intense scientific exploration and more than a decade of failed trials, Alzheimer’s disease (AD) remains a fatal global epidemic. A traditional research and drug development paradigm continues to target heterogeneous late-stage clinically phenotyped patients with single 'magic bullet' drugs. Here, we propose that it is time for a paradigm shift towards the implementation of precision medicine (PM) for enhanced risk screening, detection, treatment, and prevention of AD. The overarching structure of how PM for AD can be achieved will be provided through the convergence of breakthrough technological advances, including big data science, systems biology, genomic sequencing, blood-based biomarkers, integrated disease modeling and P4 medicine. It is hypothesized that deconstructing AD into multiple genetic and biological subsets existing within this heterogeneous target population will provide an effective PM strategy for treating individual patients with the specific agent(s) that are likely to work best based on the specific individual biological make-up. The Alzheimer’s Precision Medicine Initiative (APMI) is an international collaboration of leading interdisciplinary clinicians and scientists devoted towards the implementation of PM in Neurology, Psychiatry and Neuroscience. It is hypothesized that successful realization of PM in AD and other neurodegenerative diseases will result in breakthrough therapies, such as in oncology, with optimized safety profiles, better responder rates and treatment responses, particularly through biomarker-guided early preclinical disease-stage clinical trials.
AB - After intense scientific exploration and more than a decade of failed trials, Alzheimer’s disease (AD) remains a fatal global epidemic. A traditional research and drug development paradigm continues to target heterogeneous late-stage clinically phenotyped patients with single 'magic bullet' drugs. Here, we propose that it is time for a paradigm shift towards the implementation of precision medicine (PM) for enhanced risk screening, detection, treatment, and prevention of AD. The overarching structure of how PM for AD can be achieved will be provided through the convergence of breakthrough technological advances, including big data science, systems biology, genomic sequencing, blood-based biomarkers, integrated disease modeling and P4 medicine. It is hypothesized that deconstructing AD into multiple genetic and biological subsets existing within this heterogeneous target population will provide an effective PM strategy for treating individual patients with the specific agent(s) that are likely to work best based on the specific individual biological make-up. The Alzheimer’s Precision Medicine Initiative (APMI) is an international collaboration of leading interdisciplinary clinicians and scientists devoted towards the implementation of PM in Neurology, Psychiatry and Neuroscience. It is hypothesized that successful realization of PM in AD and other neurodegenerative diseases will result in breakthrough therapies, such as in oncology, with optimized safety profiles, better responder rates and treatment responses, particularly through biomarker-guided early preclinical disease-stage clinical trials.
KW - Alzheimer precision medicine initiative
KW - Alzheimer’s disease
KW - biomarkers
KW - integrative disease modeling
KW - precision medicine
KW - precision medicine initiative
KW - systems biology
KW - systems medicine
UR - http://www.scopus.com/inward/record.url?scp=85011851560&partnerID=8YFLogxK
U2 - 10.1080/13697137.2017.1287866
DO - 10.1080/13697137.2017.1287866
M3 - Review article
C2 - 28286989
AN - SCOPUS:85011851560
SN - 1369-7137
VL - 20
SP - 107
EP - 118
JO - Climacteric
JF - Climacteric
IS - 2
ER -