Skip to main content
Open drug discovery in Alzheimer's disease. Alzheimer's & dementia (New York, N. Y.) Axtman, A. D., Brennan, P. E., Frappier-Brinton, T., Betarbet, R., Carter, G. W., Fu, H., Gileadi, O., Greenwood, A. K., Leal, K., Longo, F. M., Mangravite, L. M., Edwards, A. M., Levey, A. I., Emory-Sage-SGC TREAT-AD Center 2023; 9 (2): e12394


Alzheimer's disease (AD) drug discovery has focused on a set of highly studied therapeutic hypotheses, with limited success. The heterogeneous nature of AD processes suggests that a more diverse, systems-integrated strategy may identify new therapeutic hypotheses. Although many target hypotheses have arisen from systems-level modeling of human disease, in practice and for many reasons, it has proven challenging to translate them into drug discovery pipelines. First, many hypotheses implicate protein targets and/or biological mechanisms that are under-studied, meaning there is a paucity of evidence to inform experimental strategies as well as high-quality reagents to perform them. Second, systems-level targets are predicted to act in concert, requiring adaptations in how we characterize new drug targets. Here we posit that the development and open distribution of high-quality experimental reagents and informatic outputs-termed target enabling packages (TEPs)-will catalyze rapid evaluation of emerging systems-integrated targets in AD by enabling parallel, independent, and unencumbered research.

View details for DOI 10.1002/trc2.12394

View details for PubMedID 37215505