A perspective on multi‐target drug discovery and design for complex diseases

TLDR

Infectious, neurodegenerative, and malignant diseases arise from complex, varied causes, and modern drug discovery can identify modulators for multiple targets using computational methods that predict compound–target associations before synthesis, contingent on validated targets. The Perspective reviews molecular and computational strategies that enable medicinal chemists to design multi‑target drugs and foster collaboration with clinical scientists.

Abstract

Diseases of infection, of neurodegeneration (such as Alzheimer's and Parkinson's diseases), and of malignancy (cancers) have complex and varied causative factors. Modern drug discovery has the power to identify potential modulators for multiple targets from millions of compounds. Computational approaches allow the determination of the association of each compound with its target before chemical synthesis and biological testing is done. These approaches depend on the prior identification of clinically and biologically validated targets. This Perspective will focus on the molecular and computational approaches that underpin drug design by medicinal chemists to promote understanding and collaboration with clinical scientists.