Drug discovery in academic institutions

Area of Science:

• Translational science
• Medicinal chemistry
• Cancer biology

Background:

• Academic science traditionally focuses on disease mechanisms.
• Growing imperative for academics to directly contribute to new medicine discovery.
• Integration of medicinal chemists with cancer biologists fosters drug discovery.

Purpose of the Study:

• To highlight the role of academic-led collaborations in drug discovery.
• To present case studies demonstrating the synthesis of chemical biology tool compounds (chemical probes).
• To elucidate the function of specific proteins and pathways in disease through chemical probes.

Main Methods:

• Case study 1: Discovery of mer kinase inhibitors for acute lymphoblastic leukemia.
• Case study 2: Development of chemical probes targeting epigenetic regulators.
• Iterative optimization of lead compounds identified through screening.

Main Results:

• Successful identification of mer kinase inhibitors for pediatric acute lymphoblastic leukemia.
• Development of novel chemical probes for studying epigenetic regulators.
• Demonstrated value of iterative optimization in drug/probe development.

Conclusions:

• Academic-industry collaborations accelerate drug discovery and development.
• Medicinal chemists and cancer biologists' synergistic efforts are crucial.
• Target selection, iterative optimization, and collaborative partnerships are key to successful therapeutic and probe discovery.