Drug discovery in academia.

Area of Science:

• Pharmacology and Drug Development
• Biomedical Research
• Medicinal Chemistry

Background:

• Drug discovery is predominantly commercial, involving target identification, high-throughput screening, and lead optimization.
• Preclinical evaluation includes animal models for efficacy, pharmacokinetics (administration, distribution, metabolism, elimination - ADME), toxicology, and drug interactions.
• Academic drug discovery fills a niche by focusing on rare diseases and developing research reagents like chemical genetics tools.

Purpose of the Study:

• To outline the practical aspects of preclinical drug discovery for academic researchers.
• To highlight the value of academic contributions to drug discovery, particularly for neglected or rare conditions.
• To present a case study of academic drug discovery for the cystic fibrosis transmembrane conductance regulator (CFTR).

Main Methods:

• Review of preclinical drug discovery processes relevant to academic settings.
• Description of target discovery, validation, high-throughput screening, and medicinal chemistry.
• Explanation of preclinical evaluation, including ADME and toxicology studies in animal models.

Main Results:

• Academic drug discovery can address targets with limited commercial value, such as rare diseases.
• Academic programs can generate novel research tools, including chemical genetics reagents.
• The cystic fibrosis transmembrane conductance regulator (CFTR) modulator program exemplifies successful academic drug discovery yielding physiological research compounds and clinical candidates.

Conclusions:

• Academic drug discovery is a vital and expanding field with unique contributions to medicine.
• Preclinical drug discovery processes are adaptable for academic research settings.
• Academic efforts in drug discovery, exemplified by CFTR modulators, advance both basic science and therapeutic development.