Targeting disease, not disease targets: Innovative approaches in tackling neurodegenerative disorders

Area of Science:

• Drug Discovery and Development
• Preclinical Research
• Pharmacology

Background:

• Preclinical drug investigation requires accurate disease models to assess therapeutic efficacy and safety.
• Current models often use artificial biochemical assays, potentially compromising biological relevance and limiting innovation.
• The predictability and cost-effectiveness of disease models are critical for successful drug development.

Purpose of the Study:

• To explore alternative or complementary approaches to traditional drug discovery methods.
• To advocate for the early integration of biological context into the drug discovery process.
• To present a case study of a 'biology-driven' drug discovery implementation.

Main Methods:

• Review of traditional reductionist approaches in preclinical drug investigation.
• Discussion of the limitations of artificial biochemical assays in mimicking disease biology.
• Presentation of a case study detailing a biology-driven drug discovery strategy.

Main Results:

• Reductionist approaches may compromise model predictability by removing biological context.
• Over-reliance on pre-defined targets can hinder innovative drug discovery programs.
• Integrating biological context early can enhance the identification of specific molecular modulators.

Conclusions:

• Introducing biological context early in drug discovery is crucial for developing effective therapeutics.
• Biology-driven approaches offer a complementary or alternative strategy to traditional methods.
• The presented case study demonstrates the practical application and potential benefits of this approach.