Designer drugs: pipe-dreams or realities?

Area of Science:

• Drug Discovery and Development
• Parasitology
• Tropical Medicine

Background:

• Urgent need for novel chemotherapies against parasitic infections in animals and human tropical diseases.
• Current drug discovery methods are resource-intensive, necessitating efficient approaches.

Purpose of the Study:

• To advocate for and justify the application of rational molecular design in discovering new chemotherapies.
• To address the challenges and potential of rational drug design versus empirical synthesis.

Main Methods:

• Discussion of rational molecular design principles and their application.
• Comparison of rational design with empirical synthesis, including combinatorial approaches.
• Emphasis on target validation as a critical step.

Main Results:

• Rational molecular design requires substantial upfront investment but is justifiable.
• While not all aspects are fully rational, the approach is viable where possible.
• It is premature to definitively compare the cost-effectiveness of rational design versus empirical synthesis.

Conclusions:

• Continued validation of drug targets is essential for advancing rational design.
• Both rational design and empirical synthesis may coexist and evolve in parallel.
• Discovery research should not be halted due to development concerns; collaborative models are encouraged.