Drug repositioning for rare diseases: Knowledge-based success stories

Area of Science:

• Pharmacology
• Genetics
• Drug Development

Background:

• Over 7000 rare diseases exist, with treatments available for only 5%.
• Most rare diseases are life-threatening, highlighting the urgent need for novel therapeutics.
• Traditional drug development is lengthy and expensive, posing significant challenges for rare disease treatments.

Purpose of the Study:

• To explore drug repositioning as a viable strategy for rare disease treatment development.
• To emphasize the importance of rational drug repurposing based on molecular disease understanding.

Main Methods:

• Review of existing literature on drug repositioning successes in rare diseases.
• Focus on rationally-driven repositioning strategies informed by disease molecular characterization.
• Discussion of drug repurposing, reprofiling, and re-tasking as synonyms.

Main Results:

• Drug repositioning is a more efficient and cost-effective alternative to de novo drug development for rare diseases.
• While serendipitous discoveries occur, rational drug repurposing yields more predictable successes.
• Molecular characterization of rare diseases is crucial for identifying new therapeutic applications for existing drugs.

Conclusions:

• Drug repositioning presents a promising avenue for addressing the unmet medical needs in rare diseases.
• Rational drug repurposing, guided by molecular insights, accelerates the development of orphan drugs.
• This strategy offers a faster and more economical pathway compared to traditional new drug discovery.