Animal models for Niemann-Pick type C: implications for drug discovery & development

Area of Science:

• Biochemistry
• Genetics
• Pharmacology

Background:

• Niemann-Pick type C (NPC) is a rare, progressive, neurovisceral lysosomal storage disease.
• Limited therapeutic options and no approved treatments exist in the US.
• Recent years have seen increased drug development efforts, with two trials completed in 2018.

Purpose of the Study:

• To review and evaluate available animal and cell models for Niemann-Pick type C.
• To prioritize these models for efficient and clinically translatable drug development.
• To discuss relevant developments and opportunities in NPC research.

Main Methods:

• Review of existing literature on Niemann-Pick type C models.
• Evaluation of in vitro and in vivo models.
• Analysis of drug development programs and clinical trial data.

Main Results:

• A variety of in vitro and in vivo models are available for NPC research.
• Several drugs with different mechanisms of action are in late-stage development or approved.
• The field is dynamic with ongoing research and development.

Conclusions:

• The availability of diverse models and advancing drug candidates presents an exciting phase for NPC research.
• Key challenges include leveraging lessons from late-stage programs and incorporating model limitations into drug discovery.
• Successful translation requires careful consideration of model utility and limitations in drug development programs.