Preclinical drug development for neurodegenerative diseases: why do we still need animal models?

Neurodegenerative diseases increase in prevalence, especially in aging western societies, but remain incurable and severely reduce life span and quality of life of patients and their caregivers. Developing disease-modifying therapy to halt neurodegeneration remains challenging and is thus far rarely successful. However, the currently available neuroprotective or symptomatic therapeutics for diseases like Parkinson’s, Alzheimer’s or Spinal muscular atrophy were developed in animal models, and the majority of compounds or strategies in pipelines still heavily rely on in vivo pre-clinical development. This is due to the complexity of brain diseases, which are expressed in motor or cognitive symptoms that cannot yet be modelled in vitro. However, every effort should be made to reduce the number of animals used for pre-clinical drug trials. This is being done by adding in vitro assays to screen for efficacy and toxicity, but also by improving the quality and validity of research with animal models. This talk will outline the state of the art and the challenges of pre-clinical drug development with a focus on Parkinson’s disease, and which strategies should be used to apply the 3R principle.