Preclinical and Clinical Studies in Translational Research

Session 2: Living with Animals
Moderator: Thomas Lecuit (Collège de France)

Abstract

Translational research aims to bridge the gap between basic scientific discoveries and their application for the benefit of patients. In neurodegenerative diseases, particularly Parkinson’s disease, this continuum relies on a rigorous link between preclinical models and clinical trials. This presentation will examine the conditions for the validity of this translation by questioning the relevance of animal models—from model organisms to non-human primates—in reproducing human pathophysiology and predicting therapeutic efficacy. We will address common causes of translation failure: incomplete models, methodological biases, overestimation of preclinical effects, and lack of reproducibility. Particular attention will be paid to quality standards (randomization, blinding, statistical power) that determine the robustness of preclinical results and their ability to guide clinical decisions. Using examples drawn from the development of symptomatic and neuroprotective strategies, we will illustrate the fruitful back-and-forth between the laboratory and the clinic. Finally, we will discuss strategies for enhancing the predictive power of preclinical studies and optimizing the design of clinical trials, within a truly bidirectional translational approach.

Erwan Bézard

Erwan Bézard is a research director of exceptional rank at Inserm and ranks among the world’s most cited neuroscientists (H-index = 107). The author of more than 370 publications, his research focuses primarily on Parkinson’s disease and related synucleinopathies. He is recognized for his research on the compensatory mechanisms that mask the progression of Parkinson’s disease, the pathophysiology of L-DOPA-induced dyskinesias, the mechanisms of cell death in Parkinson’s disease and multiple system atrophy, as well as the modeling of these diseases and the development of therapeutic strategies. As founder and director (2010–2020) of the Institute for Neurodegenerative Diseases (CNRS UMR 5293, University of Bordeaux), he has served on the boards of international organizations such as the Michael J. Fox Foundation and Parkinson’s UK. He is editor-in-chief of Neurobiology of Disease and a member of several editorial boards. He is also CEO/CSO of Motac Neuroscience and co-founder of Treefrog Therapeutics, SE Therapeutics, and QABY Biotech.