New developements in drug-based therapies 

The aim of the research program is to fill the gap between fundamental discoveries in terms of new targets or putative pharmacological hits in the field of neurological diseases and their therapeutic applications. Our major objectives will be to overcome the challenges faced by academic research when translating results from bench to bedside, thus accelerating the process.

A battery of tests, in vitro in culture dishes and in vivo in a large panel of animal models (both toxin-related and genetic) and finally in humans will be provided by NeurATRIS for clinical investigation which will allow academic or industrial researchers to begin by revalidating their target and reach up to phase I/II trials in patients.

NeurATRIS possess a large panel of both in vitro and in vivo models/tools enabling the identification of physiologic pathways and therapeutic targets involved in the major neurological diseases and the screening of potential therapeutic candidates (CR-ICM).

A  translational approach will be used for the treatment of diseases of the developing brain. This research field remaining largely unexplored due to the specificities of the immature brain models, and the many ethical and practical difficulties pertaining to clinical trials in neonates and children. Nonetheless, preclinical testing of paediatric drugs has become a compulsory step in accordance with the European regulation enforced in 2007, and with the European Medical Agency Guideline on the “need for non-clinical testing in juvenile animals of human pharmaceuticals for paediatric indications”.

NeuRATIS will combine rodent and non-human primate neonatal and juvenile models, along with human paediatric tissues and in vitro/in vivo/in silico models, articulated as follows:

  • The representation of the perinatal neuroprotection using imaging (MRI )
  • The study of various models of Blood-Brain-Barrier (BBB) during brain development and its permeability  to various drugs
  • The simulation and modelling for the optimization of new schemes of clinical trials adapted to rare diseases at the paediatric age.


Coordinators : E. Hirch, B. Zalc, J.C. Corvol

Collaborators : P. Hantraye & L. Hertz-Pannier,  N. Cartier,  C. Chiron (U1129), P. Gressens (U1141)