TY - JOUR
T1 - A white paper on a neurodevelopmental framework for drug discovery in autism and other neurodevelopmental disorders
AU - Martinez Diaz-Caneja, C.
AU - State, Matthew W.
AU - Hagerman, R J
AU - Jacquemont, S
AU - Marin, Oscar
AU - Bagni, C
AU - Umbricht, Daniel
AU - Simonoff, Emily
AU - de Andres-Trelles, Fernando
AU - Kaale, A
AU - Pandina, Gahan
AU - Gomez-Mancilla, Baltazar
AU - Wang, P P
AU - Cusak, J
AU - Siafis, Spyridon
AU - Leucht, S
AU - Parellada, Maria
AU - Loth, Eva
AU - Charman, Tony
AU - Buitelar, Jan
AU - Murphy, Declan
AU - Arango, Celso
PY - 2021/6/23
Y1 - 2021/6/23
N2 - In the last decade there has been a revolution in terms of genetic findings in neurodevelopmental disorders (NDDs), with many discoveries critical for understanding their aetiology and pathophysiology. Clinical trials in single-gene disorders such as fragile X syndrome highlight the challenges of investigating new drug targets in NDDs. Incorporating a developmental perspective into the process of drug development for NDDs could help to overcome some of the current difficulties in identifying and testing new treatments. This paper provides a summary of the proceedings of the ‘New Frontiers Meeting’ on neurodevelopmental disorders organised by the European College of Neuropsychopharmacology in conjunction with the Innovative Medicines Initiative-sponsored AIMS-2-TRIALS consortium. It brought together experts in developmental genetics, autism, NDDs, and clinical trials from academia and industry, regulators, patient and family associations, and other stakeholders. The meeting sought to provide a platform for focused communication on scientific insights, challenges, and methodologies that might be applicable to the development of CNS treatments from a neurodevelopmental perspective. Multidisciplinary translational consortia to develop basic and clinical research in parallel could be pivotal to advance knowledge in the field. Although implementation of clinical trials for NDDs in paediatric populations is widely acknowledged as essential, safety concerns should guide each aspect of their design. Industry and academia should join forces to improve knowledge of the biology of brain development, identify the optimal timing of interventions, and translate these findings into new drugs, allowing for the needs of users and families, with support from regulatory agencies.
AB - In the last decade there has been a revolution in terms of genetic findings in neurodevelopmental disorders (NDDs), with many discoveries critical for understanding their aetiology and pathophysiology. Clinical trials in single-gene disorders such as fragile X syndrome highlight the challenges of investigating new drug targets in NDDs. Incorporating a developmental perspective into the process of drug development for NDDs could help to overcome some of the current difficulties in identifying and testing new treatments. This paper provides a summary of the proceedings of the ‘New Frontiers Meeting’ on neurodevelopmental disorders organised by the European College of Neuropsychopharmacology in conjunction with the Innovative Medicines Initiative-sponsored AIMS-2-TRIALS consortium. It brought together experts in developmental genetics, autism, NDDs, and clinical trials from academia and industry, regulators, patient and family associations, and other stakeholders. The meeting sought to provide a platform for focused communication on scientific insights, challenges, and methodologies that might be applicable to the development of CNS treatments from a neurodevelopmental perspective. Multidisciplinary translational consortia to develop basic and clinical research in parallel could be pivotal to advance knowledge in the field. Although implementation of clinical trials for NDDs in paediatric populations is widely acknowledged as essential, safety concerns should guide each aspect of their design. Industry and academia should join forces to improve knowledge of the biology of brain development, identify the optimal timing of interventions, and translate these findings into new drugs, allowing for the needs of users and families, with support from regulatory agencies.
U2 - 10.1016/j.euroneuro.2021.02.020
DO - 10.1016/j.euroneuro.2021.02.020
M3 - Review article
SN - 0924-977X
JO - European Neuropsychopharmacology
JF - European Neuropsychopharmacology
ER -