Radiprodil, a selective GluN2B negative allosteric modulator, rescues
audiogenic seizures in mice carrying the GluN2A(N615S) mutation
Abstract
Background and Purpose: GRIN-related disorders are neurodevelopmental
disorders caused by mutations in the N-methyl-D-aspartate receptor
(NMDAR) subunit receptor GRIN genes. A large fraction of these mutations
leads to gain of function (GoF) of the NMDAR. Patients present with a
combination of symptoms that includes epilepsy, intellectual disability,
behavioural and motor symptoms. Controlling seizures is a significant
medical need in most patients with GRIN-related disorders. The aim of
this study was to assess the therapeutic efficacy of radiprodil, a
selective negative allosteric modulator of GluN2B-containing NMDARs, in
counteracting audiogenic seizures (AGS) in a murine model carrying the
GluN2A(N615S) mutation in homozygosity (Grin2aS/S mice). Experimental
Approach: Grin2aS/S mice were acutely treated with radiprodil at
different doses before the presentation of a high-frequency acoustic
stimulus commonly used for AGS induction. Key Results: Radiprodil
significantly and dose-dependently reduced the onset and severity of AGS
in Grin2aS/S mice. Conclusion and Implications: Our data clearly
indicates that radiprodil has the potential to control seizures in
patients with GRIN2A GoF mutations, targeting the underlying
pathophysiology of the disorder.