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Radiprodil, a selective GluN2B negative allosteric modulator, rescues audiogenic seizures in mice carrying the GluN2A(N615S) mutation
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  • Ilaria Bertocchi,
  • Lorenzo Cifarelli,
  • Alessandra Oberto,
  • Carola Eva,
  • Rolf Sprengel,
  • Mirza Rohman,
  • Pierandrea Muglia
Ilaria Bertocchi
Neuroscience Institute Cavalieri Ottolenghi

Corresponding Author:[email protected]

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Lorenzo Cifarelli
University of Turin
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Alessandra Oberto
Neuroscience Institute Cavalieri Ottolenghi
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Carola Eva
Neuroscience Institute Cavalieri Ottolenghi
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Rolf Sprengel
Max-Planck-Institute for Medical Research
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Mirza Rohman
Sygnature Discovery Ltd
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Pierandrea Muglia
GRIN Therapeutics Inc
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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.