| First Author | Finn R | Year | 2013 |
| Journal | J Child Neurol | Volume | 28 |
| Issue | 9 | Pages | 1159-68 |
| PubMed ID | 24014511 | Mgi Jnum | J:221698 |
| Mgi Id | MGI:5641324 | Doi | 10.1177/0883073813494480 |
| Citation | Finn R, et al. (2013) Treatment of the Ppt1(-/-) mouse model of infantile neuronal ceroid lipofuscinosis with the N-methyl-D-aspartate (NMDA) receptor antagonist memantine. J Child Neurol 28(9):1159-68 |
| abstractText | The neuronal ceroid lipofuscinoses, a family of neurodegenerative lysosomal storage disorders, represent the most common cause of pediatric-onset neurodegeneration. The infantile form has a devastatingly early onset and one of the fastest-progressing disease courses. Despite decades of research, the molecular mechanisms driving neuronal loss in infantile neuronal ceroid lipofuscinosis remain unknown. We have previously shown that N-methyl-d-aspartate (NMDA)-type glutamate receptors in the Ppt1(-/-) mouse model of this disease exhibit a hyperfunctional phenotype and postulate that aberrant glutamatergic activity may contribute to neural pathology in both the mouse model and human patients. To test this hypothesis, we treated Ppt1(-/-) mice with the NMDA receptor antagonist memantine and assessed their response to the drug using an accelerating rotarod. At 20 mg/kg, memantine treatment induced a delayed but notable improvement in Ppt1(-/-) mice. Much remains to be assessed before moving to patient trials, but these results suggest memantine has potential as a treatment. |
