Pitt Hopkins Syndrome

We have identified a series of calcium channel inhibitors which also inhibit the sodium channel implicated in this rare disease. One of these, nicardipine, demonstrated return of wild type phenotype in the Pitt Hopkins KO mouse. The in vitro and in vivo work has been published and was all funded by the Pitt Hopkins Research Foundation. We have an orphan drug designation and rare pediatric disease designation for this disease.

Ekins S, Gerlach J, Zorn KM, Antonio BM, Lin Z, Gerlach A. Repurposing Approved Drugs as Inhibitors of Kv7.1 and Nav1.8 to Treat PittHopkins Syndrome.Pharm Res. 2019 Jul 22;36(9):137. doi: 10.1007/s11095-019-2671-y.PMID: 31332533

Ekins S, Puhl AC, Davidow A.Pharm Res.Repurposing the Dihydropyridine Calcium Channel Inhibitor Nicardipine as a Nav1.8 Inhibitor In Vivo for Pitt Hopkins Syndrome. 2020 Jun 11;37(7):127. doi: 10.1007/s11095-020-02853-5.PMID: 32529312