BRIEF RESEARCH REPORT article
Front. Pharmacol.
Sec. Experimental Pharmacology and Drug Discovery
Volume 16 - 2025 | doi: 10.3389/fphar.2025.1592011
This article is part of the Research TopicOpportunities and Challenges in Drug RepurposingView all 6 articles
Repurposing nicardipine leads to improved development in a young patient with Pitt-Hopkins syndrome
Provisionally accepted
- 1Chair of Social Pediatrics, TUM School of Medicine, Technical University of Munich, München, Germany
- 2Center of Rare Developmental Disorders, Kbo-Kinderzentrum München, Munich, Bavaria, Germany
- 3German Center for Child and Adolescent Health (DZKJ), Partner Site Munich, Munich, Germany
- 4Collaborations Pharmaceuticals Inc., Raleigh, North Carolina, United States
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We describe a drug-repurposing treatment involving the use of nicardipine in a young patient with Pitt-Hopkins syndrome (a rare neurodevelopmental disorder that results from variants of TCF4 gene) as a bench-to-bedside approach. Loss of TCF4 function in Pitt-Hopkins syndrome leads to increased excitability of Nav1.8 in neurons. Nicardipine is normally used alone or together with other medicines to treat severe chest pain (angina) or high blood pressure 3 (hypertension), and can also be used in children to treat hypertension.Nicardipine was shown to have an inhibitory effect on Nav1.8 in vitro as well as in Tcf4 +/-mice, showing promising effects on behavior, learning and memory.In this study, nicardipine was given orally for 7 months (starting dose 0.2 mg/kg/d, maximum dose 1.7 mg/kg/d). There were no significant side effects. The patient showed mild to moderate improvement in all developmental trajectories as well as in her restlessness.Repurposing nicardipine in Pitt-Hopkins syndrome patients could be a promising approach to enhance development in these often severely affected patients.
Keywords: Pitt-Hopkins syndrome, Precision treatment, Nicardipine, TCF4 gene, drug repurposing
Received: 11 Mar 2025; Accepted: 07 Jul 2025.
Copyright: © 2025 Somorai, Ekins, Lettl, Rupprecht and Mall. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Marta Agnes Somorai, Chair of Social Pediatrics, TUM School of Medicine, Technical University of Munich, München, Germany
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