Research for Rare - Research for rare diseases

New Therapies for Neurological Ion Channel and Transporter Disorders

Treat-ION represents a network of clinicians and scientists across Germany to advance the knowledge about recognizing and treating rare neurological ion channel and transporter disorders. Those comprise a variety of neuropsychiatric diseases and symptoms including developmental delay, epilepsy, episodic and chronic ataxia, migraine and others, which often occur in combination or are caused by mutations in the same channels. Due to the common fundamental function of channels and transporters to regulate neuronal excitability and ionic homeostasis, pathophysiological and therapeutic principles are shared across diseases.
The main goal of the consortium is to translate findings from genetic and pathophysiological studies into rational, individualized therapies. Treat-ION will therefore focus on therapeutic studies in cellular, animal and human models, which will be complemented by in silico searches for new treatments, better predictions for the functional consequences of mutations for therapeutic purposes and cellular drug screens. The consortium will focus its efforts on approved and available ‘repurposed’ drugs. As a proof-of-principle, Treat-ION has successfully been performing n-of-1 trials and established three investigator-initiated trials in specific rare channel disorders with other funding, which will be of great value for the network.
The results of our research and the knowledge of experts will be systematically and directly delivered to patients through a structured molecular therapeutic board attached to the German academy of rare neurological diseases (DASNE).

Projects
Contact Prof. Dr. Holger Lerche
University of Tübingen
Department of Neurology and Epileptology, Hertie-Institute for Clinical Brain Research
Hoppe-Seyler-Strasse 3
72076 Tübingen
Tel.: +49 7071 29-80466
Fax +49 7071 29 4488.
Email: holger.lerche@uni-tuebingen.de