SMVT

Sodium-Dependent Multivitamin Transporter (SMVT, SLC5A6) mediates the uptake of essential micronutrients, including biotin, pantothenic acid, and lipoate. SMVT is expressed at the blood–brain barrier and exhibits favorable characteristics for transporter-mediated drug delivery, including broad substrate specificity and high transport capacity.

Our research focuses on utilizing SMVT as a surrogate transporter for the treatment of Creatine Transporter Deficiency Syndrome (CTDS), a rare neurological disorder caused by defects in the creatine transporter (CRT1, SLC6A8). In CTDS, impaired creatine transport into brain cells leads to cerebral creatine deficiency, resulting in developmental delays, intellectual disability, behavioral abnormalities, and motor dysfunction.

We are developing SMVT-utilizing creatine prodrugs designed to bypass the defective creatine transport pathway and enable efficient delivery of creatine across the blood–brain barrier and into the central nervous system. By harnessing alternative transport mechanisms, we aim to restore brain creatine levels and establish new therapeutic strategies for CTDS.

Beyond CTDS, this work may provide valuable insights into transporter-mediated therapeutic strategies for other disorders associated with impaired cellular energy metabolism, including neurodegenerative and cardiovascular diseases.

Selected Publications

Králová, A.; Kuorikoski, V.; Anamea, L.; Tampio, J.; Tonduru, A. K.; Cui, H.; Leinonen, H.; Rysä, J.; Poso, A.; Terasaki, T.; Jalkanen, A. J.; Kronenberger, T.; Huttunen, K.M. A Exploring Sodium-Dependent Multivitamin Transporter (SMVT)-Mediated Transport in Neurons in the Context of Creatine Transporter Deficiency Syndrome. 2026, Submitted.