This week in therapeutics




Licensing status

Publication and contact information

Endocrine/metabolic disease

Creatine transporter deficiency (CTD)

Solute carrier family 6 creatine transporter member 8 (SLC6A8; CRT)

In vitro studies suggest a dodecyl creatine ester could help treat CTD caused by SLC6A8 deficiency. Chemical synthesis and testing in rat brain cells identified a dodecyl creatine fatty ester that was nontoxic to endothelial, glial and neuronal cells. In a rat cell model for the blood brain barrier and in rat cortical neurons, uptake of the compound was greater than that of a control ethyl creatine ester analog. In fibroblasts from patients with CTD caused by SLC6A8 deficiency, the compound increased intracellular creatine levels compared with no treatment. Future studies could include developing a way to deliver the compound that protects it from degradation in plasma.

SciBX 6(25); doi:10.1038/scibx.2013.625
Published online June 27, 2013

Patent and licensing status unavailable

Trotier-Faurion, A. et al. J. Med. Chem.; published online May 22, 2013;
Contact: Aloïse Mabondzo, French Alternative Energies and Atomic Energy Commission, Gif-sur-Yvette, France