Understanding How a Transporter Allows an Acetylated Amino Acid to Act as a Drug

Clinical observations revealed that Tanganil, an over-the-counter drug approved only in France, relieved neurological symptoms in ataxia. Tanganil is a racemate of N-acetyl-leucine. As part of a drug development program by our startup biotech IntraBio, we investigated how acetylated L-leucine was effective but leucine itself was not. Using data and advice from BioIVT, we worked out that acetylation makes leucine a substrate for the monocarboxylate transporter. This combined with other work on the mechanism of action and a successful phase 3 clinical trial makes it likely that the N-acetyl-L-leucine will become an approved drug for rare and common neurological disorders.
Download a copy of the slides: info.bioivt.com/2024-adme-sma...
About the Presenter:
Dr. Grant Churchill is a professor of chemical pharmacology at the University of Oxford and a founding scientist with the startup IntraBio. He delights in both research and teaching and was awarded ‘Most Acclaimed Lecturer’ in the Medical Sciences Division. His research is cell signalling and drug discovery has produced chemical tools, drugs and even publications in glamour journals. He obtained a BSc in Agriculture and MSc in Crop Science from the University of Saskatchewan, and a PhD in Biochemistry from the University Minnesota. He then moved to the University of Oxford and can’t seem to leave.