Posted by If you are following advances in the re-discovery of cyclodextrins as APIs, there is quite a race among the candidates especially for treating Niemann-Pick C. HPBCD and its new versions (Trappsol and VTS-270) are of course in the lead, yet there are now papers, patents and publications on methylated-CDs, HPACD, pseudorotaxanes and even CD-polymers. From earlier papers we already know that other CDs with little affinity towards cholesterol (SBECD, HPGCD) are also showing positive in vivo results.
In your opinion, what is the theory behind the CD’s success and which candidate will take the prize?
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In my opinion the way forward for CD-based therapy for NPC lies in smarter delivery. Modified monomeric CDs require high dosages and some side effects are probably associated with the high concentration on administration (ototoxicity…). Also engineering the CD system might help with the crossing of the BBB. I think that CD-based macromolecular systems be a interesting route to explore further