RT Journal Article SR Electronic T1 Effects of curcumin nanoformulations on cellular function in Niemann-Pick disease type C astrocytes JF bioRxiv FD Cold Spring Harbor Laboratory SP 135830 DO 10.1101/135830 A1 Emily Maguire A1 Luke J. Haslett A1 Joanne L. Welton A1 Helen Waller-Evans A1 Jule Goike A1 Emily H. Clark A1 Harry R. Knifton A1 Ravin Shrestha A1 Kim Wager A1 Richard Webb A1 Emyr Lloyd-Evans YR 2017 UL http://biorxiv.org/content/early/2017/05/09/135830.abstract AB Niemann-Pick disease type C1 (NPC disease) is a neurodegenerative multi-lipid lysosomal storage disease caused by mutations in the NPC1 gene presenting with reduced lysosomal Ca2+ signalling and inhibited late endosome-lysosome transport. Elevating cytosolic Ca2+ levels in NPC cells has been shown to reduce lysosomal lipid storage. Treating Npc1-/- mice with the Ca2+ modulator curcumin led to reduced lipid storage, improved life expectancy and function. These studies led to reported utilisation of curcumin supplements by NPC disease families despite there being no clinical evidence of benefit and a report indicating no benefit of nanoformulated curcumin in Npc1-/- mice. The aim of this study was to determine whether various commercially available curcumin nanoformulations were capable of reproducing the findings obtained with unformulated pharmaceutical grade curcumin. We compared seven curcumin nanoformulations in Npc1-/- mouse astrocytes. All the nanoformulations elevate cytosolic Ca2+ levels but only two lowered lysosomal lipid storage. Importantly, some caused elevations in NPC lysosomal storage and/or decreased cellular viability. Although this is an in vitro study, our findings suggest that care should be taken when contemplating the use of curcumin supplements for NPC disease.