Matencio et al. reported in their recent paper that 92.8% of HPBCD administered intrathecally to a child suffering from Niemann Pick type C (NPC) disease was recovered in 88 h in urine showing that HPBCD can cross the BBB from the brain to blood direction. It is well known that the pathway in the opposite direction (blood to brain) is strongly inhibited and that is the reason why HPBCD is directly administered into the brain using an Ommaya device.
HPBCD is administered to NPC patients to reduce the unesterified cholesterol content accumulated in their brain causing neurodegeneration. This fatal lysosomal storage disorder has no therapy at present. HPBCD as cholesterol depleting agent in orphan drug status was found to control the progress of the disease.
The HPLC method using light scattering detection can detect as low as 3.4 · 10−10 mols HPBCD in urine.
Matencio, A., Alcaráz-Gómez, M.A., García-Carmona, F., Arias, B., López-Nicolás, J.M.
Application of a simple methodology to analyze Hydroxypropyl-β-Cyclodextrin in
urine using HPLC–LS in early Niemann–Pick disease type C patient
Journal of Chromatography B: Analytical Technologies in the Biomedical and Life Sciences, 2018, 1093-1094, 47-51