Direct Administration to the CNSIncreases Efficacy of Niemann-Pick Disease Treatment

As per the new information indicated that review concludes that 2-hydroxypropyl-β-cyclodextrin (HPβCD) does not cross the blood-brain barrier (BBB) in a therapeutically relevant amount up to address the neurological manifestations of Niemann-Pick Disease Type C1 (NPC1).

These manifestations change with the age of onset and include a delay in developmental motor milestones, gait problems, falls, clumsiness, vertical supranuclear gaze paralysis, cerebellar ataxia, dysarthria, dysphagia and dynamic dementia.

Based upon physicochemical properties, discoveries in animal models, early clinical examinations, and patient case reports, the evidence to date proposes that HPβCD does not cross the BBB in therapeutically relevant amounts in the NPC1 setting.

Direct administration to the central sensory system (CNS) would be relied upon to give the greatest NPC1 neurological efficacy and is upheld by a phase 1/2a clinical study. The review is published in the Current Pharmaceutical Design.

‘2-hydroxypropyl-β-cyclodextrin (HPβCD) does not cross the blood-brain barrier in therapeutically relevant amounts to address the neurological manifestations of Niemann-Pick Disease Type C1.’

NPC1 is an autosomal- recessive, uncommon lysosomal storage described by continuously debilitating and at last fatal neurological manifestations. Clinical trials of different HPβCD specialists are currently in progress and the route of administration is an important point of thought for the anticipated outcomes of these trials with respect to safety, tolerability, and efficacy in the NPC1 populace.

Cyclodextrins are complex mixtures of various chemical species and different cyclodextrin products are in this way not the same as each other. While administering drugs directly to the CNS, it is important that the agent being administered is highly purified and well characterized to avoid introducing any contaminants or potentially unknown agents that could adversely affect neurological development and additionally work.

Currently, VTS-270 is the main particular and well-characterized mixture of HPβCD, with a tightly controlled molar substitution specification and a defined molecular “fingerprint” of the different chemical species show in the mixture in light of Kleptose® HPB (RoquettePharma, France).


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