About My Health Careers Internship MedBlogs Contact us
Medindia LOGIN REGISTER
Advertisement

Direct Brain Administration Increases Efficacy of Niemann-Pick Disease Treatment

by Anjali Aryamvally on December 17, 2017 at 7:31 PM
Font : A-A+

Direct Brain Administration Increases Efficacy of Niemann-Pick Disease Treatment

A new data review concludes that 2-hydroxypropyl-β-cyclodextrin (HPβCD) does not cross the blood-brain barrier (BBB) in therapeutically relevant amounts to address the neurological manifestations of Niemann-Pick Disease Type C1 (NPC1). These manifestations vary with age of onset and include delay in developmental motor milestones, gait problems, falls, clumsiness, vertical supranuclear gaze palsy, cerebellar ataxia, dysarthria, dysphagia and progressive dementia.

Based upon physicochemical properties, findings in animal models, early clinical studies, and patient case reports, the evidence to date suggests that HPβCD does not cross the BBB in therapeutically relevant amounts in the NPC1 setting. Direct administration to the central nervous system (CNS) would be expected to provide the greatest NPC1 neurological efficacy and is supported by a phase 1/2a clinical study. The review is published in the Current Pharmaceutical Design.

Advertisement


NPC1 is an autosomal-recessive, rare lysosomal storage characterized by progressively debilitating and ultimately fatal neurological manifestations. Clinical trials of different HPβCD agents are currently underway and the route of administration is an important point of consideration for the anticipated results of these trials with regard to safety, tolerability and efficacy in the NPC1 population.

Cyclodextrins are complex mixtures of different chemical species and different cyclodextrin products are therefore not the same as one another. When 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 and/or unknown agents that could adversely affect neurological development and/or function. Currently, VTS-270 is the only specific and well-characterized mixture of HPβCD, with a tightly controlled molar substitution specification and a defined molecular "fingerprint" of the different chemical species present in the mixture based on Kleptose® HPB (Roquette Pharma, France).



Source: Eurekalert
Advertisement

Advertisement
News A-Z
A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
Advertisement
News Category
What's New on Medindia
Fermented Skin Care
Television Binge-Watching May Boost the Risk of Deadly Blood Clots
Western Diet may Augment the Risk of Autoimmune Diseases
View all

Medindia Newsletters Subscribe to our Free Newsletters!
Terms & Conditions and Privacy Policy.

More News on:
Parkinsons Disease Parkinsons Disease Surgical Treatment Brain Brain Facts Ataxia Language Areas in The Brain Ways to Improve your Intelligence Quotient (IQ) 

Recommended Reading
Niemann-Pick Disease
Niemann-Pick disease is a group of inherited metabolic disorders in which cholesterol and lipids ......
Blood Brain Barrier Similar to the Original Developed
An step-by-step process model of the blood brain barrier has been created in the laboratory dish ......
Scientists Study Blood Brain Barrier Function in Pediatric Neurological Diseases
The blood brain barrier (BBB) in the human brain is a very important aspect in human physiology....
Johns Hopkins Researchers Develop New Method to Analyze Damage to Blood Brain Barrier
A new method of analyzing standard MRI scans has been developed by John Hopkins researchers....
Ataxia
Ataxia affects coordination. Gait becomes unstable and the patient loses balance. The cerebellum or ...
Language Areas in The Brain
The mechanism of how human brain processes the language to express and comprehend the verbal, writte...
Parkinsons Disease
Parkinson’s disease is a neurodegenerative disease caused by progressive dopamine brain cells loss. ...
Ways to Improve your Intelligence Quotient (IQ)
Intelligence quotient (IQ) is a psychological measure of human intelligence. Regular physical and me...

Disclaimer - All information and content on this site are for information and educational purposes only. The information should not be used for either diagnosis or treatment or both for any health related problem or disease. Always seek the advice of a qualified physician for medical diagnosis and treatment. Full Disclaimer

© All Rights Reserved 1997 - 2022

This site uses cookies to deliver our services. By using our site, you acknowledge that you have read and understand our Cookie Policy, Privacy Policy, and our Terms of Use
open close
CONSULT A DOCTOR
I have read and I do accept terms of use - Telemedicine

Advantage Medindia: FREE subscription for 'Personalised Health & Wellness website with consultation' (Value Rs.300/-)