Because cyclodextrin treatment in humans is safe (FDA approved application of 2 hydroxypropyl - β -
cyclodextrin for the treatment of Niemann - Pick disease, type C. Phase I clinical trial) and cyclodextrin beneficially affects key mechanisms of atherogenesis, it may therefore be used clinically to prevent or treat human atherosclerosis http://stm.sciencemag.org/content/8/333/333ra50.
Based on the results of this trial and past work, Ory said a larger phase three clinical trial — that is both randomized and controlled — is already underway investigating
cyclodextrin for patients with NPC.
The Phase 2/3 trials will be sponsored by Vtesse, a new biotech company that was formed specifically to evaluate the safety and efficacy of
cyclodextrin for the treatment of NPC and ultimately obtain FDA labeling if the drug is effective.
Not exact matches
For example, it should be easy to replace hydroxy (OH) groups with carboxy groups (COO --RRB- and so allow the
cyclodextrin to form complexes with positively charged species.
«The difference between our material and activated carbons is that the
cyclodextrins are actually much better
for binding pollutants,» he says.
Doughnut - shaped
cyclodextrin molecules used as catalysts, enzyme mimics, drug carriers and food stabilisers, have been studied
for more than a decade.
Vtesse is currently running a late - stage clinical trial of the drug, a large sugar molecule known as a
cyclodextrin, in which it is injected by lumbar puncture into the spinal fluid of children with the disease (see «Why are people fighting over a promising treatment
for a fatal childhood disease»).
«Drug trial shows promise
for deadly neurological disorder:
Cyclodextrin slows progression of Niemann - Pick type C.» ScienceDaily.
Another three patients were given
cyclodextrin in the spinal column every two weeks
for 18 months.
In the combined phase one / two clinical trial, 14 NPC patients who were ages 4 to 23 years and showing neurological symptoms were given
cyclodextrin, administered into the spinal column once per month
for 12 to 18 months.
Chan emailed the twins» mother, who told him what to do to get Galya on the treatment, and then asked Wang if he'd be willing to apply on his daughter's behalf
for compassionate use of
cyclodextrin.
The Wikipedia page also linked to stories about twin 5 - year - old girls who in 2009 had become the first Americans to fight NPC with an experimental treatment called
cyclodextrin — but only after their parents had spent years pressing
for «compassionate use» of the investigational drug outside of a clinical trial.
Several months later, a few days before Christmas, Chan brought 2 - year - old Galya in
for her weekly
cyclodextrin treatment.
«
Cyclodextrin is being developed as a drug
for NPC, but it currently is being tested in the laboratory in other much more common conditions, including atherosclerosis and Alzheimer's disease.
Those researchers would not be studying
cyclodextrin if not
for the initial work that was done by the NPC community.»
The reason alpha -
cyclodextrins lower postprandial glucose and cholesterol, and the reason they got relatively easy approval
for use as food additives even in the EU, is because they aren't absorbed systemically: they work largely as soluble fiber does, binding excreted bile acids and slowing absorption of glucose.
[6] In 2013 the EU commission has verified a health claim
for alpha -
cyclodextrin.
α -
Cyclodextrin has been authorized
for use as a dietary fiber in the European Union since 2008.
Dmitry: as the paper itself notes, the most likely mechanism
for the ability of hydroxypropyl - β -
cyclodextrin to lower Aβ burden in the mouse model is AD finding is by normalizing membrane cholesterol content and reducing the appearance of abnormal cathepsin D - positive lysosomes, leading to reduced beta - secretase cleavage of APP and an upregulation of genes involved in cholesterol trafficking and Aβ clearance.