In addition to demonstrating that disease progression slowed
with cyclodextrin, Ory and his colleagues assessed biomarker measurements in the blood that showed evidence that the drug was removing cholesterol from the brain.
Not exact matches
Now researchers in the US have discovered how to modify
cyclodextrins to take other chemical groups, opening the way to making related compounds
with useful new properties.
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.
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»).
In the drug trial, researchers have shown that treatment
with a type of sugar molecule called
cyclodextrin slows progression of the disease.
A study coming out in Science Translational Medicine and led by University of Pennsylvania School of Veterinary Medicine researchers has shown that cats
with NPC — which mirrors the human version of the disease — show vast improvements when treated
with a compound called
cyclodextrin.
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 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.
My group is also working on A2E lipofuscin in the retina, and we've managed to remove it
with a different treatment (using
cyclodextrins).