3 ms·
The paper in Science Translational Medicine: http://stm.sciencemag.org/content/8/333/333ra50 http://stm.sciencemag.org/content/8/333/333ra50 This does sound p
by randcraw 11y ago
The paper in Science Translational Medicine:
http://stm.sciencemag.org/content/8/333/333ra50 http://stm.sciencemag.org/content/8/333/333ra50
This does sound promising. The compound (2-Hydroxypropyl-beta-cyclodextrin, or CD) was approved by the FDA in 2009, but only for compassionate use in children for treating Niemann-Pick disease intravenously (IV). Clinical trials are ongoing for non-compassionate access (again, only for treating Niemann-Pick).
However intramuscular (IM) use for treatment of atherosclerosis in the general population probably will have to go through the entire Phase I-II-III clinical test cycle before CD will become available to that end. That's likely to take another 5-10 years, since such trials have not yet begun. I can't guess whether the Niemann-Pick trials might expedite acceptance of CD for a secondary target using a different delivery method (IM vs IV). Maybe.