In a double-blind, placebo study, acetyl-L-carnitine was administered
to 7 probable Alzheimer's disease patients who were then compared by c
linical and P-31 magnetic resonance spectroscopic measures to 5 placeb
o-treated probable AD patients and 21 age-matched healthy controls ove
r the course of 1 year. Compared to AD patients on placebo, acetyl-L-c
arnitine-treated patients showed significantly less deterioration in t
heir Mini-Mental Status and Alzheimer's Disease Assessment Scale test
scores. Furthermore, the decrease in phosphomonoester levels observed
in both the acetyl-L-carnitine and placebo AD groups al entry was norm
alized;in the acetyl-L-carnitine-treated but not in the placebo-treate
d patients. Similar normalization of high-energy phosphate levels was
observed in the acetyl-L-carnitine-treated but not in the placebo-trea
ted patients. This is the first direct in vivo demonstration of a bene
ficial effect of a drug on both clinical and CNS neurochemical paramet
ers in AD.