Double-blind, crossover, placebo-controlled clinical trial with L-acetylcarnitine in patients with degenerative cerebellar ataxia

Despite the different generic defects underlying degenerative ataxias, it h as been suggested that mitochondrial energy production and antioxidative me tabolic dysfunction may be common biochemical alterations related to these diseases. Acetylcarnitine. a cholinomimetic substance, is involved in oxida tive metabolism and is a potential sourer of acetyl groups for the synthesi s of acetylcholine in the mammalian brain. To determine whether treatment w ith L-acetylcarnitine may improve some clinical conditions of patients with ataxia, a double-blind crossover study with L-acetylcarnitine was performe d in 24 patients with degenerative cerebellar diseases. Patients were selec ted from an ongoing prospective follow-up study at the Department of Neurol ogy at the University of Florence, Italy. Each treatment phase with L-acety lcarnitine or placebo lasted 6 months. after which patients were crossed ov er to the other treatment phase. Ataxia was documented and quantified with use of a clinical score. After the trial, we observed a statistically signi ficant improvement of some symptoms and a slow progression of the disease i n both groups of patients.