Instituut voor Neuropathische Pijn
English articles Research & Development Dexpramipexole, and dose-dependent slowing of decline in ALS
| Dexpramipexole, and dose-dependent slowing of decline in ALS |
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Dexpramipexole is an enantiomer, of the dopamine agonist pramipexole, used in Parkinson disease. It is the right-handed isomere and it is practically devoid from all the dopaminergic effect. Preclinical work indicates dexpramipexole might have neuroprotective properties, probably due to its ability to prevent mitochondria from developing leaky membranes when under stress. Therefore it has been taken into development in an orphan indication, amyotrophic lateral sclerosis, ALS, or Lou Gehring disease as the Americans call it. (Should actually been called Charcot's disease, as he first described ALS more than 100 years ago). 102 newly diagnosed ALS patients were randomized to placebo or one of three doses of dexpramipexole, 50 mg, 150 mg, or 300 mg per day, for 12 weeks of treatment. Treatment failure, defined as a 6-point decline on a specific ALS scale over 12 weeks, was seen in 33 percent of patients on placebo, 35 percent of patients on 50 mg, 15 percent on 150 mg, and 8 percent on 300 mg. The trend was significant (p=0.014). Juli 2010, Jan M. Keppel Hesselink, MD, PhD |