Tramiprosate

Current treatment options for patients with Alzheimer's disease are limited to providing symptomatic relief, with no effects on the underlying pathophysiology. However, a greater understanding of the importance of beta-amyloid peptides (Abeta) in the pathogenesis of this disease has led to the investigation of a number of potential antiamyloid therapies, of which tramiprosate (Neurochem Inc.) is in the most advanced stage of development. Tramiprosate is a glycosaminoglycan (GAG) mimetic designed to interfere with the actions of Abeta early in the cascade of amyloidogenic events. Preclinical data have shown that tramiprosate reduces brain and plasma levels of Abeta, prevents fibril formation and exerts cytoprotective effects in the brain. The pharmacological effects have also been demonstrated in clinical trials of patients with mild to moderate Alzheimer's disease. Promising findings for the efficacy of tramiprosate, indicated by improvement or stabilization of cognitive function, have been shown in phase II clinical trials and open-label extensions of these studies. Furthermore, tramiprosate appears to be well tolerated with no reports of safety concerns. This article reviews the unique mode of action of tramiprosate and summarizes the available clinical information on the effects of tramiprosate in patients with Alzheimer's disease.