Emerging drug therapies in Huntington's disease.
Huntington's disease (HD) is a relentless neurodegenerative disease that results in profound disability through a triad of motor, cognitive and neuropsychiatric symptoms. At present, there are very few therapeutic interventions available with the exception of a limited number of drugs that offer mild symptomatic relief. Although the genetic basis of the disease has been identified, the mechanisms behind the cellular pathogenesis are still not clear and as a result no candidate drugs with the potential for disease modification have been found clinically until now. One of the major limitations in assessing the usefulness of drug treatments in HD is the lack of well-designed, double-blind, placebo-controlled clinical trials. Most studies have been open-label, using a small number of patients and tend to concentrate on the motor features of the disease, primarily the chorea. This review discusses the treatments now used for HD before evaluating the newer drugs at present being explored in both the clinic and in the laboratory in mouse models of the disease.
Mason
Sarah L
SL
Cambridge Centre for Brain Repair, ED Adrian Building, Forvie Site, Robinson Way, Cambridge CB20PY, UK. slm64@cam.ac.uk
Barker
Roger A
RA
D000818:Animals; D014150:Antipsychotic Agents / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D003071:Cognition / Q000187:drug effects; D004195:Disease Models, Animal; D018491:Dopamine Agonists / Q000627:therapeutic use; D016503:Drug Delivery Systems; D015507:Drugs, Investigational / Q000627:therapeutic use*; D004777:Environment; D018691:Excitatory Amino Acid Antagonists / Q000627:therapeutic use; D016332:Fetal Tissue Transplantation / Q000379:methods; D018755:GABA Agonists / Q000627:therapeutic use; D006801:Humans; D006816:Huntington Disease / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D051379:Mice; D011618:Psychotic Disorders / Q000150:complications / Q000188:drug therapy
D014150:Antipsychotic Agents; D018491:Dopamine Agonists; D015507:Drugs, Investigational; D018691:Excitatory Amino Acid Antagonists; D018755:GABA Agonists