Cure Found for Huntington Disease in Mice Offers Hope for Treatment in Humans

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Cure Found for Huntington Disease in Mice Offers Hope for Treatmentin Humanshttp://www.huntington-assoc.com/Cure%20Found%20For%20HD%20in%20Mice%20Offers%20Hope%20for%20Treatment%20in%20Humans%20-%20JUN%202006%20Dr.%20Hayden%20etcl.pdfVANCOUVER, B.C. – June 16, 2006:Researchers at the Child and Family Research Institute’s Centre for Molecular Medicine andTherapeutics (CMMT) have provided ground-breaking evidence for a cure for Huntingtondisease in a mouse offering hope that this disease can be relieved in humans.Published today in Cell journal, Dr. Michael Hayden and colleagues discovered that bypreventing the cleavage of the mutant huntingtin protein responsible for Huntington disease(HD) in a mouse model, the degenerative symptoms underlying the illness do not appear andthe mouse displays normal brain function. This is the first time that a cure for HD in mice hasbeen successfully achieved.“Ten years ago, we discovered that huntingtin is cleaved by ‘molecular scissors’ which led to thehypothesis that cleavage of huntingtin may play a key role in causing Huntington disease”, saidDr. Michael Hayden, Director and Senior Scientist at the University of British Columbia’s Centrefor Molecular Medicine and Therapeutics.Now a decade later, this hypothesis has resulted in a landmark discovery. “This is amonumental effort that provides the most compelling evidence of this hypothesis to date”, saidDr. Marian DiFiglia, Professor in Neurology, Massachusetts General Hospital, Harvard MedicalSchool and one of the world’s leading experts on Huntington disease. “Dr. Hayden and his teamhave shown in convincing fashion that many of the changes seen in HD patients can be erasedin HD mice simply by engineering a mutation into the disease gene that prevents the proteinfrom getting cleaved at a specific site”.To explore the role of cleavage, Dr. Hayden’s team established an animal model of HD thatreplicated the key disease features seen in patients. A unique aspect of this particular animalmodel is that it embodied the human HD gene in exactly the same way seen in patients. Thisreplication allowed researchers to examine the progression of HD symptoms including theinevitable cleavage of the mutant huntingtin protein. In the study, researchers confirmed that thedeadly cleavage is caused by a key enzyme called caspase-6. By blocking the action of thistarget, they showed that the mouse did not develop any symptoms of Huntington disease.Hayden's team is now trying to test this model of prevention in a mouse using drug inhibitorsand then ultimately in humans. “Our findings are important because they tell us exactly what weneed to do next”, said Dr. Rona Graham, Post Doctoral Fellow at the CMMT and lead author inthe study.This work is also pivotal for the individuals and families affected by Huntington disease.“Patients of this disease should know that this is a research milestone for all and that this workbrings the field closer to finding effective treatment for a devastating disorder”, said Dr. DiFiglia.The Huntington Society of Canada (HSC), a national network of volunteers and professionalsunited in the fight against HD, echoed this sentiment. “This ground-breaking research providesgreat hope for the Huntington community”, said Don Lamont, the Society’s CEO and ExecutiveDirector. “This research brings us closer to treatment and ultimately a cure”.Huntington disease is a degenerative brain disease that affects one in every 10,000 Canadians.One in 1,000 is touched by HD — for example, as a person with HD, a family member, a personat risk, caregiver or friend. The disease results from degeneration of neurons in certain areas ofthe brain causing uncontrolled movements, loss of intellectual faculties, and emotionaldisturbances. Currently, there is no treatment to delay or prevent HD in patients.This research was funded by Canadian Institutes of Health Research, Hereditary DiseaseFoundation, Huntington Disease Society of America, Michael Smith Foundation for HealthResearch, High Q Foundation, Merck Frosst, Child and Family Research Institute of BC.Alexandra Howard, CommunicationsCentre for Molecular Medicine and Therapeutics (CMMT)T: 604.875.3881E: alexandrawww.cmmt.ubc.caSOURCE: http://www.cmmt.ubc.ca/news_events/news/index.php?news_id=77 http://www.huntington-assoc.com/artonresearch.htm