“On the Mind” is a series that aims to demystify biological disorders and give a platform to patients; at last week’s event at New York’s Caveat, the focus was on Huntington’s disease (HD), a slowly progressive, hereditary neurodegenerative disorder that causes cognitive, psychiatric, and motor problems. The evening’s program had three parts: the scientific story of HD, dance performances inspired by HD, and the personal story of Justin Goldberg, whose father has HD and who is himself at risk for the disease.
Approximately 30,000 people in the US are living with HD and another 200,000 are at risk, with diagnosis usually occurring when a person reaches his or her early 40s and begins to exhibit motor symptoms, said Leora Fox, Ph.D., manager of mission and research programs at the Huntington’s Disease Society of America. Presently, there are no treatments that can slow or stop Huntington’s disease.
Everyone has two copies of the huntingtin gene (one from each parent), she explained, and the existing hypothesis is that the disease is caused by a mutation on the gene that gives instructions to produce a toxic huntingtin protein (“DNA makes RNA makes protein”), which eventually leads to death of brain cells. The logical solution therefore seems to be: “Lower the amount of huntingtin in the brain,” she said.