“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.
“How do we design that medicine, if we’re trying to get at the gene or the protein?” Fox asked. “How do we actually get it into the human brain? And how do we then test that it’s working?”
People are trying lots of different approaches, she said. Some researchers are using the relatively new gene-editing tool, CRISPR (see our recent briefing paper for a general explanation of what it is), while others are looking at methods that block or remove the RNA so it can’t make the toxic protein or ways to clean out the huntingtin protein before it wreaks havoc. There has been some success reported in the initial phases of an antisense oligonucleotides (ASO) drug study aimed at lowering huntingtin protein production, said Fox, though she cautioned that bench to bedside is often a long road.
While patients wait for new HD treatments, they can adopt treatments shown to help with variety of symptoms and the general management of the disease. These include psychological support; drugs for movements symptoms; physical therapy for movement, communication, and life skills; and support for family members.
Extolling the benefits of physical therapy for HD patients and, in particular, dance, was Columbia University Ph.D. candidate, Greg Youdan, a movement scientist and former dancer.
“Through exercise, we can actually improve brain health,” said Youdan, by strengthening brain circuitry and improving motor behavior. “But exercise is not a pill, and it’s hard to get people to exercise.”
Furthermore, people often approach exercise piecemeal, he said, such as targeting flexibility but ignoring strength. Dance, he believes, offers an opportunity to address many areas—even beyond motor learning, such as cognition and quality of life. In a dance routine, you memorize steps, time the movement, improvise, move with varying levels of intensity, and have fun.
Youdan cited a small study with 18 participants with mid-stage HD who played the active video game Dance Dance Revolution as a potential exercise therapy. At the end of six weeks, the participants improved their game scores and also their ability to walk. To top it off, they enjoyed the experiment and wanted to continue playing the game even after the study ended.
An appropriate follow up to Youdan’s talk, were two moving dance performances inspired by HD, featuring dancers from Heidi Latsky Dance, which “brings together performers who are disabled, non-disabled and of varying ages, sizes, and races in works designed to lean into each individual’s strengths where everyone has an equal role.”
Former dancer Youdan joined Justin Goldberg, who is gene positive and whose father has HD, in the second routine, titled “Duet.” Following the performance, Justin spoke about his father, now “pushing 80,” who wasn’t diagnosed with HD until after he was in a car accident and sought medical attention (and even then, it took six months to a year to determine the diagnosis). He had not been aware of HD in family medical history.
Justin himself began to experience problems at work beginning in 2010, and once an avid skydiver and skydiving instructor, had to stop. He stays positive, though, and found himself trying out another physical feat: dance. Tying it all together, the woman who taught him to skydive was a dancer, he said, and told him that dancers always make the best skydivers because of their capacity for range of movement. At Caveat, Justin proved that the reverse can be true too.
-Ann L. Whitman