A shortened version of the following article appears in the Fall 2014 edition of Horizon. This is the full interview with Michelle discussing why clinical trials are so vital in finding a meaningful treatment for Huntington disease. The article was written by Josh Martin.
When Michelle signed up to be part of the PREDICT-HD study, her two kids were teenagers and Paul Martin was prime minister. A decade later, the kids are grown up, the study is wrapped up and Michelle is revved up by the progress researchers are making. “There is going to be a cure,” she says. “I really, really believe that.”
Michelle was 25 when her dad was diagnosed with Huntington’s. Recently married and in the process of renovating a new home and starting a family, Michelle’s sunny future was suddenly clouded by fear and uncertainty. On her 40th birthday, Michelle had the blood work done that would confirm she was also gene-positive. A few months later, her dad passed away, but not before she made him a promise: that she would participate in every study she could to help put an end to this disease.
Shortly after, Michelle joined PREDICT-HD. As a 10-year participant in the long-term observational study, Michelle has contributed to a mountain of data for HD investigators around the world. Deepening our understanding of how Huntington disease progresses, the study has helped researchers pinpoint the little changes that occur before HD is officially diagnosed. Being able to identify subtle motor symptoms, small structural changes in the brain and, in some cases, mild cognitive changes has huge implications when it comes to measuring whether potential drugs can delay the onset of HD.
So what does a day in the life of a research participant look like? For Michelle, it meant annual trips to the University of British Columbia Centre for Huntington Disease. There, she’d spend the day with different members of the research team, taking psychological tests, motor tests, reflex tests … even tests for her sense of smell.
As much as she gave, Michelle also got a lot out of the experience. Participating gave her more access to medical experts and updates on the latest research developments. It gave her a chance to build relationships with the staff at the Centre — “they have such an amazing group of people,” says Michelle. She felt a sense of empowerment that came from taking action, rather than just waiting for symptoms to show up. Plus, she could be proud knowing that she was doing what she could to protect her children’s future.
Michelle even noticed a drop in her blood pressure after a day at the clinic. “Every time I came out of there, I felt so positive, so full of hope,” she says.
PREDICT-HD may be officially wrapped up, but the UBC Centre is currently looking for volunteers for Enroll-HD, another observational study. According to Clinical Research Manager Joji Decolongon, they are recruiting people who are gene-positive or have HD, plus their close relatives.
Soon, they also expect to be testing potential treatments. The UBC Centre’s Director of Medicine, Dr. Lynn Raymond, points to recent lab-bench breakthroughs that could alter the course of HD — from drugs that target specific neural pathways to genetic therapy designed to lower the levels of mutant huntingtin protein. “Things are really heating up,” she says.
That means research centres like hers will need more volunteers than ever. Although a lot of progress is being made in animal models, at the end of the day it’s human trials that determine what works and what doesn’t. “We need volunteers so that we can get an approved drug for Huntington disease,” says Dr. Raymond. “We can’t move forward with treatments without these studies.”
She also points out that volunteers in these double-blind drug trials have a 50/50 chance of getting a potential new treatment. On top of that, if trial results look promising, all participants are often given the opportunity to continue taking the drug until it is approved.
The more people who sign up for trials and studies, the better. Because HD is a relatively uncommon disease that progresses slowly and presents symptoms differently in different people, researchers need a lot of participants to get enough data to produce meaningful results.
“No matter what you get involved in, it helps,” says Michelle. “I’m already signing up for the next one.”
For more information on clinical studies in your area, contact your closest HSC Resource Centre through our Family Services Team or sign up for notifications at www.HDTrials.org.