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As you may have heard, Dr. Simonetta Sipione at the University of Alberta in Edmonton made a discovery that, in her words, “literally had my colleagues and me jumping with excitement in the lab”.
Dr. Sipione and her team successfully reversed the symptoms of HD in a mouse. They took mice that were already symptomatic, including impaired motor control and coordination, treated them with a special molecule called GM1, resulting in complete restoration of motor control. Isn’t that amazing!
Just like you and all Canadians, HD research has been affected by the global financial crisis and there has been a decrease in funding. So your support helps HSC play a critical role. In fact, HSC provided the seed funding for this project! Together, we enabled this exciting research to get off the ground.
Dr. Sipione and colleagues applied for funding from us with promising preliminary results, and a really, really good idea. They just believed that it would work. And we believed in them. HSC research experts reviewed the proposal and understood what could be achieved.
As you know, HD is an inherited brain disorder where a mutant protein triggers brain cell death, causing loss of motor and cognitive skills, and eventually, death. What researchers also know is that people with HD have slightly lower levels of a brain molecule called GM1. GM1 is a molecule, in the same category as cholesterol, that helps neurons communicate with each other and interpret signals from the environment.
The science was quite simple: restore GM1 in the HD mouse to normal levels and we should see an improvement in motor skills. As Dr. Sipione said herself, “to be quite honest, we didn’t expect to see a complete recovery. It was striking to see the mice return to normal within days.”
What does this mean? Well, in our opinion, it means we have proof of principle that HD symptoms can be reversed and the progression of the disease stopped. Of course, humans are much more complex creatures than mice. And even though we have made the basic discovery, it takes further research to translate discovery into a reliable treatment. This will need to be proven in clinical trials.
Your support is incredibly powerful. Whatever you can give helps support services and research like Dr. Sipione’s. There are many accomplished researchers and scientists working together on all aspects of HD research. Your support for this cause has never been more important.
Every single gift gets us one step closer to a world without HD. Please, give today.