Since he and his colleagues identified the gene responsible for spinocerebellar ataxia type 1 (SCA1) 18 years ago, Harry Orr, Ph.D., has not tired in his pursuit of a cure.
Ataxia—characterized by uncontrolled, uncoordinated movements, balance, or speech caused by dysfunction in the way signals are transmitted to and from the cerebellum—can be devastating. Hereditary types of ataxia such as SCA1 often affect families for generations.
Orr’s team, after cloning the SCA1 gene and developing animal models to understand how ataxia affects the body, is reaching another turning point in its work—developing several promising drug compounds that could one day be used to treat the disease.
And Orr has a new partner in this quest quite literally from the other side of the world.
Hong Kong-based investor Zhao Danyang, who has a personal interest in helping people with SCA1, sought out the foremost experts on the disease. That search led him to Orr at the University of Minnesota.
Now a $50,000 gift from Zhao has allowed Orr’s team to work with the College of Pharmacy’s Institute for Therapeutics Discovery and Development to try to make a half-dozen lead candidate compounds for treating SCA1 “drug-compatible.” This means that they’d be “not just chemicals you pull off the shelf but something you could see actually giving to a human patient,” Orr explains.
Some of those candidate compounds are drugs that are already used to treat other diseases, and some are brand new. Depending on which compounds show the most promise in treating SCA1 in animal models, Orr says, human clinical trials could begin in the next five years.
“It’s an exciting time for sure,” he says.
Though solving scientific challenges in the lab certainly can be energizing, Orr says the people who suffer from SCA1 have truly kept him motivated throughout the years.
“They are a very special group of people,” he says. “You can’t help but be inspired by the courage they have.”