With help from a challenge grant, U advances research on a first-ever treatment for ataxia
There are no real treatment options for people who have ataxia—no real course of action other than coping with symptoms of the neurodegenerative condition, which can include difficulties with balance, coordination, speech, and sometimes vision.
But today researchers at the University of Minnesota are on a path to change that reality. Working with colleagues at the University of Iowa and Rush University in Chicago, U scientists are developing what could be the firstever gene therapy for people who have spinocerebellar ataxia type 1 (SCA1).
Gene-based treatment approaches are potential alternatives to drug-based therapies for some conditions. In the case of SCA1, a specific gene therapy already has been shown to be effective in mice. Now a team led by renowned U of M ataxia researcher Harry Orr, Ph.D., is taking the next steps toward determining whether the therapy could also be transferable to people.
The University has a rich history of “firsts” in ataxia research. In the 1990s, Orr and his colleagues discovered the gene and mutation that cause SCA1 and developed the first mouse model of the disease. (Investigators also discovered that the genetic mutation that causes SCA1 causes Huntington’s disease as well as five other ataxias: SCA2, SCA3, SCA6, SCA7, and SCA17). Research centers around the world have used this knowledge in their own studies.
Through experiments funded by the generosity of donors, Orr and his collaborators proved that this new gene therapy works in mice. And now they’re relying on that generosity to take the work to the next level—and, eventually, to patients through clinical studies.
Philanthropists Richard and Maureen Schulze have offered to match all gifts to advance this gene therapy research project dollar for dollar, up to $50,000.
It’s a significant opportunity to make a difference—not only for people who deal with SCA1 every day of their lives, but also for people suffering from Huntington’s disease and other forms of ataxia who stand to benefit from this therapy.