Finding new ways to treat rare childhood brain disorders

The University of Minnesota has been an international leader in using cellular therapies to treat rare, life-threatening childhood diseases. Unfortunately, some of these therapies, such as bone marrow transplantation, do not effectively treat inherited, progressive diseases that affect the brain because current techniques for delivering therapeutic cells to the brain are insufficient.

Pediatrics faculty members Paul Orchard, M.D., and Troy Lund, M.D., Ph.D., are working with medicinal chemistry faculty member Carston Wagner, Ph.D., to overcome these obstacles by increasing the efficiency at which donor cells are delivered to the brain. 

The team is developing chemically self-assembled nanorings, which have the ability to attach to stem cells and enhance the delivery of these cells to better treat rare brain diseases. They are planning to test different variations of the treatment in mouse models in the coming months and are eager to determine whether this strategy proves more effective than traditional treatment methods. 

Without support from Minnesota Masonic Charities, their research would not have been possible. 

"Getting funds to support a project this early in development is exceedingly difficult,” says Orchard. “It was a great benefit to obtain support most graciously provided by Minnesota Masonic Charities, as it is very unlikely that we would have been able to pursue these investigations otherwise."