Morgan Sherburne
February 7, 2020

ANN ARBOR—A team led by University of Michigan researchers has found that using drug compounds to target specific molecules within muscle cells can ameliorate Duchenne muscular dystrophy in mice.

The U-M team partnered with researchers from the National Institutes of Health’s National Center for Advancing Translational Sciences, or NCATS, led by Marc Ferrer, Juan Marugan and Noel Southall. The group carried out high-throughput screening and medicinal chemistry studies to produce the first-in-class TRPML1 activator that the researchers tested in cell and animal models. The team reported that activating TRPML1 either genetically or using pharmaceutical compounds protected muscle cells in mice with muscular dystrophy.

Targeting the ion channel, present in all muscle cells, could provide a broader reach to treating the disease.

“We believe that TRPML1 activators can be useful for the treatment of multiple neurodegenerative disorders as well,” Marugan said.

These novel TRPML1 compounds have been patented and are ready for licensing to the pharmaceutical industry.

To read more about the team’s research, click here.