The National Institutes of Health has awarded two five-year grants totaling $4.4 million to the Indiana University School of Medicine. The funding will support research to identify new treatments for a family of fatal genetic diseases.
The university says researchers are looking for ways to combat glycogen storage diseases, such as Lafora and Pompe diseases, which can causes fatal illnesses.
"These diseases are rare but nonetheless devastating not only for the patients but also their family and friends," said Peter Roach, distinguished professor of biochemistry and molecular biology. "Our group has met with Lafora families at workshops organized by their foundation, Chelsea’s Hope, and it is humbling to hear of their ordeals and their faith that we can find a cure or, at least, a treatment. We sincerely hope that this new funding can help us reach that goal by developing a novel therapy."
The team at the IU School of Medicine has found ways to alleviate the symptoms of these diseases in genetically modified mice by decreasing glycogen accumulation. Roach says his team is focusing developing small-molecule drugs to perform this task.
The NIH grant adds to existing funding for the research, including a $2.2 million grant from the National Institute of Diabetes, Digestive and Kidney Diseases and a $9.1 million Program Project Grant from the National Institute of Neurological Disorders and Stroke.