U of M Cardiologist's Research Uses Patient Heart Stem Cells to Understand and Treat Duchenne Muscular Dystrophy Cardiomyopathy
Author: | February 28, 2020
Duchenne muscular dystrophy (DMD) is a fatal condition first diagnosed in young boys between the ages of three and five, and it affects the skeletal muscle, causing weakness and deterioration of muscles, forcing those affected into wheelchairs by their early teens. Patients with DMD are now living into their mid 20s, and heart failure is being identified as an increasing problem and cause of death.
Forum Kamdar, MD, FACC, assistant professor in the Department of Medicine, and her colleagues conducted research on DMD heart failure and developed a cell-based model to study this deadly disease. Their research, recently published in the Journal of the American College of Cardiology (JACC), focuses on a possible treatment using beta blockers, a medication commonly used to control heart rhythms that may increase survival rates.
Dr. Kamdar’s team began their research by recreating beating heart cells in a petri dish to compare the cells of DMD patients to non-DMD cells. To do this, they used skin cells from patients with DMD and reprogrammed those cells to become heart cells. Within eight to 10 days, her team had a “beating sheet of heart cells” to study.
“The focus of this model, by using beating cells from a patient with DMD, is to help better understand what happens in the heart cells of patients with DMD versus those that don’t have the disease,” Dr. Kamdar said.
Her team noticed irregular heartbeats in the DMD heart cells, which is also seen in DMD patients and a cause of sudden death. In order to test treatments for irregular heart rhythms, Dr. Kamdar and her team tested beta blockers on mice with DMD. This experiment found that the treatment not only decreased abnormal heart rhythms but also increased survival rates. She also identified that patients with DMD are rarely currently treated with beta blockers.
“So, I went from patient cells to an animal model, with the goal to go back to the patient, because they are ultimately who I want to help,” Dr. Kamdar said. “The end goal is to potentially increase DMD patients’ survival through beta blockers with additional studies.”
Dr. Kamdar’s research, published in JACC, provides a novelty model to study DMD heart failure. She is currently using her “cardiomyopathy-in-a-dish” model to identify new treatments for DMD cardiomyopathy.
“It’s a component with having the right environment and lots of mentorship to make this happen,” Dr. Kamdar said. “This is an exciting discovery, and it wouldn’t have been possible without these patients and also the support, infrastructure and mentorship at the U of M.”