NOV 17, 2017 5:25 AM PST

Improving Heart Function for People with Duchenne Muscular Dystrophy

WRITTEN BY: Kara Marker

Duchenne muscular dystrophy - a rare, genetic, neuromuscular disease that primarily affects males - leads to degeneration and weakness of muscles. The heart, being a muscle, is not spared by this disease. From the Cedars-Sinai Medical Center, researchers offer a new treatment for this disease with an approach never before attempted.

Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Credit: CDC

Those diagnosed with Duchenne muscular dystrophy usually begin losing their ability to walk between the ages of 12 and 15, with not many surviving past 25 years. The disease is a result of a genetic mutation that eliminates dystrophin, a muscle protein. Duchenne muscular dystrophy is rare in females because the genetic mutation causing the disease is on the X chromosome, of which females have two and males only have one.

Duchenne muscular dystrophy was first described in the 1860s, and there is still no current treatment to address heart failure in people with the disease.

A new study, the HOPE Duchenne randomized clinical trial, was the first to test cell therapy as a treatment for heart disease in Duchenne muscular dystrophy patients. 25 participants between the ages of 12 and 22 joined scientists from the Cedars-Sinai Medical Center for the trial. Cardiac progenitor cells, which have the ability to grow into mature cardiomyocytes (heart cells), were delivered to twelve randomly-chosen participants. The remaining thirteen participants instead received standard care for their condition, in the form of available drugs such as steroids that slow the loss of muscle strength and other drugs focused on controlling symptoms to improve quality of life.

The results showed improvement in the heart, hands, and forearm function. In the heart, the cardiac progenitor cell treatment improved function by reducing the amount of heart tissue scarring by seven percent.

"These early results show that further research is warranted and, in fact, is being planned,” said primary investigator Ronald G. Victor, MD. Phase II of the clinical trial is already in the planning stages.

"Generally, the primary cause of death in these patients is heart failure,” explained Eduardo Marban, MD, PhD, director of the Cedars-Sinai Heart Institute. “If we can slow or reverse heart failure in Duchenne patients, it will be a step forward."

Sources: Muscular Dystrophy Association, MedlinePlus.gov, Cellular Dynamics International, Cedars-Sinai Medical Center

About the Author
I am a scientific journalist and enthusiast, especially in the realm of biomedicine. I am passionate about conveying the truth in scientific phenomena and subsequently improving health and public awareness. Sometimes scientific research needs a translator to effectively communicate the scientific jargon present in significant findings. I plan to be that translating communicator, and I hope to decrease the spread of misrepresented scientific phenomena! Check out my science blog: ScienceKara.com.
You May Also Like
NOV 24, 2021
Cardiology
Stressed Mitochondria From Fat Cells Are a Warning to the Heart
NOV 24, 2021
Stressed Mitochondria From Fat Cells Are a Warning to the Heart
While obesity is closely linked to cardiovascular disease, that connection is murky. Some people who are considered obes ...
DEC 21, 2021
Clinical & Molecular DX
More Pollution, More Heart Attacks?
DEC 21, 2021
More Pollution, More Heart Attacks?
  When air pollution levels soar, so do calls to emergency hotlines specifically for heart attacks, says a Ger ...
JAN 18, 2022
Cardiology
New Study Links Mental Stress to Cardiovascular Disease
JAN 18, 2022
New Study Links Mental Stress to Cardiovascular Disease
We all know stress is bad for us. A new study confirms that psychological stress leads to greater risk for our hearts.
FEB 22, 2022
Clinical & Molecular DX
Gut Bacteria Imbalances and Heart Disease
FEB 22, 2022
Gut Bacteria Imbalances and Heart Disease
Patients with cardiovascular disease have significant disruptions in their gut microbiomes, says a new study by an inter ...
MAR 10, 2022
Cardiology
COVID-19 and the Heart- Insights from Cardiac Imaging
MAR 10, 2022
COVID-19 and the Heart- Insights from Cardiac Imaging
Although widespread disease prevention strategies such as vaccination have proven significantly helpful in reducing COVI ...
MAY 26, 2022
Cardiology
Reducing Salt Intake Improves Heart Failure Symptoms
MAY 26, 2022
Reducing Salt Intake Improves Heart Failure Symptoms
Reducing sodium intake improves symptoms, but not hospitalizations or deaths, for heart failure patients.
Loading Comments...