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
JAN 03, 2020
Cardiology
JAN 03, 2020
Invasive Surgery isn't Worth it For All Heart Disease Patients
Invasive procedures are no better than medications and lifestyle advice at treating heart disease that’s severe bu ...
MAR 08, 2020
Cardiology
MAR 08, 2020
Cashier and Healthcare Jobs Increase Risk of Heart Problems in Women
Stress has long been known to be a major risk factor for developing heart disease. Now, researchers from Drexel Universi ...
MAR 11, 2020
Cardiology
MAR 11, 2020
People with Arthritis at Higher Risk for Heart Disease
As it turns out, rheumatoid arthritis (RA) doesn’t just affect the function of your joints. New research has found ...
APR 14, 2020
Cardiology
APR 14, 2020
Personalized Treatments for Patients with Diabetes and Heart Disease
Often, people who have Type 2 diabetes are at an increased risk for cardiovascular disease. This is because they are usu ...
APR 23, 2020
Cardiology
APR 23, 2020
Blood Pressure Spikes in Young Adults Linked to Heart Disease Later on
Researchers have found that young adults in their 20’s and 30’s who experience inconsistent blood pressure m ...
APR 23, 2020
Cardiology
APR 23, 2020
Arteries Respond in Different Ways in Females and Males
Exploring Arterial Smooth Muscle Kv7 Potassium Channel Function using Patch Clamp Electrophysiology and Pressure Myograp ...
Loading Comments...