It attacks the heart and breathing muscles in boys and young men. Those with Duchene Muscular Dystrophy will likely die well before they reach their mid-20's.
Now, one young man and his father are focusing on prolonging lives, until there's a cure. And a new ground-breaking study is possible because of their efforts.
Ryan Ballou suffers from a rare genetic disease passed from mother to son, Duchene Muscular Dystrophy. It causes breathing and heart muscles to breakdown, and fibrosis, or scarring to develop. Ryan's Dad Ty, knows it will eventually kill his boy.
Ty Ballou, Ryan's dad, "Ryan will have lung failure and or, he will have a heart attack."
But because of various medications, Ryan's damaged heart is still strong. Cardiologist Subha Ramen, saw promise in the minimally scarred muscle.
Dr. Subha Raman, Professor of Cardiovascular Medicine at Ohio State University Medical Center, describes how he began the process of the new treatment, "So, it made me wonder if it was possible to treat fibrosis at an early stage."
Along with Ohio State University Researchers Jill Rafael-Fortney and Paul Jansen, Doctor Ramen wanted to test a combination of the common heart failure drugs, spironolactone and lisinopril.
Dr. Jill Rafael-Fortney Associate Professor of Molecular and Cellular Biochemistry, Ohio State University, explains how the two drugs may help the condition, "Both of the drugs are known to be anti-fibrotic or prevent scarring."
But there was no money to do that, so, Ty and Ryan created Ballou Skies.
Ty Ballou, explains how he took charge and started the charity, "I said to hell with it. Whatever you need, you get the kids, we're gonna get the money. I started doing these crazy tri-athlons."
Now, Ballou Skies has its own tri-athalon team. Ty says with help from the athletes, Ballou Skies has raised more than $70,000for the research effort.
Ryan ballou, describes how he feels about the things his father is doing, "It's nice that we're actually kind of helping speed along the research."
Wearing a Ballou Skies bracelet for inspiration, Doctor Ramen says without that money there would be no research. Now that is it happening, the team is seeing improvement in heart muscle structure and function. And they're surprised by the effects on skeletal muscles.
Dr. Rafael-Fortney explains the results that the doctors have seen since the treatment began, "We saw a doubling of muscle force, so the muscles in the limb and the diaphragm were twice as strong."
Results made possible by Ballou Skies, that could some day prolong its co-founders life, and others just like him.
Clinical trials are expected to start in the next several months. Doctor Ramen says because both drugs are already available, the FDA can use data from the clinical trials to add MD treatment to their existing approved uses. Ty says he needs to raise more than $200,000 to help fund the work.
The link to the website to donate is ballouskies.com
TOPIC: 'BALLOU' SKIES: FUNDING THE FIGHT AGAINST MD
BACKGROUND: Duchenne Muscular Dystrophy, DMD, is an inherited disorder that involves rapidly worsening muscle weakness. It is one of nine types of muscular dystrophy. Men are more likely than women to develop symptoms of the disease; approximately one out of every 3,600 male infants develops the disease.
(SOURCE : www.ncbi.nlm.nih.gov)
CAUSES: It is caused by a defective gene for dystrophin, a protein in the muscles.
SYMPTOMS: Symptoms of DMD can include fatigue, mental retardation, muscle weakness that begins in the legs and pelvis area causing frequent falls. Also, progressive difficulty walking is a sign of DMD. (www.ncbi.nlm.nih.gov)
COMPLICATIONS: There are a series of complications related to DMD. Complications can include heart disease, congestive heart failure, deformities, heart arrthymias, mental impairment, pneumonia or other respiratory infections and also respiratory failure. (www.ncbi.nlm.nih.gov)
TREATMENT: The two drugs Spironolactone and Lisinopril are used for other forms of heart disease. The combination of the two is being studied for the treatment of DMD due to the disease causing breathing and heart muscles to breakdown and fibrosis or scarring to develop.
For the study, the drugs were tested on mice. One group received both drugs at eight weeks of life; the second group at four weeks and the third group received no treatment. At 20 weeks all mice had a normal amount of blood pumped through the heart from the right and left ventricles with each heartbeat.The treated mice showed less heart muscle tissue damage.
In general, it is suggested that individuals with DMD stay active, such as through physical therapy which can help maintain muscle strength and function. Inactivity can worsen the muscle disease.