Clinical Trial Under Way for Duchenne Muscular Dystrophy
Ohio State is currently enrolling patients in a clinical trial that may offer a new treatment option for patients with Duchenne muscular dystrophy (DMD). The goal is to improve diagnosis and treatment of heart muscle disease occurring in MD patients.
The clinical trial is based upon earlier research at The Ohio State University Wexner Medical Center through which muscle function in lab mice was preserved by using drugs much earlier in the disease.
Subha Raman, MD, teamed up with Ohio State scientists Jill Rafael-Fortney, PhD, and Paul Janssen, PhD, allowing clinical and research experts to leverage their specific areas of study to improve the research timeline and results. The team began to look into the possible benefits of spironolactone, a diuretic developed in the 1950s, and lisinopril, a heart drug from the early 1990’s.
Both drugs are often given to muscular dystrophy patients, but only after there is noticeable scarring in their heart tissue. By giving both drugs to mice earlier in the study, doctors found muscle protection throughout the body. “What was surprising to us was this was not just in the heart, where these medicines have traditionally shown a benefit” says Raman, “but also in skeletal muscle which is, potentially, a great benefit to these patients.”
Patient Success Story Fuels Clinical Trials
While clinical trials using these drugs at earlier stages of the disease have begun this year, there is already compelling evidence that the results will translate just as well.
Ryan Ballou is a 24-year old with muscular dystrophy from Pittsburgh, who has been taking the medications for more than five years.
“I’ve been ahead of the curve” he said, “I walk longer than most people and I still have a lot of strength in my arms.” But when it comes to charting the possible benefits of the drugs, seeing is truly believing. “They use an MRI scan to take a three-dimensional picture of my heart and can see what scarring is already there, caused by the muscular dystrophy” said Ballou. “There was scarring to begin with, when I first went, but since I’ve been taking the medicine there’s been no more scarring.”
It was during one of those MRI scans that Ryan’s father, Ty Ballou, became a believer. “Ryan was saying ‘Dad, how’s my heart look?’ And I’m looking at the three-dimensional image and it was – even today, that was five years ago – that never, never gets old,” he says. “It’s just utterly amazing.”
Donations Fund Research at Ohio State
The findings have so encouraged and inspired Ryan and his family that they started a charity called Ballou Skies, specifically to help fund the research by Dr. Raman and her team at Ohio State. Today, Ballou Skies has its own website, ballouskies.com.
“It’s a huge, huge impact,” says Raman. Traditionally, doing a study like this from start to finish with public grants might take up to five years. “But with Ballou Skies funding, we’ve been able to complete all of that within a year,” says Raman. “This simply would never have happened in such a short timeline without their inspiration and their support.”
As for the Ballous, they are happy to do it and say it just proves what a few people with a purpose and a passion can do. “It makes me very happy to know that I am actually having an active part in gaining funds for research,” says Ryan. “It’s kind of my outlook on life, you can’t let things keep you down and you can’t let things hold you back.”
To learn more about the research or to become involved in the clinical trial, please contact Beth McCarthy.
Give a gift online in support of this research or contact Julie Dials.