Lab tests show combination of existing drugs preserves heart, muscles

(COLUMBUS, Ohio) –   By taking two drugs that have been on the market for decades and using them in a new way, researchers may have discovered a promising new treatment option for Duchenne muscular dystrophy, according to a new study in the journal Circulation.*  

Scientists at Ohio State University Medical Center were able to preserve muscle function in lab mice by using both drugs much earlier in the disease. “Compared to mice without muscular dystrophy, these mice preserved 80% of normal muscle function” said Subha Raman, MD, the lead investigator of the study.  “We think it’s a big step, and that’s not just our opinion.  We’ve gotten good feedback from other investigators and the collective response is that this may be a tremendous advance for patients with muscular dystrophy.”

Raman teamed up with OSU scientists Dr. Jill Rafael-Fortney, and Dr. Paul Janssen to look into the possible benefits of spironolactone, a diuretic developed in the 1950’s, 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” said Raman, “but also in skeletal muscle which is, potentially, a great benefit to these patients.”

While human tests using these drugs at earlier stages of the disease are still months away, 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 3-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 3-dimensional image and it was - even today, that was five years ago - that never, never gets old” he said.  “It’s just utterly amazing.”

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.  To raise money Ty began competing in triathlons at the age of 50.

“I wore a tee-shirt at a triathlon down in Miami Beach and I think I raised $150 for that race.  And I’m like ‘OK, that’s not really going to do a lot.’ Then I did another race and another race” said Ty Ballou.

Today, Ballou Skies has it’s own website, www.ballouskies.com, and the triathlon team has grown to 13 members from California to western Pennsylvania.  All member compete to not only raise awareness of muscular dystrophy but, specifically, to raise money on Ryan’s behalf for Dr. Raman.  And that personal, private support is making a difference.

“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” said 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” said 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.”


*Early Treatment With Lisinopril and Spironolactone Preserves Cardiac and Skeletal Muscle in Duchenne Muscular Dystrophy Mice, Circulation, Volume 124, Number 5, August 2011.  Online: http://circ.ahajournals.org/