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In what may be the biggest breakthrough in muscular dystrophy in years, a patient at Cincinnati Children’s is believed to be the first in the nation with Duchenne muscular dystrophy to receive a left ventricular assist device (LVAD).
A surgical team led by David Morales, MD, chief of cardiovascular surgery and executive co-director of the Heart Institute, recently implanted a Thoratec HeartMate II LVAD for Jason Williams, a 29-year-old man with Duchenne.
Because 80 percent of boys and men with Duchenne ultimately die of heart failure, LVADs could add years, possibly decades to their lives.
“This is a major milestone in the care of Duchenne muscular dystrophy,” says John Lynn Jefferies, MD, director of the heart failure and ventricular assist device programs at the Cincinnati Children’s Heart Institute. “This treatment offers the possibility to change the outcome and the lives of these young men in a significant way.”
Worldwide, about 2,500 people a year are born with Duchenne. Symptoms of muscle deterioration and weakness generally emerge between the ages of 2 and 6, with many patients requiring a wheelchair by age 12. As the disease progresses, people with Duchenne develop heart problems and severe difficulty breathing. Many die in their late teens or early 20s.
Patients with Duchenne are not typically candidates for heart transplant. But continuing advances in mechanical heart assist devices offer a new option for extending life while researchers continue working to find a cure. Current studies are examining whether stem cell therapy can increase the heart’s pumping ability and whether bone marrow cells injected into the heart can replace malfunctioning cells.
“This operation gives Jason the most important thing: hope for a future,” Morales says. “Jason has a lot going for him. In Cincinnati Children’s, he has a broad and deep heart team; a backbone of basic, translational and clinical research working for him; international thought leaders in muscle disease to help manage his care; and institutional leadership that makes advances such as this possible. Most importantly, he has a tremendously dedicated family to support him.”
Jason’s care is managed through the Comprehensive Neuromuscular Center at Cincinnati Children’s, which cares for more than 1,100 children from around the world with a range of neuromuscular diseases. Directed by Brenda Wong, MD, the center includes specialists in pulmonary medicine, cardiology, pediatric rehabilitation, genetics, physical therapy, gastroenterology, nutrition therapy, social services, psychology, neuropsychology, endocrinology, ophthalmology, and orthopaedics.
Cincinnati Children’s growing expertise in cardiac assist devices also is attracting attention from heart surgeons worldwide. In October, leading five surgeons from Japan and Brazil visited Cincinnati Children’s to receive training on using ventricular assist devices in pediatric cases.
In Japan, heart transplantation is relatively new. In fact, children under 15 could not become organ donors until 2010. In Brazil, heart transplants have been performed regularly since the 1980s. But until now, surgeons had not been using heart assist devices in children.
“These are entire countries just coming online to do this kind of work,” says Jeffrey Towbin, MD, co-executive director of the Heart Institute. “Japan selected just three centers to do it, and they’ve all come here to receive training.”
Since 2009, the Heart Institute at Cincinnati Children’s has grown rapidly:
“Cincinnati Children’s has become one of the premier places in the world for treating children with end-stage heart failure,” Morales says. “This training visit demonstrates that we have the technology, the know-how and the team care approach that people all over the world want to learn from.”
Heart patient, Jason Williams.
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