Adult cardiologist Kory Lavine, MD, PhD, completed his advanced heart failure transplant fellowship at Washington University School of Medicine with more questions than answers. He had been researching dilated cardiomyopathy, a chronic disease that renders the heart muscle too weak to work efficiently. Eventually, the heart begins to fail, making a transplant necessary. This can happen in adults or children.
Pediatric dilated cardiomyopathy can either be acquired, such as through a virus or cancer chemotherapy, or inherited through a gene mutation of one or both parents. However, according to the Children’s Cardiomyopathy Foundation (CCF), 75 percent of cases do not have a known cause. Families faced with the diagnosis today are forced to come to terms with an ominous prognosis. The CCF says the five-year survival rate for children with dilated cardiomyopathy is just 40 to 50 percent. For those who can survive until a heart transplant can be performed, the peril continues. In fact, throughout his career, Dr. Lavine has treated patients who had transplants as children. In many cases, those patients would grow up to once again develop heart failure as young adults. Dr. Lavine’s desire to find a way to more effectively
treat children in heart failure became a CDI-funded project.
Inside the human heart are white blood cells called macrophages. Some, known as embryonic-derived macrophages, were seeded there during fetal development. Others are derived from another type of white blood cell known as monocytes. Originating in bone marrow, monocytes become macrophages once they leave the blood stream and enter a tissue.
Before applying for a CDI grant, Dr. Lavine’s lab had been modeling human dilated cardiomyopathy in mice. What he and his colleagues noticed from that work was that, in a neonatal mouse heart, very few monocytes are attracted into the heart from the blood stream. Dr. Lavine will use his CDI funding to selectively remove those monocyte-derived cells. With them out of the way, the question becomes will the embryonic-derived macrophages take over to repair the heart or slow the progression of heart failure.
“We hope to identify a mechanism we can tap into that will disable the harmful immune cells and activate the reparative immune cells,” Dr. Lavine
Cheering him on are Colleen and Mike Miller. In 2010, just two days before Christmas, at 6 months of age, their daughter Layla Rose was diagnosed with dilated cardiomyopathy. As a registered nurse with cardiac care experience, Colleen knew her family’s life would never be the same from that day forward.
“When the diagnosis was cardiomyopathy, we felt like we were given a death sentence,” Colleen says. “More than half of all children diagnosed don’t live past age 5. If they do survive, it is a fluke or du
e to a heart transplant, and that is not a cure, since the life span of a new heart is only around 10 to 15 years.”
Layla spent her first Christmas in the cardiac intensive care unit at St. Louis Children’s Hospital, and had many more hospitalizations, tests, medical and surgical procedures after that. The Miller’s lives became filled with doctors’ appointments, medication schedules and worry, knowing the whole time that a transplant loomed in their future. That time came in August of 2014.
“We have always had an idea of where the dilated cardiomyopathy road would lead,” Colleen wrote in her Caring Bridge blog after learning they were out of options. “We prayed and worked and gave meds, faithfully went to doctors’ appointments and spent many nights in the hospital and cardiac ICU. All of this was done to keep the worst at bay … knowing full well it was out of our control.”
Unfortunately, Layla passed away after going into cardiac arrest during a cardiac catheterization that was meant to determine the readiness of her lungs to handle a new heart. Since that devastating day, the Millers have channeled their grief into acts of kindness, such as installing a buddy bench at Layla’s school or purchasing a rocking chair for the cardiac ICU, where they spent so much time. The chair has a plaque with Layla’s name and a quote that has sustained the Millers through it all. It reads: “There is no foot too small that it can’t leave an imprint on this world.”
To help make that imprint, the Millers also never shy away from opportunities to raise awareness of pediatric cardiomyopathy.
“We are thrilled to do anything we can do to keep Layla’s story going,” Colleen says. “Promoting awareness for a condition that has no cure, limited treatment, high mortality and needs more funding to find a cure is a goal of ours.”