Pittsburgh doctor's discoveries could help pediatric cardiac patients
Dr. Bernhard Kühn was still toiling in medical school when he dived into pediatrics, figuring he might use the specialty someday to treat his own children.
Now breakthrough discoveries that the pediatric cardiologist brought to Pittsburgh could help safeguard more than 1 million vulnerable babies born each year with congenital heart defects, scholars say.
They say doctors have no effective way to save those youngsters when their hearts give up, even though research has delivered proven treatments for adults with similar problems.
“There are none, period,” for babies with heart failure, said Kühn, 44, a father of three who joined Children's Hospital of Pittsburgh in September from Boston Children's Hospital. “We must look at pediatric heart failure as a unique thing that's different from adults.”
Children's touts his arrival as a prime catch for its Richard King Mellon Foundation Institute for Pediatric Research, where Kühn is the third physician-scientist in the Mellon Scholars Program for gifted researchers early in their careers. Appointees announced last year, Drs. Stephen Maricich and Timothy Sanders, focus their work on sensory, neural, organ and limb development.
“The recruitment of Dr. Kühn will bring in one of the leading researchers in heart regeneration to further explore heart-cell growth and to give hope to advancing treatments to heart failure,” Mellon Scholars Director Jay Kolls wrote in a statement.
About five years ago, Kühn and his colleagues in Boston identified neuregulin1, a natural stimulant manufactured by the body that can encourage heart muscle cells to divide. The discovery was a surprise because it reversed years of conventional thought that the cells would not multiply.
Another revelation from Kühn's laboratory arrived last year, when his team reported finding that the human heart keeps producing cells through a person's 20th birthday. He said the number of heart cells explodes from 1 billion to 4 billion in the first two decades of life.
Taken together, the discoveries suggest doctors eventually could treat young cardiac patients with a cell stimulant to spark heart muscle regeneration, overcome their defects and prevent heart failure. Kühn and his growing team at Children's are working toward clinical trials of treatments to test the theory.
“It's hugely promising,” said Anita Marcelo, president of the Children's Heart Research Guild fundraising group near Seattle.
Her son Gabriel, 24, has outlived many of his peers with heart defects as he fights his own cardiac problems. A transplant is no longer an option for him, but Marcelo said Kühn's work joins staggering advancements in surgery, genetic research and pharmaceuticals that are giving pediatric heart patients new optimism.
“If you can keep your kids alive for the next five years, so much will be changed. There's going to be so much more,” she said.
Kühn credited UPMC for giving him space and time in the lab to explore concepts after a researcher in Boston told him: “You're trying to play God.”
Though the National Institutes of Health support some of his work, he said, UPMC offered resources to help get the medical exploration off the ground. UPMC would not comment on the amount of money spent on Kühn's lab, which employs three technicians, a postdoctoral fellow and a part-time research coordinator.
“If he is able to regenerate heart muscle cells that work normally and repopulate … with normal heart muscle, that would be a huge benefit. I think it could be a potential game-changer,” said Dr. Brian Feingold, medical director of a pediatric heart failure and transplantation program at Children's.
Feingold helped lead another research team that questions a common medical practice for kids with heart problems. The group found some children on transplant wait lists tended to live longer — and have lower medical bills — if they accepted the first suitable hearts available, even if they had immune system antibodies that might cause rejection of the organs.
Doctors long have tried to match patients with hearts that appear less likely to trigger the antibody reaction, Feingold said.
“We think it's really exciting times, in terms of scientific abilities and techniques,” he said, questioning entrenched approaches to treatment. “We're always working hard to try to understand what we're doing, the implications of our therapies.”
Adam Smeltz is a Trib Total Media staff writer. Reach him at 412-380-5676 or [email protected].