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Going to the Heart: Why Research Matters
As a leader in pediatric research, Children’s Memorial Research Center is dedicated to preventing childhood illness, treating pediatric conditions for which cures are not available, and promoting healthier lifestyles. Whether the cause is cancer, a birth defect, chronic illness or the environment, our interdisciplinary teams of physicians, nurses, research assistants, scientists and other staff are sharing thoughts and observations, data and publications, in order to help children and their families.
Cardiac problems, for which infants and children are diagnosed each day, are an example. Congenital heart defects, the most common type of major birth defect, might develop during pregnancy. Heart problems in children can be caused by infections, inflammatory conditions, even by malfunctions of other organs. And sometimes the causes are not known, or are poorly understood. Here we focus on five scientists who are crossing boundaries to make a difference in cardiac care, and to find more effective therapies and cures.
Kimberley Dilley, MD, MPH is a general pediatrician working in the Center for Cancer and Blood Disorders who is interested in long-term outcomes for children following cancer therapy. Two points are critical: First, approximately one in 350 children in the U.S. will be diagnosed with cancer. Second, and fortunately, the long-term survival rate is 80 percent, due to incredible advances in therapies. For this significant population of survivors, the long-term effects of therapies are also important. In a recently published report, Dilley and a colleague used the knowledge that anthracyclines, powerful anti-tumor agents used for a variety of malignancies, cause cardiotoxicity. This in turn can result in cardiomyopathy and even heart failure. In their study, the team found that it is important to screen these patients because many have abnormalities that aren’t otherwise apparent. Says Dilley, “Research is a critical component of my ability to provide care to patients. The survival rates for childhood cancer have been high for a few decades now, so we must continue to monitor very long-term outcomes and design intervention studies to prevent late effects.”
Across the street at the research center, Christine DiDonato, PhD of the Human Molecular Genetics Program is interested in proximal spinal muscular atrophy (SMA), the leading genetic cause of infant mortality. Traditionally, SMA has been described as a motor neuron disease; however, there is growing evidence that the genetic defect in SMA impacts other systems in the most severely affected infants. DiDonato’s group has identified functional cardiac abnormalities in a mouse model for SMA. When treated with trichostatin A, a drug known to benefit SMA mice, improved maturation of the heartbeat and an increase in cardiac size result. These mice eventually succumb to their disease, frequently from severe bradyarrhythmias. Clinical studies to evaluate sensory and autonomic function, including cardiac rhythm disturbances, are projected in infants with SMA. “With supportive care producing increased length of survival in infants and children with SMA, and with anticipated clinical trials likely to lead to further improvement, studies such as these are crucial to understand potentially life-threatening cardiac involvement,” says Nancy Kuntz, MD, medical director of the Mazza Foundation Neuromuscular Program at Children’s Memorial.
Kawasaki disease (KD) is an acute childhood illness characterized by a high spiking fever that usually affects previously healthy infants and children. Although the symptoms normally resolve fairly quickly, in untreated or severe cases coronary artery aneurysms, heart attacks, and/or sudden death can occur. In fact, KD has replaced acute rheumatic fever as the most common cause of acquired heart
disease in children in the U.S. and other developed nations. Unfortunately for sufferers, KD resembles other diseases and there is no diagnostic test, so diagnosis can be delayed or the condition is not treated. Anne Rowley, MD, a physician in infectious diseases, has devoted most of her research career to find the cause of KD. This would strengthen efforts to develop a diagnostic test, improve therapy and prevent KD. Rowley’s long-term goal is prevention through vaccination or other strategies. She says: “Biomedical research is important because it is the primary means to improve the health and alleviate health-related suffering of people throughout the world, including our families and loved ones. Through careful study of a health problem by scientists working together, significant advances in diagnosis, treatment and prevention of many illnesses can be achieved.”
Sunjay Kaushal, MD, PhD performs cardiovascular-thoracic surgery and conducts basic research in cardiac biology. His interest in cardiac stem cells for therapy is more than academic. Kaushal sees patients with heart failure whose long-term survival depends on replacing or repairing damaged tissue. While current therapies are amazing feats of technique and have greatly benefited patients’ lives, Kaushal says, “Resident cardiac stem cells represent one of the most promising types of cellular therapy for children with cardiac failure. Because they respond to intercellular signaling or environmental stimuli, stem cells offer a powerful advantage for regenerative therapies.”
The Pediatric Practice Research Group (PPRG) is a regional practice-based research network that includes over 70 Chicago-area community practices and health centers. Through these partnerships, PPRG is dedicated to determining best practices, delivering ongoing clinical education to providers, and evaluating the outcomes of its efforts. The alarming recent uptick in obesity rates among children has raised many questions regarding consequences and prevention. It is known that overweight and obese children exhibit type 2 diabetes, dyslipidemia and hypertension, which contribute to cardiovascular disease risk. In a recent study, PPRG found that there are close ties
between the health habits reported by mothers for themselves and their children. Because parental role modeling provides a consistent, important influence on child health behaviors, PPRG is exploring new strategies to aid pediatricians in directly addressing parental health habits. “A better understanding of physiologic, behavioral, and emotional influences on child health can refine and redirect health care providers and health care systems to provide care that addresses the complex needs of children and their families,” says PPRG director Helen Binns, MD, MPH.
All of these scientists share a commitment to the patients and families they serve, and their efforts offer hope for moving prevention, care and cures forward.
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| Kimberley Dilley, MD, MPH |
Christine DiDonato, PhD |
Anne Rowley, MD |
Sunjay Kaushal, MD, PhD |
Helen Binns, MD, MPH |