Celebrating the 2013 Batchelor Award are, from left, Dean Pascal J. Goldschmidt, recipient Michael S. Kapiloff, UM President Donna E. Shalala, and Judy Schaechter, interim chair of pediatrics.

Michael S. Kapiloff, a pediatrician who for 18 years has devoted his expertise and energies to developing drug therapies for intractable heart diseases, is the recipient of the prestigious 2013 Micah Batchelor Award for Excellence in Children’s Health Research for a research proposal that could lead to new treatments for a number of cardiovascular disorders.

Selected from 11 outstanding applicants from the Department of Pediatrics, Kapiloff, professor of pediatrics and director of the Cardiac Signal Transduction and Cellular Biology Laboratory at the Interdisciplinary Stem Cell Institute, plans to use the $300,000 award to demonstrate that a seemingly insignificant enzyme he and his team identified could serve as a therapeutic target for treating the most commonly inherited heart defect, Familial Hypertrophic Cardiomyopathy (HCM). Present in about one of every 500 people, HCM causes the heart muscle to overgrow, making it prone to heart failure and sudden death. It can be especially deadly in children and often causes sudden cardiac death in seemingly healthy young athletes.

Kapiloff, an internationally recognized cardiac researcher who has enhanced the understanding of intracellular signaling pathways, officially accepted the award at an August 22 ceremony at the Batchelor Children’s Research Institute, where Miller School Dean Pascal J. Goldschmidt and Judy Schaechter, associate professor and interim chair of pediatrics, lauded his research accomplishments, selfless mentoring, and humanity. As Schaechter noted, “He is a great scientist – that is for sure – but he is also a really, really good guy who always gives opportunities and credit to those in his lab and other trainees.”

In her congratulatory remarks, UM President Donna E. Shalala also paid tribute to the late George E. Batchelor, the aviation pioneer and philanthropist who established the award in 2001 in memory of his grandson, Micah, to support Miller School investigators who are researching children’s diseases. Seeded with a $5 million endowment, it is one of the largest awards nationally for children’s health research.

“George Batchelor wanted every child in South Florida to be healthy, and he wanted us to have an impact on every child in the world,” President Shalala said. ”This year’s recipient, Michael Kapiloff, exemplifies the kind of basic scientists and physicians who really make a difference and who George, in particular, envisioned when he established the award.”

Taking the podium, Kapiloff expressed his gratitude to the Batchelor family for investing in research and ideas that are pushing the frontiers of health forward. Turning to his own winning idea, he explained why RSK3, a little-known and little-studied enzyme, has taken center stage in his well-known research on the protein mAKAP, which orchestrates the regulation of heart muscle cell growth.

Though RSK3 is such a “bit player” in the heart that eliminating it has no ill effect on the health of mice, Kapiloff said, it happens to bind to mAKAP, and that association enables RSK3 to regulate heart muscle growth. That’s why, he said, he proposed using genetically modified mice to test the idea that mAKAP-bound RSK3 is required for abnormal heart growth in pediatric heart disease. Though Kapiloff expects the animal experiments to demonstrate RSK3’s value as a therapeutic target for the treatment of HCM, he said he also expects them to support the enzyme’s potential therapeutic use for other, more common cardiovascular diseases.

“Because cardiac myocyte cardiomyopathy is actually the common response of the heart to any type of stress, our work actually will have an impact way beyond genetically encoded cardiomyopathy,” he said. “In fact, it will be directly applicable to what my dad died of, aortic stenosis, and for those people who have high blood pressure. These experiments are part of a translational pathway that could lead to clinical trials in human patients within this decade.”

Paying tribute to his father, Bernard Kapiloff, Kapiloff warmly recalled how the Baltimore surgeon who passed away last year at age 94 would call him every day for research updates. “Sometimes, I’d have to say, ‘Dad, it’s only been 24 hours! Your patients may get better in 24 hours, but we don’t make discoveries every single day.’ ”

When the laughter subsided, Kapiloff said he looked forward to sharing news of his lab’s research success in the near future.

For the first time this year, the Batchelor Award was opened to all full-time faculty with primary appointments in pediatrics. That resulted, the dean and Schaechter both noted, in extraordinary proposals from superb applicants across the department, any one of which was worthy of funding. The other applicants and their proposals were:

• Carolyn Abitbol, professor of pediatrics, with “Early Detection of Kidney Disease and Abnormal Angiogenesis in Preterm Infants”

• Samita Andreansky, research assistant professor of pediatrics, with “Development of Influenza Specific T Cell Vaccines”

• Emmalee Bandstra, professor of pediatrics, with “Epigenetics of Prenatal Cocaine Exposure and Postnatal Environmental Adversity”

• Jeffrey Brosco, professor of clinical pediatrics, with “Newborn Screening and Health Disparities in the Genomic Era”

• Jayanthi Chandar, associate professor of pediatrics, with “Promoting Functional Health Literacy in Youth before Kidney Transplantation: Will This Improve Health Outcomes?”

• Nelson Claure, research associate professor, with “Automatic Adjustment of Inspired Oxygen for Prevention of Retinopathy of Prematurity and Bronchopulmonary Dysplasia in Premature Infants”

• Aymin Delgado, assistant professor of pediatrics, with “Determinants of HCV Testing among High Risk Children and Youth”

• Michael Freundlich, professor of pediatrics, with “Roles of Vitamin D and Fibroblast Growth Factor 23 in the Chronic Reno-Cardiac Syndrome”

• Charles Mitchell, professor of pediatrics, with “T Follicular Helper Cell Dysfunction as the Cellular Basis for Vaccine Non-Responsiveness among HIV-Perinatally Infected Children”

• Elena Perez, associate professor of pediatrics, with “The Intestinal Stem Cell Niche – Understanding the Immune, Epithelial, and Non-Epithelial Dialogs in the Intestinal Niche During Tissue Injury and Recovery”