Advancing Pediatric Cardiovascular Research Interview with Clinical Innovator: Gail D. Pearson, MD, ScD, FACC
Gail Pearson, MD, ScD, FACC, is a pediatric cardiologist, an associate director of the Division of Cardiovascular Sciences and the director of the Adult and Pediatric Cardiac Research Program at the National Heart, Lung, and Blood Institute (NHLBI). Pearson joined the NHLBI in 1997 to oversee and develop the NHLBI’s clinical pediatric cardiovascular research programs. Since 2000, she has occupied several leadership positions within the Institute. Pearson received her medical degree from Johns Hopkins School of Medicine and a doctorate in health policy from Johns Hopkins School of Hygiene and Public Health. Pearson completed her pediatric residency and pediatric cardiology fellowship at the Children’s National Medical Center in Washington, DC, where she continues to teach and provide care for children with congenital heart disease (CHD).
Katlyn Nemani, MD, talked with Pearson about her career, the NHLBI’s investment in pediatric cardiovascular research, programs she has developed and what’s ahead in the next decade.
What inspired your interest in pediatric cardiology, and more specifically in caring for patients with congenital heart disease?
After spending several years in regional health planning and regulation, a change in political winds away from regulation made it clear that I needed to think about career options. Among my friends were two women who were neonatologists, and who encouraged me to think about a career in medicine. I initially dismissed this out of hand, because I didn’t have sufficient undergraduate science courses. I was also single at the time, both parents had passed away, so the prospect of four years of medical school with no visible means of financial support was a bit daunting. Nevertheless, they were persuasive, and so I embarked on a post-baccalaureate pre-med program. I was accepted by Johns Hopkins, which fortunately was interested at the time in students who were not coming directly out of college.
"It has been terrific to watch my patients grow up over the years, and watch the grace with which their parents accommodate the challenges of pediatric heart disease." — Gail Pearson, MD, ScD, FACC
When it came time to think about residency training, pediatrics was a foregone conclusion, in part due to the influence of the two neonatologists who had encouraged me into medical school, and because I liked children and felt there was a great deal of opportunity to make a difference in a child’s life through medicine. Once in pediatrics, I was interested in a sub-specialty that had a critical care component (ironic now, as I am an outpatient pediatric cardiologist), and of those available, only pediatric cardiology provided an opportunity to follow your patients through their lives, which appealed to me greatly. I wanted to help children and their families as they grew up. In addition, during my time at Hopkins, which is one of the birthplaces of pediatric cardiology, I was inspired by that rich history and the opportunities ahead.
Please tell us a bit about the path that led to your career in health policy and leadership position within the NHLBI.
I came to NHLBI through serendipity and a network of women that led me to the position of a pediatric cardiologist when another candidate suddenly changed her mind and left the NHLBI in the lurch. When I took the job, I thought I would be there for maybe 3 years – that was 20 years ago! Over time, I was entrusted with the development of the Pediatric Heart Network (PHN) and other programs, and was promoted into various leadership positions.
Who are the mentors who’ve had the most meaningful impact on your career?
The list is long! I have been fortunate to have had many mentors and a great deal of support throughout my training and career, but I’ll just highlight a few here. First are Billie Short, MD, and Anne Fletcher, MD, the neonatologists and friends who encouraged someone with an undergraduate degree in sociology to go to medical school, and with whom I had the privilege of working on research projects during my residency. While at Hopkins, I had the opportunity to spend time in the Division of Pediatric Cardiology and work with Catherine Neill, MD. Dr. Neill, who passed away at age 84 in 2006, was one of the pioneers in pediatric cardiology, trained by Helen Taussig, MD, FACC, and an extraordinary mentor during research.
Roberta G. Williams, MD, MACC, a pediatric echocardiography pioneer, made a semi-stray comment to me during a break at a conference held by ACC advising me to not reject a position that was not 100 percent clinical, and this came back to me when I was considering my first position at NHLBI.
I would like to give credit also to Michael S. Lauer, MD, FACC, currently Deputy Director of the National Institutes of Health, who was my boss for several years at NHLBI. He played a significant role in helping me refine my leadership style, particularly because he embraced the science of management. And my husband of 29 years, Craig Hoffman, MD, whose calm and imperturbable approach to both science and management has been a model I still strive to emulate.
Please tell us about the Pediatric Heart Network, the first program you developed at NHLBI.
The PHN1 was established in 2001 to improve evidence-based treatment options and standards of care for patients of all ages with CHD and children with acquired heart disease, such as Kawasaki Disease. The PHN is a multi-center clinical research consortium that currently consists of 9 main clinical research sites in the U.S. and Canada, a data coordinating center, and a variable number of auxiliary sites.
Over the past 15 years, the PHN has changed the landscape of pediatric cardiovascular research by providing a vibrant, sustainable, nimble infrastructure for multi-center research. To date, the PHN has conducted more than 20 clinical trials and other studies, which have advanced pediatric cardiovascular science in several areas: surgical care, single ventricle physiology, Marfan syndrome, Kawasaki disease and quality improvement.
The PHN has trained several hundred investigators and study coordinators in the conduct of clinical trials, and enrolled nearly 8,000 children and young adults in our studies. It has developed a robust young investigator program, a productive nursing research program, and the Children and Clinical Studies campaign,2 which helps patients and families learn about what it means to participate in clinical research. Our success is supported by the US News & World Report’s use of participation in the PHN as a factor in naming the Best Children’s Hospitals.
In 2009 you developed two research efforts to create the Bench to Bassinet Program. What are the goals of the Cardiovascular Development Consortium (CvDC) and the Pediatric Cardiac Genomics Consortium (PCGC)?
NHLBI had funded different programs designed to promote translation of research across the basic to clinical spectrum, and we had learned that you can’t force translation in one 5-year grant program, so we were looking for a novel approach. After a great deal of deliberation and consultation with extramural investigators, the Bench to Bassinet Program3 was born. The vision for the program was to provide a framework for scientists spanning the domains of basic science, functional genomics, translational biology, and clinical research to collaborate in accelerating progress toward discovery and translation.
The CvDC is a group of four academic institutions conducting basic science research to characterize the molecular networks and pathways that control normal and abnormal heart development. Recent work by the CvDC has enabled the creation of spatiotemporal maps of gene expression at a single cell resolution during heart development, which has revealed cell line-specific gene programs that underlie normal heart development and CHD.
The PCGC is a group of five centers using state-of-the-art genomics tools to identify the genetic causes of CHD and to determine how genes affect treatment outcomes. The PCGC has enrolled more than 10,000 children and adults with CHD, and many of their parents, who have agreed to provide DNA to the PCGC’s CHD Genes study. Recent PCGC studies have uncovered new groups of genes not previously associated with CHD, and have identified a genetic link between CHD and impaired neurodevelopmental outcome.
The CvDC and PCGC were designed to provide a continuum of research that, along with the PHN, would allow focus within the three consortia on their specific scientific strengths, while also encouraging organically, through joint meetings and research projects, translation of findings as they ripen to maturity. For example, we are now using the PCGC’s genomics capabilities and the PHN’s detailed phenotyping data to help us identify genetic and genomic factors that influence outcome. In the future, we hope to add data collection regarding behavioral and environmental risk factors for CHD.
What goals do you have for the NHLBI in the coming decade?
NHLBI is in the midst of an ongoing Strategic Visioning process designed to anticipate and capitalize on emerging scientific opportunities and identify approaches to overcome new barriers to progress. By way of disclaimer, the comments here reflect my thoughts, and don’t necessarily reflect official NHLBI positions. However, they are consistent with the four NHLBI Strategic Goals and Objectives: Understand Human Biology, Reduce Human Disease, Advance Translational Research, and Develop Workforce and Resources. In fact, the Bench to Bassinet Program is perfectly aligned with these goals.
I wear several hats at NHLBI, and have corresponding goals. In the broad clinical research arena, I anticipate that NHLBI will continue to be a leader in the efficient stewardship of the clinical trials process, and hope that we can take significant steps to help streamline clinical trials and to increase patient engagement in the process from start to finish. For my scientific program, the Adult and Pediatric Cardiac Research Program, which includes heart failure, arrhythmias, coronary artery disease and valvular heart disease as well as pediatric cardiology, I think heart failure is an area that requires some re-thinking, especially heart failure with preserved ejection fraction. For pediatric cardiovascular research, it would be great to see progress in several areas, including:
- Developing an integrated data network for CHD.
- Capturing newborns who screen positive for critical CHD through screening mandated in 2011.
- Identifying social, environmental, genetic and genomic contributions to the etiology and outcomes of CHD across the lifespan.
- Enrolling the majority of children and adults with CHD into one or more research protocols.
What aspects of your clinical and research career have been the most rewarding?
The rewards are numerous. The ‘village’ that the PHN has become never ceases to amaze me. It has been nothing short of remarkable over the years to watch the selfless energy that the investigators, coordinators and data coordinating center staff pour into the research. The success of our signature programs, the PHN Scholars for young investigators, and the nursing research program also have been extremely gratifying. Every member of the PHN, every day, is pulling together in the same direction – improved outcomes for individuals affected by the conditions we cover.
And then, watching the Bench to Bassinet program mature into a research powerhouse has exceeded all expectations. Who would have thought that the PCGC would so rapidly advance our understanding of the genetics of congenital heart disease and that their inaugural publication would be a Nature paper? I have frequently said in talks, and it remains true, I feel as if I am playing for an All-Star team every day.
I really enjoy interacting with the fellows at Children’s and the early career staff at NHLBI, and helping them to sort out research and other professional issues. Finally, it has been terrific to watch my patients grow up over the years, and watch the grace with which their parents accommodate the challenges of pediatric heart disease. Continuing to practice has significantly informed the programs that I have had the fortune to be able to develop at NIH, and reminds me, every week, why the research we support here at NHLBI is so important. There continue to be so many questions for which we don’t have answers, so it is gratifying to see patients one at a time, and to have the opportunity to design programs to help get the answers.
- www.pediatricheartnetwork.org/. Accessed March 14, 2017.
- www.nhlbi.nih.gov/studies/children-and-clinical-studies. Accessed March 14, 2017.
- www.benchtobassinet.org. Accessed March 14, 2017.
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