Research and Innovation

Clinical research and innovations in care have dramatically improved the outlook for children with many forms of heart disease. Betty Irene Moore Children’s Heart Center’s Pediatric Advanced Cardiac Therapies (PACT) program is fully committed to supporting, participating in, and leading diverse forms of research.

As a part of Stanford Medicine, our physician-scientists lead investigations into the fundamental biology, diagnoses, and care of heart failure and related conditions. We advance the understanding of heart failure and heart transplantation and discover new and improved treatments that provide options for children where sometimes none existed, decrease the risk of rejection, quicken time to transplant, improve donor matches, and increase quality of life before, during, and after transplant. If you are interested in having your child participate in a research study or clinical trial, talk with one of our doctors about opportunities.

A sampling of some of our recent and ongoing research work is listed below.

Donor heart size matching

Pioneered by one of our pediatric cardiologists, John Dykes, MD, this innovation helps to expand the heart transplant donor pool and shorten waiting-list times for children in need of a heart. When a child is in heart failure, their heart is bigger, so it can sometimes match the size of an adult heart or that of an older child. We use a computed tomography (CT) scan and special software in Stanford’s sophisticated 3-D lab to measure the volume of the donor and recipient hearts to determine if there’s a good match. This unique heart-size matching approach helps save the lives of children on the waiting list by getting them hearts, and sooner. Without this innovation, many donated hearts would not even be considered for children.

Use of stem cells in pharmacogenomics

Under the direction of Daniel Bernstein, MD, for several decades, the Bernstein laboratory has conducted important investigations to understand cardiac function. The lab’s current work focuses on using stem cells to understand the basis for individual variation in response to cardiac medications. This will ultimately allow for personalized combinations of medications based upon these differences to maximize the benefits for patients while limiting side effects.

Panorama-HF

The Panorama-HF study is an industry-led investigation of a new medication to treat pediatric heart failure. Studies of this medication (Entresto) have shown good results in adults with heart failure. While this is encouraging, it doesn’t provide definite proof of its value in children. Panorama-HF is a large national study to answer that question. Beth Kaufman, MD, is the Stanford lead investigator for this study, which has completed enrollment for the primary phase and is now in the long-term follow-up stage.

Molecular basis of right-ventricular failure

Heart failure in children frequently involves diseases of the right ventricle, in contrast with heart failure in adults, which is more commonly seen in the left ventricle. Sushma Reddy, MD, is studying the cellular basis of right-ventricular failure in order to understand key differences and similarities between right- and left-ventricular failure. With this knowledge, we will be better able to treat this condition in affected children.

Teammate trial

This is a North American multicenter study designed to test a new combination of heart transplant immunosuppressive medications that promises to reduce the side effects associated with long-term treatment of heart transplantation patients. The study is designed and led by Christopher Almond, MD, Seth Hollander, MD, and Kevin Rady, MD, from Stanford Medicine Children’s Health and Boston Children’s Hospital. Enrollment for this study is ongoing.

“Changes in Myosin Biomechanics Influence Growth and Maturation of iPSC-Cardiomyocytes” study

Our researchers, led by Daniel Bernstein, MD, are studying how gene mutations lead to heart problems. White blood cells that have been genetically reprogrammed back into an immature stem cell (iPSC) and then induced to form heart muscle cells were used to better understand some of the most potentially devastating heart diseases, dilated and hypertrophic cardiomyopathy. By better understanding how gene mutations lead to these heart problems, doctors will be able to develop new precision-based drug therapies for children and adults with cardiomyopathy, helping them live a healthier life.

“The Impact of Fontan Circulatory Failure, Frailty, and Resilience on Heart Failure and Transplant Outcomes” study  

In this large, national, multicenter study, several of our specialty heart programs, including our Single Ventricle Program, Pediatric Advanced Cardiac Therapies (PACT) Program, and Pulmonary Vascular Disease Program, have teamed up to use advanced therapies to try to postpone the need for heart transplant in children with these conditions. The study, spearheaded at Stanford Medicine Children’s Health by Sharon Chen, MD, is exploring the best timing for transplant evaluation, how to reduce risk, how fragility or a patient’s overall state affects success, and other key factors to maximize a chance of a good outcome for children with Fontan heart circulation. 

Ask your Stanford Medicine Children’s Health provider if you would like to learn more about these research efforts.