Setting New Research Vision for Cedars-Sinai Guerin Children’s

Cedars-Sinai Guerin Children'sis building a formidable pediatric research enterprise aimed at identifying the genetic underpinnings of childhood diseases and developing novel therapies. Spearheading this scientific endeavor is David H. Rowitch, MD, PhD, associate director of Research at Guerin Children's and an internationally renowned neonatologist, developmental neuroscientist and authority on pediatric neurological disease.

Under the guidance of Rowitch and Guerin Children's Executive Director Ophir Klein, MD, PhD, a growing corps of research scientists is performing basic and translational research to establish high-impact investigative programs in functional genomics, neonatal and pediatric screening, pediatric single-cell biology and clinical neurological disease.

Rowitch, who oversees research at Guerin Children's, was elected recently to the prestigious National Academy of Medicine in recognition of his groundbreaking scientific contributions to the understanding of pediatric neurological diseases. Election to the academy is one of the highest honors in health and medicine, celebrating individuals who have shown exceptional professional achievement and dedication to service.

The honor was the latest accolade for the physician-scientist, who was elected to the American Association of Physicians in 2012. He was also elected as a fellow of the Academy of Medical Sciences in 2018 and a fellow of the Royal Society in 2021-both in the U.K. Rowitch served as a member of the National Advisory Council for Child Health and Development of the National Institutes of Health from 2020 to 2024.

Rowitch's research has garnered international recognition for studies focused on glial cell development, leukodystrophy, multiple sclerosis and cerebral palsy and has advanced diagnosis and treatment for neonatal brain injury.

"Dr. Rowitch is globally recognized for his extensive expertise in neuroscience and genomic research and his innovative approach to integrating physical and mental healthcare," said Klein, executive vice dean of Children's Health at Cedars-Sinai. "His leadership is propelling Guerin Children's research to new heights."

Rowitch transitioned to Guerin Children's in 2023 while continuing to serve as professor and head of the Department of Pediatrics, University of Cambridge, academic lead of Cambridge Children's Hospital and a Wellcome Trust senior investigator in the Wellcome-MRC Cambridge Stem Cell Institute in the U.K. During his time at Cambridge, Rowitch created a new approach to integrated physical-mental healthcare and genomic research at Cambridge Children's Hospital.

The Cedars-Sinai Newsroom spoke recently with Rowitch to learn more about his priorities for pediatric research at Guerin Children's.

What is your vision for Guerin Children's research?

At Guerin Children's, we have a commitment to include and integrate research in our clinical care because we believe all patients and families should have the option to participate in research, which is where innovative care is often tested and can provide long-lasting benefits. We are using basic science research to understand rare conditions and develop new therapies for poorly understood conditions, starting with tools like single cell and DNA analysis using whole genome sequencing.

What is whole genome sequencing?

Knowing genetic characteristics helps us to decide the medicines that might work best for a child. For example, a child with epilepsy and a genetic condition can respond better to certain antiepileptic drugs based on rapid genome testing. Whole genome sequencing can assess the more than 6 billion base pairs of DNA in every cell that exists in our body. The genome sequencing test, which once took 10 years and required international collaboration, can now be done through a simple blood test much faster.

The genome, containing crucial information for predicting future health risks, can be analyzed within 24 hours, making it an important tool both for rapidly diagnosing critically ill children and predicting disease before it starts, instructing interventions that can potentially delay the onset of future health conditions. This technology has evolved remarkably and is now becoming a bedside tool, and we will be testing its impact on our care at Guerin Children's.

What is the proposed Cedars-Sinai Guerin Children's birth cohort study?

The birth cohort study is proposing to address the big question: What will the newborn screen of the future look like, and how can we make it most effective and beneficial? To do that, we need to collect a lot of data through whole genome sequencing on patients and families who are willing to participate. As part of a research study, we plan to conduct a birth cohort assessment by collecting genomic data from newborn babies in the Cedars-Sinai Neonatal Intensive Care Unit, as they are more likely to face future health events.

Through the birth cohort study, we aim to capture and identify thousands of combinations of biomarkers in the bloodstream, which can potentially help connect the dots and predict health risks like respiratory infections or learning disabilities. Furthermore, at Guerin Children's, we are committed to providing long-term beneficial support and utmost discretion to families who participate in the study. Based on the findings, we will make rapid diagnoses, provide genetic counseling and recruit participants for precision medicine or gene therapy trials.

Where do you see Guerin Children's research in the next five years?

We will be performing whole genome sequencing and ideally offer it to all children who are admitted to Guerin Children's. Our goal is to eventually incorporate it at sister hospitals that exist as part of our network in the greater Los Angeles area and make this a new tool that we can use consistently throughout our system.

To meet the demand, we are recruiting a growing list of new basic science investigators who are looking at the fundamental building blocks of development from the womb to birth, so that we can get new insights into the impact of genetics on development and how that might affect our understanding of disease.

Read more on the Cedars-Sinai Blog: Innovation and Collaboration in Pediatrics at Cedars-Sinai Guerin Children's

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