Innovation and Collaboration in Pediatrics at Cedars-Sinai Guerin Children's
Nov 17, 2023 Nicole Levine
Discoveries in pediatric diseases could hold answers to some of the biggest questions in medicine.
Experts at Cedars-Sinai Guerin Children’s are committed to reshaping the understanding and treatment of a spectrum of diseases. Supported by a $100 million gift from the Shapell Guerin Family Foundation, Guerin Children’s has an ambitious vision for children’s healthcare. It couples a scientific mission with seamless and innovative clinical care that serves patients from their first breath to their first steps into adulthood.
Guerin Children’s physicians share their hopes for the program and their vision for the next wave of breakthroughs in pediatric medicine.
"In our field, we get excited about the success of new medications, but if you look in detail, most of these successes are in the 30% to 40% range. That’s not good enough. We need to reach more patients, get them into remission and achieve that deep healing of their tissues, so they’re not at risk of long-term complications.
"That’s why it’s so important to match the right patient, at the right time, with the right treatment. It’s one of the reasons it’s important to study children diagnosed with IBD before age six. They’re a unique cohort with a genetically driven disease that can be more severe. Our collaboration with our Regenerative Medicine Institute is seeking novel ways to study these patients and better understand how to effectively treat IBD.
"We work with teams in regenerative medicine, genomics, proteomics and microbiome in treatment and research. Our doctors, nurses, dietitians and social workers consider every aspect of what our patients are eating, how they are feeling and their mental health alongside medical interventions."
Sometimes, pediatrics can get buried in the big adult world. Being at a medical center with adult and pediatric patients turned out to be one of our greatest advantages because of the collaborations that are possible."
Tyler Pierson, MD, PhD: "I believe rare diseases can lead to broad breakthroughs."
Title: Pediatric Neurologist
Expertise: Pediatric Neurology, Regenerative Medicine/Stem Cells, Center for the Undiagnosed Patient
"Studying rare diseases does not only help those who are directly affected by them. In fact, the study of a rare disease led to the development of statins, which revolutionized how we treat heart disease, the No. 1 cause of adult death in the world.
"Most rare diseases are genetic, and most people with these conditions are children. Guerin Children’s has the expertise across many disciplines to facilitate important discoveries and potentially find solutions for problems that affect patients of all ages.
"An example of this is the work we’re doing with GATAD2B-associated neurodevelopmental disorder, or GAND. When I first started working with patients with GAND, there had only been about four cases of it in history, and now we’ve identified about 250. I’ve worked with Helping Hands for GAND, a family research group, to help them identify and register other patients so we can better study the condition. In the laboratory, we’re working with multiple models to better understand how the brain organizes itself, and how GAND affects that. We’re using patient-derived induced pluripotent stem cells to create cerebral organoids, and we can compare those to what we see in a mouse cortex to identify which genes the GATAD2B protein is affecting. These new technologies allow for better disease-modeling tools that could make a big difference for understanding and treating rare diseases."
"Sometimes, pediatrics can get buried in the big adult world. Being at a medical center with adult and pediatric patients turned out to be one of our greatest advantages because of the collaborations that are possible. For example, I was covering transplants in adults on weekends, and I saw that they were doing a test to predict early detection of rejection—which we’ve never had for children. It wasn’t Food and Drug Administration approved yet for pediatrics, which often happens with drugs, testing and other advances. We developed a memorandum of understanding with the company that makes the test, and they gave us 150 testing kits. We enrolled 67 patients in our study and found that this test for donor-derived, cell-free DNA is an excellent predictor of organ rejection in children."
We’re building on the Cedars-Sinai model of care, and that is premised on excellence, innovation and research."
"One of the game-changers for children with cancer is immunotherapy. We’re looking at drugs as well as methods to turn on the immune system to fight the cancer within the body, like monoclonal antibody treatments and reprogramming immune cells. We’re seeing novel results, and there’s still a lot to discover.
"I treat neuroblastomas, these massive tumors that can grow inside children and spread to organs and bone. Removing them can take 10 to 16 hours in surgery. Often, we find we can clear cancer from the abdomen, but it comes back to the bone marrow and other areas we cannot control. We’re working to develop treatments to prevent that spread, seeking new pathways and new drugs that will prevent cancer recurrence. As surgeons, we try to leverage surgical techniques to create models that will really mimic the human condition."
"When we do research in pediatrics, there are several factors to consider, and we often must study the impact to the entire family. We feel strongly that we need uniquely pediatric research.
"We say all the time that kids are not just little adults, and so the science must be specific to them. Guerin Children’s is well-positioned because we have access to all the core resources of Cedars-Sinai: We have a Regenerative Medicine Institute, Computational Biomedicine, biostatistical support, biobanks, genomic support—all the advanced, high-level tools we need, and an opportunity to use them to focus on the needs of kids.
"We’re building on the Cedars-Sinai model of care, and that is premised on excellence, innovation and research."