Leading-edge research at UCLA Health is changing children's lives
Dr. Sherin Devaskar, MD, executive chair of the department of pediatrics at UCLA, loves coming to work every day because she’s endlessly inspired by her colleagues.
“They bridge what happens in science to medicine, with a focus on disorders that affect the most vulnerable of our population, and that’s the children. They don’t have a voice, right?” Dr. Devaskar says. “And these people are so passionately dedicated, they become their voice, in a way, and try to bring cures to the bedside.”
The UCLA Children’s Discovery and Innovation Institute is the research home of the pediatrics department. Funded through state and national grants, these physician-scientists are harnessing creativity and groundbreaking science to remedy some of the most challenging health conditions youngsters face.
Here’s a look at some of their work:
Studying COVID-19’s effects on pregnant women and their newborns: Since early in the COVID-19 pandemic, Karin Nielsen, MD, has been studying how the coronavirus affects pregnant women and the developing fetus. She and her colleagues have found that COVID-19 does not cross the placenta to infect the fetus, but concerns remain about how inflammation in the mother caused by the virus could affect the child.
Early findings show that babies born to mothers who were severely ill with COVID-19 develop unique inflammatory markers, Dr. Nielsen says.
“There was no overlap between the markers babies had and markers moms had, but in both moms and babies, the cytokine profile was significantly altered,” she says. “We want to follow these babies over time to see what their neurodevelopment will be.”
Previous studies indicate that increased inflammation can be a trigger for the development of disorders on the autism spectrum, Dr. Nielsen says. Early study results, however, are “reassuring,” she says. Many of the children have completed their six- and 12-month follow-up visits.
”Most babies seem to be doing OK,” Dr. Nielsen says. “We’re especially concerned and focused on the ones who had the most abnormal blood findings associated with inflammation and who were born to mothers with severe disease. It seems like the ones whose mothers had mild to moderate disease have immune inflammatory patterns that are very similar to people who haven’t had COVID.”
Dr. Nielsen previously studied the effects of Zika virus on pregnant women and their babies.
Developing potential cures for rare immune disorders: Immunologist Caroline Kuo, MD, has been interested in the power of stem cells and gene editing to treat rare diseases since her medical training. “For a lot of these diseases, only a bone marrow transplant is curative, but you have to have a match,” says Dr. Kuo. “Not everyone has a matched donor, and even those with a donor are often very sick, making a traditional bone marrow transplant risky. I wanted to see if there was anything else that could be done to help these individuals.”
So Dr. Kuo began to research how gene editing might play a role in treating patients with a rare immune deficiency called X-linked hyper IgM syndrome. The condition, which occurs almost exclusively in boys, makes it hard for the body to fight infections. Though X-linked hyper IgM is caused by a genetic mutation, it hasn’t been successfully treated by traditional gene therapy — the process by which genetic material is inserted into an individual’s own bone marrow cells to give rise to a healthy immune system. In this case, it caused genetic overactivity.
Dr. Kuo used the Nobel Prize-winning CRISPR-Cas-9 gene-editing technology to “take the corrective gene and put it exactly where it’s supposed to be,” so the sequence of genes around it prevent it from overacting. She has demonstrated that editing this gene in bone marrow stem cells appears to regulate its expression in laboratory settings. The next step is to translate these discoveries into an early-phase clinical trial in hopes of curing X-linked hyper IgM syndrome.
Expanding the boundaries of clinical care: Treating family finances isn’t traditionally part of a regular health care visit, despite decades of research showing that poverty and the factors that create it strongly influence health outcomes. A chance conversation between Adam Schickedanz, MD, PhD, and a financial coach serving low-income families led Dr. Schickedanz to consider how to help parents in pediatric clinics address the economic challenges that directly harm their health and the health of their children.
Since 2018, Dr. Schickedanz has been studying how pairing low-income families with financial resilience coaching services through a Medical-Financial Partnership leads to improved health outcomes for parents and children, as well as improving their financial realities.
Not only are parents who receive financial coaching able to build financial stability and assets, Dr. Schickedanz’s studies find these parents report reduced symptoms of anxiety and depression. They return for clinical visits more often, he says, and there are early signs that this intervention is reducing the risk of developmental delays in children.
“It’s really about reshaping and transforming what the clinical encounter can encompass,” he says, “to address poverty-related stresses as a way to improve health outcomes.”
Originally serving families at the Harbor-UCLA Primary Care Pediatrics Clinic, the financial-coaching program has expanded to include families at Olive View-UCLA Medical Center. Dr. Schickedanz and Dr. Monique Holguin, co-director of the Medical-Financial Partnership, are seeking resources to expand to additional clinics in 2022.