By: Ada Chong
October 2022
20 years ago, Miriam Vos, MD, MSPH, started studying non-alcoholic fatty liver disease (NAFLD) when it was rare in children. It’s now the most common liver disease in children across the world. NAFLD is a condition where fat is deposited in the liver and can cause damage to the liver over time. Researchers have come a long way when it comes to screening and identifying this disease, however a cure remains elusive.
Dr. Vos’ research group focuses on the clinical needs for NAFLD. She says they are working to learn why children get fatty livers although more components of that is becoming clear. Her work focuses on better understanding of the disease and what drives the severity of the disease.
One study Dr. Vos is working on focuses on the prevention of this disease. This study is funded by a NIH R01 and Dr. Jean Welsh is the co-PI. Dr. Vos and her team are trying to understand what drives children to onset of this disease and whether they can prevent it from happening. In previous studies, children with NAFLD are often diagnosed during puberty because puberty is the time where the body changes from a child to an adult and there’s increasing deposition of fat. Healthy weight children become mildly insulin resistant during this period, while overweight children can have dramatic increases in insulin resistance. Dr. Vos and her team suspect these normal physiological drivers are fueling the onset of NAFLD. The prevention study enrolls and tests children who are pre-pubertal and have increased risk in developing this disease. The study enrolls children ages 6-9 from Hispanic backgrounds who don’t yet have NAFLD and randomizes them to either a control group or put on a low sugar diet. The children in the study get blood tests so researchers can look at their livers and other markers. The study follows the children for two years and during this time, they continue to get MRIs and blood work done. At the end of the study, Dr. Vos hopes to have a good understanding of the role sugar has in the onset of NAFLD. The top goals for this study are to understand if a reduction in sugar can improve physiology and reduce liver damage and fat in the liver over time, and to evaluate if other metabolic features such as insulin sensitivity and weight will improve from a low sugar diet over the two years.
Another NIH R01 study led by Dr. Vos is focused on improving diagnostic testing for NAFLD. Dr. Vos and her team use metabolomics to explore better biomarkers that can be translated into better diagnostic tests. Dr. Vos says it can be time consuming and expensive to get a diagnosis from a MRI or liver biopsy compared to getting a simple blood test. This study uses metabolomics to look at thousands of metabolites in the blood in children with and without NAFLD. Researchers hope it will help them identify a panel of metabolites that are more specific for NAFLD so a simple blood test can be used to get a diagnosis. There are preliminary results from this study and Dr. Vos hopes to publish in the next several months.
Dr. Vos was inspired to do this research because of the challenges she sees children and families face in her clinic. NAFLD is difficult to reverse and the diagnosis can be a complicated pathway. She wants to help children avoid the disease because treatment studies haven’t been effective enough to reverse or cure NAFLD so far.
Dr. Vos is also the Program Director for the pediatric component of the Georgia Clinical and Translational Science Alliance (Georgia CTSA). The GA CTSA supports clinical and translation research across universities and supports the Pediatric Research Unit at Children’s Healthcare of Atlanta. Researchers can leverage the CTSA for resources to increase efficiency and productivity of research and are welcome to reach out to Dr. Vos to learn more.