Advancing Pediatric Cancer Research with the Jackson Laboratory Posted on September 11, 2023 By: Ching C. Lau, MD, PhD A personal mission of pediatric cancer research Ching Lau, Ph.D., M.D., never thought he would want to be a doctor. He remembers hating going to the doctors in his youth and finding the atmosphere unacceptable. Because of his early experiences, he set out to find a solution, which led to his clinical career. Lau joined The Jackson Laboratory (JAX) faculty in 2016 to advance his research on pediatric cancers. He also serves as the Scientific Director of the Center for Cancer and Blood Disorders at and the Head of the Division of Pediatric Hematology-Oncology in the Department of Pediatrics at the UConn School of Medicine. Lau’s research focuses on understanding brain and bone tumors, as well as researching and diagnosing different blood cancers in children. His team works to determine which cells the cancers originate from, the genetic aberrations driving the disease and the treatments that will give patients the best chance to not only survive, but to thrive. He believes there is a way to fight childhood cancer without taking away patients’ normal lives after they survive their cancer. Many treatments are effective in killing the cancerous cells, but also harm healthy cells too. This affects not only the children physically but also their families. “You are dropping an atomic bomb on an anthill. You wipe out the anthill, but the price you pay is pretty steep,” says Lau. “You have a lot of collateral damage to the normal tissues in the body.” Advancing pediatric cancer practices Clinicians use such aggressive practices in treating cancer because they aren’t able to define normal and cancer cells more precisely. Lau and his team of researchers have been developing methods to find the fundamental differences between these cells. But how can cancer treatments be improved? Lau and his team intend to use their findings that distinguish normal versus cancerous cells to inform the development of personalized medicine that can specifically target cancerous cells and limit irreversible side effects. “We need to find the qualitative differences between normal and cancerous cells to contribute something to the improvement of the overall outcomes of children with cancer,” says Lau. At JAX, Lau uses various types of patient-derived xenograft (PDX) models to study the exact cancer he observes in the patients he sees at Connecticut Children’s. PDX models are created by introducing the patient’s tumor cells into a mouse engineered to accept foreign cells and tissues and, sometimes, to have a human-like immune system. He also works with cancer cell models in vitro (in cell cultures outside of a body). Both types of models are essential. Mouse models provide biological context, while cellular models yield an inexhaustible supply to study over time. The future of pediatric cancer research Lau believes immunotherapies hold much promise for the pediatric field. However, treatment is only effective if the diagnosis is accurate. Therefore, Lau is looking to improve the accuracy of his diagnoses. He has implemented several tools to help determine the characteristic profiles of each cancer he sees in the clinic. One tool proving to be an essential part of his diagnostic process is a test to profile DNA methylation of cancer cells, which regulates gene expression. The DNA methylation test results can therefore more accurately diagnose the specific subtype of cancer. The Lau lab has been working in conjunction with the Advanced Precision Medicine Laboratory to validate these tests which are currently available to Connecticut Children’s as well as other hospitals locally and around the country. “DNA methylation has proven to be a powerful diagnostic tool that we are now using routinely,” says Lau. “The Advanced Precision Medicine Laboratory at JAX has helped us in implementing this lifesaving test into the clinic. It has been a group effort. None of these projects can be accomplished alone.” For Lau, the end goal for his research is always to further develop and improve personalized pediatric treatments. “We are in a better position to design therapies that would only target the cells that have the genetic abnormalities.” Such therapies will make not just survival, but also a high quality of life more attainable for the children and families which Lau has been striving for since the beginning of his career and will continue to for as long as he can dream. Professor Ching Lau, Ph.D., M.D., envisions a world where his pediatric cancer patients receive targeted medicines with minimal side effects that give them the ability to dream big.