Translating Discoveries in the Lab to Improve Clinical Care for Children
Lurie Children’s is a research-intensive pediatric academic medical center, and research into the many diseases that affect children is a critical part of our mission to achieve optimal child health outcomes. Before becoming CEO, I led the Stanley Manne Children’s Research Institute at Lurie Children’s, and I know first-hand how passionate our researchers are in their pursuit of discoveries that will transform care and health outcomes for children.
But how does a discovery in the laboratory ultimately progress to the clinic to directly impact a child’s life? This occurs by translational research driven by collaborative partnerships that enable a bridge connecting basic science and clinical care. For example, scientists might discover a molecular mechanism that promotes brain tumor growth. In order to develop a drug that targets that mechanism, they need to test a promising agent in various preclinical models of pediatric brain tumor. Once they determine it stops tumor growth and appears to be safe, the drug can proceed to clinical trials in people. These intermediate steps are how scientific discoveries get “translated” to practical clinical applications.
Lurie Children’s investment in translational research is driven by our commitment to advance pediatric medicine. Last year, we launched the innovative Advanced Center for Translational and Genetic Medicine (ACT-GeM) to bring together researchers from various disciplines to improve our understanding of rare genetic diseases in children. They are using cutting-edge biological and bioinformatic tools to identify and interpret human genetic variation, discover their potential to cause diseases, as well as develop and test new therapies in experiments that model the consequence of the gene variants. The ultimate goal of ACT-GeM is to develop new treatments and convert what are now deadly genetic diseases into manageable conditions or ideally, cures.
Translational research comprises one of the key scientific pillars at the Manne Research Institute. The other pillars include basic science, clinical trials and community-oriented research, so our investigations span the entire spectrum of pediatric research necessary to translate discoveries from the bench ultimately into the community. Each day Lurie Children’s researchers engage in this work because every child deserves the promise of a cure and the promise of a healthier future. Together, we are determined to deliver on these promises.
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Tom Shanley, MD