Director's Message

Announcing Exciting New Funding
09/20/2017

Today, news of a relapse of T-ALL is made worse when families learn that no effective or targeted therapies are left to try. John DiPersio, MD, PhD, oncology; Robert Fulton of the McDonnell Genome Institute; and Shalini Shenoy, MD, pediatrics; want to change that conversation to one of hope by advancing the science of immunotherapy to treat T-cell leukemia.

T cells are the immune system’s most powerful tool for attacking cancer cells. In fact, when genetically engineered to produce chimeric antigen receptors (CARs) -- special proteins that help them recognize tumor cells -- T cells have been used with success to treat B-cell ALL, another type of lymphoblastic leukemia. So far, however, scientists have not found a successful way to train CAR-T cells to distinguish between normal T cells and malignant ones. The CDI researchers propose using gene editing to optimize the CAR-T cells so they limit their attack to cancer cells. The scientists also are evaluating strategies to avoid over-activating the immune response induced by the CAR-T cells, which can result in life-threatening complications that can hamper their use.

In the second project, neonatologist Cynthia Ortinau, MD, pediatrics, will use her new Faculty Scholar award to pursue a highly innovative approach, MRI scanning in-utero, to understand why fetuses with congenital heart defects (CHDs) often have developmental delays. While advances in surgical and medical care have improved survival in moderate and severe CHD, impaired brain development can affect nearly half of these children. Dr. Ortinau theorizes that the underlying CHD could influence blood flow and oxygen delivery as these babies’ brains develop. She plans to test that hypothesis by comparing measures of cerebral cortical development and cerebral blood flow in third-trimester fetuses with and without moderate to severe CHD. Understanding the underlying mechanisms of impaired fetal brain development in children with congenital heart defects could inform prenatal counseling and neuro-protective strategies.

Expect to hear more about these studies in the months ahead as they, and all the CDI high-risk, high-reward studies, lay the groundwork for extramural funding by the NIH and other sources and ultimately transform child health one discovery at a time.

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