Siteman Kids is among the first U.S. pediatric centers to offer breakthrough cancer treatment that uses genetic engineering to harness a child’s immune system to fight cancer

ST. LOUIS, MO (December 5, 2017) – Siteman Kids at St. Louis Children’s Hospital is now one of only 19 pediatric centers in the U.S. offering a breakthrough cancer treatment approved by the Food and Drug Administration (FDA) just over two months ago. CAR-T immunotherapy harnesses a child’s own immune system to fight off cancer. The FDA approved the therapy in August 2017 as a defense against an aggressive form of acute lymphoblastic leukemia (ALL) in children who have not responded to standard therapies or whose cancer has relapsed.

A person’s immune system normally fights disease and infection but in cancer patients, specialized immune cells called T-cells lose the ability to recognize and attack cancer cells. With CAR-T therapy, a patient’s own T-cells are isolated from the blood. Those cells then are genetically altered – or supercharged – enabling them to home in on cancer cells and destroy them. During this process, specialized receptors called chimeric antigen receptors are put onto patients’ T-cells, thus the name CAR-T cells. When the immune cells are infused back into patients, these receptors allow the cells to recognize and attack tumor cells, thus turning the T-cells into cancer-fighting machines. These supercharged cells also stay alive and circulate in the patient’s body for years. 

Washington University pediatric oncologists with extensive experience in treating leukemia will administer the CAR-T immunotherapy through Siteman Kids at St. Louis Children’s Hospital.

“If it looks like the leukemia is not responding to treatment or relapsing, the CAR-T cells can attack it all over again using a different mechanism than chemotherapy,” explained Shalini Shenoy, MD, a Washington University Pediatric Hematologist, Oncologist, and Director of Siteman Kids at St. Louis Children’s Hospital. “We think this is just the beginning for this kind of therapy. If we can make a patient’s own cells smart enough in more cancers, and we think we can, we believe we’ll be able to tackle even more types of cancer with immunotherapies in the future.”   

Clinical trials have shown that in children with aggressive B-cell acute lymphoblastic leukemia who have not responded to standard therapies or have relapsed, CAR-T therapy has achieved more than an 80 percent remission rate. Some patients have remained in remission for more than five years. While the treatment is considered a major advance in cancer treatment, CAR-T therapy induces a heightened immune response that can lead to a range of side effects, some of which can be severe.

“Because of the wide range of potential side effects, it’s important that patients undergoing CAR-T therapy are watched closely by physicians and care teams with extensive experience in blood cancer therapy and bone marrow transplantation,” Shenoy continued. “Siteman Kids at St. Louis Children’s Hospital is equipped to manage the therapy, which is why our center is among the first to offer it.” 

Research into the potential of CAR-T therapy continues. The Children’s Discovery Institute (CDI) – a research partnership between St. Louis Children’s Hospital and Washington University School of Medicine – has given $360,000 for further investigation into these life-saving therapies.  Dr. Shenoy has joined John DiPersio, MD, PhD, the Virginia E. and Sam J. Golman Professor of Medicine in Oncology and deputy director of Siteman Cancer Center; Matthew Cooper, PhD, an instructor in medicine; and Robert Fulton, an assistant professor of genetics and a scientist at McDonnell Genome Institute, all at the School of Medicine, in this effort. The medical team will use the CDI funding to develop an “off-the-shelf” CAR-T therapy that prevents CAR-T cells from attacking each other or non-cancer cells in the patient; strategies to overcome a life-threatening side effect of CAR-T therapy called cytokine release syndrome (CRS); and a novel “suicide gene” that will track CAR-T cells in the body using a unique form of positron emission tomography (PET) scanning and eliminate those cells, if needed.

Families may call 314.454.KIDS (5437) to request an appointment. Physicians can refer a patient through the Children’s Direct Access Line at 1-800-678-HELP (4357)


St. Louis Children’s Hospital has provided specialized care for children for more than 130 years. US News & World Report ranks St. Louis Children’s among the best pediatric hospitals in the nation. In 2015 the hospital again received the Magnet designation from the American Nurses Credentialing Center, the nation’s highest honor for nursing excellence. St. Louis Children’s Hospital is affiliated with Washington University School of Medicine, one of the leading medical research, teaching and patient care institutions in the nation. The hospital is a member of BJC HealthCare. For more information, visit, or find us on Facebook and @STLChildrens on Twitter.


Siteman Kids at St. Louis Children’s Hospital treats children and young adults with cancer through cutting-edge technology, clinical trials, specialty physician care and highly trained staff in a compassionate and family-centered environment. Bringing together the expertise of St. Louis Children’s Hospital, Siteman Cancer Center and Washington University Physicians, Siteman Kids at St. Louis Children’s Hospital provides the most advanced medical treatments and innovation for kids. U.S. News & World Report ranks St. Louis Children’s Hospital among the best pediatric cancer hospitals in the nation. Children’s is among only 7% of the nation’s hospitals with Magnet designation, the highest honor for nursing excellence. For more information, visit, or find us on Facebook and @STLChildrens on Twitter.


Siteman Cancer Center, ranked among the top cancer treatment centers by U.S. News & World Report, also is one of only a few cancer centers to receive the highest rating of the National Cancer Institute (NCI) – “exceptional.” Comprising the cancer research, prevention and treatment programs of Barnes-Jewish Hospital and Washington University School of Medicine in St. Louis, Siteman treats adults at five locations and partners with St. Louis Children’s Hospital in the treatment of pediatric patients. Siteman is Missouri’s only NCI-designated Comprehensive Cancer Center and the state’s only member of the National Comprehensive Cancer Network. Through the Siteman Cancer Network, Siteman Cancer Center works with regional medical centers to improve the health and well-being of people and communities by expanding access to cancer prevention and control strategies, clinical studies and genomic and genetic testing, all aimed at reducing the burden of cancer.