Pediatric Hematology and Oncology Research
The Division of Pediatric Hematology and Oncology is nationally distinguished for its design and conduct of NIH-funded multicenter clinical trials involving childhood cancer and blood disease.
Faculty are conducting molecular and cellular biology experiments in cancer and blood disease. Laboratory research efforts are both basic and translational studies that help to bridge the lab and clinical venues. Research is carried out in laboratories in the Division of Hematology/Oncology and also across the entire UT Southwestern Medical Center campus, including the NCI-designated Simmons Cancer Center and the Children's Medical Center Research Institute at UT Southwestern.
Active areas of basic research include:
- Using fruit fly and zebrafish models to understand the genetic defects causing rhabdomyosarcoma, Ewing sarcoma, and malignant germ cell tumor
- Using complementary pre-clinical models to dissect the key “vulnerabilities” in rhabdomyosarcoma, Ewing sarcoma, and other soft tissue sarcomas
- Understanding the molecular machinery by which normal cells can undergo “senescence” as a tumor suppressor mechanism in the presence of a cancer-causing oncogene
- Identifying novel proteins that can be “targeted” as novel therapies in childhood cancer
- Understanding how certain cancer-causing mutations influence the metabolism in childhood brain tumors and certain types of sarcoma
- Uncovering how hematopoietic and embryonic stem cells are controlled and how these control mechanisms can go awry in cancer and blood disease
- Elucidating the molecular machinery that guides erythrocyte development
- Using novel model systems to elucidate the host and bacterial factors that cause invasive bacterial and fungal infections
Physicians in the Division are engaged in a wide range of clinical research efforts spanning the cancer and blood disease programs. Clinical research efforts are supported by robust infrastructure provided by the Clinical Research Office (CRO) within the Gill Center and the Simmons Comprehensive Cancer Center at UT Southwestern, the only NCI-designated cancer center in North Texas. At any point, 75 to 100 oncology trials and 20 to 30 hematology trials are open for enrollment for Gill Center patients.
Active areas of clinical research include:
- Prospective clinical trials for children with cancer, conducted under the umbrella of the NCI-sponsored Children’s Oncology Group
- Prospective, early-phase clinical trials for children with hematological malignancies, conducted as part of the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) consortium and other academic and industry partners
- Prospective therapeutic trials for children with sickle cell disease, iron deficiency anemia, and hemophilia
- Investigator-initiated and industry-sponsored therapeutic studies of children with cancer and blood disease
- Retrospective research studies investigating molecular and clinical factors influencing late effects in childhood cancer survivors
- Prospective and retrospective studies assessing a variety of quality measures of children with chronic hematology disorders
- Early phase clinical trials of immunotherapeutics for childhood cancer, including the use of CAR T-cells for childhood leukemia
- Prospective and translational research trials in children with venous thrombosis
Clinical and laboratory research efforts are funded by a wide variety of national, regional and local organizations, such as the National Cancer Institute, the National Heart, Lung, and Blood Institute, National Eye Institute, Cancer Research and Protection Institute of Texas, American Cancer Society, St. Baldrick’s Foundation, Children’s Cancer Fund of Dallas, Children’s Medical Center Foundation, Wipe-Out Kids’ Cancer, the 1 Million for Anna Foundation, and Hyundai Hope on Wheels Foundation.
Research In The News
Drug targeting mutant cancer gene is highly effective, durable
February 21, 2018 - A drug targeting a gene fusion that occurs in lung, colon, and other cancers was effective in 75 percent of patients of all ages in clinical trials, and an even higher percentage of pediatric patients, researchers at UT Southwestern’s Simmons Cancer Center announced. The drug is being given fast-track consideration by the Food and Drug Administration.
The drug, larotrectinib, targets TRK-fusion, which can occur in lung, colon, thyroid, and many other types of cancer, said Dr. Ted Laetsch of UT Southwestern’s Department of Pediatrics and the Harold C. Simmons Comprehensive Cancer Center. Dr. Laetsch led the pediatric arm of the clinical trial for the study, which appears in the New England Journal of Medicine. Read more
CART-T clinical trial enrolling multiple myeloma patients
February 14, 2018 - UT Southwestern Medical Center is one of nine exclusive sites in the country enrolling multiple myeloma patients for a clinical trial of the CAR-T “living drug” therapy for cancer. CAR-T therapy (chimeric antigen receptor T-cell therapy) is an innovative immunotherapy that uses a re-engineered version of the patient’s own immune cells to fight cancer. A UT Southwestern cancer researcher who was among the first patients to be treated, has now been cancer-free for two years.
As one of just 49 in the country designated a Comprehensive Cancer Center by the National Cancer Institute and the only one in North Texas, UT Southwestern’s Harold C. Simmons Comprehensive Cancer Center is offering both treatments and clinical trials in several critical areas:
- Dr. Ted Laetsch, Assistant Professor of Pediatrics, led the only clinical trial site in the Southwest for a CAR-T treatment for acute lymphoblastic leukemia (ALL). Results from the trial resulted in FDA approval for the first CAR-T therapy, and Dr. Laetsch is now treating ALL patients who are 25 and under with this CAR-T therapy at the Pauline Allen Gill Center for Cancer and Blood Disorders at Children's Health.
- In November, the FDA approved a CAR-T treatment for lymphoma, and UT Southwestern physicians soon will be offering this treatment to patients through the Bone Marrow Transplantation/Hematologic Malignancies Clinic at the Simmons Cancer Center.
- As new uses for CAR-T continue to be explored, leadership and guidance on how and when to use these treatments also is needed. Dr. Ankit Kansagra, Assistant Professor of Internal Medicine, is co-leader of the Global CAR-T Initiative, a group of physicians who are meeting to draw up guidelines for CAR-T use. Read more
Simmons Cancer Center researchers part of historic CAR-T breakthrough
February 1, 2018 - A historic study involving researchers from UT Southwestern’s Harold C. Simmons Comprehensive Cancer Center demonstrates the effectiveness of CAR-T therapy, which uses genetically modified immune cells to treat acute lymphoblastic leukemia (ALL) in children and young adults. The research appears in the New England Journal of Medicine. Dr. Ted Laetsch served as the lead investigator for the only clinical trial site in the Southwest for the CAR-T trial. Read more
Good-guy bacteria may help cancer immunotherapies do their job
October 5, 2017 - Individuals with certain types of bacteria in their gut may be more likely to respond well to cancer immunotherapy, researchers at the Harold C. Simmons Comprehensive Cancer Center found in a study of patients with metastatic melanoma. Senior author Dr. Andrew Koh, Associate Professor of Pediatrics and Microbiology with the Simmons Cancer Center, found that the bacteria was able to prime the immune system to attack cancer cells and kill them. Read more
A therapy for leukemia that turns the human body into a ‘living drug’ awaits FDA approval
July 19, 2017 - A Food and Drug Administration advisory panel ushered in a new era for cancer treatment last week. The panel unanimously recommended the approval of the first treatment to fight acute lymphoblastic leukemia, a common blood cancer in children – with a patient’s own cells. For the past few years, researchers from around the world have been running clinical trials, where they genetically alter people’s T-cells, their immune cells, to attack and shut down leukemia in the body. UT Southwestern Medical Center was one of 13 research sites in the U.S. – in fact, one of the biggest. Dr. Theodore Laetsch is a pediatric oncologist at UT Southwestern Medical Center and Children’s Health in Dallas. He was also one of the study’s lead investigators. Laetsch says this first-ever gene cancer therapy could be formally approved as early as this year. Read more about this study and listen to Dr. Laetsch's interview on KERA.