HJBR Jul/Aug 2022

50 JUL / AUG 2022 I  HEALTHCARE JOURNAL OF BATON ROUGE CHILDREN’S HEALTH COLUMN CHILDREN’S HEALTH AT Children’s Hospital New Orleans, there were 90 clinical trials focused on cancer in 2021 alone. Clinical trials teams are backed by a comprehensive research team that are led by physicians and include nurses, pharmacists, psychologists. Pediatric oncology trials encompass a wide spectrum, from trials that test new drugs and drug combinations to learning how the psychological effects of cancer may impact cancer outcomes. Today, the Children’s Oncology Group (COG), a Na- tional Cancer Institute supported coop- erative clinical trials group, is the world’s largest organization devoted exclusively to childhood and adolescent cancer research. COG unites more than 10,000 experts in childhood cancer at more than 200 leading children’s hospitals. Research through the Children’s Oncology Group (COG) encom- passes most of the clinical development of new therapeutics for children and adoles- cents with cancer. One trial that may dramatically shift the treatment of childhood leukemia away from conventional chemotherapy drugs is called chimeric antigen receptor T-cell therapy (CAR T-cell therapy), which was first approved to treat certain types of leu- kemia and lymphoma in 2017 but is avail- able in only some children’s hospitals. The beauty of CAR T-cell therapy is that Pediatric Clinical Trials Enable Groundbreaking Cancer Research it uses immune cells that come directly from the patient’s own blood and are “en- gineered”to recognize cancer cells. This al- lows for the body’s own immune system to find and destroy the cancer cells. In prac- tice, the patient undergoes a peripheral stem cell collection to collect white blood cells called T lymphocytes (T cells) that normally help protect the body from infec- tion. The blood, which is full of the immune system’s T cells, is shipped to a lab where the T cells are transformed into the can- cer-killing CAR T cells by inserting a spe- cialized marker that then makes a protein that can adhere to the cancer cell. The CAR T cells then mature and multi- ply in the lab for several weeks before an army of millions of CAR T cells are infused back into the patient in a process like a blood transfusion. The CAR T cells bind to and destroy cancer cells since they now have gained the ability to recognize them. When we treat a patient with CAR T-cell therapy, it should eventually enhance the immune system’s ability to fight and kill cancer cells. This makes it possible to kill cancer cells without potentially toxic che- motherapy. In 2021, Children’s Hospital New Orleans became the first hospital in Louisiana to be approved by Novartis, the pharmaceutical corporation that manu- factures the CAR T cells, called Kymriah, or tisagenlecleucel. Kymriah is one of six approved types of CAR T cells and is used primarily to fight B-cell acute lympho- blastic leukemia (ALL), the most common type of cancer in children, and diffuse large B-cell lymphoma (DLBCL), another cancer that can affect children. One of the many positive aspects of using CAR T cells is that the side effects are much less severe compared to those that accompany chemotherapy because the treatment involves the patient’s own cells. CAR T cells have been shown to cure cancer in relapsed or refractory disease patients through previous clinical trials at specialized centers across the country. In addition to groundbreaking cancer treatments, clinical trials encompass the full continuum of care, including pain management, decreasing visits to the hos- pital, and even improving outcomes by helping kids undergoing cancer treatment to express their emotions. The SPARK Study, for example, features a symptom screening app to report symptoms that are side effects of treatment like chemothera- py or radiation. Within the app, patients or their parents can report any symptoms in real time and log how the therapy is mak- ing them feel. If the patient logs a symp- tom as “very bothersome” or “painful,” the child’s doctor will receive a notification To remain at the cutting edge of medicine, a strong clinical trials program is imperative. This is especially true in the field of pediatric hematology oncology. The treatments that many clinical trials provide are often lifesaving and innovative therapies for the patients who receive them.

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