Childhood Cancer Awareness Month: How Immunotherapy is Helping Kids and Teens Tackle Cancer

In 2012, former President Barack Obama designated September as Childhood Cancer Awareness Month, drawing attention to the phenomenon of cancer in children and adolescents. Contextually, a child is defined as someone under 18, and an adolescent is between 10-19 years old. Globally, every two minutes, a child or adolescent is diagnosed with cancer.

What drives my work and that of most pediatric oncologists is the potential to harness technological advancements to develop new drugs that can turn the incurable into something treatable.”

Robbie Majzner, MD, CRI Lloyd J. Old STAR

The most common cancers for children under 15 include acute lymphocytic leukemia, brain cancer, neuroblastoma, and non-Hodgkin lymphoma. Among adolescents between the ages of 15 and 19, the most common cancers are brain cancer, Hodgkin lymphoma, thyroid carcinoma, and testicular germ cell tumors. Immunotherapy has shown progress for certain childhood cancers while avoiding many of the long-term side effects that can be commonly found in more conventional treatments.

Recent Progress: Chimeric Antigen Receptor (CAR) T Cell Therapy is Helping More Children and Adolescents 

CAR T cell therapy, an immunotherapy that re-engineers T cells to target and kill cancer cells, has treated over 20,000 patients worldwide. Robbie Majzner, MD, director of the Pediatric and Young Adult Cancer Cell Therapy Program at the Dana-Farber Cancer Institute/Boston Children’s Hospital, principal investigator with the Majzner Lab at the Dana-Farber Cancer Institute, and CRI Lloyd J. Old STAR, says there have been several recent CAR T cell therapy breakthroughs in childhood cancers. One promising development occurred early this month, in September 2024. 

“Investigators in Singapore and Italy reported very impressive results using CAR T cells against T cell leukemia in children by targeting a protein on the leukemia cells called CD7,” Dr. Majzner explained in recent conversation with CRI. The CD7 protein plays a fundamental role in T cell interactions and in T cell/B cell interaction early in lymphoid development. He said historically, CAR T cells had been successful with B cell leukemias, but not for T cell derived leukemia. “These investigators came up with an elegant method to hide the target on the CAR T cells, making it possible to use CAR T cells for T cell leukemia. The results were outstanding.” 

A Historical Breakthrough Continues to Shape Childhood Cancer Immunotherapy 

Immunotherapy treatments, such as stem cell transplants, where healthy stem cells are used to replace damaged or destroyed ones, have been instrumental in helping nearly 80 percent of children survive their cancers. Over the years, CRI-funded scientists have made important contributions to fighting childhood cancer. Their research includes finding ways to perform effective stem cell transplants without using chemotherapy. They have also developed new ideas for immunotherapy treatments for children and adolescents. 

Carl June, MD, the Richard W. Vague Professor in Immunotherapy in the Department of Pathology and Laboratory Medicine, director of the Center for Cellular Immunotherapies at the Perelman School of Medicine, director of the Parker Institute for Cancer Immunotherapy at the University of Pennsylvania, and CRI Scientific Advisory Council member, led clinical trials that have successfully treated young cancer patients. Dr. June treated Emily Whitehead, a five-year old who was suffering from acute lymphoblastic leukemia, with CAR T cell therapy.  

Emily became the first child ever to be treated with CAR T cell therapy and was discharged from Penn Medicine at seven years old. According to The Daily Pennsylvanian, Emily is currently enrolled at the University of Pennsylvania as a college sophomore. She has been living without leukemia – the most common childhood cancer – since 2012. 

The Path Forward 

Dr. Majzner elaborated a critical difference between adult and childhood cancers as the former are driven by traceable environmental factors, such as exposure to UV rays or cigarette smoke. Childhood and adolescent cancer most often develop due to hereditary factors but can also occur because of a problem encountered in a typical environment — this makes the malignancy biologically different. For example, checkpoint inhibitor therapies that have revolutionized immunotherapy treatments in adults do not work for most childhood cancers.  

However, CAR T cell therapy has shown promise for childhood cancers, and the next generation of this immunotherapy treatment, that utilize novel techniques of cell engineering and genome editing, will continue to help academic physician-scientists develop future treatments for childhood cancers. Masakazu Kamata, PhD, associate professor of microbiology at the University of Alabama at Birmingham and CRI Technology Impact Award Grantee, agreed that immunotherapy for childhood cancer is a nuanced subject. 

“While CAR T cell therapy has demonstrated notable success in B cell acute lymphoblastic leukemia, its application in solid tumors poses considerable challenges,” Dr. Kamata explained in conversation with CRI. “There is no universally effective method for treating all childhood tumors.” 

Thanks to Drs. Mazjner, June, Kamata, and other pioneering CRI scientists who are dedicated to thwarting cancers in children and adolescents, we have a greater understanding of childhood cancers and are ever closer to creating a world immune to cancer.