Updates in Pediatric Cancer: What’s New?

Reading time: 3 minutes

Susan Egbert

Pediatric cancer is a broad term that refers to cancers originating in children younger than 18. There are different types of pediatric cancer, but they all fall into one of two categories: solid tumors and leukemia. Solid tumors are cancers arising from cells in the body’s tissues. These tumors can be benign (noncancerous) or malignant (cancerous). Malignant solid tumors can be locally advanced (meaning they have spread to only one part of the body) or metastatic (meaning they have spread to other body parts). Leukemia is cancer originating in the white blood cells (leukocytes). It is the most common cancer in children and the fourth most common cancer in adults. There are many different types of pediatric cancer, and treatments vary depending on the type of cancer and the stage of cancer. Some types of pediatric cancer can be cured with surgery, while others may require chemotherapy or radiation therapy.

Figure 1. Simplified diagram of how immunotherapy is supposed to work.

When surgery is not possible, new therapy options are being explored, as traditional chemotherapy and radiation cause harsh side effects that deter patients from wanting to continue therapy. This article will highlight some of the progress made for new therapeutic agents in pediatric patients. Immunotherapy (Figure 1) involves using the body’s own immune system to fight cancer, and it has shown great promise as a treatment for solid tumors in pediatric patients. One particularly exciting approach to immunotherapy is CAR (chimeric antigen receptor)-T cell therapy, which involves genetically engineering immune cells to recognize and attack cancer cells (as seen in Figure 2). While this therapy has shown remarkable success in some cases, it is important to closely monitor patients for potential side effects, such as being more prone to infections and cytokine release syndrome which is fatal if not treated in a timely manner. Currently, there is an FDA-approved CAR-T cell drug for pediatric patients called KYMRIAH (tisagenlecleucel).

Figure 2. CAR-T cell therapy and how it works1

Another promising new approach to immunotherapy is using checkpoint inhibitors. Checkpoint inhibitors are a type of immunotherapy that works by blocking the signals that prevent the immune system from attacking cancer cells. Normally, cancer cells can evade the immune system by producing signals that trick immune cells into thinking they are not harmful. Checkpoint inhibitors prevent these signals from being received, allowing the immune system to recognize and attack cancer cells more effectively. Checkpoint inhibition is currently being explored in solid tumors. By using checkpoint inhibitors to boost the immune response against solid tumors, it may be possible to improve treatment outcomes for pediatric patients with these types of cancers. Early research has shown promising results, with some pediatric patients experiencing significant tumor shrinkage and improved quality of life. Clinical trials are currently underway to evaluate the safety and effectiveness of these treatments in a range of pediatric cancers. So, we can expect to see these as therapy options for pediatric patients in a few years.

Figure 3. How checkpoint inhibitors work2

In conclusion, immunotherapy offers hope for improved outcomes and quality of life for pediatric patients with solid tumors. CAR-T cell therapy, in particular, has shown remarkable success in some cases, with complete remission being achieved in many pediatric patients with blood cancers. For checkpoint inhibitors, although there is no FDA approved therapy available, we can expect to see some approvals coming in the upcoming years. While more research is needed to fully understand the potential of these new immunotherapy approaches for solid tumors, the ongoing development of new treatments offers hope for children and their families who are affected by cancer. As research continues to advance and new treatments are developed, there is reason to be optimistic about the future of cancer treatment and the potential for improved outcomes for pediatric patients.

Edited by Megan Majocha


  1. Kavanagh, D. (2019) Ethical and Biosafety Oversight of New CAR T-Cell Products, https://www.genengnews.com/insights/ethical-and-biosafety-oversight-of-new-car-t-cell-products/. Genetic Engineering and Biotechnology News. 
  2. 1. National Cancer Institute. Immune Checkpoint Inhibitors. National Cancer Institute. Published April 7, 2022. https://www.cancer.gov/about-cancer/treatment/types/immunotherapy/checkpoint-inhibitors

Image Credits: https://www.pexels.com/photo/a-gold-childhood-cancer-awareness-ribbon-7723557/

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