Honoring Childhood Cancer Awareness Month: The Legacy of Innovative Treatments

The wisest person I ever met was eight years old in the pediatric oncology wing of the Children’s Hospital. It was not her first time there. She knew the halls better than I did, as a resident doing my rotations. She was fortunate, I guess, in that she kept her black hair through the series of treatments. I’m sure she would have traded it for a treatment that worked. She knew she would not be around long in this world and had come to accept it. She had been through enough ups and downs to have gone through the stages of grief that her parents still couldn’t manage. I wasn’t managing so well either when I met her. She was feeling pretty bad when I came to try to help her pain and nausea, but she looked at me with eyes as deep as her soul. Those very tired eyes melted my professional facade and plans for a quick follow-up before dinner. We shared honesty and more than a few tears. She wasn’t sad for herself but sad that she couldn’t do any more to help her parents. When I came back the next day, she was gone.

The Impact of Pioneering Research

Those were the days when we didn’t have many choices for hematology/oncology treatments for pediatric patients. Thankfully, treatments have advanced so much in the past 30 years. September is Childhood Cancer Awareness Month, and it is a time to remember children who died from childhood cancer, families who are still struggling with the aftermath, and those who are in the middle of the fight. It is also a reminder to clinical research stakeholders that we’ve worked hard, and we still have a lot of work to do against childhood cancer, which is the leading cause of disease death in children.¹

Over the past 50 years, treatments and therapies for pediatric hematology/oncology have undergone significant advancements, transforming the prognosis and quality of life for children with cancer. In the mid-1970s, 58% of children (ages 0 to 14 years) diagnosed with cancer survived at least five years.² However, with the introduction of new therapies and treatments, the overall survival rate has increased to over 80% today.² Moreover, targeted therapies, immunotherapies, and gene therapies have emerged as promising treatments for specific types of childhood cancers. For example, CAR-T cell therapy has shown remarkable efficacy in treating certain types of leukemia and lymphoma, offering new hope to families and children affected by these devastating diseases.

The development and implementation of these groundbreaking treatments would not have been possible without the rigorous research and clinical trials that have taken place over the past several decades. Institutional Review Boards (IRBs) have played a crucial role in these advancements, ensuring that all research studies involving human subjects, particularly vulnerable populations like children, are conducted ethically. By overseeing the conduct of clinical trials, IRBs have supported researchers in pursuit of innovative treatments and therapies, which have significantly improved the outcomes for children with cancer.

Conclusion

In August 2024, the White House put out a proclamation, listing both obstacles and efforts to address childhood cancer.³ There is no better time than now to reenergize our work on childhood cancer. Basic genetic profiles, epigenetic driver discovery, and single-cell RNA sequencing have uncovered potential new avenues for research.⁴ We have encouraging success stories from the recent advances in pediatric cancer treatment such as CAR-T with Emily Whitehead and immunotherapy with Isabella Snow Frasier. ^{5, 6} As an industry, we’ve come a long way since my residency days, with so much more to do. By working together, we can help families have real hope in the face of a devastating diagnosis.

References

1. Siegel RL, Giaquinto AN, Jemal A. Cancer statistics, 2024. CA Cancer J Clin. 2024; 74(1): 12-49. doi:10.3322/caac.21820.
2. https://www.cancer.gov/types/childhood-cancers/child-adolescent-cancers-factsheet#:~:text=In%20the%20mid%2D1970s%2C%2058,least%205%20years%20(2). Accessed 9 Sept 2024.
3. https://www.whitehouse.gov/briefing-room/presidential-actions/2024/08/30/a-proclamation-on-national-childhood-cancer-awareness-month-2024/. Accessed 2 Sept 2024.
4. McEachron TA, Helman LJ. Recent Advances in Pediatric Cancer Research. Cancer Res. 2021 Dec 1;81(23):5783-5799. doi: 10.1158/0008-5472.CAN-21-1191. Epub 2021 Sep 24. PMID: 34561271; PMCID: PMC9725930.
5. https://www.chop.edu/news/emily-whitehead-first-pediatric-patient-receive-car-t-cell-therapy-celebrates-cure-10-years. Accessed 3 Sept 2024.
6. https://cancerprogressreport.aacr.org/report/survivor-journeys/isabella-snow-fraser-reclaiming-childhood-thanks-to-immunotherapy/. Accessed 2 Sept 2024.