By Bill Thomas | April 4th
In recent years, one of the most promising developments in childhood cancer research has been the rise of CAR T therapy for pediatric cancers. A novel form of immunotherapy, CAR T therapy leverages the power of the patient’s own immune system to effectively target and eradicate cancer cells.
Since 2001, Pediatric Cancer Research Foundation has been at the cutting edge of CAR T cell research. Our investments into the work of Dr. Laurence Cooper and other pioneers ultimately led to the launch of the first in-human protocol using T cells to treat leukemia. This research, which proved that CAR T cells are capable of mounting immune system attacks on tumor cells when infused back into a patient, laid the foundation for decades of innovative investigations into the capabilities of CAR T therapy.
Most recently, a study published in Nature Medicine found that a four-year-old girl whose neuroblastoma went into remission after she was treated with experimental CAR T therapy back in 2006 is now a healthy, cancer-free mother of two. The patient’s 18-year remission, the longest reported cancer remission following treatment with CAR T cells, offers a source of hope for countless patients diagnosed with neuroblastoma, a devastating form of nerve cancer that accounts for an estimated 15% of all childhood cancer deaths.

While CAR T cells have proven particularly effective in treating patients diagnosed with blood cancers, previous investigations into whether CAR T therapy could produce similarly positive long-term outcomes for patients with solid-tumor cancers (like neuroblastoma) were inconclusive. These new findings, reported by researchers at Baylor College of Medicine in Houston, Texas, suggest that CAR T therapy is indeed capable of safely producing such outcomes.
Notable is the fact that numerous advances in CAR T therapy have been made, both by Baylor College of Medicine researchers and others elsewhere, since the now cancer-free woman received her treatments back in 2006. As neuroblastoma research and CAR T cell research continue to progress, our understanding of how to maximize positive long-term patient outcomes deepens. The discoveries being made today form the bedrock of tomorrow’s breakthroughs.
Here at Pediatric Cancer Research Foundation, improving patient outcomes by powering research is one of our core institutional priorities, alongside ensuring equitable care and prioritizing survivorship and mental health. Today, federal funding for childhood cancer research is more at risk than ever before. As such, it falls to all of us within the childhood cancer community to help and support one another.
If you would like to contribute to PCRF’s vision of a future where all children facing cancer are able to beat their disease and go on to enjoy happy, healthy, productive lives, please consider becoming a donor. To stay up-to-date with all the latest news shaping the future of pediatric cancer treatment, don’t forget to follow the Pediatric Cancer Research Foundation Profectus blog.