CAR-T cell therapy has made a groundbreaking breakthrough in treating childhood cancer, with one patient achieving long-term remission after receiving the treatment nearly two decades ago. This remarkable case study sheds light on the potential of CAR-T cell therapy in treating solid tumors, offering new hope for patients with neuroblastoma and other types of cancer.
A Breakthrough in Cancer Treatment: Long-term Survival of a Child Treated with CAR-T Cell Therapy
CAR-T cell therapy is a type of immunotherapy that uses genetically modified T cells to attack cancer cells.
It involves extracting T cells from a patient's blood, modifying them in a lab to recognize specific cancer antigens, and then reinfusing the modified cells into the patient.
This treatment has shown significant promise in treating certain types of blood cancers, such as leukemia and lymphoma.
According to clinical trials, CAR-T cell therapy can achieve complete remission rates of up to 90% for some patients.
CAR-T cell therapy has been a game-changer in the fight against blood cancers. However, its effectiveness in treating solid tumors has been limited. A recent study published in Nature Medicine sheds light on a remarkable case where a child who received CAR-T cell therapy 18 years ago remains disease-free.
The patient, a 4-year-old girl with neuroblastoma, was part of a clinical trial that involved infusing her with immune cells genetically engineered to target and kill cancer cells. Since then, she has defied the odds by remaining cancer-free for nearly two decades. This case study is significant not only because it highlights the potential of CAR-T cell therapy in treating solid tumors but also because it suggests that this treatment may be more effective than previously thought.
The Challenges of Treating Solid Tumors with CAR-T Cell Therapy
Solid tumors, which account for approximately 90 percent of all cancers, are notoriously difficult to treat with CAR-T cell therapy. This is because they have molecules that can hinder the engineered cells from penetrating and targeting them effectively. Helen Heslop, a physician-scientist at Baylor College of Medicine and Texas Children’s Hospital in Houston, explains that neuroblastoma, in particular, has been challenging to treat with this technology.
A New Hope for Neuroblastoma Patients

Neuroblastoma is a type of cancer that affects the nervous system, primarily in children.
According to the American Cancer Society, it accounts for about 6% of all childhood cancers.
The five-year survival rate for neuroblastoma patients varies depending on age and stage at diagnosis.
For infants under one year old, the survival rate is around 80-90%.
However, for those over one year old, the survival rate drops to around 40-50%.
Researchers are working to develop new treatments, including 'immunotherapy' and 'targeted therapy', to improve outcomes for neuroblastoma patients.
The recent study published by Heslop’s team provides new hope for patients with neuroblastoma. Of the 19 children who received CAR-T cell therapy as part of the clinical trial, one patient remains cancer-free after 18 years. This remarkable case has sparked excitement among researchers, including Carl June, a cancer immunotherapist at the University of Pennsylvania Perelman School of Medicine.
June notes that this study is significant because it suggests that CAR-T cell therapy may be effective in treating solid tumors, not just blood cancers. He emphasizes the need for further research to understand why some patients benefit from this treatment while others do not.
Next-Generation CAR-T Cells Show Promise
CAR-T cell therapy has revolutionized cancer treatment by harnessing the power of a patient's immune system to fight cancer.
Next-generation CAR-T cells are an evolution of this technology, designed to improve efficacy and reduce toxicity.
These advanced cells use novel engineering techniques to enhance their ability to recognize and kill cancer cells.
With improved manufacturing processes and new targeting strategies, next-gen CAR-T cells show promising results in clinical trials.
Studies have reported increased response rates and longer patient survival times.
Since the original study was conducted, researchers have made progress in developing next-generation CAR-T cells that can last longer and track down tumors more effectively. A recent study published by researchers at Bambino Gesù Children’s Hospital in Italy found that nine out of 27 patients with neuroblastoma had no signs of cancer six weeks after receiving these new cells.
While this is a promising development, Heslop emphasizes the need for further research to confirm whether these patients will experience long-term benefits. Nevertheless, this study provides a glimmer of hope that CAR-T cell therapy may be an effective treatment option for neuroblastoma and other solid tumors in the future.