A groundbreaking study published in the journal Nature Medicine has revealed the long-term success of CAR-T cell therapy in treating neuroblastoma, a rare and aggressive type of cancer that primarily affects children. This study, which comes from a clinical trial conducted at Baylor College of Medicine between 2004 and 2009, highlights the case of a patient who has remained cancer-free for 18 years after receiving this innovative treatment.
As detailed by Science News, CAR-T cell therapy is a type of cancer treatment that involves taking a patient’s own immune cells, modifying them in a lab to help them recognize and attack cancer cells, and then putting them back into the patient’s body. While this therapy has shown great success in treating certain types of blood cancers, using it to treat solid tumors like neuroblastoma has been much more difficult. This is because solid tumors have a more complex structure and contain molecules that can block the engineered immune cells from working effectively.
The clinical trial at Baylor College of Medicine included 19 children with neuroblastoma. Of these, 11 had active cancer that was growing, and 8 were at high risk of their cancer coming back after previous treatment. All the children received an infusion of CAR-T cells designed to target a protein called GD2, which is found on the surface of neuroblastoma cells. However, the CAR-T cells used in this trial were an early version of the therapy, known as first-generation CAR-T cells. These early cells did not include certain components, called co-stimulatory domains, that are now known to help the cells survive longer and work more effectively in the body.
Over the course of seven years after treatment, 12 of the 19 patients saw their cancer return and eventually passed away from the disease. However, seven patients remained cancer-free, with five of them showing no signs of cancer for 10 to 15 years. Of the remaining two, one stayed in remission for eight years before leaving the study, and the other, who is the focus of this report, has now been in remission for 18 years. This patient is believed to be the longest-surviving cancer patient ever treated with CAR-T therapy.
The results of this study show that even the early version of CAR-T therapy had the potential to help some patients achieve long-term remission. However, the researchers note that more work is needed to understand why some patients responded well to the treatment while others did not. They suggest that factors like how long the engineered cells lasted in the body and the tumor’s ability to hide from the immune system might have played a role in the different outcomes.
The study also reported that 31.6% of the patients were free of cancer for 15 years after treatment, and 36.8% of the patients were still alive after 15 years. Patients who did not have active cancer at the time of treatment had even higher survival rates. Importantly, the patients who remained cancer-free for the longest time also had the modified T cells in their bodies for a much longer period compared to those whose cancer returned.
Since this trial, researchers have developed newer versions of CAR-T cells that include additional molecules to help the cells last longer and target tumors more effectively. In a more recent study using these improved CAR-T cells, nine out of 27 neuroblastoma patients showed no signs of cancer six weeks after treatment, and five of those patients remained cancer-free for one to two years. Researchers are now waiting for longer-term data from this study to see how well these next-generation CAR-T cells perform over time.
This study offers hope for patients with neuroblastoma and their families. While more research is needed to fully understand how CAR-T therapy can lead to long-term remission and to make the treatment even more effective, the 18-year remission achieved by one patient in this study is a major milestone. It demonstrates the potential of this personalized approach to cancer treatment and opens the door for better outcomes not only for neuroblastoma but also for other types of solid tumors in the future.
Published: Mar 3, 2025 01:30 pm