An Experimental Treatment Offers Hope for a Young Boy with Liver Cancer
A groundbreaking experimental treatment may have provided a 3-year-old boy battling advanced liver cancer with a new chance at life. Doctors from Baylor College of Medicine in Texas and other institutions shared details of this extraordinary case earlier this month. The boy, who had metastatic cancer, achieved complete remission after undergoing a novel type of immunotherapy, and this remission has persisted for over a year now. He is part of an ongoing phase I trial aimed at assessing this treatment in several similar patients.
“This case demonstrates that a lasting complete remission in a patient with a solid tumor resistant to chemotherapy can be achieved entirely in an outpatient setting without systemic toxic effects,” the authors noted in their publication last week in the New England Journal of Medicine.
A New Kind of CAR T
The immunotherapy administered to the boy is known as chimeric antigen receptor (CAR) T cell therapy. This treatment involves genetically modifying T cells—usually harvested from the patient’s own body—to express receptors targeting specific antigens. Once reintroduced into the body, these engineered T cells can identify and destroy cells presenting that antigen, such as cancer cells, while ideally remaining in the body long-term to prevent recurrence.
CAR T cell therapy has emerged as an innovative option for treating certain severe blood cancers like leukemia and lymphoma, diseases that might be fatal otherwise. Depending on the type of cancer, research indicates that about 25% to 90% of patients respond favorably to CAR T therapy.
However, developing effective CAR T cells for solid tumors has proven to be significantly more challenging. Solid tumors present greater complexities, both in the types of antigens they exhibit and the mechanisms they employ to evade the immune system. The researchers involved in this study are experimenting with a unique kind of CAR T that they believe can address these challenges.
Their approach is actually two-fold. The CAR T cells are engineered to attack glypican-3, a protein frequently found on cancerous liver cells but absent on healthy adult cells. Additionally, the T cells are modified to produce two immune-related signaling proteins: interleukin-15 and interleukin-21. Animal studies suggest that these proteins can collectively enhance the cancer-fighting effectiveness and longevity of CAR T cells. Furthermore, these cells also include a safety switch gene, allowing clinicians to eliminate them with a separate drug if required.
The young patient had hepatoblastoma, the most prevalent form of liver cancer, which had spread from the liver to his bones and lungs. Even after surgery and multiple chemotherapy treatments, the cancer recurred. As with many cases, tests confirmed the presence of glypican-3.
The boy received two infusions of CAR T cells, derived from his own immune system, spaced eight weeks apart. Following the first infusion, he showed signs of a partial response; after the second, the remaining cancer appeared to vanish completely. Now, one year later, he still seems to be cancer-free, and notably, he didn’t experience severe side effects typically associated with CAR T therapy, such as cytokine release syndrome, a condition that can potentially be life-threatening.
The Future of This Treatment
Of course, this is just one patient in an early-stage trial. Still, the remarkable outcome here is a hopeful indicator that their treatment strategy may prove effective as intended.
“This study suggests that these innovative CAR T cells could be a safe and effective approach for hepatoblastoma, highlighting the need for further evaluation in patients with GPC3+ solid tumors,” said Andras Heczey, the study’s author and pediatric oncologist. He initially worked with Baylor College of Medicine but is now associated with the University of Washington School of Medicine and Seattle Children’s Hospital.
The boy is part of a broader trial known as the CARE study, which is set to include 18-30 patients with similar liver cancer cases. The main phase of the trial is expected to conclude by next summer. Other research groups are also exploring their own CAR T therapies for solid tumors.
CAR T therapy has already transformed cancer treatment, and we haven’t reached its full potential yet.
