An experimental treatment has made remarkable progress in the fight against liver cancer after it managed to eliminate signs of the disease in a 3-year-old child, despite the spread of the cancer and its resistance to previous treatments.
Chimeric antigen receptor (CAR-T) T-cell therapy involves taking immune cells from the patient, then genetically modifying them in the laboratory to become more capable of recognizing and attacking cancer cells, before returning them to the body.
This type of treatment has so far been primarily used to treat certain blood cancers, while its application to solid tumors, such as liver and lung cancers, has remained more challenging. However, the child's case, documented by researchers in a report published in the New England Journal of Medicine, offers an initial indication that its use could be expanded to include these tumors.
The child had hepatoblastoma, a type of liver cancer that affects children. At the time of diagnosis, the tumor was large, and the cancer had spread to the lungs, with indications of metastasis to the bones.
The child underwent three rounds of chemotherapy, along with surgery to remove the tumor in his liver and two operations to remove tumors that had spread to his lungs. However, the cancer returned quickly, and a new tumor appeared in his lungs.
The child was then enrolled in the CARE study at Baylor College of Medicine, where he received two doses of an experimental treatment known as GPC3-CAR. The treatment cells were made from his own T cells after they were genetically modified to recognize the glibecan-3 (GPC3) protein, which is found at high levels in some types of liver cancer.
After the first dose, the child showed a partial response to treatment, with the tumor shrinking and a decrease in the level of alpha-fetoprotein in the blood, an indicator used to monitor the activity of some liver tumors. After receiving the second dose eight weeks later, tests showed no clear signs of disease persistence, except for some residual scarring.
After 12 months, the child remained free of any signs of illness.
David Stephen, lead author of the report and a pediatric oncologist at Baylor College of Medicine, said the case demonstrates the possibility of achieving a complete and sustained response in a patient with a chemotherapy-resistant solid tumor without causing significant systemic toxicity.
Andras Hexzi, one of the researchers involved in the study, said that the results provide initial evidence of the potential use of CAR-T cells to treat hepatoblastoma, while emphasizing the need for further studies.
The researchers caution that this result is based on a single patient in an early trial, and therefore the treatment's effectiveness and safety in a larger number of patients cannot yet be determined. The CARE and IMPACT trials are ongoing to study this approach in other patients with solid tumors.
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