Something to look forward to: Some of the most promising cancer treatments involve genetically modifying a patient's own immune cells. As a recent study involving a pediatric patient demonstrates, the immune system can be trained to become a highly effective weapon against cancer.
A young boy with metastatic cancer was treated with an innovative immunotherapy technique, and after 12 months, the cancer appears to be completely gone. The technique is known as chimeric antigen receptor (CAR) T-cell therapy and is designed to fight cancer cells from inside the body rather than relying on external procedures.
CAR-T therapy requires physicians to take a patient's own immune cells, genetically engineer them, and then reintroduce them into the body, where they can hunt down cancer cells. The method has shown promise in the past, but primarily against blood cancers.
However, the new case could represent a significant milestone in cancer research. The very young patient had hepatoblastoma, a "solid" tumor that developed in his liver. Hepatoblastoma is a malignant cancer that commonly occurs in infants and children and can be difficult to diagnose until its later stages.
The child was first diagnosed with a solid mass in the left lobe of his liver. The cancer had spread to his lungs, as hepatoblastoma can metastasize to other parts of the body. The patient was initially treated with chemotherapy, but the cancer eventually returned, and the boy no longer responded to the treatment.
After the second diagnosis, the patient was enrolled in the experimental CARE study, which focuses on CAR-T cells. He received two doses of the GPC3-CAR treatment, which uses his own immune cells, bioengineered to target a protein commonly found in liver cancers called glypican-3. The researchers also instructed the cells to express interleukin-15 and interleukin-21, two immune proteins that can enhance CAR-T cells' ability to kill cancer cells and extend their lifespan.
After the first GPC3-CAR infusion, the patient showed a partial response to the treatment. The boy received the second infusion eight weeks later, and follow-up scans confirmed that the cancer was in remission. After 12 months, the boy still appears to be free of the disease.
According to David Steffin, a researcher at Texas Children's and first author of the study, the case "demonstrates that a durable complete response in a chemotherapy-resistant solid tumor can be achieved entirely in the outpatient setting without systemic toxicity."
Researchers are now pursuing further studies of CAR-T therapy, looking for additional evidence of its ability to treat solid tumors without causing significant toxicity. According to cancer survivors who underwent the experimental therapy, bioengineered immune cells can be extremely taxing on the body, particularly for older patients.