Hearing your child has cancer is the worst news a parent can possibly get. For one family, that nightmare became a reality when their 3-year-old son was diagnosed with hepatoblastoma — a condition the National Cancer Institute identifies as the most common type of childhood liver cancer in kids under three.
The primary tumor in his liver was over four inches long, and the disease had already spread to the toddler’s lungs, with signs it had also reached his bones. After three rounds of chemotherapy and multiple surgeries failed to keep the cancer from returning, his doctors decided to try a new approach.
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Fighting Solid Tumors With an Experimental Treatment
The medical team enrolled the toddler in the CARE study, the first human trial for an experimental treatment called Chimeric Antigen Receptor (CAR) T-cell therapy. This immunotherapy technique works by extracting a patient’s own immune cells and genetically altering them in a lab so they can actively hunt down and destroy cancer cells.
Doctors already know this therapy works well against blood cancers, but solid tumors are usually much harder to treat. To target his liver cancer, researchers programmed his T cells to recognize a specific protein called glypican-3 (GPC3) and added extra immune proteins, called interleukins, to help the cells fight harder.
After receiving his first infusion of the engineered cells, the results were immediate. Doctors saw a partial response on his CT scans and noticed a drop in blood proteins linked to tumor activity.
A Clear Scan and a Hopeful Future
Eight weeks later, the little boy received his second dose of the experimental treatment. When doctors performed his follow-up scans, the cancer was gone.
A full 12 months after the treatment, the young boy is still disease-free. Doctors recently shared the details of his recovery in The New England Journal of Medicine.
“This study provides evidence that these novel CAR T cells may be a safe and effective modality for hepatoblastoma and highlights the need for further assessment in patients with GPC3+ solid tumors,” study co-author and pediatric oncologist Andras Heczey from Seattle Children’s Hospital and the University of Washington said in a statement.
This initial report only covers one patient in an early-phase trial, but researchers are already continuing their tests at Baylor College of Medicine and Seattle Children’s Hospital. Hopefully, this is just the beginning of more life-saving options for families facing these devastating diagnoses.
