Prior to the clinical trial, the child’s liver stopped responding to chemotherapy

NEED TO KNOW

• A 3-year-old boy with chemotherapy-resistant liver cancer achieved remission after experimental CAR T-cell therapy, researchers said on Sept. 9
• The treatment was part of the CARE study, a clinical trial for pediatric patients with recurrent solid tumors
• Doctors reported no systemic toxic effects, and the child has remained disease-free for at least 12 months

A toddler with a rare form of cancer has been cleared of symptoms after undergoing an experimental treatment, according to a study published in The New England Journal of Medicine on Sept. 9.

The 3-year-old boy, whose name was not released, was diagnosed with chemotherapy-resistant metastatic hepatoblastoma.

After two infusions of glypican-3–specific CAR T-cells that coexpressed interleukin-15 and interleukin-21 were given to the child, he “had complete regression” that lasted for at least 12 months, researchers said.

“This case shows that a durable complete remission in a patient with a chemotherapy-resistant solid tumor can be achieved entirely in the outpatient setting without systemic toxic effects,” first author Dr. David Steffin, associate chief of cellular therapy and bone marrow transplant at Texas Children’s and associate professor of pediatrics – hematology and oncology in the Center for Cell and Gene Therapy at Baylor, said in a Baylor College of Medicine blog post.

The team included doctors and medical professionals from Baylor College of Medicine in Houston; Fred Hutchinson Cancer Center in Seattle; Texas Children’s Hospital in Houston; Houston Methodist Research Institute in Houston and Seattle Children’s Hospital.

Hepatoblastoma is a cancer that forms within the liver’s tissues, and is the most common type of childhood liver cancer, usually affecting children under 3 years old, according to the National Cancer Institute.

When researchers began working with the 3-year-old, there was a mass in the left lobe of his liver that measured 11.2 cm (about 4.4 inches) by 9.6 cm (about 3.7 inches) by 7.1 cm (about 2.7 inches).

The toddler received three lines of chemotherapy and underwent complete resection of the primary tumor and two pulmonary metastasectomies, the report said. However, the boy later had rapid recurrence after his last metastasectomy, resulting in a new metastatic tumor.

This prompted healthcare professionals to enroll the child in the CARE study, where he received two doses of 15.21.GPC3-CAR T-cells.

The CARE study, which refers to Chimeric Antigen Receptor Expressed in T-Cells for Pediatric Solid Tumors, is a clinical trial sponsored by Baylor College of Medicine for pediatric patients who have had cancers that have recurred.

During the trial, the child received two doses of 15.21.GPC3-CAR T-cells.

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“After the first infusion, a partial response was observed,” researchers wrote in The New England Journal of Medicine, adding that after his second infusion with lymphodepletion excluded, “imaging showed resolution of the metastatic disease with residual scarring.”

His status was “disease-free” after 12 months.

He had previously been treated with three lines of chemotherapy and underwent complete resection of the primary liver tumor and two lung metastases, and was no longer responsive to chemotherapy prior to the clinical trial.

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