A Child’s Metastatic Cancer Disappears After Novel Immunotherapy

Hepatoblastoma is the most common pediatric liver cancer. A 3-year-old battling the disease achieved extremely positive results in a new study. After receiving a new immunotherapy, the child’s cancer disappeared completely.

The 3-year-old is part of the CARE study, a first-in-human Phase 1 trial testing engineered T cells called glypican-3-specific chimeric antigen receptor (GPC3-CAR) T cells with interleukin-15 and -21 (IL-15 and IL-21).

Researchers at Texas Children’s, Baylor College of Medicine, and Seattle Children’s shared the encouraging update.

Treating the Metastatic Cancer

Originally, the child had a large liver tumor and cancer in their lungs. The patient underwent three lines of chemotherapy and surgery to remove the primary tumor and two lung spots. Unfortunately, the child became resistant to chemotherapy, and a new spot appeared on their lung. Following this, the child joined the CARE study.

The immunotherapy was engineered from the patient’s own cells at the Center for Cell and Gene Therapy. According to the researchers, they used two vectors. One encoded the GPC3-CAR, and the second encoded IL15, IL21, and a safety switch that researchers previously showed controls CAR T expansion.

The Complete Regression Continues

The patient received two infusions eight weeks apart in an outpatient setting. After the first dose, doctors saw a partial response. Then, imaging showed the spreading cancer was totally gone after the second infusion.

One year later, the complete regression continues.

“This case demonstrates that a durable complete response in a chemotherapy-resistant solid tumor can be achieved entirely in the outpatient setting without systemic toxicity,” said 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.

Corresponding author Dr. Andras Heczey, principal investigator at Seattle Children’s and professor of pediatrics in hematology-oncology at the University of Washington School of Medicine. Heczey was at Baylor and Texas Children’s at the time of research, added. “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.”