3-year-old boy’s quick-spreading cancer is cured thanks to 2 doses of new treatment

A cancer treatment has reached a remarkable milestone.

While Chimeric Antigen Receptor (CAR) T-cell therapy has shown promising effects on blood cancers, like particular types of leukemia or lymphoma, its effects are limited when treating solid tumors — until now.

A 3-year-old boy battling chemo-resistant cancer that had spread from his liver to his lungs and possibly his bones was declared cancer-free after just two doses of the experimental therapy.

The report sharing the progress researchers at Texas Children’s, Baylor College of Medicine and Seattle Children’s was published this month in The New England Journal of Medicine.

The toddler had been diagnosed with hepatoblastoma, a common form of childhood liver cancer. He had a massive tumor measuring 4.4 × 3.8 × 2.8 inches.

Despite multiple rounds of chemotherapy and surgery to remove the tumor, the cancer returned. It metastasized to his lungs, and there was evidence it had spread to his bones.

Doctors than tried CAR T-cell therapy, which involves taking a patient’s white blood cells (T cells) and genetically engineering them to become cancer-fighting machines.

After receiving two doses of the experimental GPC3-CAR treatment over eight weeks, he had no remaining signs of the disease, even after one year — indicating complete regression.

“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 said.

Liver cancer is the third leading cause of cancer-related deaths globally, yet hepatoblastoma typically relies on radiation and surgery to remove cancerous cells and tumors — methods that aren’t always successful.

And while CAR T-cell therapy is an innovative treatment, it’s only FDA-approved to mitigate certain types of leukemias, lymphomas and multiple myeloma.

This particular therapy has proven to be a difficult way to treat solid tumors due to the hostile chemical shield that blocks and destroys immune cells.

Tumor cells can also look like healthy cells, thanks to the tumors using surrounding healthy cells (like fibroblasts and regulatory immune cells) to create a barrier.

This breakthrough in cancer treatment comes after T-cell therapy was found to be an effective treatment for deadly brain tumors in children.

While the use of CAR T-cell therapy for liver cancer was in a single patient in an early-phase trial and more research is needed to see how effective and safe the treatment is, the results are promising.

Continued testing of the GPC3-CAR treatment is being carried out as part of the CARE study, the first human trial of an experimental form of CAR T-cell therapy for pediatric patients with recurrent solid tumors, at Baylor College of Medicine.