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New immunotherapy for children with hepatoblastoma or hepatocellular carcinoma


This study currently enrolls pediatric patients who have GPC3-positive liver cancers including hepatoblastoma or hepatocellular carcinoma. Patients may be considered if the cancer has come back, has not gone away after standard treatment or the patient cannot receive standard treatment.


This is a Phase I research study that uses special immune system cells called GPC3-CAR T cells, and is a new experimental treatment.

The body has different ways of fighting infection and disease. No single way seems perfect for fighting cancers. This research study combines two different ways of fighting cancer: antibodies and T cells. Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special infection-fighting blood cells that can kill other cells, including cells infected with viruses and tumor cells. Both antibodies and T cells have been used to treat patients with cancers. They have shown promise, but have not been strong enough to cure most patients.

Investigators have found from previous research that they can put a new gene into T cells that will make them recognize cancer cells and kill them. In preclinical studies, the investigators made several genes called a chimeric antigen receptor (CAR), from an antibody called GC33 that recognizes glypican-3, a proteoglycan found on solid tumors, including pediatric liver cancers (GPC3-CAR).This study will test T cells genetically engineered with a GPC3-CAR in patients with GPC3-positive liver tumors.

The purpose of this study is to find the biggest dose of GPC3-CAR T cells that is safe, to see how long they last in the body, to learn what the side effects are and to see if the GPC3-CAR T cells will help patients.

Eligibility Criteria

  • 1 Year to 18 Years   (Child, Adult)
  • Sexes Eligible for Study: All
  • Relapsed or refractory GPC3-positive liver tumors

Detailed inclusion and exclusion criteria are listed on


Andras Heczey, MD
Texas Children’s Cancer and Hematology Centers
Phone: 832-824-4233

Jacqueline Castello
Phone: 832-824-4391