Novel CAR T-cell Therapy Shows Promise for Neuroblastoma

Recently, scientists and researchers from the UCL Great Ormond Street Institute for Child Health (GOS ICH) and the UCL Cancer Institute worked to create an innovative solution for patients with cancerous tumors. According to Medical XPress, researchers performed a proof-of-concept study to evaluate modified T-cells as a potential treatment for neuroblastoma. This rare childhood tumor has a long-term survival rate of 50-60% even with treatment. Current treatments include surgery, radiotherapy, stem cell transplants, chemotherapy, and antibody therapy. If this new CAR T-cell therapy is effective, it could offer a less invasive and more beneficial therapeutic option. Read the full findings in Science Translational Medicine.

CAR T-Cell Therapy

According to the National Cancer Institute (NCI), CAR T-cell therapy is:

a type of treatment in which a patient’s T cells (an immune system cell) are changed in the laboratory so they will attack cancer cells. T cells are taken from a patient’s blood, [then] the gene for a special [chimeric antigen] receptor (CAR) that binds to a certain protein on the patient’s cancer cells is added to the T cells in the laboratory.

In this case, researchers sourced T-cells from 12 pediatric patients with treatment-averse neuroblastoma. They modified the cells to identify, attack, and kill cancerous GD2 cells. In the past, CAR T-cell therapy has been used mainly for hematologic malignancies. However, this is one of the first studies highlighting how it can also be used for solid tumors.

When researchers dosed patients with the CAR T-cells, some patients experienced tumor shrinkage. Although these benefits were not long-term, it still shows some promise. This therapy was also relatively safe and well-tolerated. It mostly attacked tumor cells. In some body tissues, GD2 is present. However, this CAR T-cell therapy did not seem to hurt this tissue.

So what’s the next step for researchers? They will collaborate with biopharmaceutical company Autolus to analyze AUTO6NG as a potential treatment for neuroblastoma.


As a fetus develops, immature nerve cells called neuroblasts play a role in that development. Typically, neuroblasts either mature or disappear by birth. However, in rare cases, these neuroblasts form a tumor called a neuroblastoma. Although researchers know that a genetic mutation changes these neuroblasts, it is not clear what causes this mutation. Neuroblastoma forms in nerve tissue. Generally, these begin in the adrenal glands, but can also develop in the abdomen, chest, neck, and spine. Typically, neuroblastoma affects children ages five and younger. Symptoms vary, but can include:

  • Bone pain
  • Restricted movement
  • Fatigue
  • Appetite loss
  • Unintended weight loss
  • Bulging eyes
  • Dark circles around the eyes
  • Failure to thrive
  • Pallor (pale skin)
  • Abdominal, neck, or chest lumps
  • Painless, bluish lumps under the skin (infants)
  • Abdominal distension (infants)
  • Difficulty breathing (infants)

Learn more about neuroblastoma.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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