Promising progress in the treatment of neuroblastoma, a common type of cancer among children


Researchers from Baylor College of Medicine published preliminary findings on the first two patients treated in a Phase I clinical trial, first of its kind in humans, with innovative immunotherapy for neuroblastoma.

The treatment is based on the use of the patient’s own T-cells (NKTs, Natural Killer T Cells) and have undergone genetic engineering. The findings were recently presented at the annual meeting of the American Society of Cell & Gene Therapy (ASGCT).

NKTs are a subset of white blood cells that can penetrate tumor tissue and indirectly inhibit its growth. GD2 is a molecule found on the surface of almost every cell of neuroblastoma.

Where is the innovation?

The researchers genetically engineered NKT cells to express a specific protein, an antigen specific to GD2, also known as GD2-specific chimeric antigen receptor, GD2-CAR. This receptor allows these immune cells to kill neuroblastoma cells directly by targeting GD2 on their surface. The GD2-CAR treatment also includes a mediator called Interleukin-15, IL-15, which allows engineered NKTs to function better in the tumor’s microenvironment and to survive longer in the body after infusion into the patient.

The main objectives of the study are to determine the maximum dose given with GD2-CAR NKT, to assess their effect on the tumor, to examine how long it can be identified in the patient’s blood and to characterize their effect on the patient’s neuroblastoma.

Initial results

The two children treated so far had no side effects associated with CAR-NKT. One of them has stabilized. The second one has experienced a very strong partial response after the first infusion with complete regression of one of its two metastases and almost complete withdrawal of the other. This patient received FDA approval for a second treatment regimen with this treatment.

Trial recruitment is still open to patients whose neuroblastoma has returned after treatment or has not responded to standard conventional treatments for this disease.

The study is designed to recruit 24 patients aged 1-21 years. The first two have already been enrolled, as stated…

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