Neuroblastoma is a cancer in children that is very difficult to treat if it spreads through the body. A new way of fighting this disease is to use some of the body’s own immune cells to kill the cancer cells. For this treatment a few teaspoons of blood are taken from the child with neuroblastoma and the cells are genetically reprogrammed to recognize and kill neuroblastoma cancer cells. These reprogrammed cells, called “chimeric antigen receptor” cells or CAR cells, have already cured several children with neuroblastoma at our hospital. However, in other children, this treatment has only partially worked and in some cases the cancer has come back. We believe that the effect of CAR cells i...

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Neuroblastoma is a cancer in children that is very difficult to treat if it spreads through the body. A new way of fighting this disease is to use some of the body’s own immune cells to kill the cancer cells. For this treatment a few teaspoons of blood are taken from the child with neuroblastoma and the cells are genetically reprogrammed to recognize and kill neuroblastoma cancer cells. These reprogrammed cells, called “chimeric antigen receptor” cells or CAR cells, have already cured several children with neuroblastoma at our hospital. However, in other children, this treatment has only partially worked and in some cases the cancer has come back. We believe that the effect of CAR cells is limited in these children, because the cells did not survive in the body long enough to kill all the cancer cells; after the CAR cells die off, the surviving neuroblastoma cells start growing back. We are planning to combine the CAR cell treatment with another gene that can provide “fuel” to the cancer-killing CAR cells, so that they can live longer and fight the cancer cells better. The “fuel” we are providing is called cytokine. Because too much cytokine can cause side effects, we are providing the cytokine in such a way that it stays only inside the CAR cell and does not cause harm to other cells or healthy tissue. When we tested the CAR cells with extra cytokine “fuel” in the lab they were able to kill cancer cells for a much longer time and prevented the tumors from growing back. We have done many laboratory experiments that show the advantages of CAR cells that are provided with extra “fuel” and we now are planning to treat patients with neuroblastoma with this improved treatment. We are hoping that this method will result in more cures for children with neuroblastoma and other cancers.

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