Neuroblastoma is a cancer of the sympathetic nervous system which can occur in the chest, neck, abdomen and adrenal glands, and can metastasize to the bone marrow and other organs. Children with low or intermediate risk neuroblastoma can be cured through surgical intervention and/or chemotherapy, however, at least half of all children with neuroblastoma have high risk disease, which often requires combined surgical, radio-, immuno-, and chemotherapy, in addition to autologous stem cell transplantation.
GD2 is a molecule expressed on almost all neuroblastomas, and a substantial fraction of small cell lung cancer and melanoma, with restricted expression on normal tissues, making it a good target for CAR-NKT cell therapy.
We are developing CMD-501 for the treatment of high risk, relapsed, refractory neuroblastoma in children. CMD-501 is an autologous cell therapy targeting GD2, which utilizes our natural killer T (NKT) cell platform technology in combination with genetically engineered chimeric antigen receptors (CARs). A clinical trial is currently open to recruitment at Baylor College of Medicine and Texas Children’s Hospital, Houston, Texas.
More information about the Neuroblastoma Trial can be found here https://www.bcm.edu/research/clinical-trials/h-41033
See our press release regarding the first dosed patient here.