A fourteen-year-old cancer patient has gone into full remission after partaking in the clinical trial of a stem cell therapy conducted at the Children’s Hospital of Philadelphia. The recently approved FDA treatment, also known as chimeric antigen receptor T-cell [CAR T cell] therapy, works by obtaining autologous (the patient’s own) immune stem cells, genetically altering and expanding them to recognize a specific molecule on the surface of cancer cells and become targeted cancer killers. In this case, it was used to treat acute lymphocytic leukemia (ALL) originating in the B cells.
The FDA panel’s unanimous recommendation to approve an autologous leukemia treatment represents a paradigm shift in medicine in which gene therapies and stem cells will play leading roles.
The treatment involves altering the genes of T cells, which are highly specialized stem cells obtained from bone marrow, to target a specific protein on the surface of defective immune cells that cause leukemia. It works by harvesting the cells from the patient, engineering them to target the protein CD-19 on the surface of B Cells, and intravenously administering the cells back into the patient, where they multiply and essentially eradicate the B Cells. Showing promise, over 80% of the patients in the trial have gone into remission.