Gene Modified Cell Therapy

In Gene-modified cell therapy, specific cells are genetically modified outside the body in order to help the patient fight a disease. After removing specific cells from the body, the cells are transferred to a laboratory where a new gene can be introduced or a faulty gene can be corrected in the cells. Therapies created this way can also be called Ex Vivo gene therapies. The modified cells then returned to the patient in order to help the patient fight a disease, for example, in Chimeric antigen receptor T-cell (CAR T-cell) therapy for cancers.

Gene-modified cell therapy includes:

  • Chimeric antigen receptor (CAR T-cell) therapies: This a way of modifying the patient’s immune cells (T cells) to recognize structures (antigens) on the surface of cancer cells. Once the T-cell receptor binds to a tumor antigen, the T cell is stimulated to attack the cancer cell.
  • T-cell receptor (TCR) therapies: T cell Receptors (TCRs) can recognize tumor-specific proteins on the inside of cells.
  • Tumor infiltrating lymphocytes (TILs): TILs infiltrate solid tumors. The therapy removes T cells from a patient and treats them to the T-cells are primed to recognize tumors. The TILs are reintroduced into the patient, generally after the patient has a low white blood cell count due to treatment with chemotherapy. TILs have been demonstrated to be effective in some forms of cancer.
  • Natural killer (NK) cell therapies: NK cells in the body can naturally recognize mutated or infected cells and eliminate them. NK cells can escape some immune attacks that might prevent them from being effective against diseased cells. They release signaling proteins that recruit and activate the body’s immune system. NK cells derived from stem cells are being tested at present.
  • Marrow derived lymphocytes (MILs): MILS are bone marrow-derived cells that house a reservoir of T-cells. The T-cells can be prepared to fight disease.
  • Dendritic cell vaccine: Dendritic cell vaccines, made from dendritic cells, promotes antitumor immune responses. Dendritic cells are rare cells found in the circulation system and in tissues. These specialized immune cells play a critical role in promoting an immune response, including an ability to regulate and control T-cell responses.

How to Learn More

Gene Therapy: Your Questions Answered – This video is presented by NORD’s RareEDU™ to address a vital topic to today’s rare disease community.