Europe Endorses Cell-based Gene Therapy to Treat Multiple Myeloma
The European Medicines Agency (EMA) announced on June 25, 2021; it had recommended granting a conditional marketing authorization in the European Union for Abecma (idecabtagene vicleucel) for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three previous therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and whose cancer has worsened since receiving the last treatment.
Despite the development and approval of a range of new medicines for the treatment of multiple myeloma over the past few years, there are limited therapeutic options for patients who have already received three major classes of drugs (immunomodulatory agents, proteasome inhibitors, and monoclonal antibodies) and no longer respond to these medicines.
Therefore, new medicines are needed for patients whose disease returns after treatment.
Abecma is a genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy and the first cell-based gene therapy to treat adult patients with multiple myeloma. Each dose of Abecma is created by collecting a patient’s own T-cells (i.e., white blood cells that help the body fight infections) and genetically modifying them to include a new gene that helps the body target and kill the body myeloma cells. These modified immune cells are then infused back into the patient’s blood.
Multiple myeloma is a rare cancer of a type of white blood cell called plasma cells. Normal plasma cells are found in the bone marrow and are an important part of the immune system. Plasma cells make the antibodies that enable the body to recognize and attack germs such as viruses or bacteria. In multiple myeloma, the proliferation of plasma cells is out of control, resulting in abnormal, immature plasma cells multiplying and filling up the bone marrow. When plasma cells become cancerous, they no longer protect the body from infections and produce abnormal proteins that can cause problems affecting the kidneys, bones, or blood.
The U.S. Food and Drug Administration approved Abecma on March 27, 2021.