Modern developments in cell and gene therapy products are transforming the treatment of cancers and genetic diseases, and in the process they have helped create a multi-billion dollar global industry that is poised to more than double over the next ten years, according to 2020 estimates by leading medical market research firm Kalorama Information as reported in Cell Therapy and Gene Therapy Markets. Therapies for cancer generate almost half of the cell and gene therapy market total, led by oncology drugs including Kymriah, Provenge, and Yescarta. Now a new entrant into the market aims to join the ranks of these notable cell and gene therapy products for cancer.
In March 2020, the U.S. Food and Drug Administration (FDA) approved a new cell-based gene therapy idecabtagene vicleucel, which will be sold as Abecma. The new therapy is for adult patients with relapsed myeloma (myeloma that returns after completion of treatment) or refractory myeloma (myeloma that does not respond to treatment) who have not responded to at least four different types of therapy. Abecma is the first cell-based gene therapy for the treatment of multiple myeloma that has been approved by the FDA. The safety and effectiveness of Abecma were established in a study of 127 patients with relapsed myeloma and refractory myeloma who received at least three prior antimyeloma lines of therapy.
Multiple myeloma is one of several areas in the oncology sector that Kalorama Information identified as having strong development pipelines, alongside hematologic cancers (leukemia, lymphoma), brain cancer, and ovarian cancer.
Though the exact cause is unknown, multiple myeloma is a cancer that forms in a type of white blood cell called a plasma cell. Plasma cells help people fight infections by making antibodies that recognize and attack germs. Multiple myeloma causes cancer cells to accumulate in the bone marrow, where they crowd out healthy blood cells. Rather than produce helpful antibodies, the cancer cells produce abnormal proteins that can cause complications, including damage to the bones, kidneys, and immune system.
Multiple myeloma is not curable but is treatable and patient outlook is often dependent on age and the stage of the condition when diagnosed. Myeloma accounted for approximately 1.8% (32,000) of all new cancer cases in the U.S. in 2020, according to the National Cancer Institute. For people with multiple myeloma who require treatment, several treatments are available to help control the disease—now including Abecma. Multiple myeloma is one of several areas in the oncology sector that Kalorama Information identifies as having strong development pipelines, alongside hematologic cancers (leukemia, lymphoma), brain cancer, and ovarian cancer.
In the case of Abecma as a treatment option, the therapy is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. Abecma doses are customized treatments created using a patient’s own T-cells, which are a type of white blood cell, to help fight the myeloma. The T-cells are collected and genetically modified to include a new gene that facilitates targeting and killing myeloma cells. Once modified, the cells are infused back into the patient.