First Patient Dosed with Experimental Treatment for Multiple Myeloma in Clinical Trial

According to a story from Myeloma Research News, the first patient has been dosed with an experimental allogeneic (donor derived) CAR-T cell therapy called UCARTCS1 as part of a phase 1 clinical trial. This therapy is being developed as a treatment for relapsed or refractory multiple myeloma, a rare type of blood cancer. This investigational product candidate is being developed by Cellectis.

About Multiple Myeloma

Multiple myeloma, which is occasionally referred to as plasma cell myeloma, is a blood cancer that affects plasma cells. These are white blood cells that produce antibodies. The overall cause of multiple myeloma is not well understood, however, some risk factors have been identified. These include obesity, family history, smoldering myeloma, and monoclonal gammopathy of undetermined significance. These last two conditions have the potential to develop into multiple myeloma. Symptoms of this cancer include bone pain, infections, anemia, kidney failure, overly thick blood, confusion, fatigue, headaches, and amyloidosis. Treatment includes chemo, stem cell transplant, and other medications for relapsed disease, which is common. Five year survival rate is 49 percent in the US. To learn more about multiple myeloma, click here.

CAR-T Cell Therapy and UCARTCS1

CAR-T cell therapy has been gaining traction in recent years as an effective form of immunotherapy treatment for various forms of cancer. The process involves the extraction of immune system cells called T-cells from a cancer patient. These cells are then modified in the lab setting to target the patient’s cancer cells. The modified cells are then propagated in the lab before being reintroduced into the patient’s body. UCARTCS1 is a different variation on this method in that the T-cells that are used are obtained from a separate donor. 

It is the first donor-derived CAR-T cell therapy that has been cleared for trials for this disease by the US Food and Drug Administration (FDA). The trial is expected to include a total of 18 patients who have relapsed disease. The researchers will evaluate the effect of the therapy at various different dose levels.

The use of cells from healthy donor is designed to counteract some of the common issues with CAR-T cell therapy, such as the long time it takes to develop the cells and the fact that some patients do not have enough T-cells to collect. UCARCS1 also is designed to account for potential issues such as graft vs host disease and cross reactions with the patient’s own cells.


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