CAR T-Cell Therapy for Leukemia

(PLAT-04 Trial)

This trial explores a new treatment for individuals with leukemia who haven't responded to other therapies. It employs a special type of cell therapy, modifying a patient's own immune cells to target and destroy cancer cells. This treatment, known as patient-derived CD22-specific CAR T-cells also expressing an EGFRt, targets cancer cells with a specific protein called CD22. Suitable candidates for this trial have relapsed or refractory leukemia and have not succeeded with other treatments. As a Phase 1 trial, the research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

The trial protocol does not specify if you need to stop taking your current medications. However, you must be free from active GVHD and off immunosuppressive GVHD therapy for 4 weeks, and you should be at least 7 days post your last chemotherapy and systemic corticosteroid administration.

Research shows that CD22 CAR T-cells, like those used in this trial, are generally well-tolerated. In studies with patients who have similar blood cancers, these CAR T-cells have demonstrated promising safety results. Both children and adults with B-cell acute lymphoblastic leukemia (B-ALL) have shown high response rates and good safety outcomes. Some side effects can occur, but they are usually manageable. This trial is in its early stages, focusing primarily on ensuring the treatment's safety for people. So far, evidence suggests that CD22 CAR T-cells are safe for use in humans, but ongoing research will provide more detailed safety information.¹²³⁴⁵

Unlike standard leukemia treatments like chemotherapy or stem cell transplants, this CAR T-cell therapy uses a patient's own immune cells that are engineered to target CD22, a specific protein found on leukemia cells. Researchers are excited because this approach offers a new mechanism of action by directly harnessing the body’s immune system to fight cancer cells. Additionally, these engineered cells also express EGFRt, which allows for better control and safety of the therapy. This innovative strategy has the potential to provide a more targeted attack on cancer cells, potentially leading to improved outcomes.

Research shows that a special treatment called CD22-specific CAR T-cells can help treat leukemia, particularly in patients whose cancer has returned or who haven't responded to other treatments. Lab-modified T-cells target and destroy cells with the CD22 protein. Studies have found that this treatment can extend life for some patients, and in certain cases, their cancer symptoms have greatly reduced or disappeared. In this trial, participants will receive autologous CD22-specific CAR T-cells expressing EGFRt. Overall, CD22 CAR T-cells offer hope for patients with hard-to-treat leukemia.²³⁶⁷⁸