In short
Allogeneic T cells expressing a chimeric antigen receptor targeting CD19, with the genes TRAC and CD52 disrupted, also known as UCART19, are an investigational cell therapy being studied in advanced lymphoid leukemia. The therapy uses donor-derived immune cells that are modified in the laboratory. One Phase 3 clinical trial is currently authorised to evaluate the long-term safety of this treatment in 30 patients who previously received UCART19.
Key points
- Allogeneic T cells (UCART19) are an investigational cell therapy for advanced lymphoid leukemia.
- The only registered trial is a Phase 3 study focusing on long-term safety.
- The trial includes 30 participants who have previously been treated with UCART19.
- The study measures adverse events for up to 12 months after the last infusion.
- The trial is currently authorised but not yet recruiting.
- The therapy is given by intravenous administration.
What is allogeneic T cells?
Allogeneic T cells expressing a chimeric antigen receptor targeting CD19, with the genes TRAC and CD52 disrupted, also known as UCART19, are a type of investigational cell therapy. The term "allogeneic" means the T cells come from a healthy donor rather than from the patient themselves. These donor T cells are modified in the laboratory to recognise and attack cancer cells that carry a protein called CD19. The therapy is not yet authorised as a medicine and is still being studied in clinical trials.
How does allogeneic T cells work?
UCART19 works by using T cells, which are a type of immune cell that normally helps the body fight infections. In this therapy, the T cells are taken from a donor and genetically modified to express a chimeric antigen receptor (CAR) that targets CD19. CD19 is a protein found on the surface of B cells, including cancerous B cells in certain types of leukemia. By disrupting the genes TRAC and CD52, the modified T cells are designed to avoid being rejected by the patient's immune system and to survive longer in the body. This allows the donor T cells to find and attack the cancer cells.
Which conditions is it used or studied for?
- Advanced lymphoid leukemia — a cancer of the blood and bone marrow that affects lymphocytes, a type of white blood cell. The term "advanced" indicates the disease has progressed or has not responded well to previous treatments.
How is allogeneic T cells given?
UCART19 is given by intravenous administration, which means it is delivered directly into a vein. In the registered trial, the therapy was administered at different cell concentrations, including 0.6 megaCells/ml, 6 megaCells/ml, 15 megaCells/ml and 20 megaCells/ml. The exact dose for each patient would be determined by the treating team as part of the clinical study.
What do the clinical trials show?
One Phase 3 clinical trial is registered for UCART19. This trial is designed to evaluate the long-term safety of the therapy in patients with advanced lymphoid leukemia who have previously been administered UCART19. The study plans to include 30 participants and is currently authorised but not yet recruiting.
The main outcomes measured in the trial include the number, duration and outcome of all adverse events (side effects) within 12 months after the last UCART19 infusion. The study also records adverse events of special interest, the proportion of patients with adverse events leading to death, and, for paediatric patients, the potential impact on growth curve and puberty.
Because this is a long-term safety study, the focus is on monitoring patients who have already received the therapy. The trial does not compare UCART19 against another treatment but instead follows patients over time to understand any lasting effects.