Studies detailing patient outcomes with chimeric antigen receptor (CAR) T cell engineering are beginning to provide evidence that supports this therapy as a viable strategy for long-term disease control.

The CAR process requires extracting T cells from the blood of a patient and outfitting them with two powerful features: a receptor on the cell surface that recognises the CD19 protein present on most leukaemia cells, and a powerful mechanism inside the cell that triggers it to expand and proliferate once attached to the targeted protein.

With these engineered features, the T cells are injected back into the patient to hunt and then destroy cancer cells.

This study report details long-term, follow up data from a trial using CAR T cell therapy in 39 children and young adults (median age 10 years) with relapsed, treatment-resistant acute lymphocytic leukemia (ALL).

After the experimental treatment, 36 of 39 children (92%) achieved a complete response. Six months after treatment, more than two-thirds (70%) of children enrolled in the study remained cancer free, and 75 percent have survived. Only five children have received subsequent treatment.

“With this longer follow up, we now have children who remain in remission a year or more after treatment solely because of this T cell therapy,” said lead study author Stephan Grupp, MD, PhD, of The Children’s Hospital of Philadelphia.

“Our next step is to conduct a Phase II, multi-site trial to assess the safety and efficacy of this treatment in multiple centres and to further evaluate its long-term potential to become a replacement for stem cell transplant for children with relapsed, treatment-resistant disease.”

Watch the interview and commentary here.

Source: ASH