CAR T-cell therapy leads to excellent results for children with leukemia

St. Jude Children’s Research Hospital researchers developed a CAR T-cell therapy for children with relapsed B-cell acute lymphoblastic leukemia. The results were extraordinary.

Nearly all of the children in the study – which was a collaboration between St. Jude and Shanghai Children’s Medical Center in China – has complete remission after receiving CAR T-cell treatment.

The remission rate was 99% for the 225 children in the study. Remission is defined as a decrease in tumor volume or a complete disappearance of symptoms and signs of a cancer. Additionally, only 25% of the children had a relapse (recurrence) of their cancer within one year after treatment.

What is CAR T-cell therapy?

CAR T-cell therapy involves engineering our own T cells to better fight cancer. Scientists remove our T cells and add genetic code that leads to a new antigen receptor. This receptor directs the T cells to search for cancer cells expressing a specific protein.

CAR T-cell therapy, part of an emerging field of cancer treatment known as cell and gene therapy, is FDA-approved for blood cancers such as leukemia, lymphoma, and multiple myeloma. As of February 2023, there are six CAR T-cell therapies approved to treat these cancers.

In the St. Jude and Shanghai Children’s study, patients received two different types of CAR T cells. The hope was by combining the two, they would address all cancer cells.

“We have learned that you must hit the cancer cells with combination therapies with different mechanisms of action at the same time to cure patients,” said Ching-Hon Pui, MD, chair of the St. Jude Department of Oncology.

Read the full story on the Alliance for Cancer Gene Therapy website.