CAR-T and Targeted Drugs May Help High-Risk Childhood Leukemia

Dr. Lisa Niswander, a pediatric oncologist and physician-scientist at Roswell Park Comprehensive Cancer Center and the Roswell Park Golisano Children’s Buffalo Cancer and Blood Disorders Program, explained that while childhood leukemia treatment has improved significantly, leukemia in infants younger than 1 year can behave differently from leukemia in older children.

Infants with acute lymphoblastic leukemia (ALL) can often reach remission, meaning leukemia can no longer be detected. However, the leukemia may develop changes that allow it to resist treatment and return. Traditional chemotherapy can also affect healthy parts of the body, which is important for infants who are still developing.

CAR-T cell therapy has helped some children with leukemia that has not responded to other treatments. The therapy uses a patient’s own T cells, which are modified in a laboratory to recognize and attack leukemia cells. However, leukemia can still return if cancer cells learn to evade CAR-T cells or if the CAR-T cells do not remain in the body long enough.

Niswander’s research is examining whether CAR-T cells can be combined with targeted treatments, including drugs known as menin inhibitors. These medicines can target certain genetic changes found in leukemia, including KMT2A rearrangements that occur in some infant leukemias.

The goal is to attack leukemia in multiple ways, potentially making it more difficult for cancer cells to develop resistance. Researchers are also studying how targeted drugs affect the CAR-T cells themselves and how the treatments should be timed.

For patients and families, Niswander emphasized that these combinations are still being studied in the laboratory. CAR-T therapy is not currently a standard first-line treatment for most newly diagnosed children with leukemia, although researchers hope these approaches can eventually move into clinical trials and earlier treatment settings.

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