Insights into Early Childhood Leukemia

Early T cell precursor acute lymphoblastic leukemia has myeloid aspects

(RxWiki News) A project to sequence genetic mutations in a particularly aggressive form of childhood leukemia developed a list of common mutations - the first step in developing adequate pharmaceutical targeted therapy.

In research published by a team from St. Jude's Children's Research Hospital, early T Cell precursor acute lymphoblastic leukemia, known as ETP ALL, was genetically sequenced to find common mutations for for the first time.

Most notably, although the cancer belongs to the lymphoblastic family (immune cells that go on to form B-cells and T-cells), using treatments originally developed for myeloid leukemias (immune cells that go on to fight infection like neutrophils or basophils) may be the best option.

"Ask your oncologist about recently developed treatments."

The study was part of the Pediatric Cancer Genome Project, which sequenced the DNA of 600 children with some of the most poorly understood and aggressive cancers.

The project was founded in order to develop the foundation for the next generation of clinical tools to treat pediatric cancers.

DNA from over a hundred cases of ETP-ALL were sequenced, and the results showed that the cancerous mutations were most similar to the myeloid leukemias, not lypmphoblastic leukemias.

Most of the mutations were found in genes that regulate cytokine receptor and growth signaling, disruptions of normal hematopoesis, histone modification and regulatory protein inactivation.

“The mutations and gene expression profile we identified in this study suggest that patients with ETP-ALL might benefit from treatment that includes drugs developed for treatment of acute myeloid leukemia,” said Charles Mullighan, M.D., Ph.D., one of the study's authors from the St. Jude Department of Pathology.

ETP-ALL is a particularly aggressive and rare form of childhood leukemia, with survival rates averaging at less than 40 percent. Treatment success for other childhood leukemias typically range around 90 percent. Researchers hope their findings will help close the gap.

Changing the treatment approach will tailor cancer therapy for the best individual results, improving patient treatment success rates for ETP-ALL.

The results were published in the journal Nature. The authors of this research denied any financial conflict of interest.

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Review Date: 
March 8, 2012