New Possible Treatment Addresses Specific Type of Leukemia

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A subtype-specific weakness of T-cell acute lymphoblastic leukemia (T-ALL) could enable improved treatment, according to results from a a recent study using a mouse model. T-ALL is a heterogeneous cancer, though treatments are typically uniformly applied across all subtypes. Genetic abnormalities, gene expression differences, and prognoses can all be used to differentiate the subtypes.1

This study, published in Genes and Development, examined a subtype of T-ALL called TAL-1. When researchers transplanted cancer cells from people with this subtype of T-ALL into normal mice, they found that administration of a compound called GSK-J4 killed the leukemic cells quickly and efficiently. The mice did not display short-term side effects.

"It's very exciting because this is the first time anyone has found a potential personalized treatment for this aggressive disease. Unlike current therapies, ours targets the offending gene without harming the rest of the body," said Marjorie Brand, PhD, a senior scientist at The Ottawa Hospital Research Institute and professor at the University of Ottawa in Ontario, Canada.

The researchers discovered GSK-J4 by first determining molecularly how TAL-1 works. The researchers learned that sometimes the TAL-1 gene acted as an oncogene, causing white blood cells to grow uncontrollably. GSK-J4 is able to inhibit the mechanism by which the TAL-1 gene induces excessive white blood cell growth.

Results from this study suggest a potential benefit in milder side effects over other T-ALL therapies. The pharmaceutical industry developed GSK-J4 for research purposes. This is its first oncology application, though it has not been approved for use in humans.

ALL is the most common childhood cancer. T-ALL is different than other subtypes of ALL because this subtype affects T-cell type of white blood cells.

"Learning how a disease works at a molecular level needs to happen before any kind of successful drug can be developed," said Brand.

"You need to do laboratory studies to find the right treatment and prove it works."


1. Benyoucef A, Palii CG, Wang C, et al. UTX inhibition as selective epigenetic therapy against TAL1-driven T-cell acute lymphoblastic leukemia. Genes Dev. 2016;30(5):508-521. doi:10.1101/gad.276790.115.  

Editor's Note: The original version of this article misidentified the most common childhood cancer in the sixth paragraph; the correct type of cancer is ALL. The article was corrected onMarch 29, 2016, to reflect this.
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