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New Drugs for Alzheimer’s May Work for Leukemia

By Kate Ruder

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Leukemia and Lymphoma
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Drugs being developed to treat Alzheimer’s disease may help children who have an uncommon but aggressive form of leukemia.

Scientists have identified genetic mutations in the tumors of children with T cell acute lymphoblastic leukemia (T-ALL) that both cause disease but may also make the tumors responsive to the experimental drugs.

The mutations occur in a gene called NOTCH1 and were present in 54 out of 96 tumors examined in the study. The researchers believe this gene may play a role in other cancers, and they are screening a variety of tumors for similar mutations.

The research is promising because some drugs under development for Alzheimer’s may work better and be less toxic for these children than their current chemotherapy treatments.

“Our study suggests that the same type of drugs in development for Alzheimer’s could be used to treat cancers that involve too much NOTCH activity,” says Jon Aster of Harvard Medical School in Boston, who led the study. The research appears online in Science today.

Clinical trials to test these drugs in leukemia patients should be underway within a year at the Dana Farber Cancer Institute, also in Boston. These drugs are not currently on the market, and it’s not clear when they will be.

While the new findings will not change how leukemia patients are treated today, the study is the latest in a series in which cancer researchers have identified drugs that seem to work best against tumors that have certain genetic mutations.

“This is one of a number of recently discovered cancers where you might be able to specifically hit, with drugs, the mutation that is important in driving the cancer,” says Aster.

Aster and his colleagues honed in on the mutations because other rare cases of T-ALL are caused by a mutation in the NOTCH1 gene. They hypothesized that the gene must somehow be important, at least some of the time.

It is thought that the newly discovered mutations cause the NOTCH1 gene to become hyperactive and that this contributes to the growth of tumor cells.

So why would an Alzheimer’s drug work for a leukemia patient? The Alzheimer’s drugs may help because the protein that interacts with the NOTCH1 gene in leukemia patients also apparently contributes to the toxic accumulation of proteins in the brains of people with Alzheimer’s. Drugs that inhibit this protein may help in both diseases.

Weng, A.P. et al. Activating mutations of NOTCH1 in human T cell acute lymphoblastic leukemia. Science 306, 269-271 (October 8, 2004).

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