Gene mutation causes Childhood Leukemia, Alzheimer's drugs may help

Main Category: Public Health
Article Date: 08 Oct 2004 - 10:00 PDT

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A gene mutation causes Childhood Leukemia (T-cell acute lymphoblastic leukaemia), say researchers at Brigham and Women's Hospital and Harvard Medical School, Boston, USA. They suggested that Alzheimer's drugs (in development) could be effective for the treatment of childhood leukaemia.

T-cell acute lymphoblastic leukaemia (T-ALL) is currently treated with chemotherapy. Although the treatment is effective, children are prone to health problems later in life.

60% of T-ALL tumours have a mutation in a gene called NOTCH1, this makes it overactive, said Andrew Weng, team leader. NOTCH1 assists in the regulation of T-cells.

For NOTCH1 to work it has to be chopped up. Proteins involved in Alzheimer's Disease are chopped up by a gamma-secretase enzyme. This enzyme also chops up NOTCH1.

Current research into Alzheimer's drugs has been focussing on developing a medication that can affect gamma-secretase.

You can read about Dr. Weng and team's research in the journal Science.

. Dr. Weng said "This discovery is significant because first, it tells us that NOTCH1 mutations are very important in all forms of T-ALL and secondly, gamma-secretase inhibitors, a class of drugs known to turn off abnormal NOTCH1 activity caused by the mutation, are already in the pipeline," said pathologist Jon Aster, who worked on the report. We are very hopeful that these drugs will prove to be a safe and effective treatment for T-ALL in the next year or so."

The team shut down NOTCH1 in cells taken from T-ALL patients grown in the lab with gamma-secretase inhibitors. They now want to see if abnormal NOTCH proteins might be found in other kinds of human cancer.

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