WASHINGTON (Reuters) - Researchers have discovered a genetic mutation that causes an aggressive form of childhood leukemia and said on Thursday it may mean that drugs being developed for Alzheimer’s disease could also provide a better treatment for the cancer.
The cancer, called T-cell acute lymphoblastic leukemia or T-ALL, is cured about 75 percent of the time with chemotherapy, but it is a toxic treatment that leaves children vulnerable to other health problems later in life.
Andrew Weng of Brigham and Women’s Hospital and Harvard Medical School in Boston discovered that mutations in a gene called NOTCH1, which made it overactive, could be found in nearly 60 percent of all T-ALL tumors.
NOTCH1 helps control the development of T-cells, the immune cells that proliferate out of control in this form of cancer.
“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 published in Friday’s issue of the journal Science.
“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 researchers used gamma-secretase inhibitors to shut down NOTCH1 in cells taken from T-ALL patients and grown in the lab.
“The findings also make us curious to know if abnormal NOTCH proteins might be found in other kinds of human cancer,” Aster said.
Dr. Daniel DeAngelo of Harvard’s Dana-Farber Cancer Institute and colleagues are planning a small phase I/II safety trial of a gamma-secretase inhibitor in patients whose T-ALL has relapsed.
MeSH Headings:Leukemia, Lymphocytic, Acute: Leukemia, T-Cell: Leukemia, T-Cell, AcuteCopyright © 2002 Reuters Limited. All rights reserved. Republication or redistribution of Reuters content, including by framing or similar means, is expressly prohibited without the prior written consent of Reuters. Reuters shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon. Reuters and the Reuters sphere logo are registered trademarks and trademarks of the Reuters group of companies around the world.
