Treatment reverses blood disorder in mice, study finds
Tue Apr 8, 2008 12:11am BST Email | Print | Share| Single Page|
Recommend (0) [-] Text [+] By Julie Steenhuysen
CHICAGO (Reuters) - An experimental treatment in mice showed promise
in reversing a rare blood disease that can cause leukemia, U.S.
researchers said on Monday, offering a glimpse of how the drug may
work as it begins testing in humans.
In experiments at Harvard Medical School in Boston and the University
of California, San Diego, researchers found the compound blocked a
genetic mutation that causes three kinds of leukemias.
The studies tested a compound supplied by privately held
biotechnology company TargeGen in San Francisco, which has just begun
early testing in humans.
It targets a genetic mutation in the JAK2 gene that causes most cases
of the leukemias polycythemia vera, essential thrombocytosis and
primary myelofibrosis, which affect up to 100,000 people in the
United States.
The mutation in the JAK2 gene causes an overproduction of blood
cells. The TargeGen compound TG101348 is designed to block the JAK2
mutation that causes this.
The idea is to set errant cells back on the right path, said Dr.
Catriona Jamieson of the University of California, San Diego, whose
paper appears in the journal Cancer Cell.
"By using how stem cells work and how they can go awry, we can
redirect them using very targeted inhibitors that get these cells
back on track," Jamieson said in a telephone interview.
Her experiments showed the compound reversed the disease in cell
cultures. Her team also found the drug reversed disease in mice that
had an engineered version of the mutant gene and those injected with
stem cells from humans with the disorder polycythemia vera
In a separate study in the same journal, Dr. Gary Gilliland and
colleagues induced polycythemia in 168 mice. They gave high and low
doses of the compound TG101348 daily for seven weeks to 112 mice. All
the mice that got the higher dose survived, six in the placebo group
died and one in the low dose group died.
"We saw that the compound alleviates and reverses the symptoms in
mice," said researcher Gerlinde Wernig, who worked on the study.
"These are completely complimentary results," Jamieson said of the
two studies, which helped lay the foundation for the human clinical
trials which began last month.
Researchers at a half dozen prestigious medical centers will
participate in the human studies, which will test a small dose of the
drug in a few patients to see if it causes toxic side effects.
"Our goal is to get an efficacious drug into use for humans," said
Gilliland, who is also a Howard Hughes Medical Institute researcher.
http://uk.reuters.
http://uk.reuters.
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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