Scientists pinpoint malfunctioning enzyme in blood disorder that is precursor to leukemia

CINCINNATI — The Cincinnati Children's Hospital Medical Center announced today that scientists have successfully targeted a malfunctioning immune system enzyme to kill diseased cells from patients with myelodysplastic syndrome — a blood disorder and precursor to leukemia.

Reporting their results July 8 in Cancer Cell, researchers say their successful laboratory tests in human MDS cells and mouse models of MDS provide a molecular target for designing new drugs to battle a syndrome with few effective treatments.

"There is an urgent need to develop new targeted therapies that can eliminate MDS-initiating clone cells and provide a durable therapeutic response," said Daniel Starczynowski, PhD, lead researcher and a member of the Division of Experimental Hematology and Cancer Biology at Cincinnati Children's Hospital Medical Center. "Not only does our research implicate errant immune system signaling in MDS cells, it strongly indicates that inhibiting the function of this hijacked immune pathway may become an effective treatment option for MDS."

MDS is a group of syndromes in which a person's immature blood cells (blood stem cells) do not mature into healthy red or white blood cells. Instead, the immature cells die off in the bone marrow or blood, leaving an insufficient number of healthy cells in the body. This can cause infections, anemia, bleeding disorders or acute myeloid leukemia.

MDS can affect children, but is more common in people ages 60 years and older. Some research studies indicate that the prevalence of MDS is increasing as life expectancies become longer. Caused by various gene mutations, the only cure for MDS is bone marrow transplant — a risky procedure that often is not a viable option, especially for older people.

 

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