FLT3, which controls the development of healthy blood cells, was identified as a treatment target in patients with acute myeloid leukemia, a blood cell cancer, several years ago by the same Johns Hopkins investigators. In the current work, the Hopkins team has confirmed that the gene is activated in dendritic cells, whose role is to distribute "look here" information about unwanted foreign invaders to soldiering T-cells.
"Someday, using a drug to block FLT3 gene signaling could stop dendritic cells from triggering harmful responses against a patient's own body," says Donald Small, M.D., Ph.D., professor at the Johns Hopkins Kimmel Cancer Center, whose findings appear in the November 15 issue of the Proceedings of the National Academy of Sciences. Preliminary clinical tests in people with autoimmune diseases with just such a drug could begin in the next year, Small said.
A characteristic of autoimmune diseases is that patients' immune T-cells mistake normal cells in the body for foreign ones. Current therapies, such as steroids, are designed to suppress T-cell responses. But the Hopkins investigators believe that targeting dendritic cells may stop the faulty immune response at a higher "upstream" level since T-cells frequently receive their information from dendritic cells.
Testing their idea, Small and his Hopkins colleague Katherine Whartenby used an experimental compound called CEP-701, already known to block actions of the growth-promoting FLT3 gene, on human dendritic cells and in mice engineered to mimic m
Source:Johns Hopkins Medical Institutions