An experimental drug designed to help regulate the blood's iron supply shows promise as a viable first treatment for anemia of inflammation, scientists say.
Anemia is a condition that occurs when red blood cells are in short supply or do not function properly.
When an individual has anemia, the body does not get enough oxygen, since there are fewer red blood cells to carry the iron-rich protein hemoglobin that helps distribute oxygen throughout the body.
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When the body fights a disease, it deploys an inflammatory response that triggers increased secretion of a hormone called hepcidin that reduces the amount of iron available in the bloodstream.
As iron is needed for the production of red blood cells in the bone marrow, many patients develop anemia.
The only current treatment strategy for anemia of inflammation involves targeting the underlying disease or infection.
As the principal regulator of iron, hepcidin has become a target for researchers developing novel therapies for blood disorders.
One hepcidin inhibitor, called lexaptepid pegol (lexaptepid), has demonstrated efficacy in treating anemia of inflammation in animal studies.
Lexaptepid inactivates hepcidin, thereby maintaining the transport of iron to the bloodstream.
In order to evaluate lexaptepid's potential in humans, investigators induced a safe and temporary model of anemia of inflammation in 24 healthy male adults and randomised them to receive lexaptepid or placebo.
Volunteers received a low dose of Escherichia coli (E coli) endotoxin to induce controlled inflammation and received either lexaptepid or placebo 30 minutes later.
After nine hours, iron in the blood stream had decreased in the placebo group, whereas this decrease could be prevented by treatment with lexaptepid.
In addition to determining whether lexaptepid interfered with hepcidin production, researchers also sought to determine whether the drug influenced the immune response.
All volunteers experienced similar flu-like symptoms, increased body temperature and white blood cell count, and higher concentrations of inflammatory and signalling proteins, demonstrating to investigators that lexaptepid did not interfere with the immune response process.
"It is quite encouraging that lexaptepid helped maintain appropriate levels of iron in the bloodstream of healthy volunteers without compromising the immune response," said lead study author Lucas van Eijk, of Radboud University Medical Center in Nijmegen, Netherlands.
The study is published in Blood, the Journal of the American Society of Hematology.