Transferrin therapy ameliorates disease in β-thalassemic mice

This is incredible. And seemingly so simple that I wonder why it hasn't gotten more publicity yet. If there was ever a therapy that deserves immediate funding and fast track development, this is it.

http://www.ncbi.nlm.nih.gov/pubmed/20098432

Transferrin therapy ameliorates disease in beta-thalassemic mice.

Li H, Rybicki AC, Suzuka SM, von Bonsdorff L, Breuer W, Hall CB, Cabantchik ZI, Bouhassira EE, Fabry ME, Ginzburg YZ.

Erythropoiesis Laboratory, Lindsley F Kimball Research Institute, New York Blood Center, New York, New York, USA.
Abstract

Individuals with beta-thalassemia develop progressive systemic iron overload, resulting in high morbidity and mortality. These complications are caused by labile plasma iron, which is taken up by parenchymal cells in a dysregulated manner; in contrast, erythropoiesis depends on transferrin-bound iron uptake via the transferrin receptor. We hypothesized that the ineffective erythropoiesis and anemia observed in beta-thalassemia might be ameliorated by increasing the amount of circulating transferrin. We tested the ability of transferrin injections to modulate iron metabolism and erythropoiesis in Hbb(th1/th1) mice, an experimental model of beta-thalassemia. Injected transferrin reversed or markedly improved the thalassemia phenotype in these mice. Specifically, transferrin injections normalized labile plasma iron concentrations, increased hepcidin expression, normalized red blood cell survival and increased hemoglobin production; this treatment concomitantly decreased reticulocytosis, erythropoietin abundance and splenomegaly. These results indicate that transferrin is a limiting factor contributing to anemia in these mice and suggest that transferrin therapy might be beneficial in human beta-thalassemia.

Hi andy how r u donig ....?
Nice shering but i dont understand ....
wht is Transferrin therapy 
so now we are in oct ...gene therapy in started with first pat im thl major ....i think sooo ........!
im wating 4 update .........?

Transeferrin is the protein that transports iron through the blood. What this study implies is that it may be possible to correct many of the problems in thalassemia by injecting transferrin into patients. This study was done using mice, but hopefully these results can eventually lead to trials in humans. My surprise is that this research has not gotten more notice, as this could prove to be a simple treatment for thalassemia. Let's hope this does get attention and is fast tracked. In theory, if this works in humans, it would eliminate many of the therapies used today.

"Transferrin could offer an alternative way to overcome these treatment-induced complications by redirecting iron from inefficient or deleterious storage places to the active synthesis of hemoglobin."
http://www.sanquin.nl/sanquin-eng/sqn_news.nsf/All/Sanquin-S-Transferrin-Shows-Positive-Results-In-%CE%92-Thalassaemia-Disease-Model.html

so what it'lll do is that the excess iron in the body will be taken up by transferrin and transferred to the haemoglobin synthesizing area and then the abnormal hemoglobin will be formed?
what about beta zeros?
will it reduce chelation needs and transfusion needs for some thals?
i'm confused...

Basically, what has been shown with transferrin injections in mice is that this corrects several of the problems present in thalassemia.

Specifically, transferrin injections normalized labile plasma iron concentrations, increased hepcidin expression, normalized red blood cell survival and increased hemoglobin production; this treatment concomitantly decreased reticulocytosis, erythropoietin abundance and splenomegaly.

"... increased hepcidin expression" would allow the body to properly regulate iron absorption resulting in "normalized labile plasma iron concentrations". The body normally does not absorb more iron than it needs but in thalassemia this gets out of control, as hepcidin levels drop. Normalizing hepcidin levels would eliminate this excess iron absorption. Iron deposits would be cycled into red cell production but at a normal rate. The other side is a normalization of bone marrow activity, resulting in reduction or elimination of the production of defective red cells.

These changes would greatly reduce or eliminate the need for chelation, reduce the amount of bad red cells produced and reduce the problems associated with ineffective erythropoiesis and hemolysis. I can't say if this has any potential for increasing hemoglobin levels in beta zero but it would reduce the other problems, which are the main dangers of thalassemia.

thank you Andy!