May 4, 2018
Today the treatment of people with hemophilia is restrictive and expensive. A new study could be a game-changer.
Changing the daily life of people with hemophilia
The blood of people with hemophilia does not clot. Hemophilia is a genetic disease that mainly affects men and can lead to bleeding, sometimes very severe, which can lead to death if not treated in time. Time-consuming and expensive, the current treatment is difficult to live with (especially for children and adolescents) and loses effectiveness over time.
Today, people with hemophilia must receive frequent injections every week to fill the lack of proteins usually present in plasma and allow the blood to form a clot. These injections contain animal cells and help restore coagulation.
The study that could make a difference
But a study could change this painful daily life. Researchers affiliated with Salk Institute for Biological Studies, in California, conducted work on mice and their findings are encouraging for further clinical research. They plan to treat hemophilia with a single injection liver stem cells.
In mice, the researchers transplanted these cells through the spleen. It is in fact this organ which sends these cells to the liver by following the “portal system”. Results: the cells produced the clotting factor well and the protein was in sufficient quantity. Research continues and soon more work will lead to clinical trials in men.
Maylis Choné
Read: What is hemophilia?
