Revolting Against Hemophilia, ‘The Royal Disease’ – Researching Better Treatments

April 25th, 2010

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Genetic Changes Made By Researchers Eliminate Hemophilia

Beyond these approaches, other research efforts underway involve genetic modifications to a patient’s blood clotting system. Quite obviously, eliminating the disease is the surest way to eliminate all ongoing treatment costs.

One such approach involves modifying patient bone marrow to produce platelets with the clotting protein built in. Platelets are the cells that normally accumulate at the site of an injury to form the physical blood clot. The clotting protein controls their ability to form a clot. In contrast to gene therapies where patients are infected with a virus that alters their genes, this specific technique involves extracting bone marrow, altering it genetically and reintroducing it to the body.

In this case, scientists at the Wisconsin Blood Research Institute exposed the extracted platelet-producing stem cells to the clotting factor protein. This effectively “trained” these stem cells to produce platelets equipped with the clotting protein. Their research determined, in animal studies, that the coagulating abilities of the blood became permanent. The disease was effectively eliminated.

Another approach that uses genetic alteration and could also rid patients of the disease involves targeted nanoparticles. In this case, researchers from the University of Minnesota attached chemicals to the nanoparticles so that they would target the specific liver cells normally responsible for creating the clotting protein. Also attached to these nanoparticles was a gene responsible for correcting the deficiency in the liver cells that limits their production of clotting protein.

Once the nanoparticles were delivered, the liver cells were genetically altered to correct their genetic error. In their studies, the researchers found that almost 1 year after the treatment, formerly Hemophiliac mice had normal blood clotting abilities. Again, the disease was effectively eliminated.

Conclusions

Hemophilia is still very much a disease controlling the lives of patients and taxing the resources of health care systems. However, knowledge and research are slowly chipping away at its power and offering the hope of freedom from the tyranny of the disease. While all quiet revolutions take time, Hemophilia now seems to be a disease whose reign is quickly coming to an end. For those affected by Hemophilia, that time cannot come soon enough.