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

2010-04-25 | |
Last updated: 2010-04-25

As a very well known, yet rare, medical condition, Hemophilia gained its notoriety because the disease affected members of several European royal families of the last century. The blood clotting or coagulating disorder is often fatal if untreated, but since the 1960’s, life expectancy for people with the condition has increased dramatically due to the creation of reasonably effective medical treatments.

However, even with the medical advances achieved to date, the effect that this genetic disorder has on health is still considerable. In the last article on Hemophilia costs and treatments, several significant problems with the current treatments were described. Because of these important remaining issues, research is still underway in an effort to tame the disease.

What Are The Problems With Current Hemophilia Treatments?

What Are Researchers Doing to Improve The Treatment Of Hemophilia

Researchers Working To Make Hemophilia Treatments Safer

To improve the safety of existing treatments, researchers at the University of Florida and University of Arizona are attempting to the improve the tolerance of patient immune systems to the current treatment. Treatment normally involves patients receiving infusions of clotting protein, but the immune system can attack the protein either rendering it ineffective or causing a potentially deadly allergic reaction.

Their current efforts have involved creating genetically altered plants that generate the coagulating protein inside the plant cells. When these plants are later consumed, the intact plant cells and protein can make it past the acid and digestive juices of the stomach into the intestines. Once there, bacteria break down the cell wall releasing the protein.

At that point, the immune system learns to recognize the protein and becomes tolerant of it. In the studies involving mice, subsequent infusions of clotting protein into the blood have caused no allergic reactions. This offers the hope of treatment to individuals who might otherwise have no further options.

While not eliminating the costs of the treatment, this advance might soon mean that infusion of the clotting protein need not require hospital supervision.

Hemophilia Research Efforts Seek To Improve Body’s Creation Of Clotting Proteins

In contrast to these efforts to train the body to more readily accept current treatments, other approaches involve enabling the bodies of those with Hemophilia to generate clotting protein directly. In this case, the potential treatment developed at Yeshiva University requires transplanting some specific cells from a donor liver to the liver of the patient. In those without Hemophilia, it is these liver cells that generate clotting protein.

In their research, the scientists found that mice livers accepted the transplants readily and within 3 months were producing levels of clotting protein equivalent to that in mice without Hemophilia. The treatment effectively eliminates the disease, but, as is the case for any transplant, requires immune suppressors to be taken for life. Though, not ideal, it is far less invasive and expensive than requiring protein transfusions several times each week.

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Category: Disease Information, Medical Research, Medical Treatment

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