05 December 2016

Hemophilia treatment: pills instead of injections

Researchers at the University of Texas at Austin, working under the guidance of Professor Nicholas A. Peppas, have developed a biodegradable capsule that can replace patients with hemophilia In the injection of the coagulation factor they need. Switching from injections to such capsules will not only reduce the cost and simplify therapy, but also make it less traumatic.

Hemophilia A and B are hereditary diseases in which the human body lacks one of the proteins necessary for blood clotting. According to statistical estimates, there are about 400,000 people with hemophilia in the world. To eliminate symptoms such as bleeding and joint pain, as well as to prevent further joint damage, these people need regular injections of blood clotting factors. Hemophilia affects people all over the world, but globally access to adequate treatment of the disease is limited by the cost of drugs, the need for qualified medical personnel and possible complications of injections.

The authors have developed an alternative approach to hemophilia B therapy, which is a biodegradable system for oral delivery of blood clotting factor IX, which is absent in the body of patients with this type of disease. It is a capsule filled with micro- and nanoparticles carrying therapeutic protein.

The main difficulty in developing such a system was the instability of protein in conditions of changing acidity levels of the human digestive tract. The capsule shell developed by the authors protects its contents from enzymes and the acidic environment of the stomach, however, in the small intestine, under conditions of increasing pH (medium alkalinization), the capsule gradually swells and degrades under the action of digestive enzymes, slowly releasing the drug into the intestinal lumen.


Schematic representation of a biodegradable system that delivers blood clotting factor IX to the body, which is necessary to prevent bleeding in patients with hemophilia B.

According to the authors, in today's form, 2 capsules are approximately equivalent to 1 injection of clotting factor. However, they hope that in the future they will be able to increase the capacity of the system and reduce the number of capsules.

They are currently planning to conduct preclinical studies of the system and eventually hope to get approval from the U.S. Food and Drug Administration (FDA) for its clinical use.

Article by Sarena D. Horava et al. Biodegradable hydrophilic carriers for the oral delivery of hematological factor IX for hemophilia B treatment is published in the International Journal of Pharmaceuticals.

Evgeniya Ryabtseva
Portal "Eternal youth" http://vechnayamolodost.ru Based on University of Texas at Austin: UT Austin Engineers Develop First-Ever Capsule to Treat Hemophilia.


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