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Capsule technology designed to treat hemophilia

Hemophilia A or hemophilia B affects some 400,000 people world wide. These are rare blood disorder where the affected individual’s blood doesn’t clot normally because it lacks sufficient blood-clotting proteins (clotting factors). It is primarily an inherited genetic disorder. The result is people bleeding longer after an injury, easy bruising, and an increased risk of bleeding inside joints or the brain. With the two main types of haemophilia: haemophilia A occurs due to not enough clotting factor VIII, and haemophilia B, which occurs due to not enough clotting factor IX.

The primary treatment, for more severe cases, involves the administration of clotting factors. These are isolated from human blood serum, recombinant (DNA molecules formed by laboratory methods of genetic recombination), or a combination of the two. These factors replace the missing factor in the blood clotting cascade of the person with hemophilia.

The new development moves away from the need to receive regular injections and it is based on nanotechnology. It is based on a capsule made up of micro and nanoparticles. These particles administer a protein therapy called human factor IX (which means it is designed for treating hemophilia B).

Trials conducted to date show the capsule travels through the stomach to the small intestine. Here it starts to swell as the result of an increase in pH levels. The major intestinal enzyme then begins to degrade the capsule’s shell. This leads to a slow release of the protein-based drug. At present approximately two capsules would be equivalent to one injection, although the longer-term aim is to reduce the administration down to one capsule.

Interviewed by Bioscience Techniques, the lead researcher, Dr. Sarena Horava, explains: “Based on the current capabilities of this system, approximately two capsules would be equivalent to one injection. However, we anticipate that we will make further improvements to the delivery capacity of the oral delivery system and therefore decrease the capsule amount.”

The technology is primarily aimed at developing countries, which cannot afford expensive therapeutics. The new process is described in the International Journal of Pharmaceutics. The research is headed “Biodegradable hydrophilic carriers for the oral delivery of hematological factor IX for hemophilia B treatment.”

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Dr. Tim Sandle is Digital Journal's Editor-at-Large for science news. Tim specializes in science, technology, environmental, business, and health journalism. He is additionally a practising microbiologist; and an author. He is also interested in history, politics and current affairs.

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