Breakthrough Biodegradable Capsule Aids Hemophilia Treatment

A new study describes how an innovative biodegradable capsule that uses hydrophilic carriers for oral delivery of hematological factor IX (hFIX) can treat hemophilia B.

Developed by researchers at the University of Texas (UT; Austin, USA), the oral delivery system is based on a pH-responsive polymer hydrogel microcarrier system that carries the prophylactic hFIX particles. The poly (methacrylic acid)-grafted-poly(ethylene glycol) [P(MAA-g-EG)] polymer, synthesized using ultraviolet (UV) polymerization, is subsequently joined with an enzymatically degradable peptide crosslinking agent that allows for site-specific degradation (by trypsin) in the small intestine.

As it moves through the body, the particle-containing capsule resists the major gastric enzyme in order to remain intact while in the stomach, protecting the encapsulated drug. When in the small intestine, the capsule begins to swell with the increase in pH, and is subsequently degraded by the minor intestinal enzyme, slowly releasing the drug over time. In study models, the system successfully transported hFIX, and was able to deliver adequate levels of the drug to the target site in the small intestine. The study was published on November 15, 2016, in the International Journal of Pharmaceutics.

“While an oral delivery platform will be beneficial to all hemophilia B patients, patients in developing countries will benefit the most,” said lead author Sarena Horava, PhD, of the UT Cockrell School of Engineering. “In many developing countries, the median life expectancy for hemophilia patients is 11 years due to the lack of access to treatment, but our new oral delivery of factor IX can now overcome these issues and improve the worldwide use of this therapy.”

Hemophilia B is a blood clotting disorder caused by a mutation of the factor IX gene, leading to a deficiency of factor IX. It is the second-most common form of hemophilia, and is sometimes called Christmas disease, after Stephen Christmas, the first patient described with the affliction. Presentation of hemophilia B is consistent with easy bruising, urinary tract bleed, and nosebleeds. Current protein replacement therapies rely on intravenous (IV) injections and infusions.

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