Scientists Develop Oral Delivery System to Treat Hemophilia

Treatment for hemophilia can now be administered via a biodegradable, pH-responsive hydrogel microcarrier system, a capsule, thanks to a breakthrough by researchers at the University of Texas at Austin. They describe their system in the Nov. 30, 2016 issue of the International Journal of Pharmaceutics.

Illustration of the degradable system carrying treatment for hemophilia B. Image credit: University of Texas at Austin.

Illustration of the degradable system carrying treatment for hemophilia B. Image credit: University of Texas at Austin.

There are about 400,000 people worldwide living with either hemophilia A or hemophilia B, both caused by a missing protein in their blood.

Hemophilia B is caused by a missing or defective factor IX, a clotting protein.

The bleeding disorder affects people throughout the world, but global accessibility to therapy is limited by cost, the need for trained medical personnel and possible complications associated with needle-based drug administration.

Thousands of people endure multiple injections weekly to keep symptoms, such as excessive bleeding and pain in the joints, at bay and prevent future joint disease.

The team, headed by Dr. Lisa Brannon-Peppas, designed a capsule, which contains micro- and nanoparticles, to carry a protein therapy that treats hemophilia B.

“While an oral delivery platform will be beneficial to all hemophilia B patients, patients in developing countries will benefit the most,” said study’s first author Sarena Horava.

“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.”

The team’s work has been patented and builds upon their published and patented system for the oral delivery of human factor IX (hFIX), a prophylactic treatment for hemophilia B patients.

That system was successful in transporting hFIX, and it was able to deliver adequate levels of the drug to the target site in the body.

The biggest challenge in delivering hFIX is that it is extremely delicate and unstable in the body’s various pH environments.

The team’s improved system is designed to capitalize on the body’s pH and changes in enzymes inside the gastrointestinal tract for a smooth delivery.

As it moves through the body, the particle-containing capsule resists the major gastric enzyme to remain intact while in the stomach, providing protection for the encapsulated drug.

In the small intestine, the capsule begins to swell with the increase in pH and is then degraded by the major intestinal enzyme, slowly releasing the drug over time.

“Based on the current capabilities of this system, approximately two capsules would be equivalent to one injection,” Horava said.

“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 scientists plan to further test their system before clinical trials.

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Sarena D. Horava et al. 2016. Biodegradable hydrophilic carriers for the oral delivery of hematological factor IX for hemophilia B treatment. International Journal of Pharmaceutics 514 (1): 220-228; doi: 10.1016/j.ijpharm.2016.05.056

This article is based on a press-release from the University of Texas at Austin.

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