First biodegradable capsule developed to treat haemophilia B

A capsule has been developed that can treat people affected by the hereditary bleeding disorder haemophilia.

Researchers from the Cockrell School of Engineering at The University of Texas developed a treatment for haemophilia that can be administered via a biodegradable capsule. The hope is that the capsule will result in both a less expensive and a less traumatic treatment in contrast to traditional treatments such as injections and infusions.

The capsule contains micro and nanoparticles which carry a protein therapy that treats haemophilia B. The capsule is able to remain intact whilst in the stomach before moving down to the small intestine. Whilst there the capsule begins to swell with the increase in pH before being degraded by the major intestinal enzyme to slowly release the drug over time.

Whilst current treatment is available developing countries are limited due to the cost and need for trained medical personnel.

The disease, which affects around 400,000 people worldwide, is caused by a missing protein in patients’ blood. Heamophilia B is specifically caused by a missing or defective factor IX, a clotting protein.

Sarena Horava, the lead author of the study said: “While an oral delivery platform will be beneficial to all haemophilia B patients, patients in developing countries will benefit the most. In many developing countries, the median life expectancy for haemophilia 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.”

Horava collaborated with co-author and co-inventor Nicholas A. Peppas, the director of UT Austin’s Institute for Biomaterials, Drug Delivery and Regenerative Medicine and Katie J. Moy, an undergraduate in the Cockrell School’s Department of Biomedical Engineering, and who is also a co-author on the study.

Peppas’ incentive for the research was to alleviate the burden of injections for children.

“My most pressing concern was the treatment of younger patients who suffer from haemophilia and who have to apply injections every two days.” Peppas said.

“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 team intend to test the capsule further before clinical trials. Together with the UT Austin Office of Technology Commercialisation they are working towards advancing the technology for clinical use before positioning it for Food and Drug Administration approval.

The researchers describe their system in the Nov. 30 issue of the International Journal of Pharmaceutics.