Can Hemophilia B Be Treated with an Oral Capsule? Researchers Hope So

Researchers have developed a biodegradable capsule to deliver a protein missing in patients with hemophilia B, according to a new study published in the International Journal of Pharmaceutics. If it proves to be successful, this capsule may provide a cheaper and less painful option to injections or infusions.

The study, “Biodegradable Hydrophilic Carriers For The Oral Delivery Of Hematological Factor IX For Hemophilia B Treatment,” was conducted by researchers at the Cockrell School of Engineering at the University of Texas at Austin.

Hemophilia B is caused by a missing or defective protein called factor IX, which plays an important role in blood clotting, and hemophilia B patients bleed longer, often dangerously so, than other people when injured. The main treatment for this disease is the delivery of externally produced factor IX by intravenous administration. However, these procedures are expensive and painful.

The new capsule contains micro- and nanoparticles to carry factor IX, and opens the possibility of treating hemophilia orally and less expensively.

According to the authors, the capsule takes advantage of changes in acid levels throughout the gastrointestinal tract. While it remains intact in the stomach, resisting the action of gastric enzymes, the capsule swells in the small intestine with the increase in acidic levels. The intestinal enzymes then degrade the capsule, gradually releasing the protein inside and ensuring a smooth delivery of the treatment.

“While an oral delivery platform will be beneficial to all hemophilia B patients, patients in developing countries will benefit the most,” Sarena Horava, the lead author of the study, said in a news release.

“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,” she said.

The capsule would also be a better option for children, who have a hard time with current treatment injections.

“My most pressing concern was the treatment of younger patients who suffer from hemophilia and who have to apply injections every two days,” said Nicholas Peppas, the other inventor of the capsule.

“The original idea of the project was conceived when Dr. Lisa Brannon-Peppas, who at the time was a biomedical engineering faculty member, discussed with me the side effects of the disease and the psychological impact it has on mothers.”

According to Horava, two capsules are approximately equivalent to one injection, but the team has plans to improve the delivery system and decrease the capsule amount. They also plan to test the new technology in clinical trials to receive approval from the U.S. Food and Drug Administration (FDA).