Researchers from the Cockrell School of Engineering at the University of Texas at Austin have developed a biodegradable capsule to deliver a protein therapy for hemophilia. The research, published in the International Journal of Pharmaceutics, could provide a cheaper, less painful alternative to injections or infusions.
The capsule contains micro and nanoparticles which carry a protein therapy to treat hemophilia B. The disease is caused by a missing or defective factor IX, a clotting protein, in the blood. Access to the protein therapy is limited due to its cost and the complications brought on by needle-based drug delivery.
The team’s work has been patented, building on a previous system for the oral delivery of human factor IX for hemophilia B patients. The new system is designed to take advantage of the body’s change in acid levels and enzymes inside the gastrointestinal tract. As the capsule moves through the body, it can resist a major gastric enzyme and stay intact while in the stomach. In the small intestine, the capsule swells as the acid level increases. The major intestinal enzyme then slowly degrades the capsule, releasing the drug over time.
“Based on the current capabilities of this system, approximately two capsules would be equivalent to one injection,” lead author Sarena Horava said in prepared remarks. “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 said they will continue to test the system before moving into clinical trials, with an ultimate goal of FDA approval.
“While an oral delivery platform will be beneficial to all hemophilia B patients, patients in developing countries will benefit the most,” Horava added. “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.”