UMass Amherst Chemical Engineering alumna Dr. Sarena Horava, now employed at Triton Systems of Chelmsford, Massachusetts, was the lead author of an article published in the November 30 issue of the International Journal of Pharmaceutics that describes groundbreaking research leading to the first-ever capsule to treat hemophilia. Horava worked on the historic process during her doctoral studies at the University of Texas Austin Cockrell School of Engineering, where she collaborated with Nicholas A. Peppas, the director of UT Austin’s Institute for Biomaterials, Drug Delivery, and Regenerative Medicine and a Cockrell School professor.
Horava is a recent Ph.D. graduate from the Cockrell School’s McKetta Department of Chemical Engineering and a National Science Foundation graduate research fellow, who finished her doctoral degree in three and a half years. The work of Peppas and Horava 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.
“While an oral delivery platform will be beneficial to all hemophilia B patients, patients in developing countries will benefit the most,” said 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.”
According to an article in the UT News, posted by the University of Texas, the breakthrough will mean “that the treatment for hemophilia can now be administered via a biodegradable system, a capsule, giving people affected by the hereditary bleeding disorder hope for a less expensive, less painful treatment option than conventional injections or infusions.”
Horava and colleagues designed the oral delivery system, which contains micro- and nanoparticles, to carry a protein therapy that treats hemophilia B. The UT News article noted that there are approximately 400,000 people worldwide living with either hemophilia A or 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.
Peppas said that alleviating the burden of injections for children was the impetus for the research project, which started about nine years ago.
The biggest challenge in delivering hFIX is that it is extremely delicate and unstable in the body’s various pH environments. The researchers’ new and 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.” (December 2016)