Illustration of the degradable system carrying treatment for hemophilia B. Credit: Cockrell School of Engineering
In the near future, hemophiliacs could be able to treat their disease by simply swallowing a capsule. Thanks to a breakthrough led by researchers in the Cockrell School of Engineering at The University of Texas at Austin, 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.
They designed the oral delivery system, which contains micro- and nanoparticles, to carry a protein therapy that treats hemophilia B. There are ~400,000 people worldwide with hemophilia A or B. Hemophilia B is caused by a missing or defective factor IX, a clotting protein. People endure multiple injections weekly to keep symptoms, such as excessive bleeding and pain in the joints, at bay and prevent future joint disease. “While an oral delivery platform will be beneficial to all hemophilia B patients, patients in developing countries will benefit the most,” said Sarena Horava, PhD. “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.”
“My most pressing concern was the treatment of younger patients who suffer from hemophilia and who have to apply injections every two days,” Peppas said. “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.”
Peppas and Horava’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 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 2 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.” They plan to further test this system before clinical trials. They are working with the UT Austin Office of Technology Commercialization to further advance the technology for clinical use. The researchers indicate that their ultimate goal is to position this technology for FDA approval.
https://news.utexas.edu/2016/11/28/engineers-develop-first-ever-capsule-to-treat-hemophilia
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