- Hemophilia is an inherited bleeding disorder where the blood does not clot properly due to the absence of a clotting protein or a defective human factor IX.
- Injections used for Hemophilia treatment are often painful and expensive.
- Researchers find new capsule to deliver the clotting protein for treating hemophilia.
A new capsule containing micro- and nanoparticles to treat hemophilia B was designed by a team of researchers from The University of Texas at Austin.
Hemophilia is a bleeding disorder that affect millions of people around the world. Around 400,000 people are living either with Hemophilia A or Hemophilia B in the world. It is a hereditary condition where the blood does not clot properly due to a missing defective factor IX or clotting protein.
‘Oral capsule shows hope to develop less expensive and less painful treatment for hemophilia.’
The study findings were published in the International Journal of Pharmaceutics.
Treatment options for Hemophilia are expensive and require the help of health care professionals for administering injections. Thousands of people suffer due to continuous bleeding and joint pains due to hemophilia.
The research team led by the Cockrell School of Engineering at The University of Texas at Austin were able to develop a biodegradable capsule which can help in the treatment of hemophilia.
Sarena Horava, the study author said,"While an oral delivery platform will be beneficial to all hemophilia B patients, patients in developing countries will benefit the most."
"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."
Peppas, director of UT Austin's Institute for Biomaterials, Drug Delivery and Regenerative Medicine, Cockrell School professor also said that this research project was mainly carried out to reduce the pain of injections.
"My most pressing concern was the treatment of younger patients who suffer from hemophilia and who have to apply injections every two days," he 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."
The research work was patented for the oral delivery of human factor IX (hfIX) to hemophilia B patients. This system was successfully able to send the drug to the targeted site. However, the only drawback lies in delicately delivering the hfIX in the body's different pH system.
The researchers have designed the drug in such a way that it enters the body's pH and also resist the gastric enzyme to remain inside the stomach, protecting the encapsulated drug. When the drug reaches the intestine, it swells, increases pH and releases the drug over time.
The author stated that two capsules would be equivalent to one injection that is used for hemophilia and further research will be carried out to reduce the capsule content and clinical trials will be conducted to indicate its use and receive approval from the Food and Drug Administration.