Discovery of a pill, requiring oral intake to treat type B hemophilia is underway, which will put an end to the current tedious treatment of administration of weekly injections to patients.
What Is Type B Hemophilia?
Type B hemophilia is a hereditary bleeding disorder caused by deficiency of blood clotting factor IX. It results in improper clotting of blood, leading to prolonged bleeding. The other symptoms of hemophilia B include nose bleeding, blood in stool or urine, bleeding into joints accompanied with swelling and pain, spontaneous bleeding, etc.
Hemophilia B disorder occurs in boys who have a defective factor IX gene that is found on the X chromosome obtained from the mother. The factor IX protein is needed in the body for clotting of blood. Mothers are usually the carriers of the disease but they don't have the disorder because they have a functioning factor IX protein gene on their other X chromosome. In extremely rare cases, daughters might inherit the disorder if their father has the disorder and the mother carries the gene too.
Oral Pills May Replace Weekly Injections
The treatment of hemophilia B involves weekly administration of several injections of factor IX protein. However, this technique is not an easy and convenient one for patients to follow on a regular basis.
Looking at the sufferings of hemophilia patients and their families, NIBIB-funded researchers are developing a pill which can help in the treatment of this debilitating bleeding disorder. The patients will merely need to swallow a pill to increase the factor IX protein in their body.
Nicholas Peppas, Professor of Biomedical Engineering, Chemical Engineering and Medicine, accompanied by his team is determined to perfect the pill that can be used for treating hemophilia B. The existing work is published in the International Journal of Pharmaceutics.
Peppas explained that the motivational factor in designing the pill was not only seeing the sufferings of the patients and their families, but also the emotional stress levels of mothers.
"However, in working with these families, we soon learned that there was also an emotionally draining aspect for mothers, who carry the burden that they passed this disorder on to their sons. This has added an urgency to our research because we know that oral administration of factor IX would be a great relief for these families" said Peppas in a press release.
Challenges Faced By The Researchers
The researcher and his group are facing a number of challenges in developing the factor IX proteins pill. The important factor is to protect the proteins from getting dissolved in the acidic background of the stomach so that they can be released rightfully when they reach the small intestine.
The researchers have been trying for years to develop an inventive polymer case that can shield the protein content and help it reach its intended destination in the digestive tract.
