A Pill Is Underway To Treat The Serious Inherited Bleeding Disorder

A pill is being developed by scientists to treat the serious inherited bleeding disorder. So, instead of being having several weekly injections of factor IX, a pill would control the fatal bleeding episodes of those individuals with hemophilia B.

The development of the pill is led by Nicholas Peppas, a professor of Biomedical Engineering, Chemical Engineering and Medicine and his colleagues. Meanwhile, David Rampulla, Ph.D., the Director of the National Institute of Biomedical Imaging and Bioengineering Program in Delivery Systems and Devices for Drugs and Biologics, explained that the problem with oral delivery is the need to protect proteins such as factor IX in the acidic environment of the stomach but then release them when they reach the small intestine. He further said that this is an extraordinary challenge and the Peppas group has spent years developing innovative polymer shells capable of shuttling the protein to its destination in the digestive tract.

Hemophilia B or also known as Christmas disease is derived from Stephen Christmas, the first patient diagnosed with this disease. It is a blood clotting disorder and caused by mutation of the factor IX gene, which leads to a deficiency of factor IX that the body needs for blood to clot.

It mostly occurs in boys and inherited from mothers. On the other hand, the mothers do not have the disease because they have a normal factor IX gene on their other X chromosome. Meanwhile, daughters who have this disease might inherit it from their father who has it and her mother carries the gene for hemophilia, too.

Dr. Peppas explained that multiple weekly injections of factor IX are very difficult for the boys who need the clotting factor to avoid potentially bleeding episodes, as well as for their families. They also learned that this was an emotionally draining aspect for mothers, who carry the disease and passed this on to their sons. He said that this has added an urgency to their research because they know that oral administration of factor IX would be a great relief for these families.

Currently, the researchers are using their skills in advanced materials and chemical and biomolecular engineering to develop a capsule. Their work is now described in the International Journal of Pharmaceutics, according to Science Daily.