Hemophilia Treatment Breakthrough: Pill For Blood Disease May Soon Be On The Market
Hemophilia is a hereditary disease characterized by a reduced ability of blood to clot. Also known as the “Royal Disease” for its widespread prevalence within European royal families in the 19th and 20th centuries, the condition is treatable, although current therapies are both expensive and painful. However, according to a new study, this disorder may soon be treated by simply swallowing a pill.
The pill contains micro- and nanoparticles to carry a protein therapy that treats hemophilia B, a form of the disease that is about four times less common than hemophilia A. Hemophilia B is caused by a missing or defective factor IX, a clotting protein.
"While an oral delivery platform will be beneficial to all hemophilia B patients, patients in developing countries will benefit the most," said Sarena Horava, the study's lead author in a recent statement. "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."
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Today, treatment for hemophilia involves frequent injections, which are costly and uncomfortable. However, the team was able to figure out a way to deliver the medication orally, yet keep the pill intact until it entered the intestines where it then slowly released over time. For now, the team wants to keep improving the effectiveness of the pill.
Though rare, the psychological effects of hemophilia, especially on children and their parents, can be intense. The team hopes that making treatment more accessible and less intrusive may also improve the emotional effects of the disease on families.
Without treatment, individuals with hemophilia have trouble forming blood clots. Although small scrapes are not a big issue, internal bleeding can be life-threatening, The Mayo Clinic reported. Although there is no cure yet, regular treatment helps individuals with this condition maintain a healthy, active life.
The study will be published in the November 30 issue of the International Journal of Pharmaceutics.
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