Gene Therapy Promises Effective Hemophilia Treatment

A recent landmark study, researchers found out the gene therapy can successfully treat patients with severe hemophilia, a blood-clotting disorder.

So far, hemophilia (hémophilie, hemofilie, hemofili, hemofilia, hämophilie, emofilia) is an inherited, fatal disorder affecting mostly males. The cause is indicated for a faulty gene. In severe hemophiliacs, they can spontaneously start bleeding internally, even in the brain. Internal bleeding in the joints leads to debilitating movement problems and intense pain.

Replacement therapy is commonly known as the treatment for Hemophilia by  the expensive protein injection until the latest method made gene therapy a milestone ever.

Many questions and articles are released to discuss about this topic and come into a common conclusion that: gene therapy can successfully treat hemophilia.

How was the study process?

That new study was led by researchers at the University College London Cancer Institute and St. Jude Children’s Research Hospital in Memphis, Tennessee with participation of six male patients who had severe forms of type B hemophilia.

Hemophilia B is the second most common type of hemophilia and a hereditary bleeding disorder caused by a lack of blood clotting factor IX.

All six had increased levels of FIX in their blood after treatment, and these levels continued throughout the follow-up period (up to 16 months). Four of the six participants were able to stop their regular injections of FIX protein, and the other two required fewer injections than before even though these two also faced some side effects after the experimental treatment including mild immune responses that required steroid treatment.

In Dr.Andrew Davidoff, leader researcher’s word, it was a small trial in that only six patients had been treated thus far, but it had been rather successful and it would encourage further use of this approach in the very near future, both by us and others. Anyway, it was a good start.


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