According to a recent article in the British publication Latest Page News, doctors at the University College in London and Royal Free London believe that a ‘cure’ is possible within three years for most adults who have hemophilia B.
NORD (National Organization for Rare Disorders) also featured an article describing hemophilia as a deficiency in the Factor IX protein.
Bleeding from a cut is usually resolved when clotting factors together with platelets stop the bleeding. If a person has hemophilia their clotting factors are not sufficient and they are at serious risk of uncontrolled bleeding.
Stated otherwise, changes in the factor IX gene which is on the X chromosome cause hemophilia. The disease is known for slowing the body’s natural blood clotting process.
Another name for the disorder is “Christmas Factor”. It originated in 1952 when young Stephen Christmas who was found to be deficient in the IX Factor became the first person to be diagnosed with hemophilia.
Continued Infusions
Currently, most hemophilia treatments require continued infusions of the IX Factor protein. A description of the new one-time form of therapy appeared in an article published in the NEJM. Researchers believe that within three years most people with hemophilia B may be cured.
The credit belongs to FLT180a (verbrinacogene setparvovec). FLT180a is a liver-directed AAV one-time gene therapy that normalizes factor IX levels.
Factor IX is the blood protein known to be lacking in sufficient quantities in hemophilia patients. The clinical trial initially conducted for twenty-six weeks included ten patients who had either severe or moderately severe hemophilia B.
The initial trial was followed by a twenty-seven-month study involving the same group of patients. With one exception, all other patients had sustained factor IX levels at the end of the twenty-seven-month period.
About Adverse Events
Ten percent of the adverse events that were reported during the study were due to the treatment. However, one-quarter of other adverse events were related to immunosuppression (glucocorticoids) which are man-made steroids occurring naturally in the body.
Projections about the efficacy of FLT180a indicate that its benefits could last for a decade. These estimates have yet to be proven but researchers remain optimistic. Gene therapies in general have proven to have lasting benefits. Current clinical trials include studies of varied diseases from glaucoma to cancer.
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Rose Duesterwald August 3, 2022
