Just one jab could be enough for the people suffering haemophilia B and they need no further medication, revealed an early study in US and UK. It has been shown that a single treatment with gene therapy which is an experimental method of fixing flawed genes, improved output of crucial blood clotting factor, potentially offering long-term solution for haemophilia b.
People suffering haemophilia B have a fault in their genetic code, means they cannot produce a protein known as factor IX, which is significant for blood-clotting. The patients of haemophilia B are at presently treated with injections of factor IX, sometimes such patients are given that injection, multiple times a week.
However, the process of its manufacturing is very expensive. Therefore researchers from at University College London and St Jude Children’s Research Hospital in the US were searching for a more permanent solution for the condition. Researchers replaced the faulty gene, causing the bleeding disorder with a correct edition of gene through the virus in liver cells of the patient.
Liver cells are the only cell in human body, which are capable of producing certain clotting factors that are absent in people suffering haemophilia The two main types of the disease are haemophilia A, occurred due to lack of clotting factor VIII and hemophilia B that occurred due to lack of clotting factor IX.
So far, six people were injected with the tailored virus at the Royal Free Hospital in London. Two of them were given a low dose, two a middle dose and two were given a high level of doses. The normal levels of factor IX is less than one in healthy people. After injecting, levels of factor IX ranged from two to twelve percent.
The first patient treated has maintained levels of factor IX at two percent for more than sixteen months.The patients receiving the highest dose, maintained levels which from eight percent to twelve percent for twenty weeks. The study findings published in the New England Journal of Medicine revealed that levels of factor IX could be increased.
Patients with twelve percent of normal factor IX production would no longer be seen in the clinic. They would be able to go about their normal daily lives without any problems. In the absence of severe major trauma these people would not know that they have haemophilia, expalined Dr. Andrew Davidoff and Dr Amit Nathwani, co-researchers from University College London
The aim of their research was to take patients from a severe form of haemophilia to a mild one. All the patients have actually benefited from this gene replacement therapy, even the patients who have not been able to stop protein concentrate infusion. Such individuals required smaller number injections of factor IX, added Dr Nathwani.
This is the first study that has shown that you can actually achieve stable, long-term, therapeutic level of expression in people suffering severe haemophilia B. This was truly a landmark study, since it is the first to achieve long-term expression of a blood protein at therapeutically relevant levels, stated Dr Katherine Ponder, from the Washington University School of Medicine.