Can Haemophilia Be Cured? New Gene Therapy Might Have Some Answers

Scientists have been trying for long now to find a treatment option for haemophilia, a disorder in which the blood of the patient doesn’t clot normally.

In a recent development, scientists have been able to develop a gene therapy for haemophiliacs where patients can inject themselves using intravenous infusions that will plant new gene in the cells and help them against this condition.

But the not-so-good news is that in certain trails, the therapy stopped working in 2 out of 12 patients. Despite these setbacks, the scientists and researchers are still hopeful that new treatments would be approved within a few years.

It is important to keep in mind that the new gene therapy is not a ‘cure’ for haemophilia and the treatment in it’s current version can only be used once. The benefits of the treatment would wear off if it stops working.

Speaking to The New York Times, Dr Steven Pipe, Director of the Hemophilia and Coagulation Disorders Program at the University of Michigan, said:

For now, we are anticipating that this is a once-in-a-lifetime treatment.

Dr Steven Pipe

For now, it is difficult to predict the lasting effects of the treatment.

Normally when healthy people bleed, substances called clotting ‘factors’ in the blood combine with platelets to make the blood sticky and eventually the bleeding stops.

An inherited mistake in the genes stops blood from clotting in haemophiliacs – so even minor cuts, bruises and fall can result in internal bleeding of the joints, muscles and brain accompanied with external bleeding.

The patients with severe haemophilia usually need to inject themselves every couple of days as the quantity of the necessary proteins drop between two injections.

The latest gene therapy aims to reduce the dependability on injections and also try to decrease the number of bleeds. The inserted gene would depend on the type of haemophilia a patient has.

Haemophilia in India

Did you know that even though haemophilia is a rare disease, India has the highest burden of patients in the world?

Approximately one in 10,000 Indians suffer from haemophilia, and yet, there is no comprehensive public health intervention to prevent or control the genetic disorder, and there is absolutely no national policy on this disease.

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