Hemophilia can be cured with ‘miracle’ therapy –

Doctors say today that a new “miracle” gene therapy could effectively cure people with hemophilia.

Researchers say a one-time transfusion could prevent people with the rare condition that prevents blood clots from taking weekly injections to control it.

A Phase 1-2 study of 10 patients with hemophilia B found that after more than two years of treatment known as FLT180a, nine patients no longer needed injections to replace their clotting agents.

Lead author Professor Pratima Chowdary, a hematologist at University College London, said treating the condition “will be a reality for most adults in the next one to three years”.

Gene therapies are already showing promise for treating people with hemophilia A, who make up 85 percent of those with the condition.

But this new treatment is one of the first treatments to be tried for people who have emoilia B, which is caused by a missing protein called a clotting factor.

None are yet available on the NHS, and there are concerns about price, as treatment for hemopia A, which showed similar results earlier this month, was estimated at between £1.5m and £2.5m per dose.

Current clotting factor injections can cost between £150,000 and £200,000 per year.

About 6,000 people in the UK have hemophilia, while 20,000 people in the United States have the condition.

It is usually inherited and usually affects males because the missing protein is located on the X chromosome.

Normally, when a person cuts himself, clotting factors mix with blood cells called platelets to stop bleeding.

However, people with hemophilia lack clotting factors and are at risk for heavy bleeding. This can be fatal and even cause severe joint pain in some patients.

Professor Chowdary, who also works at the Royal Free Hospital in London, told the BBC: “We have many young patients suffering and there is nothing we can do to reverse the joint damage.”

The study, published in the New England Journal of Medicine, was led by experts from UCL, the Royal Free Hospital, and biotech company Freeline Therapeutics.

All ten patients selected for the study had “severe or moderate” hemophilia B, the rarest form of the condition, accounting for 15% of cases.

It is caused by a deficiency of clotting factor IX, while patients with hemophilia A do not have factor VIII.

At the start of the study, they were given one of four different doses of the treatment, and at the end, the doctors measured their factor IX levels to see how it affected their long-term condition.

After an average of 27 months, five patients had normal factor IX levels (51 to 78 percent), three patients had 23 to 43 percent and one patient had 260 percent levels.

Nine patients no longer required weekly injections. Professor Chowdary said: “We are very excited about the results.

‘Removing the need for people with hemophilia to regularly inject the missing protein is an important step in improving their quality of life.

“The long-term follow-up study will monitor patients for duration of expression and monitor for late effects.”

FLT180a works by using a small portion of the virus to deliver a copy of a gene directly into a patient’s tissue to make up for a missing copy.

This new gene can then produce the missing FIX proteins that allow the blood to clot normally.

Patients participating in the study had to take immunosuppressant drugs for several weeks or months to keep their immune systems from rejecting the treatment.

Although the treatment was generally well tolerated, all patients experienced some side effects with an abnormal blood clot in one patient who received the highest dose of FLT180a and had the highest protein levels.

“Gene therapy is still a young field that is pushing the boundaries of science for people with serious genetic diseases,” said Freeline co-founder Professor Amit Nathwani, a UCL haematologist and one of the study’s authors.

He said the new study adds “to the growing evidence that gene therapy has the potential to free patients from the challenges of sticking to a lifelong treatment or providing care where it doesn’t exist today.”