I have £2.6million of blood and it has ‘cured’ my haemophilia

A transformational gene therapy that reverses the bleeding disorder haemophilia B is being made available by the NHS.

Elliott Collins was one of the first to receive the treatment when he took part in the studies that showed it worked.

He says he “feels cured” and that the gene therapy has given him a “new lease on life.”

The treatment is one of the most expensive in the world, with an official cost of £2.6 million.

When you cut yourself, the body senses the injury and activates a series of clotting proteins in the bloodstream.

This makes the blood sticky at the wound site and then seals it with a clot.

But from the day he was born, Elliott was unable to produce enough clotting factor IX (nine).

Without this crucial clotting component, bleeds are larger and longer. Bleeding also occurs spontaneously in joints, such as the knee or elbow, and causes long-term damage.

Elliott, who is 34 and from Colchester, rebelled against his diagnosis as a child by secretly playing rugby or skateboarding.

But each injury risked damaging bleeding, such as when he was tackled while playing football.

“I have studs all over my shin and it came out looking like a cartoon, like Tom and Jerry,” he said.

Elliott required factor IX injections twice a week for 29 years, and more often when he was injured.

“I thought about it all the time,” he said.

Elliott Collins at home in the sunElliott Collins at home in the sun

Elliott Collins is one of the few people treated with the gene therapy during the clinical trials (Elliott Collins)

But he participated in trials of the gene therapy when it was just an experimental idea in 2019.

He has not needed factor IX injections since and says it has changed his life.

He told me, “I don’t think you can go through something like that for 29 years before it completely disappears and doesn’t change someone mentally and physically.

“I feel healed.”

He knew it worked when he rammed his knee into a locker. He hit it so hard that it swelled and needed a factor IX injection, but instead he watched as only a small mark appeared.

“That reinforced (the realization) that this is real, this works and I can relax a little bit.”

Tests showed that Elliott’s blood levels of factor IX had risen from zero to 60% of normal.

How it works

An image of a virus containing a DNA strand that represents the correct copy of the factor IX instructions given to the bodyAn image of a virus containing a DNA strand that represents the correct copy of the factor IX instructions given to the body

The gene therapy uses a virus to “infect” Elliott’s liver with DNA that contains the ability to make factor IX (Getty Images)

Elliot was born with a mutation in his DNA – his genetic code – which meant his body’s instructions for making factor IX were incorrect.

So doctors gave him engineered viruses that contained copies of the fully functional instructions for factor IX.

The viruses act like a fleet of microscopic postmen, delivering the blueprints to the liver.

The organ is then able to produce the clotting protein.

It was a one-time infusion, lasting about an hour, that Elliott is benefiting from five years later.

Not everyone will respond as well as Elliott. In studies involving 54 men, two more had to take factor IX injections.

And no one knows how long it will last; some studies indicate more than ten years.

“I’m very aware that this is new. I’m almost preparing myself for it to possibly go away to avoid any kind of major disappointment,” Elliott says.

How can the NHS afford this?

Uncertainty about how long patients will benefit makes it difficult to know whether the gene therapy is worth the money.

Normal injections of clotting factors cost between £150,000 and £200,000 per patient per year for life.

But the gene therapy – branded Hemgenix – has an official list price of £2.6 million.

Elliott says it’s “wild to think it costs that much” and now that he had millions of pounds of blood, he should “probably get life insurance”.

The treatment is made available through an agreement between the company CSL Behring, NHS England and the National Institute of Health and Care Excellence, the body that assesses cost-effectiveness.

I have asked all parties involved for details of the deal, but it is contained in confidentiality agreements.

As I understand it, the NHS will track how many patients benefit over the next ten years and that will determine how much CSL Behring is paid. If factor IX levels fall earlier than expected, the NHS will pay less. It is essentially a performance-based pay for medicines and it is the first deal the NHS has done.

The therapy will be available immediately at eight centres: Oxford, Manchester, Leeds, Bristol, Birmingham, Cambridge and two in London.

In the UK, around 2,000 people suffer from haemophilia B and the NHS expects to treat 250 of them.

Not everyone will be suitable for the gene therapy, as some are too young or have other health problems that rule them out.

Prof Sir Stephen Powis, the NHS’s national medical director, said the “transformative” gene therapy could be “truly life-changing” and had been delivered “at an affordable price”.

‘Real milestone’

Hemophilia runs in families.

Elliott’s younger brother was six days old when he died of a cerebral hemorrhage. His great-uncle died of the condition at the age of 14.

Those with hemophilia, usually but not exclusively men, pass on the affected genes to their daughters, who become carriers.

This therapy will not change that genetic inheritance, so it is not considered a true “cure.”

But Kate Burt, chief executive of the Haemophilia Society, said it was a “real milestone”.

“It is life-transforming at its most effective, it can last for decades and put the bleeding disorder into complete remission.”

Similar gene therapies are being developed for haemophilia A, which is becoming more common and affects almost 9,000 people in Britain.

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