Man effectively 'cured' of life-threatening haemophilia following clinical trial

Darren Lapicki was born with a severe case of haemophilia A – a hereditary condition where the body does not produce a protein which helps blood to clot.

Haemophiliacs risk excessive bleeding from even tiny injuries and knocks and bumps can cause internal bleeding. It is potentially life-threatening and there was thought to be no cure.

However, Darren – the sixth generation in his family to suffer the condition – became the first success story in an incredible clinical trial which effectively ‘cured’ him.

In 2015, Darren enrolled as a participant in a clinical trial at Barts Health NHS Trust.

He said: “I was very keen to sign up. I’d read lots about the different research going on and was interested to learn about possible new treatments. My consultant, Professor John Pasi, is involved in a lot of clinical research but had said I wasn’t suitable for several of the trials I’d read about.

“When he said I was eligible for the BMN270 trial, I jumped at the chance.”

He was injected with a copy of the missing gene, which doctors hoped would allow cells to produce the missing clotting factor.

“Within a few days, my levels had already increased”, Darren said. “I started noticing that minor knocks were not bruising like they used to.

“Just a week after treatment, I was at work when a child accidently rammed me with a shopping trolley. I thought ‘oh no, that’s really going to be bad’ and I was all prepared for a bad ankle bleed. But instead of needing four days of bed rest it heeled within 24 hours, I couldn’t believe it,” he said.

Darren’s factor VIII levels continued to rise and a month later, his readings were 90 per cent normal.

“The treatment has changed my life considerably”, Darren beamed.

“I already had damage and arthritis in some of my joints and it was such a relief to know it wouldn’t continue to get worse. It’s great not having to inject anymore.

“I’d always tried to lead as normal a life as I could but it’s brilliant not having to worry all the time whether I might bump or scratch myself. There’s no more need to panic.”

“Think of the people who did the research years ago, to help discover the drugs we use now. If not for pioneers in the 1960s I wouldn’t have had hope of an anywhere near normal life. I want the younger generations to live an even better life than me. Taking part in research now is my way of returning the favour.”

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