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HealthMost PopularPrimary School Teacher Who Took A Life-Changing Wonder Drug As Part Of A Clinical Trial Has Now Been Denied It By The NHS

Primary School Teacher Who Took A Life-Changing Wonder Drug As Part Of A Clinical Trial Has Now Been Denied It By The NHS

A mum-of-two who took a life-changing wonder drug as part of a clinical trial to treat her rare illness has spoken of her anger at being denied it by the NHS.

Louise Moorhouse, 35, spent three years of her life as a “guinea pig” for a US pharmaceutical company to help get the medication granted a European licence.

The primary school teacher, who suffers with a rare genetic condition called phenylketonuria (PKU), said taking the drug Kuvan made her feel like “superwoman.”

But despite it now being readily available across Europe after being granted FDA approval, health bosses in the UK have now decided it is too expensive.

The US biotech company also stopped giving it to once the trial was over meaning Louise now faces having to take 80 pills a day to treat her condition.

PKU is caused by a gene defect that creates the enzyme needed to break down the amino acid phenylalanine.

If untreated it can lead to brain damage and total mental degeneration, seizures and epilepsy.

Louise, of Birmingham, has avoid 80 per cent of normal foods such as bread, pasta, eggs and her protein intake is restricted to 12 grams a day.

But she found Kuvan allowed her to eat a normal diet and gave her a freedom she had never experienced before.

So she was left devastated to be told after her trial she wouldn’t be given the drug after being promised it for life by the company BioMarin.

She and other PKU suffers have also been denied the drug by the NHS, which refuses to fund it after the company put a £70,000 per adult price tag on it.

Louise is heading to parliament next Wednesday (24/4) with a group of supporters in a bid get the price of the drug reduced.

She said: “When I took part in the trial I was told that as a thank you for proving that it worked, I would be given access to the drug for life.

“So, half of me did it for that reason and half of me did it just because it was the right thing to do to try and do further research into my condition as it’s so rare.

“I took part because I just thought, ‘Wow, if this works it’s just going to change lives.’

“Kuvan allowed me to eat a completely normal diet.

“It was it was almost like someone had opened curtains on my life and I could see everything in Technicolor.

“It just freed me up so much. I felt like superwoman. I was healthier and I was happier.

“I felt like I could everything and anything, and I had time to think for myself and time to do things with my family.

“Then after the trial finished, that promise wasn’t kept and I was told it wouldn’t be available to me.

“I just remember thinking at the time, why have I had this snatched away from me, it was just full-stop done.

“I had tested this drug for them and helped get it FDA (US Food and Drug Administration) approval and a European licence.

“I was essentially their guinea pig to test that it worked for the UK market – and it clearly did.

“Because the trial had gone so well and because it made such a difference, I was convinced that it would only be a matter of time before this drug would be available to everyone who responded to it.”

Louise, who lives with husband Adrian, 36, a postman, and their two sons, aged 14-year-old and 17-months-old, said coming off drug in her 20s was a “shock to the system.”

She added: “I had to relearn almost everything.

“It had a massive effect on my concentration, my mood, my focus at work, my relationship.

“When I’m not on the drug I was walking around with a black cloud over my head, my brain was in a fog.

“One of the hardest things is knowing that I’m not being myself, I feel like a shadow of the person I was when I was on Kuvan.

“The drug was like a mini cure. But then it was just taken away from me.

“The only reason why the NHS is refusing to allow me to take the drug is because of it’s price tag.

“I know the NHS is under financial pressure right now but what they don’t release it that if they don’t do something now there will a large group of people in their 60s and 70s that will need care.

“There are so many people out there that are suffering.

“We are being priced out of it, there’s no ethics, there’s no human side to this at all.”

Louise’s diet has to be restricted and weighed and means she can only have 12 grams of natural protein a day.

She added: “I can’t have meats, cheese, eggs, nuts, beans, pasta or bread – a slice of bread has four grams of protein in it, so there is no point in me having a slice of toast in the morning.

“It’s pretty tricky, I can’t really eat out. I basically live on fruits and vegetables.

“I avoid socialising with food if there’s people there who I don’t know.

“I have prescription foods that are protein free or we have our supplements which are the tablets or drinks.

“The nasty thing about PKU is that it can have a violent, slow brain damage effect if your blood levels aren’t in control.

“But the cost of that put together is near the cost of Kuvan for the NHS to provide, so it’s a bit of a paradox.

“Following a low protein diet and only having 12 grams a day is not a treatment, it’s a sentence.”

An NHS England spokesperson said: “The NHS does not offer a blank cheque to pharmaceutical companies.

“Instead the NHS works hard to strike deals which give people access to the most clinically effective and innovative medicines, and at a price which is fair and affordable.

“Which is exactly what our patients and the country’s taxpayers would expect us to do.”

A petition set up by those effected by PKU in a bid to get BioMarin to reduce the price and the NHS to revise its stance currently has over 13,300 signatures.

Anyone wishing to sign the petition can do so at: https://www.change.org/p/biomarin-pharmaceutical-make-kuvan-affordable-to-the-nhs-allowing-people-with-pku-to-lead-healthy-lives

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