Wonder drug treating Barnstaple woman's rare kidney disease may be stopped

A WONDER-drug helping a woman deal with a terminal kidney disease is in danger of being withdrawn over funding issues.

Elena Lilley, 27, from Barnstaple, was diagnosed with a rare condition called atypical haemolytic uraemic syndrome (aHUS) when she was 19.

The disease currently affects around 170 people in the UK, with up to 20 additional cases being diagnosed each year.

For the first five years after being diagnosed, the only treatment available to Elena was plasma exchange.

This was a four hour process which involved blood being taken through one needle and re-entering Elena's body through another.

"That was gruelling," she said. "It felt like I was vibrating. I couldn't eat because I felt so poorly. Looking back now I was not well at all."

Elena had to travel to Exeter for the treatment, relying on friends or family to get her there.

"In a worst case scenario I was going down three times a week, struggling to work and feeling poorly all the time," she said.

"You feel lethargic. You think I have got to go because it is keeping me alive but I really can't be bothered.

"I really tried to stay as normal as possible."

Elena worked in a children's nursery but was forced to quit her job due to her illness.

But in 2010 she was offered the chance to take part in a trial being run by a company called Alexion. It involved a drug called eculizumab, which it was hoped would combat Elena's aHUS.

She began the trial in April 2010 along with three others to see how the new drug would affect her kidney condition.

"They gave me one last plasma exchange and then that was it, which was kind of scary because it had kept me alive for so long," said Elena.

"It is like a drip and it takes 35 minutes, then you wait for an hour afterwards."

Elena was so convinced it would work she returned to her old job just one month after the trial began.

"It is an amazing drug," she added. "When I started the trial I had 5 per cent kidney function. Now I am approaching 20 per cent.

"I have gone back to the nursery and now I am a supervisor. I managed to get married, go to the Olympics and do so many things I couldn't do before."

But despite the huge improvement the drug has had on Elena, it has still not been nationally commissioned.

Despite having undergone three years of research by AGNSS, a company which advises health ministers on which services should be offered nationally, eculizumab has been referred on for yet more tests.

Elena has been told the drug has been referred to the National Institute for Health and Clinical Excellence (Nice) over affordability issues.

"The issue of funding has been discussed for a long time in government," she said. "In the UK there are only four people being given the drug so there are lots of people who would benefit from this.

"We have decided we should fight for this. We feel responsible to help the others that haven't experienced it yet."

Elena will still receive the drug while further tests are being undertaken. But she and another woman on the trial have set up a petition to see the drug nationally commissioned as soon as possible.

Nick Harvey, MP for North Devon, has already signed an early day motion to get eculizumab available without delay. An e-petition is also running at www.epetitions.direct.gov.uk/petitions/46252.

A Department of Health spokesman said: "Ministers have accepted the advice of AGNSS on the clinical effectiveness of eculizumab for treating patients with aHUS, but considered they needed further, rounded advice which takes account of the cost, benefits and affordability of the drug.

"From April, Nice will be responsible for looking at very high cost drugs for patients with very rare conditions, and that is why ministers intend that Nice will carry out an evaluation of eculizumab."