Drug helps Darlington woman avoid kidney failure after 'shock diagnosis'

A new kidney disease drug can prevent organ failure and significantly improve patient outcomes, a university study has found.

The drug, known as eculizumab, is given to patients with rare condition Atypical Haemolytic Uraemic Syndrome (aHUS) with studies showing it prevents 86% of people from going into kidney failure.

Kidney patient Chloe Pratt, 25, of Darlington, thought her chances of ever living a normal life, let alone starting a family, had been dashed when she was diagnosed with aHUS last year.

The human resources assistant’s diagnosis came out of the blue as she was the first in her family to have presented with the disease after becoming seriously ill and being admitted to intensive care.

Within days of being diagnosed, she was given eculizumab and is doing so well on the treatment that she has regained hope for a future she once feared might be out of reach.

Had she not been given the treatment she would have been on permanent dialysis, and this makes it less likely to become pregnant as well as more likely to have complications.

Ms Pratt said: “I’d never heard of aHUS, so it was quite overwhelming to be told that I had a life-threatening illness and would need eculizumab. I was extremely tired, and I was just not feeling myself, but it wasn’t until my eyes went yellow that I went to seek medical advice, leading to my shock diagnosis.

“Since I have been taking eculizumab I feel much better, and I am getting back to myself again. I can’t imagine not being on this treatment, it has prevented kidney failure and it has saved my life.”

A research team at Newcastle University carried out clinical trials into eculizumab and the National Institute for Health and Clinical Excellence (NICE) approved its use by the NHS from 2015.

Now, a study by Newcastle experts, published in Blood, revealed that eculizumab prevents 86% of patients going into kidney failure.

Professor David Kavanagh, who led the study, said: “Our study confirms the effectiveness of eculizumab in preventing kidney failure for those with aHUS. It further strengthens the importance of patients receiving early treatment as it’s life-saving and helps significantly improve quality of life without the need for dialysis or a kidney transplant."

Ms Pratt said: “It’s fantastic that the research into aHUS and eculizumab has been led in Newcastle and I feel very lucky that I have been able to benefit from this.”

She added: “I’m now really excited about the prospect of living a normal life and even hope one day to be able to start a family. After my diagnosis, I feared that this dream would not be possible, but thanks to eculizumab, I see a brighter path ahead.”

When the drug was approved, NHS England commissioned the National Renal Complement Therapeutics Centre, a collaboration between Newcastle University and Newcastle upon Tyne Hospitals NHS Foundation Trust, to run the highly specialised aHUS service.

Professor James Palmer, national medical director for specialised services at NHS England, said: “These important findings provide renewed evidence that eculizumab is helping people to live longer without kidney failure, and could help enable doctors to identify those who are unlikely to benefit from the drug and could be spared the side effects.

“Collaborative research projects like this are helping to drive real progress for NHS patients with the support of a nationally commissioned service that provides both comprehensive clinical advice and a national patient registry, and it’s fantastic to see the results, which will help save and improve patients’ lives.”

Professor Kavanagh added: "Two decades ago, we began researching aHUS and it’s wonderful to see that our efforts have benefitted those with the illness with the NHS approving the drug all those years ago.”

At least nine different genes have been identified to be associated with aHUS. Several members of Ms Pratt's family were genetically tested for the condition and do have the affected genes, putting them at risk of developing the disease in the future.

In the largest study of its kind, which was funded by Kidney Research UK and the Medical Research Council, more than 2,000 aHUS patients between 1995 and 2019 were analysed.

A total of 244 were given eculizumab and responded very well to the treatment. However, a small proportion did not with 14% of patients still requiring long-term dialysis.

What the experts say

Dr Vicky Brocklebank, from Newcastle University, said: “This study is a wonderful example of the benefits of close collaboration between patients, clinicians, researchers, and charities.

“Our pioneering research has allowed for genetically targeted treatments to improve patient outcomes and revolutionise the way the condition is managed.”

Dr Aisling McMahon, executive director of research and policy at Kidney Research UK, said: “This work by David, Vicky and the Newcastle team is a really important example of how, with collaboration between several partners, laboratory research can lead to clinical benefits for patients.

“We are delighted to have been a significant partner in this research and look forward to future projects supporting patients with aHUS."

Further research by the Newcastle team will now focus on finding a cure for the small proportion of patients who do not respond to the drug.

For some of those who did not benefit, a new genetic cause for aHUS was identified which allowed the drug to be stopped to avoid the potential side effects of ineffective treatment.

Want a quick and expert briefing on the biggest news stories? Listen to our latest podcasts to find out What You Need To know...