New hope for kidney patients in 2-a-day pill

PATIENTS with a potentially fatal kidney disease have been handed a lifeline after a breakthrough drug that slows down the disease was approved for NHS use.

Tolvaptan has been given the green light as the first-ever treatment for autosomal dominant polycystic kidney disease (ADPKD), an incurable genetic condition that can cause heart attacks, stroke and kidney failure.

The move has given hope to an estimated 60,000 Britons living with ADPKD, who until now have faced the prospect of gruelling kidney dialysis or even a kidney transplant.

The disease leads to multiple cysts developing on the kidney, which can cause it to grow up to 50 times its normal size.

Tolvaptan was last month approved by medicines watchdog the National Institute for Health and Care Excellence (NICE). It is expected to be available from early 2016 to patients in England and Wales who show signs of rapidly progressing disease.

The twice-daily tablet works by slowing the rate at which cysts develop. A three-year trial found it reduced the increase in kidney size by approximately 50 per cent. It also reduced the rate of worsening kidney function, pain and infections. Tess Harris, CEO of the Polycystic Kidney Disease Charity, has ADPKD and says: ‘Until now there hasn’t been a treatment that can delay its progression. This has the potential to fundamentally change the outlook for patients with ADPKD and their families and brings great hope for the future.

‘Tolvaptan is the first therapy to target the underlying disease and not just the management of symptoms and complications.’

ADPKD accounts for about one in ten people on dialysis in the UK and one in eight transplants.

Abnormal kidney development is thought to start soon after birth, but symptoms – which include high blood pressure, abdominal pain and blood in the urine – tend not to begin until adulthood, when the disease is already well advanced.

The outlook varies, but for many, life with these large cystic kidneys is debilitating and painful. Kidney cysts can burst and bleed, causing severe pain and infections and sometimes hospitalisation. Surgical interventions may be needed, such as cyst aspiration or even removal of one or both kidneys.

Kidney failure or end-stage renal disease occurs in most ADPKD patients before 60, with dialysis or kidney transplant the only lifesaving options.

Tolvaptan, which is the result of four decades of research into ADPKD, aims to reduce the need for dialysis or transplant and make the disease more manageable. It has been recommended by NICE for patients who show signs of the disease progressing rapidly.

Dr Richard Sandford, a consultant clinical geneticist at Addenbrooke’s Hospital, Cambridge, said: ‘This drug will give us the opportunity to help some patients to reduce the number of symptoms they experience and potentially slow their progression towards kidney failure.’

Because the disease is genetic, there is a one in two chance of it being passed to children of a parent with the gene. One British family holds the world record for the number of members having a transplant. So far, across three generations, 11 have had new donor kidneys.

Dr Sandford adds: ‘For many people with ADPKD, kidney failure at a relatively young age has been a daunting prospect which we’ve been unable to guard against.

‘They have often seen other close family members require dialysis or a kidney transplant.’