Stem cell jab that repairs liver without need for transplant to be trialled on sick British children

Claire Bates


11:21 GMT, 9 April 2012



13:21 GMT, 9 April 2012

Professor Dhawan hopes they will see improvements in the 18 sick children within a few months

Pioneer: Professor Dhawan hopes they will see improvements in the 18 sick children within a few months

Doctors have developed a pioneering treatment for liver disease that could save hundreds of lives a year and avoid the need for transplant surgery.

Liver specialists desperately need new approaches to the epidemic of liver diseases that is leading to huge demand for donor livers.

Eighteen British children suffering from rare and life threatening liver conditions are to receive infusions of specially treated liver cells removed from the organs of dead donors.

Doctors believe they will make vital stem cells – the building blocks of life – and repair the damaged organ.

The worlds first trial using liver stem cells is to take place at London's King's College Hospital and will be headed by paediatric liver consultant Professor Anil Dhawan.

He described the use of stem cells to treat liver disease as an 'exciting breakthrough'.

He said: 'We have many very sick children and babies who need transplants. If we can cure them without a transplant that will a fantastic development.

'We have tried using ordinary liver cells with limited success, but is the first time a treatment has been developed that gets the liver to re-grow using stem cells.'

It comes after they successfully treated on baby Iyaad Syed last year.

Iyaad was born healthy in February 2011 but his liver had started to fail after he caught the herpes-simplex virus. Professor Dhawan injected donor liver cells into the boy's abdomen when he was just two weeks old. In November, doctors announced that Iyaad had recovered well and his own liver was functioning normally.

Liver specialist, Professor Etienne Sokal, who developed the technique at the Catholic University of Louvain in Belgium said: 'Some patients with liver disease are unable to produce appropriate stem cells to repair the liver.

'We have been able to show the cells we infuse into the liver last and continue to supply the liver with new stem cells, which are able to correct the missing functions of the liver in these children.

'Our early trials in the laboratory were successful enough for us to get the go ahead to start human trials.'

If the trials in children are successful
it is hoped the treatment can be extended to patients who have livers
damaged by alcohol abuse, viral diseases like hepatitis or the growing
numbers of patients who going into liver failure due to obesity.

Survivor: Iyaad Syed was the first patient to be treated with liver stem cells

Survivor: Iyaad Syed was the first patient to be treated with liver stem cells

At present large numbers of liver patients die each year waiting for an organ transplant – while some don't even make the transplant list due to organ shortages.

For those lucky to get an organ there is the risk of the liver rejecting putting them back on the transplant list.

The UK – along with many countries – is facing an epidemic of serious liver disease much of it due to alcohol abuse and obesity which is dramatically increasing the need for donor livers.

Latest figures show that around one fifth – or around 150 – of 700 organs transplanted each year go to recovering alcoholics.

The death toll due to liver disease has risen by a quarter in the last decade and many of the victims are only middle-aged. Just over 11,500 men and women now die of liver disease every year -up from 9,200 in 2001.

It is estimated that just under 80 per cent of these deaths are caused by alcohol, and obesity and the remaining 20 per cent by hepatitis and inherited conditions.

A culture of overeating is also putting the lives of more than 500,000 obese young people at risk of serious liver disease according to a recent report by the Department of Health.

While alcohol is a major contributor to liver damage, many people are unaware of non-alcoholic fatty liver disease, which is linked to being overweight.

The world-first trial will be held at King's College Hospital in London

The world-first trial will be held at King's College Hospital in London

It can progresses to a life-threatening condition called cirrhosis of the liver and is now almost prevalent as alcohol abuse as a cause of liver disease.

The new treatment developed by Professor Sokal offers the hope of an alternative to liver transplants for the growing numbers of people who face death due to liver failure.

The stem cells are better tolerated than a organ transplant and require less immuno- suppression drugs- reducing risks of rejection.

From a small number of cells gathered from one liver, experts have been able to grow millions of special liver cells capable of making stem cells when infused into a patient's liver.

Trying to treat liver disease by infusing donor liver cells is not new. But until now only adult liver cells have been used – with limited success as the cells die off after a period of time.

By using specially treated liver cells that are capable of becoming stem cells, scientists believe they may have found a permanent cure for patients who would normally need a transplant.

Doctors will trial the stem cell treatment on children with an inherited metabolic disorder that affect the liver called Crigler-Najjar syndrome.

Children with this condition are unable to eliminate toxins from their bodies and therefore must undergo daily 12-hour exposure to special blue lights, just to survive.

Without daily treatments, a child would suffer brain damage, muscle and nerve damage and death.

The treatment will also be tried on children with urea cycle disorders who are unable to process liver toxins because of a genetic defect. This condition can lead to brain damage and death without a special diet.

Experts believe that up to 20 per cent of cot deaths may be due to undiagnosed urea cycle disorders.

Professor Dhawan, said: 'If all goes well the children we are treating with the cells will show an improvement within a couple of months. We would expect those children to come off their medicines and therapy. It will mean the liver cells have done their job and corrected the defects that made them ill.

'Then we will have to see how long the effect lasts and whether we have to top up these children with further infusions. I am optimistic the treatment will work.'

Professor Max Malago, a liver transplant surgeon at London's Royal Free Hospital, said: 'There is enormous demand for donor livers at present which is impossible to meet. There patients who are desperate to transplant but unfortunately not everyone can get an organ.

'If there was an alternative treatment to transplant where you could save the liver it would offer hope to patients who are at present dying waiting for an organ.'