Modern medicine can now save the lives of more children with liver disease than ever before. But that’s not enough for researchers based at our paediatric research laboratories, the MowatLabs. They want to find ways to improve quality of life for children too.
“Survival rates for children’s liver disease have improved from 20% to more than 90% in the last four decades,” says Professor Anil Dhawan, Corporate Medical Director of the Variety Children’s Hospital at King’s.
“We are giving children quantity of life but we are not completely giving them quality of life. That’s what our research is aiming to do; we want to create a model of care to help children live happy, fruitful and long lives.”
Seeking answers to important questions
Professor Dhawan and his team use a variety of research methods to increase their understanding of liver disease and they are greatly helped by the MowatLabs’ unique and purpose-built location, next to the Children’s Liver Ward.
“We need to know why certain things happen in children’s liver disease.” says Professor Dhawan.
“Once we have worked out how and why something occurs or has happened then it is a lot easier for us to find out what we can do about it; how we can change it or stop it.
“Then we can take that concept that we created in the laboratory and take it to the bedside and test it with patients. And once we have done that; answered a question, come up with a solution and proved that it works, we can then help to prolong a child’s life and improve their day-to-day living.”
Ground-breaking early successes
The research team has already made some promising discoveries and has developed treatments that are the first of their kind in the world but they need to do more.
“We have created microbeads, containing liver cells from organ donors,” says Professor Dhawan.
“These can be transplanted into a child experiencing liver failure in order to support their liver function while they receive treatment or wait for a transplant. We’re now trying to find ways to freeze these microbeads so they can be ready to use whenever we need them.
“60 per cent of our current treatments are non-liver transplant and our ultimate goal is to end the need for transplants altogether. Transplantation is a treatment, not a cure and although it is very effective, it does come with its own problems.
“So we are working on techniques that will hopefully enable us to grow healthy liver cells from a patient’s own cells (a process known as cell therapy). This could mean that we no longer have to replace the whole liver; we could remove the damaged area and then use the healthy liver cells (without the defective gene that caused their liver disease) to help rebuild the liver.”
Filling knowledge gaps
Professor Dhawan and his team are also determined to reveal the secrets of liver disease in very young patients.
“We still haven’t really looked into the effects of liver disease in early life."
"Most children with liver disease are diagnosed before they are 12 months old. That’s the time when the brain is growing very fast and if, at that time, something happens – even something very subtle – this can cause significant cognitive function issues which could appear later on in life when they are going to school, college or working. Certain skills may not develop that well.
“So we need to find out exactly what could happen and, if it happens, how big is the problem and why does it happen? Once we have figured out why something is happening we will then be able to design interventions to tackle the issue. Examples could range from music therapy to medication. But we have a very small window of opportunity; if we don’t act on problems in the first two years of life then we may miss the boat.”
A unique partnership
The positioning of laboratories so close to a children’s ward is unique to King’s. It is the only hospital in the UK to have this advantage.
This provides researchers with many benefits.
“Researchers can sometimes work in academic silos, unable to gain a personal understanding of how their work is actually helping to transform lives,” says Professor Dhawan.
“But my staff say that the most important and fulfilling thing for them is to see their work actually being used to treat somebody on the ward. So they take great pride in their work because sometimes when they are working on cells in the lab, they get to meet the patient who has provided these cells and they get to meet the families too.
“So that satisfaction, enthusiasm and motivation makes our researchers even more productive – they are inspired by having patients so close by. Sometimes we invite the children to see the labs. They are fascinated by our work – and it may even inspire them to become future scientists themselves.”
Helping the children of today and tomorrow
Unsurprisingly, Professor Dhawan and his team gain huge job satisfaction from knowing that their life-changing research has helped a young patient – but they also have their eyes firmly set on the future.
“We feel elated when we know that we have helped a child in some way; we get a tremendous sense of achievement from that” says Professor Dhawan.
“But ultimately we are making these advances, these platforms, so that future technologies can build upon them. The building blocks that we lay down today will make sure that the future of regenerative medicine is secured.”
Funding the crucial questions
As with most healthcare research, the MowatLabs do not receive money from the NHS.
“Charity support is vital to our work because this funding enables us to develop new ideas,” says Professor Dhawan.
“It enables us to produce proof of a concept. Some potential ideas may not be scientifically very solid at first and may need some exploration before larger funding bodies will agree to fund a larger study or clinical trial.
“Ultimately I hope that we will be able to treat children’s liver disease with more non-invasive methods that improve quantity and quality of life. These advances will take time but our work is ensuring that this exciting field of research is going in the right direction.”
By making a donation to our TLC Appeal, your support could help Professor Dhawan and his team make a scientific breakthrough that will transform the lives of young liver patients.