Your donations will help support further pioneering research at King’s.
Understanding the Origins of Paediatric Liver Disease
The King’s Liver Molecular Genetics group, led by Dr Richard Thompson, undertakes groundbreaking research into genetic causes of liver disease in children. Through the identification of new gene defects involved in liver disease, diagnosis can be made earlier and treatment can be better targeted to the patient’s condition.
Two of the four main genetic defaults found to cause liver disease in children were discovered and described at King’s. This research has led to direct clinical benefits for children with these gene mutations. Using a patient’s blood sample, we can now simultaneously sequence seven different gene types to provide a genetic diagnosis within a matter of hours. As soon as a diagnosis has been made, doctors can start personalised treatments.
Developing Treatments To Combat Autoimmune Liver Disease
Working within the Institute of Liver Studies, the Liver Immunopathology Research Group is led by Professor Giorgina Mieli Vergani and Professor Diego Vergani. They are global leaders in the field and have been conducting research at King’s College Hospital for over thirty years. The group has trained over thirty Postgraduate Students and numerous Research Fellows as well as publishing over 500 peer-reviewed papers.
Professors Vergani and Mieli-Vergani are recognised worldwide for their work in autoimmune hepatitis. Extremely rare in children, autoimmune hepatitis is a condition in which the body’s immune system malfunctions and attacks the liver, causing inflammation. Continuing cell destruction results in scarring of the liver and can lead to cirrhosis (long-term liver damage).
Current research focusses on developing a new treatment for autoimmune hepatitis. Within the next few years, researchers hope to begin clinical trials, in which the deficient immune cells are removed from the patient, are re-educated in the test-tube and then are re-injected back into the patient to stop the autoimmune attack. Researchers believe this form of treatment could be particularly successful in children, who have a maturing immune system.
Pioneering Liver Cell Transplantation in Children
Recent breakthroughs in liver cell transplantation have produced an alternative to full organ transplantation in the management of liver-based metabolic conditions and acute liver failure. Researchers at King’s have pioneered a new technique to encapsulate liver cells in alginate microbeads, which can be transplanted into the abdomen cavity of a child.
There are numerous benefits to patients undergoing this treatment:
- The patient’s liver remains in place. In the case of acute liver failure, this allows the patient’s own liver to regenerate over time
- Hepatocyte transplantation is much less invasive than organ transplantation
- Alginate beads protect the transplanted cells from host immune cells, avoiding the need for immunosuppressive drugs
- Unlike whole organs, liver cells can be frozen and thawed on demand, allowing ‘off the shelf’ treatment
- This technique makes use of donor liver tissues that are unused or would normally be rejected for transplantation
This clinical liver cell transplantation programme started at King’s in 2001. To date, twenty-three children have undergone hepatocyte transplantation (sixteen for liver-based metabolic disease and seven for acute liver failure). The team continues their research to improve cell grafts while avoiding the body’s immune system.
A World First in Children’s Liver Care at King’s
At just twelve-days-old, Iyaad, pictured here with his parents, went into liver failure after contracting a virus. Transplantation was not an option as he was too sick and no donor was available.
Professor Anil Dhawan, Consultant Paediatric Hepa
tologist and Clinical Director of Child Health at King’s, and his team injected donor liver cells into Iyaad’s abdomen that acted as a temporary liver, processing toxins and producing vital proteins. The cells were coated with a natural chemical found in algae so there was no risk of the cells being attacked by his immune system. Iyaad’s parents noticed a positive difference in his health within forty-eight hours and after two weeks his own liver had begun to recover.
This was the first time such a procedure had ever been performed and its success is testament to the expertise of Professor Dhawan and the team at King’s, who are constantly working on translating their findings into treatment improvements for our patients.
Supporting the World’s Largest Paediatric Bio Bank
The paediatric bio bank at King’s is the largest of its kind in the world and is a vital resource to all researchers within the field of child health. Professor Mowat founded the bio bank in the 1970s and today it stores the most extensive collection of children’s liver samples in the world. Due to the high volume of patient referrals to the children’s liver service at King’s, the hospital is in a unique position to collect, prepare and store a wide variety of patient samples.
Over the past forty years, the bio bank has been a valuable resource for our researchers, underpinning all the major breakthroughs in understanding more about the origins of disease, improving methods of diagnosis and the development of new treatments. Given the pivotal role bio banks play in promoting scientific discovery, it is essential that we invest in the ongoing development and upkeep of the King’s paediatric bio bank.