Stem cell research
The Great Ormond Street Hospital in London is pioneering the engineering and transplantation of tissue engineered organs for sick children.
Great leaps have been made in the field of stem cell research in recent years as scientists and doctors strive to find cures for some of the major diseases of our time. Stem cells have the remarkable potential to develop into many different cell types in the body.
They can serve as a sort of internal repair system, dividing to replenish other cells. The enormous potential of stem cells has yet to be fully discovered, but it is hoped they could help cure many diseases, including diabetes, schizophrenia, Parkinson’s disease, cancer and multiple sclerosis. Oak Foundation is supporting stem cell research in the hope that this cutting-edge treatment will benefit the lives of millions of people.
New York Stem Cell Foundation
The New York Stem Cell Foundation (NYSCF) is a world-class research institute. It works with major medical research institutions in the US and around the world to support and enable the most advanced stem cell research. Collaborative projects include research on diseases such as heart disease, diabetes, schizophrenia, Parkinson’s disease, Alzheimer’s disease, cancer, retinopathies and spinal cord injury. NYSCF aims to better understand the underlying causes of disease and to then accelerate development of more effective therapies.
Great Ormond Street Hospital
On the day he was born, Ciaran’s lungs collapsed and he was rushed to Great Ormond Street Hospital (GOSH), London. He was diagnosed with a rare disorder known as long segment tracheal stenosis – he had been born with a very narrow windpipe and had great difficulty breathing. This condition has been described as being similar to running 100 metres while breathing through a straw.
At just six days old, Ciaran underwent major surgery to reconstruct his airways, remaining in intensive care for a further four months. He continued to have difficulties breathing throughout all of his childhood and had to undergo several operations at GOSH. In 2010, by the time he was 12, he was mostly confined to a wheelchair and there did not seem to be much hope of saving his life.
However, the GOSH is an international centre of excellence in child healthcare and he had therefore come to the right place. Professor Paolo De Coppi, Consultant Clinical Senior Lecturer at GOSH and Head of the Surgery Unit at the University College London Institute of Child Health was determined to do something for Ciaran. In 2010 he was part of the team that performed the first successful transplantation of a tissue-engineered windpipe on Ciaran, dramatically improving the young boy’s quality of life.
By working with a research team to understand better how tissue-engineered organs can be transplanted with the help of stem cell research, Professor De Coppi led the team that injected a donor trachea with some of Ciaran’s bone marrow, which contained vital stem cells. To encourage growth, the cells were first mixed with chemicals in the laboratory before being injected into the windpipe, which was then transplanted into Ciaran. This procedure was Ciaran’s only option; it was the first time ever that such an operation was performed on a child anywhere in the world.
“My hope is that soon we might build complex organs such as the heart, gullet or intestine; functional organs that can grow with children, eliminating any need for organ donors. We’ve got a patient population that really needs these alternatives, urgently, if we’re to offer them long-term quality of life.”
- Professor Paolo De Coppi Great Ormond Street Children’s Hospital
Remarkably, the body recognises the stem cells as its own and does not reject the new organ, as is the case with regular organ transplants, so no immune-system repressing drugs are needed. Indeed, two years later, the follow-up appointment with Ciaran showed that the graft had not triggered an immune rejection response at all, but that rather the windpipe had formed an adequate lining, serving to greatly improve Ciaran’s quality of life. In 2014 Professor De Coppi said that the windpipe had not yet achieved normal rigidity but, nonetheless, was allowing Ciaran to lead the life of a normal teenager. Indeed, it seems that Ciaran is doing just that – the latest news from his family included a video of him playing the drums in a band at a family wedding! In addition, the team expects that his body’s natural tissue repair mechanisms will continue to strengthen Ciaran’s trachea over time.
Ciaran enjoys a much better quality of life thanks to the groundbreaking work in stem cell research of Professor Paolo De Coppi and team at the Great Ormond Street Children’s Hospital, London. 2. Professor Paolo De Coppi, who, along with his team, pioneered the first successful transplant of a tissueengineered windpipe.
© The New York Stem Cell Foundation / © Great Ormond Street Hospital
Source: Oak Foundation Annual Report
Year of publication: 2014