A team of doctors in Britain is to become the first in the world to modify pig organs to treat newborn babies with birth defects.
Babies born missing a section of their oesophagus, the tube linking the mouth to the stomach, are to receive transplants harvested from pigs and then modified using the child’s stem cells.
The landmark life-saving treatment will be used next year by doctors at Great Ormond Street Hospital in London on about 10 children born with severe cases of oesophageal atresia.
The research team also plans to develop the treatment for adults suffering oesophageal cancer, a far more common and often fatal condition.
Oesophageal atresia can be diagnosed in the foetus at around 20 weeks. The first children for next year’s trial could be identified in coming months. Oesophagi of varying sizes have been taken from pigs at a British farm in readiness. These “animal scaffolds” will be modified to remove all their cells.
They will then be re-engineered using the child’s stem cells, making them suitable for transplant and avoiding rejection by the patient’s body.
The stems cells will be taken at the moment after birth from the babies’ muscle and residual oesophagus.
The tissue engineering takes about eight weeks and doctors hope to implant the modified oesophagi at around two to three months after the child is born.
Although 90% of oesophageal atresia cases are treatable via a relatively simple procedure to close the gap, in the most severe cases the only option is for doctors to surgically move the child’s stomach up into their chest cavity.
This can create complications throughout the patient’s life and place them at a greater risk of oesophageal cancer, according to Professor Paolo De Coppi, a consultant paediatric surgeon at Great Ormond Street who is behind the new treatment.
De Coppi previously pioneered a similarly groundbreaking transplant in 2010 in which a 13-year-old boy was given a new trachea, which had been created from a deceased human donor using the teenager’s stem cells. He said: “This is completely new. Pigs have been used for heart valve replacement for many years, but nobody has received an organ developed from an ‘animal scaffold’ this way.”
Each treatment is expected to cost £100,000. Costs will likely be recouped through the prevention of long-term conditions associated with the current procedure.
Initial work was funded by the Cell and Gene Therapy Catapult and the UK Stem Cell Foundation and the clinical translation has been supported by the National Institute for Health Research.
Before the first patient can receive a transplant, the treatment must be approved by the Medicines & Healthcare Products Regulatory Agency.
“We are confident we will be treating our first patients in 2018,” De Coppi said. “The focus is on newborns at the moment, but in the next 10 years we hope to apply this to adults with conditions such as cancer of the oesophagus.”
De Coppi is also developing a treatment to help children born with shortened bowels, which will again use pig organs and stem cells, either harvested from the bowel or even the child’s skin, to build a new intestine for transplants. He hopes to introduce this in 2020.
Ultimately De Coppi would like to treat foetuses with congenital defects while still in the womb — including children suffering a congenital diaphragmatic hernia, a hole in the diaphragm that can result in heart and lung complications.
De Coppi proposes using stem cells from the foetus’s amniotic fluid to rebuild the diaphragm before the child is even born.
Dr Peter Steer, chief executive of Great Ormond Street, said: “This new oesophageal transplant procedure has the potential to transform the lives of children with extremely complex health conditions.
“Our researchers are now working closely with hospitals across London to develop, and make available, this pioneering procedure.”