One Year On – Professor Paolo De Coppi

For last year’s One Day at GOSH, Professor Paolo De Coppi’s team were filmed in the lab as they continued their groundbreaking research into regenerative medicine.

Professor De Coppi’s research into regenerative medicine looks at the possibility of engineering or repairing tissue using your own stem cells. This form of treatment reduces the risk of the body rejecting organs that have been transplanted and improves patient outcomes, such as reducing recovery time.

One year on, we spoke with Professor De Coppi – Head of Stem Cell and Regenerative Medicine at the UCL Great Ormond Street Institute of Child Health (ICH) and Professor of Paediatric Surgery at Great Ormond Street Hospital (GOSH) – to discover more about his research.

Why is regenerative medicine and paediatrics so well matched?

Paediatric medicine is where regenerative medicine can really make a difference. The tissue is much more malleable in younger patients, so it’s more prone to regenerate and repair without forming a scar.

Today, we’ve developed practices where we can take stem cells from a patient and grow new tissue and organs. Our long-term goal is to make the stem cells in the body learn how to build these new structures inside the body without having to be removed.

Paolo De Coppi and a colleague in a lab

GOSH was the first hospital in the world to successfully transplant a stem-cell supported trachea (windpipe) into a patient. What are some of the other types of conditions that regenerative medicine could treat?

It’s much easier to engineer simpler, tube-like organs than larger and more complex organs like the liver or lung. There are three other treatments that we’re currently working on. The first is for children born without an oesophagus (known as oesophageal atresia). The treatment we currently offer is to stretch the oesophagus, or if that’s not possible, we pull up the stomach, but we need to produce longer lengths of oesophagi to account for the missing parts.

The second treatment is bladder regeneration. At the moment, we use the intestine to enlarge the bladder, but the intestine is not made to be a container – its role is to produce and absorb. When used as a wall for the bladder, the intestinal wall produces mucus, which in the long-term can cause children to develop cancer.

For the bladder regeneration, we’re still investigating the use of the intestine – which has a similar muscle wall to the bladder – but we’re hoping to replace the mucus with cells from the bladder. We could be 18 months away from being able to offer this as a solution.

Finally, I’m co-ordinating a large European grant with different centres around the world to create a new intestine for children who are born without one or lose it early in life. We take stem cells from either the native intestine or the patient’s skin if they don’t possess an intestine. We can then use those cells to create all the parts of the intestine. Clinical trials for this procedure could begin in 2020.

Of course, before we can move forward with these treatments, we’re ensuring that all of our research is validated, ethical and safe.

Why is GOSH so well placed to be carrying out this research?

At GOSH and the ICH, we’re at the forefront of this research, thanks in part to the strong link between the two institutions.

A benefit of that link is that clinicians such as myself can spend their time in the lab working for our young patients and then going to clinic to see those patients and families in the building next door. What else is unique about this partnership, particularly in Europe, is to have so much of the expertise in-house, from a clinical perspective and an ethical one.

How many children a year would benefit from your research?

Regenerative medicine is a rare disease treatment. Thinking about the treatments for oesophagus, the bladder and the intestine, we could help as many as 30 children a year at GOSH, which is huge. For context, with the trachea transplant, which we use in life-saving cases when nothing else can be done, we’ve managed to carry out three in eight years.

Paolo De Coppi in a lab

How does research have the power to support children across the UK?

As a surgeon, I’m always very careful to not offer false hope. We want to make families understand that the research we do may not directly support their child, but it could help a child in the future who has a certain condition.

Families are all too aware of how a condition affects their child and their support network, and they don’t want other families to experience the same challenges. We must see research – whether that be a clinical trial or simply filling in a survey – as helping each other and progressing treatments for children. We wouldn’t be where we are today without other children and families being selfless in our pursuit of new treatments through research.

We last saw you for One Day at GOSH a year ago. How far have some of your projects come in the past 12 months?

A year in science is a very short time. We’ve moved ahead with research into the oesophagus. We’ve also progressed our work into joining up more complicated organs with blood vessels. For an organ like the intestine, it requires a blood stream within it that is then connected to the body’s blood stream. We’re excited about where our work in that area is going.

What’s your ultimate goal with research into regenerative medicine?

Oesophageal atresia is what I’m passionate about – I’d be very happy if we’re able to successfully implant new oesophagi in children. GOSH is one of the few referral centres for these children from around the world.

Our current method – which is to essentially bring the stomach up into the chest – is not an ideal solution for these children. These are normal children who have a problem swallowing, and it’s a condition they could have all their life. If we can fix that, then I can retire.

In the past year, have any of the children, young people and families left an impression on you?

The children and families I see have a huge impact on my life every day. Today, in clinic, I saw a child I operated on when she was a newborn. The family came in with a nice letter and some lovely pictures on a card to show how far she had come, and to say thank you. Mum was originally told not to continue with the pregnancy because some of her little girl’s organs were growing outside of her tummy – now, she’s almost four years old and jumping up and down!

The outcome we all want in paediatrics is to discharge our patients – for today’s patient, it meant that she doesn’t need to be seen by other doctors. I’m very humbled to be able to work at GOSH and have the opportunity to look after these children.

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