‘Stem cell plasters’ could revolutionize treatment of children with congenital heart disease

Researchers at the University of Bristol funded by the British Heart Foundation (BHF) have developed ‘stem cell plasters’ to revolutionize the way surgeons treat children with congenital heart disease, so they don’t have to you don’t need as many open-heart surgeries.

Heart defects are the most common type of abnormality that develops before a baby is born, with around 13 babies diagnosed with congenital heart disease every day in the UK. These include abnormalities in the baby’s heart valves, major blood vessels in and around the heart, and the development of holes in the heart.

Currently, for many of these children, surgeons can perform open-heart surgery to temporarily repair the problem, but the materials used for patches or replacement heart valves are not completely biological and cannot grow with the baby. . This means that they can be rejected by the patient’s immune system, causing surgical equipment to gradually degrade and fail within months or years.

A child may therefore have to undergo the same heart operation several times during his childhood, which keeps him in the hospital for weeks, has a huge impact on his quality of life and causes a lot of stress for the family.

Now, BHF Professor Massimo Caputo has developed the first type of stem cell patch to repair abnormalities in the valve of the large blood vessel that controls blood flow from the heart to the lungs and to repair the holes between the two main chambers of blood. heart pumping. .

Stem cell dressings are designed to be sewn into the area of ​​a child’s heart that needs to be repaired during surgery. The stem cells might then boost the repair of heart tissue without being rejected by the child’s body.

These patches have the potential to adapt and grow with the child’s heart as they age, eliminating the need for repetitive heart surgeries and the many days in the hospital to recover following each one.

There are around 200 repeat operations for people living with congenital heart disease each year in the UK. The technology might save the NHS around £30,000 for each operation that is no longer needed, saving millions of pounds every year.

The BHF has awarded Professor Caputo nearly £750,000 to prepare these patches for patient testing so that clinical trials can begin within the next two years, enabling more children and babies to benefit from life-changing technology. The materials have already been proven to work safely in animals.

The team is also in the early stages of developing other stem cell technologies using 3D bioprinting and gene therapy to one day be able to repair more complex congenital heart defects.

For years, families have asked us why their child has to have heart surgery time and time once more. Although each operation can save lives, the experience can place incredible stress on the child and their parents. We believe that our stem cell patches will be the answer to solving these problems.

Our ultimate vision over the next decade is to create a paradigm shift in the way physicians treat congenital heart disease, developing personalized stem cell and genetically engineered treatments for the most complex heart defects. »

Massimo Caputo, BHF Professor of Congenital Heart Surgery, Bristol Heart Institute, University of Bristol

Dr Sonya Babu-Narayan, Associate Medical Director at the British Heart Foundation, said: “If successful, this new stem cell therapy that acts as a healing bandage might revolutionize heart surgery outcomes for children and adults. adults living with congenital heart disease.

“It might offer a solution that means their heart is repaired once and for all in one operation, saving people from facing a future of repeat surgeries and giving them the gift of a happier, healthier life.” »

Patient Story #1

Finley, 2, from Corsham in Wiltshire, was the first baby in the world with a congenital heart defect to receive a special type of stem cell injection treatment to try to keep his heart rate up. It is these donor stem cells that Professor Massimo Caputo of the BHF now uses to develop his stem cell plasters.

Finley was born with a congenital heart defect called transposition of the great arteries, where the two main arteries that supply blood to the lungs and body are in the wrong position. At just four days old, he underwent his first open-heart surgery to return the major arteries to their normal position.

Finley’s mother, Melissa Hudd, explained: “Everything happened so quickly following giving birth. It was such a shock to learn that Finley had a heart condition and needed open heart surgery. When he was born, I might only hold his hand for a few seconds through the incubator before being taken to the ambulance.

“We were told to prepare from the start that the chances of him surviving without urgent surgery were not good and the operation would take around 4-6 hours. After 12 long hours, Finley was finally out of surgery but he was dependent on a machine that took over the function of his heart and lungs. »

There were complications during the first open-heart surgery, and Finley’s heart function deteriorated significantly. Doctors tried a number of treatments, but Finley was still stuck in intensive care for many weeks, relying on medication and a ventilator to keep his heart going. So for compassionate reasons as a world first, Professor Caputo used donor stem cells to inject directly into Finley’s heart in a second surgery, in the hopes they would help the blood vessels damaged to grow to increase blood supply to the left side of his heart. .

Melissa continued: “We almost lost Finley when he was just 2 months old. The doctors called us into a room and told us they had done everything they might. It was then that Massimo came to us and explained that there was only one option left – to inject stem cells into the left side of Finley’s heart. He warned us that he might not predict what the outcome would be. But we had absolutely nothing to lose. We had to try to give Finley every possible chance to live.

“Within two weeks of the stem cell treatment, we noticed a change in Finley. He first came home when he was just 6 months old on a machine that still helps him breathe at night.

“We can’t thank Massimo enough. I believe that if it weren’t for the stem cell treatment, then Finley wouldn’t be here with us today. Finley is very feisty and very funny – he’s a real warrior from the heart and I tell him that all the time.

“We don’t know what the future holds, but we are very grateful that Finley’s life has changed following stem cell treatment, as he now has a chance to live that he might not have. had otherwise. The stem cell dressings that Massimo is now developing looks amazing. If they might give the gift of life and save other families from the emotional turmoil of their children having undergone repeated open-heart surgery, it would change their lives.

Patient Story #2

Louie, 13, from Cardiff, was born with a number of congenital heart defects. He has already undergone two open-heart operations to correct them, which were carried out by Professor Massimo Caputo. But Louie knows he will need the same operation for the third time in the near future.

Louie’s mother, Lottie Stokes, explains: “We thought everything was fine, but the day following Louie gave birth we were told that her heart was plugged in the wrong way, a large blood vessel in her heart was too narrow and he had a hole in his heart, which needed to be repaired. We talked quickly regarding what the doctors were going to do to keep him alive right now, what they would do in the future, and how many operations he would need.

Louie underwent his first open-heart surgery two weeks before his first birthday where surgeons used a synthetic patch to close the hole and a prosthetic valve to allow blood to flow properly around and out of the heart. Louie then underwent the same operation at the age of 4 to replace the materials securing his heart, with several procedures in between.

Lottie continued: “The worst part is saying goodbye to Louie with every operation. It’s basically handing your beautiful little baby over to surgeons just hoping they’ll take care of them.

“Now Louie is older, he’s not letting his heart condition stop him. He lives and breathes football and enjoys playing it with his friends, but he finds it exhausting at times. With his t-shirt, you suspect nothing. , but when you see his surgery scars you just think wow, this boy has been through a lot.

“We’ve always spoken to Louie regarding his ‘special heart’. He needs checkups every 6 months, and we know he’ll need a third open-heart surgery at some point, but we don’t know when – it’s a waiting game. It’s not a fun game to play at all.

“Our heart is with Massimo, he is truly our hero. If Massimo’s stem cell research can eliminate the need for other babies to have these multiple surgeries, it would mean the world to families so they don’t have to go through that heartache over and over like we did it. »

BHF-funded research has turned ideas that once sounded like ‘science fiction’ into treatments and cures that save lives every day. But despite all our progress, millions of people are still waiting for tomorrow’s scientific breakthroughs, so they can spend more time with the people they love. It is only through generous donations from the public that we can give hope to children like Finley and Louie and find the cures and treatments of tomorrow.

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