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Stem cell breakthrough could help treat conditions in babies before birth

UCL scientists have created ‘organoids’ (pictured) using stem cells (Giuseppe Cala/Paolo di Coppi/Mattia Gerli/PA Wire)
UCL scientists have created ‘organoids’ (pictured) using stem cells (Giuseppe Cala/Paolo di Coppi/Mattia Gerli/PA Wire)

London scientists have discovered a way to potentially treat conditions in babies before birth using stem cells collected in late pregnancy.

Researchers at University College London and Great Ormond Street Hospital used human stem cells to grow complex cell models called organoids that retain a baby’s biological information.

The organoids will be used to monitor fetal development in late pregnancy and test new treatments for fatal diseases such as congenital diaphragmatic hernia (CDH). The condition causes a hole to open up in the diaphragm, meaning that organs such as the intestine get displaced into the chest and disrupt growth.

Until now, organoids have only been made using adult stem cells or fetal tissue taken after the termination of a pregnancy. In the UK this can be done up to 22 weeks after conception, the legal limit for abortion.

However, scientists say these restrictions have limited opportunities to use organoids to study diseases at a point when they may still be treatable.

For the study, the researchers extracted live cells from amniotic fluid in the wombs of 12 women. A technique called single-cell RNA sequencing was then used to identify where in the body these cells came from, such as the lungs or kidneys.

These cells were used to grow organoids that had the functional features of these organs.

Dr Mattia Gerli, first author of the study from UCL Surgery & Interventional Science, said: “The organoids will allow us to study what is happening during development in both health and disease, which is something that hadn’t been possible before. We know so little about late human pregnancy, so it’s incredibly exciting to open up new areas of prenatal medicine.”

The researchers also worked with scientists at KU Leuven in Belgium to study the development of babies with CDH.

Organoids from babies with CDH both before and after treatment were compared to organoids from healthy babies to study the biological characteristics of each group.

Scientists identified "significant development differences" between the healthy organoids and those that had not received treatment.

However, the organoids in the post-treatment CDH group were much closer to healthy ones, providing an estimate of the treatment’s effectiveness.=

Professor Paolo de Coppi, senior author of the study from UCL Great Ormond Street Institute of Child Health, said: “When we meet families with a prenatal diagnosis, we’re often unable to tell them much about the outcome because each case is different. We’re not claiming that we can do that just yet, but the ability to study functional prenatal organoids is the first step towards being able to offer a more detailed prognosis and, hopefully, provide more effective treatments in future.”