April 2024 • PharmaTimes Magazine • 7
// RESEARCH //
Researchers from University College London (UCL) and Great Ormond Street Hospital (GOSH) have developed a new approach to observing human development in late pregnancy to potentially monitor and treat congenital conditions before birth.
Published in Nature Medicine, the study used collected stem cells from late pregnancy to create and grow cell models, known as organoids, to retain babies’ biological information.
The study of stem cell organoids aims to facilitate the monitoring of foetal development in late pregnancy, model disease progression and test new treatments for diseases including congenital diaphragmatic hernia (CDH).
Affecting one in 5,000 babies born in the UK, CDH occurs when there is a hole in the diaphragm, which can lead to the foetus’ bowel, stomach or liver being moved into the chest cavity during pregnancy.
Researchers extracted and characterised live cells from 12 pregnancies as part of routine diagnostic testing and used single-cell RNA sequencing to identify which tissues they came from.
The stem cells were collected through the amniotic fluid surrounding the child to overcome sampling restrictions, as the child should not be touched during the collection process.
Collected stem cells from the lungs, kidneys and intestines were successfully extracted and used to grow organoids that contained functional features of these tissue types.
Working with researchers from KU Leuven, Belgium, the team studied the development of babies with CDH both pre- and post-treatment to compare organoids from healthy babies to understand the biological characteristics of each group.
Results found that there were significant developmental differences between the healthy and pre-treatment CDH organoids. However, the organoids in the post-treatment CDH group were much closer to healthy ones, providing an estimate of the treatment’s effectiveness at a cellular level.
Senior author, professor Paolo de Coppi, UCL Great Ormond Street Institute of Child Health and GOSH, said: “Functional prenatal organoids is the first step towards being able to offer a more detailed prognosis and, hopefully, provide more effective treatments in [the] future.”
The National Institute for Health and Care Research (NIHR) has announced a new phase 1/2 trial, delivered by the NIHR Clinical Research Network and sponsored by Moderna, to test the effectiveness of an investigational mRNA vaccine for mpox.
The mPower trial will evaluate the safety and immune response to mRNA-1769, which aims to protect against illness caused by the mpox virus.
Currently a global public health threat, mpox is an infectious disease caused by the monkeypox virus, which is spread through physical contact with someone who is infected, leading to symptoms including painful rashes, enlarged lymph nodes and fever.
Since May 2022, over 3,700 cases of mpox have been identified in the UK, the majority of which have been from the Clade II B.1 lineage, predominantly in gay, bisexual and other men who have sex with men.
In the UK and Europe, there is only one vaccine licensed for the immunisation against mpox, called Imvanex, manufactured by Bavarian Nordic, along with Jynneos in the US and Imvamune in Canada.
Undertaken as part of the Moderna-UK Strategic Partnership, the trial aims to recruit around 350 healthy participants between the ages of 18 and 49 years old who have not been previously vaccinated for mpox and who have not had a suspected or confirmed mpox infection to receive the investigational vaccine or a placebo.
Researchers from University Hospitals Bristol and Weston NHS Foundation Trust are hoping to recruit a further 175 volunteers for the study, aiming to complete enrolment by April this year.
Dr Matthew Hallsworth, NIHR Director of Strategic Partnerships, said: “Our partnership with Moderna ensures UK research is at the cutting edge of new vaccine technologies with the potential to protect against global health threats such as mpox and future pandemics.
“We hope that recruitment to this trial will be as successful as the COVID-19 vaccine trials that were run in the UK and we encourage the public to help out where they can.”