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Developmental Biology and Cancer Research & Teaching Department Latest News

Latest News

Lab-grown mini-organs could offer treatment hope for children with intestinal failure

Photograph of Paolo De Coppi
7 September 2020

Pioneering scientists at Great Ormond Street Hospital (GOSH), the UCL Great Ormond Street Institute of Child Health (ICH) and the Francis Crick Institute have grown human intestinal grafts using stem cells from patient tissue that could one day lead to personalised transplants for children with intestinal failure, according to a study published in Nature Magazine

Many colleagues from the Developmental Biology and Cancer Research and Teaching Deaprtment have been involved in this highly promising study, and full details may be found at this link

 

Dr Erwin Pauws appointed to be a new UCL MSc Programme Director

 
Dr Erwin
Dr Erwin Pauws
Pauws of the developmental BIOLOGY of birth defects section (craniofacial malformation research group) to co-lead a UCL MSc Programme

Making the announcement on 18th June 2020 regarding Dr Pauws new role, Prof. Ros Smyth, Director and Professor of Child Health, Great Ormond Street UCL Institute of Child Health stated:

I am delighted to announce the appointment of two new Programme Directors for the MSc in Paediatrics and Child Health and the MSc in Personalised Medicine and Novel Therapies. Dr Erwin Pauws and Dr Haiyan Zhou respectively will be taking on these important roles as co-leads alongside the existing Directors, Professor Helen Bedford and Dr Mark Kristiansen. These are important leadership roles which will be crucial to our success in education over the upcoming years and in particular with our new online intake commencing in the new academic year. Congratulations to both Haiyan and Erwin who have both shown great enthusiasm for the opportunities offered by the positions and who I believe will make many positive contributions to GOS ICH in these roles.

The Developmental Biology and Cancer Research and Teaching Department congratulates Dr Pauws on his well deserved appointment.

 

Professor Paolo De Coppi elected to the prestigious Academy of Medical Sciences Fellowship

Photograph of Paolo De Coppi

The Academy of Medical Sciences announced on 13th May 2020 they had elected 50 of the UK’s most prominent biomedical and health scientists to their Fellowship, including the internationally renowned Professor Paolo De Coppi, Head of the Stem Cells & Regenerative Medicine Section at UCL Institute of Child Health, Developmental Biology and Cancer.

Developmental Biology and Cancer Research and Teaching Department warmly congratulates Prof. De Coppi on this most laudable achievement.

Further details may be found at the Academy's press release, at the following web address https://acmedsci.ac.uk/more/news/50-leading-biomedical-and-health-scientists-elected-to-the-prestigious-academy-fellowship

 

GOSH Charity and Sparks award over £1 million for child health research at the Institute

GOSH Charity and Sparks, the children’s medical research charity, have announced funding for five projects at the UCL Great Ormond Street Institute of Child Health (ICH), as part of the UK’s largest funding scheme dedicated to child health research.

Image of Dr Jane Sowden

 

 

 

 

 

 

Projects include pioneering research to save the hearing of children who are born without sight, supporting the UK arm of a global clinical trial to test whether the breast cancer drug tamoxifen could help children with a rare muscle disorder, and creating an implantable device that supports liver function and could hold the key to reducing toxic chemical levels in the blood and avoiding the need for liver transplant.

This year, the charities’ annual funding scheme will invest £2.3 million in 11 pioneering child health projects around the UK. After an open call for applications in 2019, each proposal was rigorously assessed by expert committees and researchers around the globe. Those selected, including the five projects at the ICH, were rated extremely highly and recognised as having the potential to significantly impact child health.

Projects funded at the ICH:

  • Professor Nicholas Greene will test a new compound derived from cinnamon that could help lower toxic levels of ammonia and glycine in children with the rare metabolic diseases non-ketotic hyperglycinemia and Urea Cycle Disorders.
  • Dr Giovanni Baranello’s project will support the UK arm of a global trial understanding if the breast cancer drug tamoxifen could help children with the muscle disorder X-linked myotubular myopathy.
  • Professor Jane Sowden (pictured above) will investigate whether replacing the faulty gene that causes deafness in Norrie Disease could save the hearing of boys with the condition, who are born blind. This is a continuation of the pioneering work done by Professor Maria Bitner-Glindzicz, who sadly passed away in 2018.
  • Professor Paul Gissen aims to tackle all symptoms of a complex multi-organ disorder, Arthrogryposis Renal Dysfunction and Cholestasis Syndrome (ARC), with two types of gene therapy at once. The condition affects the liver, kidneys and bone marrow cells. Most patients die in early childhood so new treatments are desperately needed.
  • Dr Hassan Rashidi hopes to develop an implantable and removable ‘liver patch’ to provide liver support to patients with the metabolic condition non-ketotic hyperglycinemia, helping them reduce the toxic levels of glycine without the need for liver transplant.

Links

Research at the UCL Great Ormond Street Institute of Child Health

Professor Nick Greene's academic profile

Dr Giovanni Baranello's academic profile

Professor Jane Sowden' s academic profile

Professor Paul Gissen's academic profile

Dr Hassan Rashidi's academic profile

GOSH Charity Press Release

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UCL-PKU Strategic Partner Funds: Prof. Nick Greene

UCL professors Nick Greene and Andrew Copp have teamed up with colleagues at Peking University (PKU) to help prevent one of world’s most common birth defects.

Image of Nick Greene and Andy Copp and colleagues from Peking University

*Update 9/3/2020: Professor Nick Greene received around £300,000 from the Joint Global Health Trials scheme, which is funded by the Department for International Development, the Medical Research Council, the National Institute for Health Research and Wellcome Trust. Prof Greene is the principal investigator with co-applicants from UCL GOS Institute of Child Health, UCL Comprehensive Clinical Trials Unit and Peking University. The team also obtained funding from Bo Hjelt Foundation, which will go towards supporting a two-year development trial in Shanxi Province, China as a precursor to a larger study. The development trial is intended to start on 1 April 2020.


UCL has teamed up with Peking University (PKU) in a bid to prevent tens of thousands of babies suffering from spina bifida, one of the world’s most common birth defects.

Researchers at UCL’s Great Ormond Street Institute of Child Health and Comprehensive Clinical Trials Unit are proposing a £5m study of up to 9,000 women in China to confirm initial findings that a vitamin common in fruit, vegetables, meat and nuts could prevent neural tube defects (NTDs) such as spina bifida.

“If successful, it’s a global treatment that could be rolled out and prevent thousands of pregnancies being affected by spina bifida,” said Professor Nick Greene, who is leading the UCL team.

Image of ICH Building
Worldwide, it is estimated that around one in every 1,000 pregnancies are affected by NTDs, with more than 260,000 babies born every year with spina bifida, a severely disabling malformation of the spinal cord.

Women are advised to take supplemental folic acid before and during early pregnancy to reduce the risk of an affected pregnancy. A number of countries, including the USA, Canada and Australia, have also introduced mandatory fortification of food with folic acid and seen a reduction in NTD prevalence.

Nevertheless, some babies still develop spina bifida despite folic acid supplementation or fortification. For example, there are still between 700 and 900 pregnancies affected by neural tube defects each year in the UK.

Research by the UCL team focussed on identifying possible ways to prevent ‘folic acid resistant’ spina bifida. Leading on from their laboratory-based studies on inositol (vitamin B8), they carried out a pilot clinical trial in the UK, among women who had previously experienced one or more affected pregnancies and were therefore at high risk of recurrence. In their next pregnancy, women were randomly given either folic acid and a placebo or folic acid and inositol.

None of the babies born to women who took inositol had spina bifida, but it did occur in some pregnancies where only folic acid was taken. Published in 2016, the findings of this first randomised trial of inositol for prevention of spina bifida indicated the need for a large-scale ‘fully powered’ clinical trial.

 

None of the babies born to women who took inositol had spina bifida, but it did occur in some pregnancies where only folic acid was taken. Published in 2016, the findings of this first randomised trial of inositol for prevention of spina bifida indicated the need for a large-scale ‘fully powered’ clinical trial.

Now Professor Greene and his colleagues hope, with their collaborators at PKU, to use China’s unparalleled database of information on birth defect occurrence to mount a large-scale study to establish whether inositol will work as an effective global treatment to prevent spina bifida, alongside folic acid.

The prevalence of spina bifida in Northern China, where the study will be carried out, is among the highest in the world.

Because of China’s systematic collection of data, the team will be able to find the necessary 9,000 women who have previously suffered a spina bifida pregnancy and are planning another pregnancy so that they can be given inositol.

“Together with its large population, China has a very comprehensive coverage of pregnancy outcomes, which means it is one of the few places that this trial would be possible,” said Professor Greene.

A bid to fund the clinical trial is currently being considered.*

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Research Highlights

Paper of the month

The paper of the month this month goes to a group from Developmental Biology and Cancer. Both joint first authors were undergraduates when they completed the work reported in this paper in Dr Gabriel Galea's lab. Nina Short, who was awarded a Child Health Research Summer Internship, and Max Butler was awarded a Wellcome Biomedical Vacation Studentship.  The last author in the paper is Gabriel Galea and the other authors are also from this Institute. 
 
The study focusses on the role of the enzyme Rho-associated protein kinase (ROCK) enclosing the posterior neuropore (PNP) in the neural tube.  The main finding from the study is that ROCK dependent apical constriction compensates for the PNP widening effects of the inter-kinetic nuclear migration to enable progression of closure of the neural tube. 

 There is also a first person interview with Max and Nina published in the same edition of the journal.  A very well done to students and the rest of the research team!

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