Funding success for Parkinson’s research

24 Sep 2020, 1 p.m.

Two multi-million-pound research grants will fund a pioneering new programme of research aimed at understanding and tackling Parkinson’s disease, including vital genetic investigations at the UCL Great Ormond Street Institute of Child Health (ICH).

While the root cause of Parkinson’s is still poorly understood, it is clear that certain genetic factors increase the risk of developing the disease and can be passed on between generations. With world-renowned expertise in the discovery, diagnosis and interpretation of genetic syndromes that affect children, the ICH/UCL Genomics team are ideally placed to contribute to these studies.

Getting to the root of Parkinson's 

Totalling more than $14 million (more than £10 million), the awards for global research initiative Aligning Science Across Parkinson's will help researchers understand the root cause of Parkinson’s, why the condition progresses so quickly in some patients but not others, and how we might slow its devastating effects.

Dr Mina Ryten, Professor of Clinical Genetics at the ICH and member of the National Institute of Health Research (NIHR) Great Ormond Street Hospital Biomedical Research Centre (BRC) Genomics and Systems Medicine theme, will provide genetic expertise for the project by analysing adult brain tissue to understand the mechanisms underlying Parkinson’s disease.

‘This research could make a real difference to families who currently face an uncertain and difficult future after a Parkinson’s diagnosis,’ says Professor Ryten. ‘As specialists in rare and complex childhood conditions, many of which have a genetic root cause, our team at GOSH has the expertise to carry out detailed genetic sequencing and molecular analysis. I hope these studies will also provide useful data for degenerative brain disorders affecting children, as well as important methods and analyses that could be applied to our work in paediatrics.’

Studying cells to map the brain

The grants will provide approximately £1.5 million for three years of genetic research at the ICH, with much of the analysis taking place in the Zayed Centre for Research into Rare Disease in Children. This newly opened centre is a partnership between Great Ormond Street Hospital (GOSH), University College London (UCL) and Great Ormond Street Hospital Children’s Charity (GOSH Charity).

The ICH team will study individual brain cells taken from adults during the early stages of Parkinson’s. They will look at the genetic instructions inside these cells and how the instructions are interpreted by the body. This could reveal key genes and processes that are responsible for the most severe symptoms of Parkinson’s, as well as why it progresses so quickly in some patients but not others.

The research team hope to produce the first open-access map of the Parkinson’s brain, including the genes and cell pathways that most influence its progress. This unique resource will support the research community to better understand the condition and, crucially, develop new ways of tackling its devastating effects.

Read more on the UCL website.

Patients and public help create multilingual resources for expectant parents

A group of clinicians and researchers at GOSH have developed information about a test offered in pregnancy called prenatal sequencing in the UK’s 12 most common languages.

New VR game to help children rehabilitate after brain tumour treatment

The National Institute for Health and Care Research Great Ormond Street Hospital Biomedical Research Centre (NIHR GOSH BRC) is working with partners to develop a new virtual reality (VR) game.

Gene therapy offers potential to extend lives of children with rare immune disorder

Children have had their lives changed by a ground-breaking gene therapy treatment at Great Ormond Street Hospital for a rare immunodeficiency.

Cutting-edge genomic technology saves girl from rare brain infection

Great Ormond Street Hospital has launched the UK’s first accredited ‘metagenomics’ testing service, that allows clinicians to identify infections that are otherwise undetectable.