Newcastle to co-ordinate national consortium in rare diseases following £14m investment

Newcastle is set to be the central co-ordination and administrative hub for a new platform bringing together UK strengths in rare disease research. It will also make a major contribution to the research programmes that make up the platform.

The UK Rare Disease Research Platform has been established with a £14 million investment over five years by the Medical Research Council (MRC) and the National Institute for Health and Care Research (NIHR).

The aim of the new platform is to bring together expertise from across the UK rare disease research system to foster new and innovative treatments for those directly and indirectly impacted by rare conditions. It will overcome some of the most common challenges in the diagnosis, management and treatment of rare diseases.

As a centre of research excellence, Newcastle has a distinguished history of research and clinical care in rare disease. It is home to the Newcastle Centre of Research Excellence for Rare Disease (NUCoRE RD), which benefits from decades of collaboration between Newcastle University and Newcastle Upon Tyne Hospitals NHS Foundation Trust, which form part of Newcastle Health Innovation Partners (NHIP), the Academic Health Science Centre (AHSC) for our area.

The new national Rare Disease Research Platform is made up of a central coordination and administrative hub, which will be based in Newcastle, as well as 11 specialist nodes across the UK.  Newcastle will also host two of these nodes, each of which involve several universities and organisations working together across the UK. They are:

  • Epigenomics Rare Diseases (EpiGenRare) led by University of Exeter and The University of Manchester
  • mTOR Pathway Diseases led by King’s College London
  • Changing clinical practice in rare diseases through innovative trial designs (CAPTIVATE) led by University of Birmingham
  • Histiocytic Disorders (HistioNode) led by Newcastle University and The University of Nottingham
  • Lipidomics and Metabolomics led by Swansea University
  • Ethical Legal and Social Issues in Rare Conditions Research and Clinical Practice (ELSI) led by The University of Manchester
  • Early Assessment, Diagnosis and Treatment of Parkinson’s Plus Related Syndromes (ExPRESS) led by University College London
  • Rare Early Onset Lower Urinary Tract (REOLUT) led by The University of Manchester
  • Renal Ciliopathies National Network (RCNN) led by Newcastle University
  • Cardiovascular Rare Disease led by Imperial College London
  • UK Platform of Nucleic Acid Therapy for rare disease treatment (UPNAT) led by University College London

The platform hub will bring these specialist nodes together and deliver wider activities for all the UK interests in rare disease research.

The hub contract was awarded through an open competitive procurement exercise to a consortium led by Newcastle University, with Newcastle NHS Trust and Genetic Alliance UK.

Professor David Jones OBE, Director of Newcastle Centre for Rare Disease, said: “We have a long and distinguished history of excellence in rare disease research in Newcastle. This includes world class programmes in muscular dystrophy, mitochondrial disease, immuno-deficiency, rare liver disease, rare cancer and rare renal disease – to name but a few.

“We set up the Newcastle Centre of Research Excellence for Rare Disease to bring these areas of excellence together – working across Newcastle University and Newcastle Hospitals to collaboratively improve the lives of people living with a rare disease.

“We are therefore delighted with the announcement of this new platform as it strengthens our vision of bringing experts together for the benefit of patients. It is also an honour to co-ordinate the whole platform and host two nodes.”

Professor David Burn, NHIP Director, added: “We are very proud of our outstanding reputation in rare disease research here in Newcastle and this announcement bolsters this even further. The new platform is a great way of bringing experts together and I look forward to seeing how this innovative, cross sector working will benefit patients.”

The Newcastle Centre of Research Excellence for Rare Disease unites over 100 experts from across Newcastle University and Newcastle Hospitals – working collaboratively to improve the lives of people living with a rare disease. By bringing together a breadth of scientific expertise alongside patient cohorts, the centre can provide a single front door in the design and execution of clinical research, providing a better disease understanding, developing and delivering better treatments, optimising care and influencing policy.

This wider investment includes support for a range of rare diseases research through NIHR’s Biomedical Research Centres – including the NIHR Newcastle Biomedical Research Centre, which sits at the heart of Newcastle’s world-leading centre for the study and treatment of rare diseases.

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