LifeArc Invests $50 Million to Launch Rare Disease Research Centers

Rare Daily Staff

The UK charity LifeArc is investing $50 million (£40 million) to launch four new LifeArc Translational Centres for Rare Diseases to provide people living with rare medical conditions access to improved tests, treatments, and potentially cures.

The virtual centers will focus on areas where there are significant unmet needs to tackle barriers that ordinarily prevent new tests and treatments reaching patients with rare diseases and speed up the delivery of rare disease treatment trials.

The centers will bring together leading scientists and rare disease clinical specialists from across the United Kingdom for the first time, encouraging new collaborations across different research disciplines and providing improved access to facilities and training.

Rare disease research can be fragmented. Researchers can lack access to specialist facilities, as well as advice on regulation, trial designs, preclinical regulatory requirements, and translational project management, which are vital in getting new innovations to patients.

“Each center has been awarded funding because it holds real promise for delivering change for people living with rare diseases,” said Catriona Crombie, head of Rare Disease at LifeArc. “These centers also have the potential to create a blueprint for accelerating improvements across other disease areas, including common diseases.”

The LifeArc Centre for Rare Respiratory Diseases is a UK-wide collaborative center co-created in partnership with patients and charities, led by Kev Dhaliwal, professor of Healthcare Technology at the University of Edinburgh, Institute for Regeneration and Repair and honorary consultant in Respiratory Medicine, NHS Lothian. The Centre is a partnership between universities and NHS Trusts across the UK.

It will provide a single center that will connect children and adults with rare respiratory disease with clinical experts, researchers, investors and industry leaders across the UK. The $11.7 million (£9.4 million) center will create a UK-wide biobank of patient samples and models of disease that will allow researchers to advance pioneering therapies and engage with industry and regulatory partners to develop innovative human clinical studies.

The LifeArc-Kidney Research UK Centre for Rare Kidney Diseases is being launched in partnership with co-funder Kidney Research UK and it will be led by Louise Oni, senior lecturer in Pediatric Nephrology at the University of Liverpool and honorary consultant pediatric nephrologist at Alder Hey Children’s Hospital. Rare kidney diseases need urgent attention as a major report from Kidney Research UK has shown that kidney failure could overwhelm the health care system within ten years.

Rare kidney diseases collectively account for at least 10 percent of adults with kidney failure and almost all children with kidney failure. They also present a lifelong impact.  The new $13 million (£10.4 million) center will unite researchers, patients and healthcare professionals and build on strong established resources, including the national registry of rare kidney diseases (RaDaR), the national renal sample biobank (NURTuRE) and care guidelines. Kidney Research UK is contributing an additional $1.25 million (£1million) to be used to support the work of the center over the next five years.

The LifeArc Centre for Rare Mitochondrial Diseases is a national partnership with the Lily Foundation and Muscular Dystrophy UK. It will be led by Patrick Chinnery, professor of Neurology at the University of Cambridge and honorary consultant neurologist at Cambridge University Hospitals NHS Trust, and key partners at University College London, Newcastle University and three other centers (Oxford, Birmingham and Manchester).

Mitochondrial diseases often cause progressive damage to the brain, eyes, muscles, heart, and liver, leading to severe disability and a shorter life. There is currently no cure for most conditions, however, new opportunities to treat mitochondrial diseases have been identified in the last five years, meaning that it’s a critical time for research development. The $9.3 million (£7.5 million) center will establish a national platform that will connect patient groups, knowledge and infrastructure in order to accelerate new treatments getting to clinical trial.

The LifeArc Centre for Acceleration of Rare Disease Trials brings together a consortium of three universities from across the UK. Newcastle University, Queens University Belfast, and University of Birmingham are pooling their expertise in a partnership coordinated by David Jones, professor of Liver Immunology at Newcastle University. Clinical trials for rare diseases are challenging and can be a major limiting step in getting new treatments to patients. The $15 million (£12 million) center will deliver trials of new treatments using one-stop, patient friendly models. The research team will create a rare disease trial recruitment portal and will design and deliver trials in partnership with patients. This will speed up the delivery of clinical trials for people with rare diseases and enable more rapid approval of new therapies for use in the NHS.

Photo: Catriona Crombie, head of Rare Disease at LifeArc