£12m research centre fighting rare diseases
ANEW £12m research centre led from Newcastle could provide hope for millions living with rare diseases.
The new LifeArc Centre for Acceleration of Rare Disease Trials will be run by Newcastle University Professor of Liver Immunology Dave Jones, in collaboration in teams at Birmingham University and Queen’s University Belfast.
The idea is to invest millions in creating specialist facilities to boost life-saving trials, and to give more patients the chance to take part.
Hopefully, the experts said, this would mean new treatments and therapies rolled out more quickly for those who need them.
Prof Volker Straub, an expert in neuromuscular genetics at Newcastle University, said the new centre was “fantastic news” for those with rare diseases.
He added: “The funding also acknowledges Newcastle’s long-standing expertise and excellence in the rare disease field.
“I’m very much looking forward to working with colleagues in Birmingham, Belfast and other rare disease centres, to develop safe and more effective treatments for patients.”
Kerry Leeson-Beevers is the parent of a child with the rare genetic condition, Alstrom Syndrome.
This can cause loss of vision and hearing, and can lead to life-threatening organ problems.
Kerry, who runs Alstrom Syndrome UK, added: “We have no specific treatment for Alstrom Syndrome and when my son, Kion, was a baby, I was told it could take around 10 years for any treatment to be developed - 20 years later, we are still waiting.
“People living with rare conditions don’t have the luxury of time and the mainstream way of delivering healthcare and drug development rarely works for people with rare conditions. As a mum, and the Chief Executive of Alstrom Syndrome UK, having a centre that will deliver a coordinated, inclusive and supportive approach to accelerate clinical trials gives me great hope.”
The non-profit medical research charity LifeArc has awarded the centre a share of nearly £40m, and the idea is that, along with similar specialist centres for rare respiratory, kidney, and mitochondrial disease, it will tackle “unmet” needs.
Dr Catriona Crombie, head of rare disease at LifeArc, said: “We’re extremely proud to be launching four new LifeArc Translational Centres for Rare Diseases.
“Each centre has been awarded funding because it holds real promise for delivering change for people living with rare diseases. These centres also have the potential to create a blueprint for accelerating improvements across other disease areas, including common diseases.”