LifeArc to launch rare disease centres across the UK

Medical research organisation and charity LifeArc has announced the launch of a new programme that will invest more than £100m by 2030 in a bid to improve the lives of people living with a rare disease.

One in 17 people will be affected by a rare disease at some point in their lives, amounting to around 3.5 million people in the UK, yet the lack of scientific knowledge and quality information on rare diseases often results in a delay in diagnosis, as well as limited treatment and management options.

LifeArc said its ‘Rare Disease Translational Challenge’ will offer funding and support to researchers to help accelerate rare disease research, find new diagnostic technologies, develop new treatments and remove some of the obstacles to receiving better care.

An initial £40m will go towards the creation of up to five ‘Translational Rare Disease Centres’ across the UK to “bring together experts in the field that specialise in different aspects of rare disease research, such as new diagnostic approaches and innovative treatments”.

Catriona Crombie, associate director of technology transfer and Rare Disease Challenge lead at LifeArc, said: “Through our Rare Disease Translational Challenge, we will leverage our expertise in drug discovery, diagnostics and translational science.

“We will actively seek partnerships with other charities, academic institutions, industry and patient advocacy groups, forming a network of dedicated individuals and organisations pursuing the same mission – to transform the lives of people living with a rare disease.”

Among the first projects to receive funding through the programme will be a £2.5m commitment in partnership with patient organisation charity DEBRA Austria to fund projects focused on repurposing drugs to help treat the rare skin disease epidermolysis bullosa (EB).

Affecting around 500,000 people worldwide, EB is a group of rare inherited disorders that causes the skin and internal mucous membranes to become extremely fragile, leading to chronic tissue damage, a persistent inflammatory response and fibrosis.

Progressive systemic disease can result in multi-organ complications, LifeArc outlined, and aggressive squamous cell carcinoma may initiate in chronically inflamed, non-healing wounds.

Crombie said: “While there has been encouraging progress in the development of gene and cell therapies targeting the underlying defects causing EB, there remains a number of serious consequences of the disease, such as chronic inflammation and non-healing wounds that can lead to fibrosis and skin cancer.

“We hope this funding will help unlock the potential of existing therapies that can be repurposed or repositioned to accelerate the development of new treatments for young people living with EB.”

Applications will be accepted from research groups at academic institutions or hospitals globally, LifeArc said, and should be based on a “strong non-clinical data package” with a clear strategy for clinical development.

“The projects should take advantage of data that is already available on how the drug works, that shows how safe the drug is and at what dose it would need to be given,” LifeArc said. “This should provide the basis for these drugs to progress rapidly to testing in people with EB.”

There will be a priority focus on projects aiming to repurpose therapies to resolve non-healing wounds, prevent or reduce chronic inflammation, lower fibrosis or decrease the risk of skin cancer.

Rainer Riedl, managing director, DEBRA Austria, commented: “Children born with EB are often called ‘butterfly children’ because their skin seems as fragile as a butterfly wing, leaving them susceptible to infection and disease that can severely reduce their quality of life. In all our research funding efforts, the patient has to be at the centre.

“We are excited to be working with LifeArc on this £2.5m funding programme and look forward to supporting the development of new treatments for this serious disorder.”

Emily Kimber is a deputy editor at PMGroup