LifeArc rare disease programme to invest £100m improving patient lives

rare disease patients
National Cancer Institute

Self-funded, non-profit medical research organisation and charity LifeArc has announced today that it is to launch the Rare Disease Translational Challenge, which will invest over £100 million by 2030 in the improvement of the lives of people living with rare disease.

The Challenge is set to offer funding and support to researchers in order to help find new diagnostic technologies, develop new treatments, accelerate new research, and remove some of the obstacles to receiving better care that currently exist.

Globally, more than 300 million people are thought to live with a rare disease, with approximately 3.5 million in the UK affected. More than 7,000 rare diseases have so far been identified and there is clear and unmet need for new treatments and innovative technologies.

An initial commitment of £40 million will go towards the creation of up to five Translational Rare Disease Centres across the UK, bringing together experts in the field that specialise in different aspects of rare disease research.

A £2.5 million commitment in partnership with DEBRA Austria kicks things off, inviting researchers focused on repurposing drugs to help treat the rare skin disease, Epidermis Bullosa (EB).

The Rare Disease Translational Challenge aims to bring together researchers in rare disease with the expertise that LifeArc can offer in translational science. It specifically looks to address some of the issues people living with rare disease face, particularly as pertains to ending the ‘diagnostic odyssey’ that rare disease patients go through to get access to clinical trials.

Close collaboration with the rare disease research ecosystem, patients and their families, patient groups, and more will be crucial.

CEO of LifeArc, Melanie Lee, said: “LifeArc has been supporting rare disease research for a number of years, committing over £32 million since 2019, and I am thrilled to be launching our Rare Disease Translational Challenge today.”

Lee continued: “[The Challenge] represents a significant step forward in our commitment to advancing rare disease research and do our best to serve the millions of individuals and families affected by these often devastating conditions.”

LifeArc’s chief project and portfolio officer, Karen Skinner, commented: “Our Translational Challenges are ambitious, collaborative research programmes shaped by what patients tell us they need and [are] designed to tackle complex health issues by taking the best scientific ideas out of the lab and helping turn them into medical breakthroughs that can be lifechanging.”

Previous LifeArc programmes have included the Neurodegenerative Translational Challenge, focused on conditions such as motor neurone disease (MND) and dementia, as well as the Chronic Respiratory Infection Translational Challenge, aimed at improving care for people living with conditions like cystic fibrosis, and a Global Health programme, targeting antimicrobial resistance and emerging viral threats.

Catriona Crombie, head of the Rare Disease Translational Challenge at Life Arc, said: “Through our Rare Disease Translational Challenge, 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.”

Additional comment came from Louise Fish, chief executive of Genetic Alliance UK, who said: “We’re delighted by LifeArc’s commitment to support early career researchers in the UK and help them establish careers in an area where there is so much unmet need, and to make sure this amazing investment focuses on the areas that matter most to people living with rare conditions.”

Dr Gail Marzetti, director of science, research, and evidence at UK Department of Health and Social Care, added: “Pioneering research is crucial for improving care and treatment for people living with rare diseases and is an underpinning theme of the UK Rare Diseases Framework […] We welcome LifeArc’s funding, which will complement our existing investments, and help further the UK’s position as world-leader in research into rare diseases for patient benefit.”

In May, pharmaphorum spoke with Jennifer Schranz, SVP and global head of rare disease at Ipsen, about how, in the rare and ultra-rare disease space, the paucity of both data and patients makes everything about research and development more complicated. Watch the video here or below.