Action Medical Research and LifeArc announce joint funding awards for translational research into children’s rare diseases

Children’s charity Action Medical Research and British life science medical research charity LifeArc are pleased to announce a third joint funding round of up to £1 million. The funding aims to support UK-based translational research projects that could lead to new interventions for children with rare diseases.

The joint fund will be used to support researchers seeking additional funding to progress projects that have moved beyond the basic understanding of disease mechanisms into the development of an intervention (therapeutic, diagnostic or device) aimed at addressing a clear, unmet medical need for children and young people with a rare disease.   

The joint call is open to a broad range of intervention types and modalities. Examples of eligible applications could include projects focused on the development of gene therapies which could benefit the high proportion of monogenic paediatric rare diseases. Further information on eligibility, funding and how to apply can be found on the Action Medical Research website. 

Dr Tracy Swinfield, Research Director at Action Medical Research said: “Through this ongoing partnership with LifeArc, we aim to accelerate cutting-edge medical research that could have a significant impact on the treatment of infants, children and young people living with rare and sometimes devastating disease. Action Medical Research and LifeArc strive to improve the lives of children suffering from rare disease and are working together to co-fund translational research projects.” 

This is the third funding call issued through this partnership. In total, Action and LifeArc have invested over £1 million in rare disease research and awarded funding to five projects since 2019. One optimising new drug treatments for Duchenne muscular dystrophy, another aiming to improve diagnosis and monitoring of hypertrophic cardiomyopathy in children and a project looking to develop new drug treatments for pyridoxine dependent epilepsy. A further two projects are now under-way, one investigating gene therapy for the rare and devastating disease Hunter syndrome and another investigating a new drug treatment for childhood leukaemia. 

Dr Catriona Crombie, Head of LifeArc’s Philanthropic Fund said: “Our renewed partnership with Action Medical Research demonstrates our focus on improving the lives of people living with rare diseases. Through our collaboration we can continue to fund high quality research and provide our expertise to translate exciting science towards vital new interventions.”   

The deadline for submitting an outline application is 9th June 2021. 

-ENDS- 

Notes to editors 

Applications can be made via the Action Medical Research website and will need to meet the guidelines provided. Projects costing a maximum of £250,000 and with a duration of up to three years will be considered. Final funding decisions for applications made in this round are expected to be announced in July 2022. 

Projects funded in the 2020 joint call:  

Duchenne muscular dystrophy – optimising new drug treatments
Led by Professor AJ Russell and Professor Dame KE Davies at the Departments of Chemistry and Pharmacology and Department of Physiology, Anatomy and Genetics, University of Oxford. 

Hypertrophic cardiomyopathy in children – improving diagnosis and monitoring
Led by Dr JP Kaski at the Institute of Cardiovascular Science, University College London. 

Paediatric Epilepsy – developing new drug treatments for pyridoxine dependent epilepsy (PDE) 
Led by Professor WW Yue and co-investigators based at Newcastle University Biosciences Institute Centre for Medicines Discovery and Nuffield Department of Medicine, University of Oxford. 

For more information on Action Medical Research:  

Grace Wardley
Communications and Engagement Officer 
+44(0)7725 090 008 
gwardley@action.org.uk  
 action.org.uk 

Action Medical Research is a leading UK-wide charity saving and changing children’s lives through medical research. For nearly 70 years we’ve helped pioneer ways to prevent disease and develop treatments benefiting millions of people. Our research has helped to beat polio in the UK, develop ultrasound in pregnancy, fight meningitis and prevent stillbirths. But we urgently need to develop more new treatments and cures for sick babies and children and we can’t do it without you. 

Join our fight for little lives today. 

Charity reg. nos 208701 and SC039284 

For more information on LifeArc: 

Surinder Maan
Communications Manager  
+44 (0)20 7391 2754 
surinder.maan@lifearc.org
www.lifearc.org 

LifeArc is a self-funded medical research charity. Our mission is to advance translation of early science into health care treatments or diagnostics that can be taken through to full development and made available to patients. We have been doing this for more than 25 years and our work has resulted in a diagnostic for antibiotic resistance and four licensed medicines. Our success allows us to explore new approaches to stimulate and fund translation. 

LifeArc are proud to be renewing joint funding with Action Medical Research, which is the latest in a series of strategic collaborations that leverage LifeArc’ s expertise in translational science – advancing strong discoveries from the lab to a point where rare disease patients can benefit. This includes a recent £8M commitment made by LifeArc to support the delivery of new gene therapy treatment through a unique partnership with the MRC. Through this partnership, LifeArc is partly funding a network of cutting-edge facilities – also known as Gene Therapy Innovation Hubs – to enable the progress of novel, academic gene therapy research into early-stage clinical trials and bring a new generation of medicines to healthcare.

It is hoped that promising gene therapy approaches, such as those supported by LifeArc’s joint fund with Action Medical Research, may benefit from interaction with the Gene Therapy Innovation Hubs as they progress towards clinical trials. 

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