Action Medical Research and LifeArc announce another round of funding to support translational research into children’s rare diseases

Children’s charity Action Medical Research and independent medical research charity LifeArc are pleased to announce a fourth joint funding round aimed at supporting UK-based translational research projects that could lead to new interventions (therapeutics, diagnostics and devices) for infants, children or young people with a rare disease.

The charities have created a £1m fund for researchers seeking additional funding to progress promising translational research projects aimed at addressing a clear unmet medical need for children and young people with a rare disease.

The joint call is open to projects developing a broad range of intervention types (therapeutics, diagnostics, and devices), including the development of targeted gene therapy approaches that could be particularly well-placed to benefit the high proportion of monogenic paediatric rare diseases. Further information on remit, eligibility and how to apply can be found on the Action Medical Research website.

This is the fourth joint funding call issued by the charities. Projects successfully funded in the last round are expected to be announced this summer. In the 2020 funding round three projects were jointly funded; one optimising new drug treatments for Duchenne muscular dystrophy, another aiming to improve diagnosis and monitoring of hypertrophic cardiomyopathy in children, and a third project looking to develop new drug treatments for pyridoxine dependent epilepsy.

Two projects in the 2019 funding round were funded; one assessing whether the combination of existing drugs could be used as a new treatment for childhood leukaemia and another aiming to improve the effectiveness of a stem cell gene therapy for Hunter Syndrome.

A recent report for 2022 Rare Disease day focused on the impact an improved stem cell gene therapy could have for children with Hunter syndrome, highlighting how research funded by this joint scheme could have real benefit for patients.

Dr Catriona Crombie, Head of LifeArc’s Philanthropic Fund said: “Through this joint funding call, LifeArc and Action Medical Research have already committed over £1.1m to support five translational research projects focused on developing improved therapies and diagnostics for children and young people with rare disease.

“Our continued partnership with Action Medical Research will provide the funds and expertise to help translate further high-quality research into interventions that could benefit paediatric patients with rare disease.”

Dr Tracy Swinfield, Research Director at Action Medical Research said: “Our ongoing partnership with LifeArc enables us to fund cutting-edge medical research that could have a significant impact on the treatment of babies, children and young people living with rare and sometimes devastating disease. ”

The deadline for submitting an outline application is 21 June 2022. Please click here for more information on the joint fund.