Charities grant £137,000 for Leigh Syndrome research

The Leigh Syndrome International Consortium (LSIC) was launched in September 2019 with the aim of advancing scientific research specifically related to Leigh Syndrome. The founding members include The Lily Foundation (UK), Mito Foundation (Australia), Mitocon Onlus (Italy), People Against Leigh Syndrome (USA) and the United Mitochondrial Disease Foundation (USA).

The group invited leading research institutions to submit proposals that related specifically to Leigh's disease research. More than 20 research proposals were submitted, and subjected to rigorous peer review by an international steering committee of scientists with deep expertise in Leigh Syndrome. Review criteria included factors such as scientific rigor, innovation, and potential to have a positive impact on the lives of patients.

Six projects were selected to receive grants ranging from $15,000 to $50,000. The successful awardees include:

Institution: University of Pittsburgh
Project: Novel dietary and pharmacological therapies in a Drosophila model of Maternally Inherited Leigh Syndrome

Institution: Regents of the University of California
Project: Fumarates for Leigh Syndrome

Institution: Heinrich Heine University (HHU)
Project: iPSC-driven repositioning of PDE5 inhibitors for Leigh syndrome patients carrying MT-ATP6 mutations

Institution: Wellcome Centre for Mitochondrial Research
Project: Leigh syndrome: Investigating Outcome measure & Natural history (LION) - a prospective, longitudinal cohort study

Institution: University of Iowa
Project: The role of microglia, NAD+ and mitochondrial ROS in Leigh Syndrome

Institution: Fondazione Telethon
Project: Therapeutic efficacy of miR-181a/b down regulation in Leigh syndrome

Commenting on the Consortium's decision to award the grants, Alison Maguire, Head of Research & Finance at The Lily Foundation, said:

"The Lily Foundation is proud to be involved in such a forward-thinking project, and to be working together with such a distinguished group of charities and research institutions. International collaboration of this sort is the best way to advance research and bring real, positive change to the lives of patients. The quality of proposals submitted was extremely high, and we are confident the grants we have awarded will lead to improved treatments and therapies for people affected by Leigh syndrome."

The Leigh Syndrome International Consortium has pledged $1 million USD (£770,000 GBP) to fund research with a focus on improving diagnosis, therapeutic development and optimised patient care for Leigh’s patients.