The Leigh Syndrome International Consortium, a patient-driven research network steered by five of the world’s leading mitochondrial disease patient advocacy groups, including Mito Foundation, Mitocon ODV, People Against Leigh Syndrome (PALS), The Lily Foundation, and the United Mitochondrial Disease Foundation (UMDF), is pleased to announce it will grant a total of $150,000 USD to five research teams that are actively working toward improving diagnosis, developing treatments and optimizing clinical care for Leigh syndrome patients. This is the second round of research grants awarded by the group since 2019 for a grand total of $329,000 USD in funding.
Fifteen institutions from around the world answered the Consortium’s request for proposals seeking Leigh syndrome specific research that fell into one of two areas:
- Translational Research, defined as projects that contribute new tools for advancing basic research from the benchtop to the clinic, especially novel cellular and animal models or their application to deepen insights, biomarkers, diagnostics, or therapeutics for mitochondrial disease; and
- Clinical Research with a focus on patient-centered projects that explore means of achieving improved diagnoses, the understanding of the natural history of specific mitochondrial diseases or the development of therapeutic approaches for treating mitochondrial disease; retrospective analyses on existing data sets or well-designed prospective analyses that will help inform the understanding of Leigh syndrome natural history; and/or projects that utilize existing outcome measures to assess disease progression as well as development of new clinically relevant outcome measures that can serve as endpoints for future clinical trials.
An international scientific steering committee with deep expertise in Leigh syndrome conducted rigorous peer review on the research proposals submitted and chose five proposals to fund. Scoring/review criteria included, but wasn’t limited to, responsiveness to the RFP, relevance to Leigh syndrome, scientific novelty, scientific premise and rigor, and potential to impact the lives of those affected with Leigh syndrome. The awardees are (listed by amount awarded in USD):
“Gene therapy in mouse models of Leigh syndrome”
Massimo Zeviani, University of Padova, Italy
“Induced pluripotent stem cells (iPSC)-driven drug repositioning for Leigh syndrome”
Dao-Fu Dai/Alessandro Prigione*, University of Iowa/Heinrich Heine University (HHU)
*2019 LSIC Grant Awardees
“Combining gene replacement and focused ultrasound to treat Leigh syndrome”
Michael Decressac, Université Grenoble-Alpes, France.
“Safety, Efficacy and Mechanism of a Nicotinamidated Fumarate for Leigh Syndrome”
Gino Cortopassi*, University of California – Davis.
*2019 LSIC Grant Awardee
“Using a variety of pre-clinical disease models to identify novel drug candidates for the treatment of Leigh Syndrome”
Roan Louw, North-West University, South Africa.
For a full brief on each project, visit https://leighsyndrome.org/grant-cycle-2021/
ABOUT THE LEIGH SYNDROME INTERNATIONAL CONSORTIUM
The Leigh Syndrome International Consortium’s mission is to accelerate research aimed at uncovering better treatments and therapies for Leigh syndrome patients. Together, the Consortium has pledged more than 1 million USD to fund research with a focus on improving diagnosis, therapeutic development and optimized patient care for Leigh syndrome patients. For additional information on the Consortium, visit www.leighsyndrome.org.
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