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LUCA Science Inc., in collaboration with Osaka University and Washington University School of Medicine in St. Louis, announces the publication of a transformative study in Nature Metabolism demonstrating the potential of mitochondria transfer-based therapies to alleviate symptoms and reduce the morbidity and mortality in a Leigh Syndrome (LS) mouse model. LS is a severe and often fatal inherited mitochondrial disease for which no effective therapies currently exist.

The study, titled “Mitochondria Transfer-Based Therapies Reduce the Morbidity and Mortality of Leigh Syndrome,” was conducted by an international team of researchers led by principal investigator Dr. Jonathan Brestoff. The research offers compelling evidence that transplantation of healthy wild-type (WT) mitochondria can ameliorate the symptoms and extend the lifespan of Ndufs4–/– mice, a well-established model for studying mitochondrial disease in Leigh Syndrome.

Leigh Syndrome is a devastating neurodegenerative mitochondrial disease caused by mutations in genes essential for mitochondrial metabolism, leading to severe metabolic dysfunction. It typically presents in infancy or early childhood with severe symptoms such as muscle weakness, developmental delays, breathing difficulties, and seizures, often leading to a shortened lifespan. This study breaks new ground by showing that wild-type bone marrow transplantation, or administration of isolated mitochondria, can restore mitochondrial function in multiple organs, improve neurological outcomes, enhance energy expenditure, and extend lifespan in LS-affected mice.

“Patients with LS and their families are in need of new therapies, and I hope that our studies open the possibility of using mitochondria transplantation to treat LS and other mitochondrial diseases,” said Dr. Jonathan Brestoff, senior author and Assistant Professor of Pathology and Immunology at WashU School of Medicine in St. Louis.

“We are excited to present these findings, which demonstrated a convincing preclinical proof-of-concept in the therapeutic potential of mitochondria transfer in treating inherited mitochondrial diseases,” said Dr. Rick C. Tsai, one of the co-authors and CEO at LUCA Science. “We have established a manufacturing process that enables the production of off-the-shelf MRC-Q, and we are ready to initiate GMP development activities to advance MRC-Q towards clinical development.

The study’s key findings include:

– Wildtype Bone Marrow Transplantation: WT bone marrow transplantation led to significant improvements in the morbidity and mortality of Ndufs4–/– mice, correlating with the release of extracellular mitochondria and transfer of healthy mitochondria to diseased cells across multiple organs.

– Autologous Mitochondria Transfer: Administration of isolated WT mitochondria extended lifespan, improved neurological function, and enhanced energy expenditure in LS-affected mice.

– Cross-Species Mitochondria Transfer: Remarkably, storable human Mitochondria oRganelle Complex Q (MRC-Q) by LUCA Science Inc. administered to Ndufs4–/– mice also ameliorated the symptoms of LS, suggesting the broader applicability of MRC-Q as biopharmaceutical therapies.

This publication marks a significant milestone in the field of mitochondrial medicine and underscores the potential of mitochondria transfer as a therapeutic strategy for treating complex mitochondrial disorders. LUCA Science remains committed to advancing this research with the ultimate goal of developing effective treatments for patients suffering from mitochondrial diseases.

The full paper is available in the latest issue of Nature Metabolism

TITLE: Mitochondria transfer-based therapies reduce the morbidity and mortality of Leigh syndrome

DOI: 10.1038/s42255-024-01125-5

LINK: https://www.nature.com/articles/s42255-024-01125-5

About LUCA Science Inc.

LUCA Science is a pioneering biotechnology company focused on developing innovative therapies based on mitochondrial biology. The company’s mission is to harness the power of mitochondria to treat a wide range of diseases, including neurodegenerative disorders, cardiovascular diseases, and metabolic conditions. With cutting-edge research and a commitment to improving patient outcomes, LUCA Science is leading the way in mitochondrial medicine.

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