The Role of Oxygen in Disease and Metabolism: Developing Hypoxia as a Treatment for Mitochondrial Dysfunction
Event details
| Date | 28.06.2018 |
| Hour | 10:00 › 11:00 |
| Speaker | Prof. Isha H. Jain, UCSF Sandler Faculty Fellow, Dept. of Physiology, University of California, San Francisco (USA) |
| Location | |
| Category | Conferences - Seminars |
SEMINAR of the LAUSANNE INTEGRATIVE METABOLISM and NUTRITION ALLIANCE (LIMNA)
Abstract:
Mitochondrial disease affects 1 in 4800 live births, often leading to death within the first few years of life. Currently, there are no proven therapies that work broadly for this class of disorders. We perform a genome-wide screen and find that the cellular hypoxia response is protective against mitochondrial dysfunction. Moreover, chronic hypoxia extends the life of a mitochondrial mouse model of Complex 1 deficiency by five-fold. Starting hypoxia therapy at a late-stage of disease can even reverse the neurodegenerative phenotype and MRI-detectable lesions in this animal model. At the other extreme, mild hyperoxia greatly exacerbates disease and leads to death within several days.
These findings have now led to a phase 1 clinical trial in healthy volunteers, with the ultimate goal of human translation. We believe we have identified a new mode of treatment that will be broadly applicable to different forms of mitochondrial dysfunction, ranging from rare inborn errors of metabolism to more common, age-associated pathologies. We believe that “turning the oxygen dial” to low or high oxygen will serve as a novel therapeutic for a range of conditions in the coming years.
(*) IMPORTANT NOTICE: All external participants have to pass through SV Reception/Welcome Desk to be able to access to AI 1153. Contact person to call at arrival at SV Reception Desk: Administrative Assistant (ext. 3 95 22).
Abstract:
Mitochondrial disease affects 1 in 4800 live births, often leading to death within the first few years of life. Currently, there are no proven therapies that work broadly for this class of disorders. We perform a genome-wide screen and find that the cellular hypoxia response is protective against mitochondrial dysfunction. Moreover, chronic hypoxia extends the life of a mitochondrial mouse model of Complex 1 deficiency by five-fold. Starting hypoxia therapy at a late-stage of disease can even reverse the neurodegenerative phenotype and MRI-detectable lesions in this animal model. At the other extreme, mild hyperoxia greatly exacerbates disease and leads to death within several days.
These findings have now led to a phase 1 clinical trial in healthy volunteers, with the ultimate goal of human translation. We believe we have identified a new mode of treatment that will be broadly applicable to different forms of mitochondrial dysfunction, ranging from rare inborn errors of metabolism to more common, age-associated pathologies. We believe that “turning the oxygen dial” to low or high oxygen will serve as a novel therapeutic for a range of conditions in the coming years.
(*) IMPORTANT NOTICE: All external participants have to pass through SV Reception/Welcome Desk to be able to access to AI 1153. Contact person to call at arrival at SV Reception Desk: Administrative Assistant (ext. 3 95 22).
Practical information
- Informed public
- Free
Organizer
- Johan Auwerx on behalf of the Lausanne Integrative Metabolism and Nutrition Alliance - LIMNA