Author Archives: Magdalena Kegel

Stealth BioTherapeutics, a biopharma that focuses on drugs for mitochondrial dysfunction, recently announced the start of a Phase 2 clinical trial exploring elamipretide (previously known as Ocuvia) in the form of eye drops for the treatment of Leber’s Hereditary Optic Neuropathy. Stealth’s lead candidate, elamipretide, works by preserving energy processes…

Individuals with Down syndrome and those with type 2 diabetes have one thing in common — dysfunctional insulin secretion in pancreatic beta cells. Now researchers have identified a gene, involved in mitochondrial function, controlling this disturbance in both conditions. In addition to explaining the high rates of diabetes in people with…

Scientists discovered that the compound Nicotinamide riboside boosts the function of mitochondria in stem cells, allowing older cells to regain their regenerative capacities. The findings have long-reaching implications for regenerative medicine, as well as for diseases such as muscular dystrophy, where researchers desperately seek new solutions to improve the lives of…

Mitochon Pharmaceuticals announced the completion of a second year of  financing, with investments totaling $1.6 million that will advance the company’s lead compound MP101 — a neuroprotective drug targeting mitochondria  — into clinical studies. The funds will also be used to further develop MP201 in preparation for the filing of an…

Mitochondrial replacement techniques may have taken a major step closer to clinical reality with a key study by the institute that invented the method — The New York Stem Cell Foundation (NYSCF) Research Institute. The findings demonstrated the compatibility of mitochondrial and nuclear DNA taken from two unrelated…

Russian researchers discovered why a natural antioxidant fails to protect a mitochondrial protein, cardiolipin, from the actions of reactive oxygen species, while a synthetic drug, Visomitin, does a good job. The discovery may pave the way for improved treatments of mitochondrial conditions linked to oxidative stress. Their study, “Impact of…

Mitochondrial DNA (mtDNA) mutations in individual cells may be more complex than previously thought. Their variability, seen in individuals with sporadic inclusion body myositis, might also be present in other mitochondrial conditions — a finding, by researchers at Newcastle University, United Kingdom, that offers new insights into role of mtDNA mutations, both in disease processes…

A mitochondrial mutation in the MT-TL1 gene, previously doubted by scientists to cause illness, was found to be linked to severe mitochondrial disease in a 15-year-old girl. The case report showed that the gene variant might indeed cause symptoms, and suggested that clinical manifestations caused by the mutation are dependent…

Early onset and extensive brain lesions are predictors of a poor prognosis in patients with Leigh syndrome, a mitochondrial disease affecting infants and small children. But findings also suggest that clinical outcomes of the syndrome, in terms of function, might be better than previously believed. Leigh syndrome is a neurodegenerative…

A study using mathematical analysis of mitochondrial morphology in malignant mesothelioma found that inhibiting mitochondria might be an effective treatment strategy against the deadly cancer — and suggested that the appearance of mitochondria might predict the response to such inhibition. The findings indicated that selectively blocking mitochondria might be a promising new…