19 February 2013
EPI-743 Vatiquinone is a new drug that is based on vitamin E. Tests have shown that it can help improve the function of cells with mitochondrial problems. It may be able to treat people with genetic disorders that affect metabolism and mitochondria
Edison Pharmaceuticals and Bambino Gesu Children’s Hospital have announced the commencement of EPI-743 Phase 2 cobalamin C deficiency syndrome trial.
EPI-743 is an orally bioavailable small molecule and a member of the para-benzoquinone class of drugs.
The trial’s principal investigator, Bambino Gesu Children’s Hospital, division of metabolism Professor Carlo Dionisi-Vici said, “Given the central role of glutathione in cellular redox balance and antioxidant defense systems, we are eager to explore whether a therapeutic that increases glutathione such as EPI-743 will provide clinical benefit.”
Improvement in visual function is the primary endpoint of the placebo-controlled study while secondary outcome measurements assess neurologic and neuromuscular function, glutathione biomarkers, quality of life, in addition to safety parameters.
The investigation is aimed at assessing the efficacy of EPI-743 in disorders of intermediary metabolism that also result in redox disturbances.
EPI-743 is an orally absorbed small molecule that readily crosses into the central nervous system. It works by targeting the enzyme NADPH quinone oxidoreductase 1 (NQO1). Its mode of action is to synchronize energy generation in mitochondria with the need to counter cellular redox stress
This drug has proven to keep my daughter alive, who is suffering with a terminal illness called Alpers disease. We lost our 5 year old after a 6 month battle with her illness before the EPI trial was available. Her younger sister was able to get on it after having her first seizure requiring a medicated coma. We have been seizure free and without disease progression symptoms for over 2 years!!!!!!!!! This is amazing given that the disease generally snowballs once symptoms start. The EPI gives hope for not only Leigh’s patients etc, but mitochondrial diseases in general! 🙂 We remain ever so hopeful that the EPI will be an OTC medication in the near future for mitochondrial diseases!
My son is denied treatment in South Africa by a doctor who havent even seen him. Who can help my Son with this medecine please?
I just wanted to update that 7 months after my prior post Morgan is still alive and well, she has had no visible progression nor seizures. The EPI is saving her. I thank God every day for Edison Pharmaceuticals and all those involved in the trial!
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