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Protonophores against Prader-Willi-Syndrome (PWS)

Prader-Willi-Syndrome (PWS) is a devastating disease leading to developmental delay, psycho-pathological disorders and morbid obesity. Recent studies hint that so-called protonophores could both alleviate obesity as well as improve neurological conditions. Building on these seminal insights, novel and safe protonophores are currently in development.

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The research aims to develop protonophoric molecules to tackle metabolic syndrome (obesity, diabetes, NAFLD) and other related chronic diseases as well as certain neurodegenerative conditions. Protonophores act as mitochondrial proton uncouplers. These compounds hijack mitochondria thereby increasing basal metabolic rate, which eventually leads to elevated calorie expenditure. Furthermore, proton uncoupling is known to reduce reactive oxygen species (ROS) and intra-mitochondrial calcium overload, thus making it a promising mechanism of action for treating many neurodegenerative diseases among others.

With a myriad of urgent therapeutic needs at choice, a main aspect of the project is devoted to develop a treatment for the Prader-Willi-Syndrome (PWS) – a complex genetic disorder caused by chromosomal anomalies. From early childhood on PWS patients suffer from developmental delay, hyperphagia, psychopathological disorders and morbid obesity. While there currently is no cure for PWS available, a diverse repertoire of pharmacological treatments and psychotherapy alleviate a number of symptoms. However, drugs targeting hyperphagia and PWS-related morbid obesity are mostly acting through the central nervous system by reducing kcal intake. These types of substances, which are typically stimulants, are not suitable for children due to their severe adverse effects on a developing brain.

A recent study (doi:10.1002/ajmg.a.40526) revealed that the reduced energy expenditure in PWS patients may be caused by a lack of endogenous proton leak, i.e. proton uncoupling. Repairing the cellular metabolism and balancing the disturbances in mitochondrial bioenergetics by increasing proton uncoupling could bring energy expenditure of PWS patients back to an adequate level.

Taking into account the latent neuroprotective properties of protonophoric entities, proton uncoupling may also benefit the cognitive development of PWS patients during their early life. This apparent synergism may represent an ideal treatment for PWS patients.


BSc Marlon Millard
University of Vienna
E: millardmarlon@yahoo.de

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