Neurodegenerative diseases, gold nanoparticles slow down progress of Friedreich's ataxia
The Friedreich’s ataxia study, co-ordinated by the State University of Milan and the Policlinico di Milano, together with the University of Milan-Bicocca, the University of Turin and the University of Miami Miller School of Medicine, found that gold nanoparticles reduce oxidative damage and improve mitochondrial function in patients with Friedreich’s ataxia. The research was published in Science Translational Medicine
Nanoparticles composed of clusters of gold atoms improve mitochondrial function and reduce oxidative damage in Friedreich’s ataxia patients
Friedreich’s ataxia is a neurodegenerative disease caused by an abnormality in the gene that codes for a protein and mainly affects the central and peripheral nervous systems: the study was recently published in Science Translational Medicine.
The research was initiated in 2019 by Chiara Villa, a researcher at the State University of Milan, afferent to the Stem Cell Laboratory of the “Centro Dino Ferrari” Fondazione IRCCS Ca’ Granda – Ospedale Maggiore Policlinico, directed by Yvan Torrente, Professor of Neurology at the State University, with the aim of investigating the role of nanoparticles of gold atoms in the treatment of Friedreich’s Ataxia (FRDA).
This study is the result of a collaboration between the research groups of the University of Milan-Bicocca (directed by Angelo Monguzzi), the University of Turin (directed by Giorgio Merlo) and the University of Miami directed by Carlos Moraes.
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FRDA (Friedreich’s Ataxia) is a neurodegenerative disease caused by an abnormality in the gene coding for a protein called fratassin (FXN)
The disease primarily affects the central and peripheral nervous systems and usually occurs at the age of puberty, manifesting as ataxia of the limbs and walking, an axonal sensory neuropathy, dysarthria and weakness.
Non-neurological symptoms include progressive cardiomyopathy with associated arrhythmias and diabetes.
There is no definitive therapy for FRDA; the symptom picture is now treated in a multidisciplinary manner.
“Although fratassin is a well-characterised protein,’ Yvan Torrente explains, ‘its function has not yet been fully elucidated.
We know from the literature that fratassin is important for proper mitochondrial function and plays a key role in intracellular iron homeostasis.
Lack of FXN results in increased exposure to oxidative stress with consequent accumulation of metals and reduced activity of the mitochondrial respiratory chain.
This process increases the generation of free radicals causing neuronal cell death and neurodegeneration through different mechanisms.
Neural tissues and the heart are susceptible to oxidative stress, and an accumulation of iron has been found in most tissues of animal models and patients with FRDA”.
“In this work,” explains Chiara Villa, “it has been demonstrated that the administration of nanoparticles composed of clusters of gold atoms leads to a reduction in oxidative damage and improvement in mitochondrial function both in cells from subjects with FRDA and in mouse models of FRDA (YG8sR).
The results allowed us to identify a significant improvement in neuromotor and cardiac function in YG8sR models several months after a single administration of nanoparticles.”
“This study represents a major breakthrough that paves the way for new studies on FRDA and the development of new drugs capable of having a long-lasting beneficial effect for the treatment of neurodegenerative diseases”, concludes Yvan Torrente.
The study was supported by, among others, the Association “OGNI GIORNO – per Emma – Onlus”, from Treviso, Italy, and the Association “Per il Sorriso di Ilaria da Montebruno – Onlus”, from Genoa, Italy.
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