Novel Pharmaceutical and Pharmacological Strategies to Induce Autophagy

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Drug Targeting and Design".

Deadline for manuscript submissions: closed (31 December 2021) | Viewed by 2593

Special Issue Editors

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Department of Cellular, Computational and Integrative Biology (CIBIO), University of Trento, 38123 Trento, Italy
Interests: protein aggregation; neurobiology; Parkinson’s disease; autophagy
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Istituto di Scienze e Tecnologie Chimiche “Giulio Natta” (SCITEC), National Research Council (CNR), 20133 Milan, Italy
Interests: protein-protein interactions; medicinal chemistry; nanoparticles

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Chemistry Department, Università Statale di Milano, Via Golgi 19, I-20133 Milan, Italy
Interests: rational drug design; chemical genetics; medicinal chemistry
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

The accumulation of protein aggregates and dysfunctional mitochondria within the brain have long been recognized as critical components of the molecular etiology of several neurodegenerative disease. The catabolic pathways collectively known as autophagy represent a key determinant of whether protein aggregates persist, or whether dysfunctional mitochondria are disposed of correctly. Both genetic and molecular evidence have implicated autophagy in the pathogenesis of neurodegenerative disorders. Thus, the pharmacological induction of autophagy may result a potential therapeutic approach relevant to a number of neurodegenerative disorders. Autophagy inducers have been characterized: rapamycin and trehalose proved to be effective in vitro and in vivo. However, those compounds have a limited clinical applicability because of safety and pharmacokinetic issues. Meanwhile, new autophagy-connected molecular targets were identified and validated, and drug discovery efforts targeted against them are appearing at a fast pace. Clearly, autophagy induction may be a promising therapeutic opportunity, but clinically relevant drugs are still to be discovered.

This Special Issue invites international researchers in the area of new strategies to induce autophagy in therapeutic, authentic models. We welcome studies focused on the identification of natural and synthetic autophagy inducers from HTS campaigns, on the rational design and structure-activity relationships of small molecule autophagic inducers, biologicals, nanocarriers, alternative systems to deliver putative autophagic drugs, and preclinical evaluation of promising leads. 

Prof. Dr. Giovanni Piccoli
Dr. Daniela Arosio
Dr. Pierfausto Seneci
Guest Editors

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  • autophagy
  • nanoassemblies
  • prodrugs
  • in vitro models
  • in vivo models
  • pharmacokinetics
  • trehalose
  • rapamycin
  • pharmacological chaperones
  • delivery systems
  • rational drug design

Published Papers (1 paper)

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17 pages, 2829 KiB  
Squalene-Based Nano-Assemblies Improve the Pro-Autophagic Activity of Trehalose
by Giulia Frapporti, Eleonora Colombo, Hazem Ahmed, Giulia Assoni, Laura Polito, Pietro Randazzo, Daniela Arosio, Pierfausto Seneci and Giovanni Piccoli
Pharmaceutics 2022, 14(4), 862; - 14 Apr 2022
Cited by 7 | Viewed by 2040
The disaccharide trehalose is a well-established autophagy inducer, but its therapeutic application is severely hampered by its low potency and poor pharmacokinetic profile. Thus, we targeted the rational design and synthesis of trehalose-based small molecules and nano objects to overcome such issues. Among [...] Read more.
The disaccharide trehalose is a well-established autophagy inducer, but its therapeutic application is severely hampered by its low potency and poor pharmacokinetic profile. Thus, we targeted the rational design and synthesis of trehalose-based small molecules and nano objects to overcome such issues. Among several rationally designed trehalose-centered putative autophagy inducers, we coupled trehalose via suitable spacers with known self-assembly inducer squalene to yield two nanolipid-trehalose conjugates. Squalene is known for its propensity, once linked to a bioactive compound, to assemble in aqueous media in controlled conditions, internalizing its payload and forming nanoassemblies with better pharmacokinetics. We assembled squalene conjugates to produce the corresponding nanoassemblies, characterized by a hydrodynamic diameter of 188 and 184 nm and a high stability in aqueous media as demonstrated by the measured Z-potential. Moreover, the nanoassemblies were characterized for their toxicity and capability to induce autophagy in vitro. Full article
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