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Mechanisms of Intranasal Deferoxamine in Neurodegenerative and Neurovascular Disease

1
School of Medicine, University of Virginia, 200 Jeanette Lancaster Way, Charlottesville, VA 22903, USA
2
HealthPartners Neuroscience Center, HealthPartners Institute, Saint Paul, MN 55130, USA
*
Author to whom correspondence should be addressed.
Academic Editor: Woo-Yang Kim
Pharmaceuticals 2021, 14(2), 95; https://0-doi-org.brum.beds.ac.uk/10.3390/ph14020095
Received: 6 January 2021 / Revised: 20 January 2021 / Accepted: 22 January 2021 / Published: 27 January 2021
(This article belongs to the Special Issue New Drugs and Biologics For Treatment of Central Nervous Dysfunction)
Identifying disease-modifying therapies for neurological diseases remains one of the greatest gaps in modern medicine. Herein, we present the rationale for intranasal (IN) delivery of deferoxamine (DFO), a high-affinity iron chelator, as a treatment for neurodegenerative and neurovascular disease with a focus on its novel mechanisms. Brain iron dyshomeostasis with iron accumulation is a known feature of brain aging and is implicated in the pathogenesis of a number of neurological diseases. A substantial body of preclinical evidence and early clinical data has demonstrated that IN DFO and other iron chelators have strong disease-modifying impacts in Alzheimer’s disease (AD), Parkinson’s disease (PD), ischemic stroke, and intracranial hemorrhage (ICH). Acting by the disease-nonspecific pathway of iron chelation, DFO targets each of these complex diseases via multifactorial mechanisms. Accumulating lines of evidence suggest further mechanisms by which IN DFO may also be beneficial in cognitive aging, multiple sclerosis, traumatic brain injury, other neurodegenerative diseases, and vascular dementia. Considering its known safety profile, targeted delivery method, robust preclinical efficacy, multiple mechanisms, and potential applicability across many neurological diseases, the case for further development of IN DFO is considerable. View Full-Text
Keywords: intranasal; deferoxamine; Alzheimer’s disease; Parkinson’s disease; ischemic stroke; intracranial hemorrhage; iron chelation; brain iron dyshomeostasis intranasal; deferoxamine; Alzheimer’s disease; Parkinson’s disease; ischemic stroke; intracranial hemorrhage; iron chelation; brain iron dyshomeostasis
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MDPI and ACS Style

Kosyakovsky, J.; Fine, J.M.; Frey, W.H.; Hanson, L.R. Mechanisms of Intranasal Deferoxamine in Neurodegenerative and Neurovascular Disease. Pharmaceuticals 2021, 14, 95. https://0-doi-org.brum.beds.ac.uk/10.3390/ph14020095

AMA Style

Kosyakovsky J, Fine JM, Frey WH, Hanson LR. Mechanisms of Intranasal Deferoxamine in Neurodegenerative and Neurovascular Disease. Pharmaceuticals. 2021; 14(2):95. https://0-doi-org.brum.beds.ac.uk/10.3390/ph14020095

Chicago/Turabian Style

Kosyakovsky, Jacob, Jared M. Fine, William H. Frey, and Leah R. Hanson 2021. "Mechanisms of Intranasal Deferoxamine in Neurodegenerative and Neurovascular Disease" Pharmaceuticals 14, no. 2: 95. https://0-doi-org.brum.beds.ac.uk/10.3390/ph14020095

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