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Review

Neurodegeneration in Niemann–Pick Type C Disease: An Updated Review on Pharmacological and Non-Pharmacological Approaches to Counteract Brain and Cognitive Impairment

by
Ida Cariati
1,2,
Laura Masuelli
3,
Roberto Bei
2,
Virginia Tancredi
4,5,
Claudio Frank
6 and
Giovanna D’Arcangelo
4,5,*
1
Medical-Surgical Biotechnologies and Translational Medicine, “Tor Vergata” University of Rome, Via Montpellier 1, 00133 Rome, Italy
2
Department of Clinical Sciences and Translational Medicine, “Tor Vergata” University of Rome, Via Montpellier 1, 00133 Rome, Italy
3
Department of Experimental Medicine, University of Rome “Sapienza”, Viale Regina Elena 324, 00161 Rome, Italy
4
Department of Systems Medicine, “Tor Vergata” University of Rome, Via Montpellier 1, 00133 Rome, Italy
5
Centre of Space Bio-Medicine, “Tor Vergata” University of Rome, Via Montpellier 1, 00133 Rome, Italy
6
UniCamillus-Saint Camillus International University of Health Sciences, Via di Sant’Alessandro 8, 00131 Rome, Italy
*
Author to whom correspondence should be addressed.
Int. J. Mol. Sci. 2021, 22(12), 6600; https://doi.org/10.3390/ijms22126600
Submission received: 27 May 2021 / Revised: 15 June 2021 / Accepted: 17 June 2021 / Published: 20 June 2021
(This article belongs to the Special Issue Advances in Knowledge in Niemann-Pick Disease Type C: Facts and Perspectives- 2nd Edition)
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Abstract

Niemann–Pick type C (NPC) disease is an autosomal recessive storage disorder, characterized by abnormal sequestration of unesterified cholesterol in the late endo-lysosomal system of cells. Progressive neurological deterioration and the onset of symptoms, such as ataxia, seizures, cognitive decline, and severe dementia, are pathognomonic features of the disease. In addition, different pathological similarities, including degeneration of hippocampal and cortical neurons, hyperphosphorylated tau, and neurofibrillary tangle formation, have been identified between NPC disease and other neurodegenerative pathologies. However, the underlying pathophysiological mechanisms are not yet well understood, and even a real cure to counteract neurodegeneration has not been identified. Therefore, the combination of current pharmacological therapies, represented by miglustat and cyclodextrin, and non-pharmacological approaches, such as physical exercise and appropriate diet, could represent a strategy to improve the quality of life of NPC patients. Based on this evidence, in our review we focused on the neurodegenerative aspects of NPC disease, summarizing the current knowledge on the molecular and biochemical mechanisms responsible for cognitive impairment, and suggesting physical exercise and nutritional treatments as additional non-pharmacologic approaches to reduce the progression and neurodegenerative course of NPC disease.
Keywords: Niemann–Pick type C; neurodegeneration; cognitive decline; lipid trafficking; pharmacological treatment; physical exercise; nutritional approach Niemann–Pick type C; neurodegeneration; cognitive decline; lipid trafficking; pharmacological treatment; physical exercise; nutritional approach

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MDPI and ACS Style

Cariati, I.; Masuelli, L.; Bei, R.; Tancredi, V.; Frank, C.; D’Arcangelo, G. Neurodegeneration in Niemann–Pick Type C Disease: An Updated Review on Pharmacological and Non-Pharmacological Approaches to Counteract Brain and Cognitive Impairment. Int. J. Mol. Sci. 2021, 22, 6600. https://doi.org/10.3390/ijms22126600

AMA Style

Cariati I, Masuelli L, Bei R, Tancredi V, Frank C, D’Arcangelo G. Neurodegeneration in Niemann–Pick Type C Disease: An Updated Review on Pharmacological and Non-Pharmacological Approaches to Counteract Brain and Cognitive Impairment. International Journal of Molecular Sciences. 2021; 22(12):6600. https://doi.org/10.3390/ijms22126600

Chicago/Turabian Style

Cariati, Ida, Laura Masuelli, Roberto Bei, Virginia Tancredi, Claudio Frank, and Giovanna D’Arcangelo. 2021. "Neurodegeneration in Niemann–Pick Type C Disease: An Updated Review on Pharmacological and Non-Pharmacological Approaches to Counteract Brain and Cognitive Impairment" International Journal of Molecular Sciences 22, no. 12: 6600. https://doi.org/10.3390/ijms22126600

APA Style

Cariati, I., Masuelli, L., Bei, R., Tancredi, V., Frank, C., & D’Arcangelo, G. (2021). Neurodegeneration in Niemann–Pick Type C Disease: An Updated Review on Pharmacological and Non-Pharmacological Approaches to Counteract Brain and Cognitive Impairment. International Journal of Molecular Sciences, 22(12), 6600. https://doi.org/10.3390/ijms22126600

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