Pharmaceutical Research

, Volume 30, Issue 10, pp 2459–2474 | Cite as

Nanoparticles in the Brain: A Potential Therapeutic System Targeted to an Early Defect Observed in Many Neurodegenerative Diseases

Expert Review

ABSTRACT

Currently, there are no effective treatments or cures for many neurodegenerative diseases affecting an aging baby-boomer generation. The ongoing problem with many of the current therapeutic treatments is that most are aimed at dissolving or dissociating aggregates and preventing cell death, common neuropathology often seen towards the end stage of disease. Often such treatments have secondary effects that are more devastating than the disease itself. Thus, effective therapeutics must be focused on directly targeting early events such that global deleterious effects of drugs are minimized while beneficial therapeutic effects are maximized. Recent work indicates that in many neurodegenerative diseases long distance axonal transport is perturbed, leading to axonal blockages. Axonal blockages are observed before pathological or behavioral phenotypes are seen indicating that this pathway is perturbed early in disease. Thus, developing novel therapeutic treatments to an early defect is critical in curing disease. Here I review neurodegenerative disease and current treatment strategies, and discuss a novel nanotechnology based approach that is aimed at targeting an early pathway, with the rationale that restoring an early problem will prevent deleterious downstream effects. To accomplish this, knowledge exchange between biologists, chemists, and engineers will be required to manufacture effective novel biomaterials for medical use.

KEY WORDS

anterograde transport axonal transport dynein kinesin neurodegenerative disease neurons/axons organically modified silica particles retrograde transport synthetic vesicle therapeutic vesicle 

ABBREVIATIONS

AD

Alzheimer’s disease

ALS

Amyotrophic lateral sclerosis

APP

Amyloid precursor protein

Amyloid beta

HD

Huntington’s disease

HTT

Huntingtin

KHC

Kinesin heavy chain

KLC

Kinesin light chain

MT

Microtubules

ORMOSIL

Organically modified silica

PD

Parkinson’s disease

Notes

ACKNOWLEDGMENTS AND DISCLOSURES

The author regrets that space limitations restricted some work from being cited. The development of ideas presented here was supported in part by the J.R. Oishei Foundation. The author thanks Kathryn Kowalski for editorial assistance, members of the Gunawardena laboratory for helpful discussions and Priyantha Karunaratne for constant support.

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Copyright information

© Springer Science+Business Media New York 2013

Authors and Affiliations

  1. 1.Department of Biological SciencesThe State University of New York at BuffaloBuffaloUSA

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