Mitochondria targeting approach for delivery of therapeutics to the central nervous system

S. Dhar
University of Georgia,
United States

Keywords: drug delivery, gene delivery, brain disease


Numerous therapeutics are ineffective in treating cerebral diseases due to the inability of current approaches to effectively deliver and sustain the therapeutic doses within the brain. Inability of the available drugs to cross the blood brain barrier (BBB), targeting a single pathophysiological mechanism, short time window to deliver the therapy, and inadequate testing in rodents with brains that are dissimilar to humans, contributed to failed translation of drugs to the clinic for diseases of central nervous system. One potential option is the use of a nanoparticle (NP) with properties to cross the BBB and to reach specific intracellular targets to deliver drugs to the target and evaluation of such NP system in an animal model such as piglet with brains similar to human. In this presentation, we will discuss the potential of a biodegradable mitochondria targeted-NP with ability of transversing the BBB resulting in NP localization in the brain white matter, the site where inflammation and injury are diffused, in piglet and dog models. Therapeutic potential of the targeted NPs containing mitochondria-acting antioxidant, or an anti-inflammatory agent or a chemotherapeutic will be discussed for possible use in traumatic brain injury or glioblastoma. This nanoparticle can be tailored for plethora of central nervous system diseases such as neurodegenerative disorders like Parkinson disease or Huntington disease, diseases with localized cerebral dysfunction, such as stroke.