The complexity and organization of the central nervous system (CNS) is widely modulated by the presence of the blood–brain barrier (BBB) and the blood–cerebrospinal fluid barrier (BCSFB), which both act as biochemical, dynamic obstacles impeding any type of undesirable exogenous exchanges. The disruption of these barriers is usually associated with the development of neuropathologies which can be the consequence of genetic disorders, local antigenic invasions, or autoimmune diseases. These disorders can take the shape of rare CNS-related diseases (other than Alzheimer’s and Parkinson’s) which exhibit relatively low or moderate prevalence and could be part of a potential line of treatments from current nanotargeted therapies. Indeed, one of the most promising therapeutical alternatives in that field comes from the development of nanotechnologies which can be divided between drug delivery systems and diagnostic tools. Unfortunately, the number of studies dedicated to treating these rare diseases using nanotherapeutics is limited, which is mostly due to a lack of interest from industrial pharmaceutical companies. In the present review, an overview of some of these rare CNS diseases is provided, the physiopathology of these disorders is discussed. It is also highlighted how nanotherapies could be of interest as a credible line of treatment, and finally the major issues which can hinder the development of efficient therapies in that area are also addressed.
The review mentions cyclodextrin in the treatment of Niemann–Pick Type C Disease acting via nanocapturing cholesterol.
Faouzzi A, Roullin FG: Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS Diseases. Pharmaceuticals 2021, 14(2), 109; https://doi.org/10.3390/ph14020109