Recombinant proteins for neurodegenerative diseases: the delivery issue

Abstract

Tackling neurodegenerative diseases represents a formidable challenge for our ageing society. Recently, major achievements have been made in understanding the molecular mechanisms responsible for such diseases, and, simultaneously, numerous proteins such as neurotrophic factors, anti-apoptotic or anti-oxidant have been identified as potential therapeutic agents. Although many neurotrophic factors have been tested on individuals suffering from various neurodegenerative disorders, to date none has shown efficacy. Inadequate protein delivery is believed to be part of the problem. Recent improvements in pump technology, as well as in cell and gene therapy, are providing innovative ways to allow localized, regulatable delivery of proteins in brain parenchyma, opening new avenues for clinical trials in the not so distant future.