Theranostics

Several classes of amyloid-directed compounds have been shown to inhibit a-syn aggregation and been investigated as potential new drugs, despite this, not a single molecule has reached the market [1, 2]. In recent years, the close cooperation between drug delivery/treatment and molecular imaging disciplines have moved towards relatively new classes of compounds known as theranostics. The term theranostic indicates the concomitant capability of a single molecule to act as therapeutic and diagnostic agent at the same time. Several examples of theranostic system have already been reported in literature for the treatment of cancer, atherosclerosis and gene delivery. In our recent work, we developed a new theranostic compound (denoted ad G8) able to simultaneously detect and inhibit Ab and PrPSc plaques in in vitro study.

Scrapie infected cells, treated with G8 compound showed lower levels of scrapie deposits and the compound was able to stain the scrapie plaques in the same infected cells, confirming its in vitro capability to inhibit and at the same time to detect the accumulated deposits. Using a similar approach, as in our reported work, among several libraries received from our collaborators from University of Bologna and Siena we are screening compounds in order to identify lead compounds able to detect and disaggregate amyloid deposits typical of neurodegenerative diseases (Aβ plaques, PrPSc, and α-syn). We seek to develop our methods into a novel pipeline for rapid drug discovery that can be applied to any target protein involved in neurodegenerative diseases.

 


1. Aulic, S., M.L. Bolognesi, and G. Legname, Small-molecule theranostic probes: a promising future in neurodegenerative diseases. Int J Cell Biol, 2013. 2013: p. 150952.

2. Latawiec, D., et al., Modulation of alpha-synuclein aggregation by dopamine analogs. PLoS One, 2010. 5(2): p. e9234.

 

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