1. Academic Validation
  2. Making Protein Degradation Visible: Discovery of Theranostic PROTACs for Detecting and Degrading NAMPT

Making Protein Degradation Visible: Discovery of Theranostic PROTACs for Detecting and Degrading NAMPT

  • J Med Chem. 2022 Dec 8;65(23):15725-15737. doi: 10.1021/acs.jmedchem.2c01243.
Junfei Cheng 1 Shipeng He 2 Jun Xu 3 Min Huang 3 Guoqiang Dong 1 Chunquan Sheng 1
Affiliations

Affiliations

  • 1 School of Pharmacy, Second Military Medical University (Navy Medical University), Shanghai 200433, China.
  • 2 Institute of Translational Medicine, Shanghai University, Shanghai 200444, China.
  • 3 State Key Laboratory of Drug Research, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, University of Chinese Academy of Sciences, 555 Zuchongzhi Road, Shanghai 201203, China.
Abstract

Proteolysis-targeting chimera (PROTAC) is emerging as a promising technology in targeted protein degradation and drug discovery. However, there is still a lack of effective chemical tools to real-time detect and track the protein degradation. Herein, the first fluorescent and theranostic PROTACs were designed for imaging the degradation of nicotinamide phosphoribosyltransferase (NAMPT) in living cells. Compound B4 was proven to be an environmentally sensitive fluorescent PROTAC, which efficiently degraded NAMPT (DC50 = 8.4 nM) and enabled the visualization of degradation in A2780 cells. As a theranostic agent, PROTAC B4 led to significant reduction of nicotinamide adenine dinucleotide (NAD+) and exerted potent antitumor activities both in vitro and in vivo. Collectively, this proof-of-concept study provides a new strategy for the real-time visualization of the process of protein degradation and the improvement of diagnosis and therapeutic efficacy of PROTACs.

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