Design and biological and structural characterization of novel HDAC6-specific inhibitors

Jakub Ptacek1, Jiri Schimer2, Sida Chen3, Kseniya Ustinova1, Werner Tueckmantel4, Jiri Pavlicek, Jana Mikesova1, Lucia Motlova1, Petra Baranova1, Barbora Havlinova1, Alejandro Villagra5, Alan Kozikowski4 and Cyril Barinka1 *

1Institute of Biotechnology CAS, BIOCEV, Prumyslova 595, 252 50 Vestec, Czech Republic

2Institute of Organic Chemistry and Biochemistry of the Academy of Sciences of the Czech Republic, Flemingovo n. 2, 166 10 Prague 6, Czech Republic

3 Department of Medicinal Chemistry and Pharmacognosy, College of Pharmacy, University of Illinois at Chicago, Chicago, IL 60612, United States

4 StarWise Therapeutics LLC, University Research Park, Inc., Madison, WI 53719, United States

5 Department of Biochemistry and Molecular Medicine, The George Washington University, Washington, DC 20052, United States

* cyril.barinka@ibt.cas.cz

 

Histone deacetylases also known as lysine deacetylases (HDACs or KDACs) catalyze the hydrolysis of acyl groups from ε-N-acetyl lysine of protein substrates. Histone deacetylase 6 (HDAC6) is a unique member of the HDAC family due to its complex domain organization and cytosolic localization. HDAC6 exerts its enzymatic activity on many non-histone substrates such as tubulin, heat shock protein 90 (Hsp90), survivin, and cortactin. It has been shown that inhibition of HDAC6 enzymatic activity can be used potentially for the therapy of several neurological conditions including peripheral neuropathies, Alzheimer`s disease, and Parkinson’s disease. Unfortunately, however, currently no truly HDAC6-specific compounds exist to meet clinical needs. We have implemented in our laboratory an innovative screening cascade comprising in vitro isotype specificity testing and in vitro/in vivo toxicity and tubulin biomarker assays. Assisted by our X-ray crystallography data we designed and optimized novel HDAC6 inhibitors bearing an alternative non-hydroxamate zinc-binding group. Such compounds have nanomolar potency and exquisite 10,000-fold selectivity for HDAC6 over other HDAC isoforms and can serve as either research tools or leads for the development of compounds used in the clinical practice.