Personalized drug discovery: HCA approach optimized for rare diseases at Tel Aviv University

Comb Chem High Throughput Screen. 2014 Mar;17(3):253-5. doi: 10.2174/1386207317666140109123249.

Abstract

The Cell screening facility for personalized medicine (CSFPM) at Tel Aviv University in Israel is devoted to screening small molecules libraries for finding new drugs for rare diseases using human cell based models. The main strategy of the facility is based on smartly reducing the size of the compounds collection in similarity clusters and at the same time keeping high diversity of pharmacophores. This strategy allows parallel screening of several patient derived - cells in a personalized screening approach. The tested compounds are repositioned drugs derived from collections of phase III and FDA approved small molecules. In addition, the facility carries screenings using other chemical libraries and toxicological characterizations of nanomaterials.

MeSH terms

  • Drug Discovery* / organization & administration
  • Drug Evaluation, Preclinical / methods*
  • Drug Repositioning
  • Dysautonomia, Familial / drug therapy
  • Dysautonomia, Familial / pathology
  • Humans
  • Intestinal Pseudo-Obstruction / drug therapy
  • Intestinal Pseudo-Obstruction / pathology
  • Israel
  • Mitochondrial Encephalomyopathies / drug therapy
  • Mitochondrial Encephalomyopathies / pathology
  • Muscular Dystrophy, Oculopharyngeal
  • Ophthalmoplegia / congenital
  • Precision Medicine / methods
  • Rare Diseases / drug therapy*
  • Rare Diseases / pathology
  • Small Molecule Libraries / pharmacology*
  • Universities / organization & administration*

Substances

  • Small Molecule Libraries

Supplementary concepts

  • Visceral myopathy familial external ophthalmoplegia