Knowledge-based approaches to drug discovery for rare diseases.

Journal: Drug discovery today
Published Date: Oct 27, 2021

Abstract

The conventional drug discovery pipeline has proven to be unsustainable for rare diseases. Herein, we discuss recent advances in biomedical knowledge mining applied to discovering therapeutics for rare diseases. We summarize current chemogenomics data of relevance to rare diseases and provide a perspective on the effectiveness of machine learning (ML) and biomedical knowledge graph mining in rare disease drug discovery. We illustrate the power of these methodologies using a chordoma case study. We expect that a broader application of knowledge graph mining and artificial intelligence (AI) approaches will expedite the discovery of viable drug candidates against both rare and common diseases.

Authors

  • Vinicius M Alves
    Laboratory for Molecular Modeling, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC, USA.
  • Daniel Korn
    Laboratory for Molecular Modeling, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA.
  • Vera Pervitsky
    Laboratory for Molecular Modeling, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA.
  • Andrew Thieme
    Laboratory for Molecular Modeling, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA.
  • Stephen J Capuzzi
    UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, North Carolina 27599.
  • Nancy Baker
    ParlezChem, 123 W Union Street, Hillsborough, NC 27278, USA.
  • Rada Chirkova
    Department of Computer Science, North Carolina State University, Raleigh, NC 27695-8206, USA.
  • Sean Ekins
    Collaborations in Chemistry, 5616 Hilltop Needmore Road, Fuquay-Varina, NC 27526, USA; Collaborative Drug Discovery, 1633 Bayshore Highway, Suite 342, Burlingame, CA 94010, USA; Collaborations Pharmaceuticals, Inc., 5616 Hilltop Needmore Road, Fuquay-Varina, NC 27526, USA; Phoenix Nest, Inc., P.O. Box 150057, Brooklyn, NY 11215, USA; Hereditary Neuropathy Foundation, 401 Park Avenue South, 10th Floor, New York, NY 10016, USA. Electronic address: ekinssean@yahoo.com.
  • Eugene N Muratov
    Laboratory for Molecular Modeling, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, North Carolina, United States of America.
  • Anthony Hickey
    UNC Catalyst for Rare Diseases, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599, USA. Electronic address: ahickey@unc.edu.
  • Alexander Tropsha
    Laboratory for Molecular Modeling, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC, USA.

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