Development of an Orally Bioavailable LCK PROTAC Degrader as a Potential Therapeutic Approach to T-Cell Acute Lymphoblastic Leukemia

J Med Chem. 2024 Jul 25;67(14):11868-11884. doi: 10.1021/acs.jmedchem.4c00481. Epub 2024 Jul 8.

Abstract

Despite significant advances over recent years, the treatment of T cell acute lymphoblastic leukemia (T-ALL) remains challenging. We have recently shown that a subset of T-ALL cases exhibited constitutive activation of the lymphocyte-specific protein tyrosine kinase (LCK) and were consequently responsive to treatments with LCK inhibitors and degraders such as dasatinib and dasatinib-based PROTACs. Here we report the design, synthesis and in vitro/vivo evaluation of SJ45566, a potent and orally bioavailable LCK PROTAC.

MeSH terms

  • Administration, Oral
  • Animals
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use
  • Biological Availability
  • Cell Line, Tumor
  • Humans
  • Lymphocyte Specific Protein Tyrosine Kinase p56(lck)* / antagonists & inhibitors
  • Lymphocyte Specific Protein Tyrosine Kinase p56(lck)* / metabolism
  • Mice
  • Precursor T-Cell Lymphoblastic Leukemia-Lymphoma* / drug therapy
  • Precursor T-Cell Lymphoblastic Leukemia-Lymphoma* / metabolism
  • Protein Kinase Inhibitors / chemical synthesis
  • Protein Kinase Inhibitors / chemistry
  • Protein Kinase Inhibitors / pharmacokinetics
  • Protein Kinase Inhibitors / pharmacology
  • Protein Kinase Inhibitors / therapeutic use
  • Structure-Activity Relationship

Substances

  • Lymphocyte Specific Protein Tyrosine Kinase p56(lck)
  • Protein Kinase Inhibitors
  • Antineoplastic Agents
  • LCK protein, human