Initial testing (stage 1) of LCL161, a SMAC mimetic, by the pediatric preclinical testing program

Peter J. Houghton, Min H. Kang, C. Patrick Reynolds, Christopher L. Morton, E. Anders Kolb, Richard Gorlick, Stephen T. Keir, Hernan Carol, Richard Lock, John M. Maris, Catherine A. Billups, Malcolm A. Smith

Research output: Contribution to journalArticlepeer-review

74 Scopus citations


LCL161, a SMAC mimetic, was tested against the PPTP in vitro panel (1.0nM to 10.0μM) and the PPTP in vivo panels (30 or 75mg/kg [solid tumors] or 100mg/kg [ALL]) administered orally twice in a week. LCL161 showed a median relative IC 50 value of >10μM, being more potent against several leukemia and lymphoma lines. In vivo LCL161 induced significant differences in EFS distribution in approximately one-third of solid tumor xenografts (osteosarcoma and glioblastoma), but not in ALL xenografts. No objective tumor responses were observed. In vivo LCL161 demonstrated limited single agent activity against the pediatric preclinical models studied.

Original languageEnglish (US)
Pages (from-to)636-639
Number of pages4
JournalPediatric Blood and Cancer
Issue number4
StatePublished - Apr 2012
Externally publishedYes


  • Developmental therapeutics
  • Preclinical testing
  • SMAC mimetic

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Pediatrics, Perinatology, and Child Health


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