The PARP inhibitor olaparib induces significant killing of ATM-deficient lymphoid tumor cells in vitro and in vivo

VJ Weston, CE Oldreive, A Skowronska… - Blood, The Journal …, 2010 - ashpublications.org
VJ Weston, CE Oldreive, A Skowronska, DG Oscier, G Pratt, MJS Dyer, G Smith, JE Powell…
Blood, The Journal of the American Society of Hematology, 2010ashpublications.org
Abstract The Ataxia Telangiectasia Mutated (ATM) gene is frequently inactivated in lymphoid
malignancies such as chronic lymphocytic leukemia (CLL), T-prolymphocytic leukemia (T-
PLL), and mantle cell lymphoma (MCL) and is associated with defective apoptosis in
response to alkylating agents and purine analogues. ATM mutant cells exhibit impaired DNA
double strand break repair. Poly (ADP-ribose) polymerase (PARP) inhibition that imposes
the requirement for DNA double strand break repair should selectively sensitize ATM …
Abstract
The Ataxia Telangiectasia Mutated (ATM) gene is frequently inactivated in lymphoid malignancies such as chronic lymphocytic leukemia (CLL), T-prolymphocytic leukemia (T-PLL), and mantle cell lymphoma (MCL) and is associated with defective apoptosis in response to alkylating agents and purine analogues. ATM mutant cells exhibit impaired DNA double strand break repair. Poly (ADP-ribose) polymerase (PARP) inhibition that imposes the requirement for DNA double strand break repair should selectively sensitize ATM-deficient tumor cells to killing. We investigated in vitro sensitivity to the poly (ADP-ribose) polymerase inhibitor olaparib (AZD2281) of 5 ATM mutant lymphoblastoid cell lines (LCL), an ATM mutant MCL cell line, an ATM knockdown PGA CLL cell line, and 9 ATM-deficient primary CLLs induced to cycle and observed differential killing compared with ATM wildtype counterparts. Pharmacologic inhibition of ATM and ATM knockdown confirmed the effect was ATM-dependent and mediated through mitotic catastrophe independently of apoptosis. A nonobese diabetic/severe combined immunodeficient (NOD/SCID) murine xenograft model of an ATM mutant MCL cell line demonstrated significantly reduced tumor load and an increased survival of animals after olaparib treatment in vivo. Addition of olaparib sensitized ATM null tumor cells to DNA-damaging agents. We suggest that olaparib would be an appropriate agent for treating refractory ATM mutant lymphoid tumors.
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