Ex vivo functional drug screens were performed on freshly isolated mononuclear cells from AML samples. Briefly, 10,000 cells per well were arrayed into three, 384-well plates containing 122 small-molecule inhibitors. This panel contained graded concentrations of a drugs with activity against two-thirds of the tyrosine kinome as well as other non-tyrosine kinase pathways, including mitogen activated protein kinases (MAPKs), phosphatidylinositol-4,5-bisphosphate 3-kinase/AKT serine/threonine kinase 1/mechanistic target of rapamycin kinase (PIK3C/AKT/MTOR), protein kinase AMP-activated (AMPK/PRKAA), ATM serine/threonine kinase (ATM), Aurora kinases, calcium/calmodulin dependent protein kinases (CAMKs), cyclin-dependent kinases (CDKs), serine/threonine protein kinase 3 (GSK3), I-kappaB kinase (IKK), cAMP dependent protein kinase (PKA), protein kinase C (PKC), polo-like kinase 1 (PLK1), and RAF proto-oncogene serine/threonine kinase (RAF). In addition, the library contained small molecule inhibitors with activity against the BCL2 family, bromodomain containing 4 (BRD4), Hedgehog, heat shock protein 90 (HSP90), NOTCH/gamma-secretase, proteasome, survivin, signal transducer and activator of transcription 3 (STAT3), histone deacetylase (HDAC), and WNT/beta-catenin. Drug plates were created using inhibitors purchased from LC Laboratories and Selleck Chemicals and master stocks were reconstituted in dimethyl sulfoxide (DMSO) and stored at −80 °C. Master plates were created by distributing a single agent per well in a seven-point concentration series, created from three-fold dilutions of the most concentrated stock resulting in a range pf 10 μM to 0.0137 μM for each drug (except dasatinib, ponatinib, sunitinib, and YM-155 which were plated at a concentration range of 1 μM to 0.00137 μM). DMSO control wells and positive control wells containing a drug combination of Flavopiridol, Staurosporine and Velcade were placed on each plate, with the final concentration of DMSO ≤0.1% in all wells. Daughter plates were created using a V&P Scientific 384-well pin tool head operated by the Caliper Sciclone ALH 3000 and equipped with 0.457mm diameter, 30 nanoliter, slotted stainless steel pins (cat num: FP1NS30). Daughter and destination plates were sealed with pealable thermal seals using a PlateLoc thermal sealer. Destination plates were stored at −20 °C for no more than three months and thawed immediately before use. Primary mononuclear cells were plated across single-agent inhibitor panels within 24 h of collection. Cells were seeded into 384-well assay plates at 10,000 cells per well in Roswell Park Memorial Institute (RPMI) 1640 media supplemented with fetal bovine serum (FBS) (10%), l-glutamine, penicillin/streptomycin, and β-mercaptoethanol (10−4 M). After 3 d of culture at 37 °C in 5% CO2, MTS reagent (CellTiter96 AQueous One; Promega) was added, optical density was measured at 490 nm, and raw absorbance values were adjusted to a reference blank value and then used to determine cell viability (normalized to untreated control wells).
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Tyner J.W., Tognon C.E., Bottomly D., Wilmot B., Kurtz S.E., Savage S.L., Long N., Schultz A.R., Traer E., Abel M., Agarwal A., Blucher A., Borate U., Bryant J., Burke R., Carlos A., Carpenter R., Carroll J., Chang B., Coblentz C., d’Almeida A., Cook R., Danilov A., Dao K.H., Degnin M., Devine D., Dibb J., Edwards DK V., Eide C.A., English I., Glover J., Henson R., Ho H., Jemal A., Johnson K., Johnson R., Junio B., Kaempf A., Leonard J., Lin C., Liu S.(., Lo P., Loriaux M.M., Luty S., Macey T., MacManiman J., Martinez J., Mori M., Nelson D., Nichols C., Peters J., Ramsdill J., Rofelty A., Schuff R., Searles R., Segerdell E., Smith R.L., Spurgeon S.E., Sweeney T., Thapa A., Visser C., Wagner J., Watanabe-Smith K., Werth K., Wolf J., White L., Yates A., Zhang H., Cogle C.R., Collins R.H., Connolly D.C., Deininger M.W., Drusbosky L., Hourigan C.S., Jordan C.T., Kropf P., Lin T.L., Martinez M.E., Medeiros B.C., Pallapati R.R., Pollyea D.A., Swords R.T., Watts J.M., Weir S.J., Wiest D.L., Winters R.M., McWeeney S.K, & Druker B.J. (2018). Functional Genomic Landscape of Acute Myeloid Leukemia. Nature, 562(7728), 526-531.
Publication 2018
Corresponding Organization :
Other organizations :
Oregon Health & Science University, Oregon State University, University of Florida, The University of Texas Southwestern Medical Center, Fox Chase Cancer Center, University of Utah, National Heart Lung and Blood Institute, National Institutes of Health, University of Colorado Denver, University of Kansas, Sylvester Comprehensive Cancer Center, University of Miami, Stanford University, University of Kansas Medical Center
Positive control: Drug combination of Flavopiridol, Staurosporine, and Velcade
Negative control: DMSO-only wells
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