High-throughput CRISPR-mediated 3D enrichment platform for functional
interrogation of chemotherapeutic resistance
Abstract
Cancer is a disease of somatic mutations. These cellular mutations
compete to dominate their microenvironment and dictate the disease
outcome. While a therapeutic approach to target specific driver
mutations helps to manage the disease, subsequent molecular evolution of
tumor cells threatens to overtake therapeutic progress. There is need
for rapid, high-throughput, unbiased in-vitro discovery screening
platforms that capture the native complexities of the tumor and rapidly
identifiy mutations that confer chemotherapeutic drug resistance.Taking
the example of CDK4/6 inhibitor (CDK4/6i) class of drugs, we show that
the pooled in-vitro CRISPR screening platform enables rapid discovery of
drug resistance mutations in a 3D setting. Gene edited cancer cell
clones assembled into an organotypic multicellular tumor spheroid
(MCTS), exposed to CDK4/6i caused selection and enrichment of the most
drug resistant phenotype in a 3D setting, detectable by next gen
sequencing after a span of 28 days. The platform was sufficiently
sensitive to enrich for even a single drug resistant cell within a
large, 2500-cell, drug-responsive complex 3D tumor spheroid. The
genome-wide 3D CRISPR-mediated knockout screen (>18,000
genes) identified several genes whose disruptions conferred resistance
to CDK4/6i. Further, multiple novel candidate genes were identified as
top hits only in the microphysiological 3D enrichment assay platform and
not the conventional 2D assays. Taken together, these findings suggest
that including phenotypic 3D resistance profiling in decision trees
could improve discovery and reconfirmation of drug resistance mechanisms
and afford a platform for exploring non-cell autonomous interactions,
selection pressures, and clonal competition.