[HTML][HTML] PD-0332991, a CDK4/6 inhibitor, significantly prolongs survival in a genetically engineered mouse model of brainstem glioma

KL Barton, K Misuraca, F Cordero, E Dobrikova… - PloS one, 2013 - journals.plos.org
KL Barton, K Misuraca, F Cordero, E Dobrikova, HD Min, M Gromeier, DG Kirsch, OJ Becher
PloS one, 2013journals.plos.org
Diffuse intrinsic pontine glioma (DIPG) is an incurable tumor that arises in the brainstem of
children. To date there is not a single approved drug to effectively treat these tumors and
thus novel therapies are desperately needed. Recent studies suggest that a significant
fraction of these tumors contain alterations in cell cycle regulatory genes including
amplification of the D-type cyclins and CDK4/6, and less commonly, loss of Ink4a-ARF
leading to aberrant cell proliferation. In this study, we evaluated the therapeutic approach of …
Diffuse intrinsic pontine glioma (DIPG) is an incurable tumor that arises in the brainstem of children. To date there is not a single approved drug to effectively treat these tumors and thus novel therapies are desperately needed. Recent studies suggest that a significant fraction of these tumors contain alterations in cell cycle regulatory genes including amplification of the D-type cyclins and CDK4/6, and less commonly, loss of Ink4a-ARF leading to aberrant cell proliferation. In this study, we evaluated the therapeutic approach of targeting the cyclin-CDK-Retinoblastoma (Rb) pathway in a genetically engineered PDGF-B-driven brainstem glioma (BSG) mouse model. We found that PD-0332991 (PD), a CDK4/6 inhibitor, induces cell-cycle arrest in our PDGF-B; Ink4a-ARF deficient model both in vitro and in vivo. By contrast, the PDGF-B; p53 deficient model was mostly resistant to treatment with PD. We noted that a 7-day treatment course with PD significantly prolonged survival by 12% in the PDGF-B; Ink4a-ARF deficient BSG model. Furthermore, a single dose of 10 Gy radiation therapy (RT) followed by 7 days of treatment with PD increased the survival by 19% in comparison to RT alone. These findings provide the rationale for evaluating PD in children with Ink4a-ARF deficient gliomas.
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