Blood in press

PRMT5 supports multiple oncogenic pathways in mantle cell lymphoma.

Sloan SL, Brown F, Long ME, Weigel C, Koirala S, Chung JH, Pray B, Villagomez L, Hinterschied C, Sircar A, Helmig-Mason J, Prouty A, Brooks E, Youssef Y, Hanel W, Parekh S, Chan WK, Chen Z, Lapalombella R, Sehgal L, Vaddi K, Scherle P, Chen-Kiang S, Di Liberto M, Elemento O, Meydan C, Foox J, Butler D, Mason CE, Baiocchi RA, Alinari L

Mantle cell lymphoma (MCL) is an incurable B-cell malignancy with overall poor prognosis particularly for patients that progress on targeted therapies. Novel more durable treatment options are needed for patients with MCL. Protein Arginine Methyltransferase 5 (PRMT5) is overexpressed in MCL and plays an important oncogenic role in this disease via epigenetic and post-translational modification of cell cycle regulators, DNA repair genes, components of pro-survival pathways, and RNA splicing regulators. The mechanism of targeting PRMT5 in MCL remains incompletely characterized. Here we report on the anti-tumor activity of PRMT5 inhibition in MCL using integrated transcriptomics of in vitro and in vivo models of MCL. Treatment with a selective small-molecule inhibitor of PRMT5, PRT-382, led to growth arrest and cell death and provided a therapeutic benefit in MCL patient derived xenografts. Transcriptional reprograming upon PRMT5 inhibition led to restored regulatory activity of the cell cycle (p-RB/E2F), apoptotic cell death (p53-dependent/p53-independent), and activation of negative regulators of BCR-PI3K/AKT signaling (PHLDA3, PTPROt, and PIK3IP1). We propose pharmacologic inhibition of PRMT5 for patients with relapsed/refractory MCL and identify MTAP/CDKN2A deletion and WT TP53 as biomarkers that predict a favorable response. Selective targeting of PRMT5 has significant activity in preclinical models of MCL and warrants further investigation in clinical trials.