PKMYT1 is a Targetable Vulnerability in del(17p) High-Risk Multiple Myeloma

A Anaïs Schavgoulidze (Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse, France) J Jian Cui J Jessica Encinas (Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts., United States) V Vanessa Katia Favasuli (Candiolo Cancer Institute, FPO-IRCCS, Candiolo (TO), Italy) S Srikanth Talluri (DFCI, Boston, Massachusetts, United States) S Sabrina Mahéo (IUCT, TOULOUSE, France) C Chloe Cerutti (Institut Universitaire du Cancer de Toulouse-Oncopole, 31100, France) M Masood A Shammas (Dana Farber Cancer Institute, Boston, Massachusetts, United States) D Daniel Primo (Vivia Biotech, Madrid, Spain) C Carmen Vicente (University of Navarra, Pamplona, Spain) M Marta Larrayoz J Jose A Martínez-Climent (Center for Applied Medical Research (CIMA), University of Navarra, Pamplona, Spain) K Kenneth C. Anderson A Anil Aktas Samur (Dana Farber Cancer Institution, Boston, Massachusetts, United States) M Mehmet K. Samur (Dana-Farber Cancer Institute and Harvard School of Public Health, Boston, Massachusetts, United States) H Hervé Avet-Loiseau (Unité Génomique du Myélome, Hôpital Universitaire de Toulouse Oncopole, Université de Toulouse, Toulouse, France) J Jill Corre (Unité Génomique du Myélome, Hôpital Universitaire de Toulouse Oncopole, Université de Toulouse, Toulouse, France) N Nikhil C. Munshi M Mariateresa Fulciniti (Dana Farber Cancer Institute, Boston, Massachusetts, United States)

Abstract

Deletion of 17p is among the most adverse cytogenetic abnormalities in multiple myeloma (MM). By integrating RNA-seq data from patient MM cells with genetic dependency data from MM cell lines, we identified the protein kinase membrane-associated tyrosine/threonine 1 (PKMYT1) kinase, a member of the Wee family, as a potential therapeutic target in MM cells harboring del(17p). Genetic suppression or pharmacological inhibition of PKMYT1 activity with the selective inhibitor RP-6306 triggered accumulation of DNA damage, micronucleus formation and mitotic catastrophe, resulting in preferential cell death in del(17p) MM cells while largely sparing del(17p)-negative MM cells and healthy cells. RP-6306 also reduced tumor burden and extended survival in vivo in both xenograft and TP53-deficient syngeneic models. Collectively, our findings nominate PKMYT1 as an actionable target and support PKMYT1 inhibition as a biomarker-driven therapeutic strategy for patients with del(17p)/TP53-deficient MM.

Article Details

Journal Blood
Volume / Issue Vol. 1, Issue 1
Published June 03, 2026
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (19)

A

Anaïs Schavgoulidze

Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse, France

J

Jian Cui

J

Jessica Encinas

Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts., United States

V

Vanessa Katia Favasuli

Candiolo Cancer Institute, FPO-IRCCS, Candiolo (TO), Italy

S

Srikanth Talluri

DFCI, Boston, Massachusetts, United States

S

Sabrina Mahéo

IUCT, TOULOUSE, France

C

Chloe Cerutti

Institut Universitaire du Cancer de Toulouse-Oncopole, 31100, France

M

Masood A Shammas

Dana Farber Cancer Institute, Boston, Massachusetts, United States

D

Daniel Primo

Vivia Biotech, Madrid, Spain

C

Carmen Vicente

University of Navarra, Pamplona, Spain

M

Marta Larrayoz

J

Jose A Martínez-Climent

Center for Applied Medical Research (CIMA), University of Navarra, Pamplona, Spain

K

Kenneth C. Anderson

A

Anil Aktas Samur

Dana Farber Cancer Institution, Boston, Massachusetts, United States

M

Mehmet K. Samur

Dana-Farber Cancer Institute and Harvard School of Public Health, Boston, Massachusetts, United States

H

Hervé Avet-Loiseau

Unité Génomique du Myélome, Hôpital Universitaire de Toulouse Oncopole, Université de Toulouse, Toulouse, France

J

Jill Corre

Unité Génomique du Myélome, Hôpital Universitaire de Toulouse Oncopole, Université de Toulouse, Toulouse, France

N

Nikhil C. Munshi

M

Mariateresa Fulciniti

Dana Farber Cancer Institute, Boston, Massachusetts, United States