Type 2 calreticulin mutations activate ATF6 to promote BCL-xL–mediated survival in myeloproliferative neoplasms

N Nicole S. Arellano (The University of Utah) W William L. Heaton (1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT) M Mirielle C. Nauman (3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL) A Abigail E. Runnels (1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT) J Jacky Gomez-Villa (3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL) D Daniele Vanni (3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL) M Melissa Gaviria (3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL) M Maihi Fujita (1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT) N Nathan M. Krah M Michele Ciboddo (3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL) S Saveg Yadav (1Huntsman Cancer Institute, University of Utah, Salt Lake City, United States) C Callie T. Brown (1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT) P Parker D. Bowden (1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT) A Amy K. Chen (3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL) C Christopher Henning (3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL) S Silvia Catricalà (6Department of Hematology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy) I Ilaria Carola Casetti (4Department of Molecular Medicine, University of Pavia, Pavia, Italy) O Oscar Borsani (4Department of Molecular Medicine, University of Pavia, Pavia, Italy) E Elisa Rumi (19Department of Molecular Medicine, University of Pavia, Pavia, Italy) D Daniela Pietra (6Department of Hematology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy) I Isabelle Plo (INSERM Unité Mixte de Recherche 1287, Villejuif, France) C Caroline Marty M Marco Marchetti (Department of Human Genetics, Immunology, Inflammation and Infections Disease Initiative-Utah Center for Genetic Discovery Bioinformatics Core, University of Utah) A Ami B. Patel (1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT) C Caner Saygin (9Department of Medicine, University of Chicago, Chicago, IL) S Shannon E. Elf (The University of Utah)

Abstract

Abstract Most calreticulin (CALR) mutations in myeloproliferative neoplasms are classified as either type 1, a 52–base pair deletion (CALRdel52); or type 2, a 5–base pair insertion (CALRins5). Both are gain-of-function (GOF) mutations that generate an identical mutant C-terminal tail, which mediates the binding to, and activation of, the thrombopoietin receptor myeloproliferative leukemia protein (MPL). We recently reported that despite this shared GOF, CALRdel52 but not CALRins5 mutations cause loss of calcium binding function, leading to activation of, and dependency on, the inositol-requiring enzyme 1/X-box binding protein 1 pathway of the unfolded protein response (UPR). This led us to ask whether CALRins5 mutations activate and depend on a different UPR pathway, and whether this is likewise mediated by a mutation type–specific loss-of-function (LOF). Here, we show that CALRins5 mutations lead to activation of the activating transcription factor 6 (ATF6) pathway of the UPR due to loss of CALR chaperone function. This LOF is caused by interference of the CALRins5 mutant C terminus with key chaperone residue H170. Furthermore, we show that CALRins5 cells are partially dependent on ATF6 for cytokine-independent growth, and identify B-cell lymphoma extra large as a transcriptional target of ATF6 that promotes type 2 CALR-mutant cell survival.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 8
Published August 21, 2025
Pages 971-983
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (26)

N

Nicole S. Arellano

The University of Utah

W

William L. Heaton

1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT

M

Mirielle C. Nauman

3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL

A

Abigail E. Runnels

1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT

J

Jacky Gomez-Villa

3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL

D

Daniele Vanni

3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL

M

Melissa Gaviria

3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL

M

Maihi Fujita

1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT

N

Nathan M. Krah

M

Michele Ciboddo

3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL

S

Saveg Yadav

1Huntsman Cancer Institute, University of Utah, Salt Lake City, United States

C

Callie T. Brown

1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT

P

Parker D. Bowden

1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT

A

Amy K. Chen

3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL

C

Christopher Henning

3Ben May Department for Cancer Research, The University of Chicago, Chicago, IL

S

Silvia Catricalà

6Department of Hematology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy

I

Ilaria Carola Casetti

4Department of Molecular Medicine, University of Pavia, Pavia, Italy

O

Oscar Borsani

4Department of Molecular Medicine, University of Pavia, Pavia, Italy

E

Elisa Rumi

19Department of Molecular Medicine, University of Pavia, Pavia, Italy

D

Daniela Pietra

6Department of Hematology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy

I

Isabelle Plo

INSERM Unité Mixte de Recherche 1287, Villejuif, France

C

Caroline Marty

M

Marco Marchetti

Department of Human Genetics, Immunology, Inflammation and Infections Disease Initiative-Utah Center for Genetic Discovery Bioinformatics Core, University of Utah

A

Ami B. Patel

1Division of Hematology and Hematological Malignancies, The University of Utah, Salt Lake City, UT

C

Caner Saygin

9Department of Medicine, University of Chicago, Chicago, IL

S

Shannon E. Elf

The University of Utah