Arginine dependence of acute myeloid leukaemia blast proliferation: a novel therapeutic target

Acute myeloid leukemia (AML) is one of the most common acute leukemias in adults and children, yet significant numbers of patients relapse and die of disease. In this study, we identify the dependence of AML blasts on arginine for proliferation. We show that AML blasts constitutively express the arg...

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Main Authors: Mussai, Francis, Egan, Sharon A., Higginbotham-Jones, Joseph, Perry, Tracey, Beggs, Andrew, Odintsova, Elena, Loke, Justin, Pratt, Guy, Pong U, Kin, Lo, Anthony, Ng, Margaret, Kearns, Pamela R., Cheng, Paul, De Santo, Carmela
Format: Article
Published: American Society of Hematology 2015
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Online Access:https://eprints.nottingham.ac.uk/37630/
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author Mussai, Francis
Egan, Sharon A.
Higginbotham-Jones, Joseph
Perry, Tracey
Beggs, Andrew
Odintsova, Elena
Loke, Justin
Pratt, Guy
Pong U, Kin
Lo, Anthony
Ng, Margaret
Kearns, Pamela R.
Cheng, Paul
De Santo, Carmela
author_facet Mussai, Francis
Egan, Sharon A.
Higginbotham-Jones, Joseph
Perry, Tracey
Beggs, Andrew
Odintsova, Elena
Loke, Justin
Pratt, Guy
Pong U, Kin
Lo, Anthony
Ng, Margaret
Kearns, Pamela R.
Cheng, Paul
De Santo, Carmela
author_sort Mussai, Francis
building Nottingham Research Data Repository
collection Online Access
description Acute myeloid leukemia (AML) is one of the most common acute leukemias in adults and children, yet significant numbers of patients relapse and die of disease. In this study, we identify the dependence of AML blasts on arginine for proliferation. We show that AML blasts constitutively express the arginine transporters CAT-1 and CAT-2B, and that the majority of newly diagnosed patients' blasts have deficiencies in the arginine-recycling pathway enzymes argininosuccinate synthase and ornithine transcarbamylase, making them arginine auxotrophic. BCT-100, a pegylated human recombinant arginase, leads to a rapid depletion in extracellular and intracellular arginine concentrations, resulting in arrest of AML blast proliferation and a reduction in AML engraftment in vivo. BCT-100 as a single agent causes significant death of AML blasts from adults and children, and acts synergistically in combination with cytarabine. Using RNA sequencing, 20 further candidate genes which correlated with resistance have been identified. Thus, AML blasts are dependent on arginine for survival and proliferation, as well as depletion of arginine with BCT-100 of clinical value in the treatment of AML.
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spelling nottingham-376302020-05-04T17:06:59Z https://eprints.nottingham.ac.uk/37630/ Arginine dependence of acute myeloid leukaemia blast proliferation: a novel therapeutic target Mussai, Francis Egan, Sharon A. Higginbotham-Jones, Joseph Perry, Tracey Beggs, Andrew Odintsova, Elena Loke, Justin Pratt, Guy Pong U, Kin Lo, Anthony Ng, Margaret Kearns, Pamela R. Cheng, Paul De Santo, Carmela Acute myeloid leukemia (AML) is one of the most common acute leukemias in adults and children, yet significant numbers of patients relapse and die of disease. In this study, we identify the dependence of AML blasts on arginine for proliferation. We show that AML blasts constitutively express the arginine transporters CAT-1 and CAT-2B, and that the majority of newly diagnosed patients' blasts have deficiencies in the arginine-recycling pathway enzymes argininosuccinate synthase and ornithine transcarbamylase, making them arginine auxotrophic. BCT-100, a pegylated human recombinant arginase, leads to a rapid depletion in extracellular and intracellular arginine concentrations, resulting in arrest of AML blast proliferation and a reduction in AML engraftment in vivo. BCT-100 as a single agent causes significant death of AML blasts from adults and children, and acts synergistically in combination with cytarabine. Using RNA sequencing, 20 further candidate genes which correlated with resistance have been identified. Thus, AML blasts are dependent on arginine for survival and proliferation, as well as depletion of arginine with BCT-100 of clinical value in the treatment of AML. American Society of Hematology 2015-04-09 Article PeerReviewed Mussai, Francis, Egan, Sharon A., Higginbotham-Jones, Joseph, Perry, Tracey, Beggs, Andrew, Odintsova, Elena, Loke, Justin, Pratt, Guy, Pong U, Kin, Lo, Anthony, Ng, Margaret, Kearns, Pamela R., Cheng, Paul and De Santo, Carmela (2015) Arginine dependence of acute myeloid leukaemia blast proliferation: a novel therapeutic target. Blood, 125 (15). pp. 2386-2396. ISSN 1528-0020 Arginase depletion BCT-100 pegylated recombinant human arginase AML blasts http://www.bloodjournal.org/content/125/15/2386 doi:10.1182/blood-2014-09-600643 doi:10.1182/blood-2014-09-600643
spellingShingle Arginase depletion
BCT-100 pegylated recombinant human arginase
AML blasts
Mussai, Francis
Egan, Sharon A.
Higginbotham-Jones, Joseph
Perry, Tracey
Beggs, Andrew
Odintsova, Elena
Loke, Justin
Pratt, Guy
Pong U, Kin
Lo, Anthony
Ng, Margaret
Kearns, Pamela R.
Cheng, Paul
De Santo, Carmela
Arginine dependence of acute myeloid leukaemia blast proliferation: a novel therapeutic target
title Arginine dependence of acute myeloid leukaemia blast proliferation: a novel therapeutic target
title_full Arginine dependence of acute myeloid leukaemia blast proliferation: a novel therapeutic target
title_fullStr Arginine dependence of acute myeloid leukaemia blast proliferation: a novel therapeutic target
title_full_unstemmed Arginine dependence of acute myeloid leukaemia blast proliferation: a novel therapeutic target
title_short Arginine dependence of acute myeloid leukaemia blast proliferation: a novel therapeutic target
title_sort arginine dependence of acute myeloid leukaemia blast proliferation: a novel therapeutic target
topic Arginase depletion
BCT-100 pegylated recombinant human arginase
AML blasts
url https://eprints.nottingham.ac.uk/37630/
https://eprints.nottingham.ac.uk/37630/
https://eprints.nottingham.ac.uk/37630/