Leukemia Fact Sheet

CIRM funds many projects seeking to better understand leukemia and to translate those discoveries into new therapies.


Leukemia is a cancer of the blood cells. Although leukemia is among the most common childhood cancers, it most often occurs in older adults and is slightly more common in men than women. In 2016 there were an estimated 60,000 new cases with around 24,000 people dying of the disease.

The stem cells in the bone marrow normally form all cells of the blood system, including the red blood cells, platelets, and immune cells. In people with leukemia, the bone marrow stem cells create abnormal immune cells that aren’t able to carry out their normal job of fighting infection.

Eventually, these abnormal cells crowd out the normal blood cells. Without a sufficient population of working blood cells, people with leukemia develop symptoms such as anemia, bleeding and infections.

Recent research has shown that in addition to these abnormal white cells, leukemia patients also have a small population of cells called leukemia stem cells. Scientists suspect that these cells evade treatments that kill leukemia cells and then later go on to cause a relapse. The goal of stem cell research for leukemia is to find ways of destroying these leukemia stem cells.

Catriona Jamieson of the UC San Diego Moores Cancer Center discusses a clinical trial for a pre-leukemia condition that was based in part on CIRM funding

Clinical Stage Programs

University of California, San Diego

A team at UC San Diego is testing the safety of a monoclonal antibody called cirmtuzumab that targets cancer stem cells in a Phase 1 clinical trial. It’s named after CIRM because we helped fund the research that led to its development. Cirmtuzumab is designed to attach to a protein, called ROR1, that is found on the surface of chronic lymphocytic leukemia (CLL) cells but is rarely found on healthy cells. The team hopes cirmtuzumab will target the cancer cells, blocking their ability to grow and/or survive.

The same UCSD team is also testing cirmtuzumab in combination with an approved cancer fighting drug call Ibrutinib, to target cancer stem cells in a separate Phase 1 trial. The hope is that combining Cirmtuzumab with Ibrutinib will improve cancer remission and long-term cancer control in patients.

CIRM Grants Targeting Leukemia

Researcher NameInstitutionGrant TitleGrant TypeAward Amount
Dr. Camilla ForsbergUniversity of California, Santa CruzDetermining how age-specific heterogeneity of human hematopoietic stem cells and megakaryocyte progenitors contribute to thrombotic disease upon agingFoundation – Discovery Stage Research Projects$1,336,800
Dr Dinesh S RaoUniversity of California, Los AngelesNovel methods to eliminate cancer stem cellsQuest – Discovery Stage Research Projects$1,384,347
Dr Julia CarnevaleUniversity of California, San FranciscoGenerating deeper and more durable BCMA CAR T cell responses in Multiple Myeloma through non-viral knockin/knockout multiplexed genome engineeringQuest – Discovery Stage Research Projects$1,463,368
Dr. Tannishtha ReyaUniversity of California, San DiegoTargeting Cancer Stem Cells in Hematologic MalignanciesQuest – Discovery Stage Research Projects$1,960,560
Markus MüschenCity of Hope, Beckman Research InstituteLgr5-mediated self-renewal in B cell selection and leukemia-initiationQuest – Discovery Stage Research Projects$2,186,520
Dr. John P ChuteUniversity of California, Los AngelesProtein tyrosine phosphatase – sigma inhibitors for hematopoietic regenerationQuest – Discovery Stage Research Projects$2,115,735
Dan S KaufmanUniversity of California, San DiegoTargeted off-the-shelf immunotherapy to treat refractory cancersQuest – Discovery Stage Research Projects$1,936,936
Dr. David J BaylinkLoma Linda UniversityBone Marrow Targeting of Hematopoietic Stem Cells Engineered to Overexpress 25-OH-VD3 1-α-hydroxylase for Acute Myeloid Leukemia TherapyInception – Discovery Stage Research Projects$178,967
Dr. Jacob Ellery Corn Ph.DUniversity of California, BerkeleyGenome editing for causation and reversion of MPN-associated mutations in human hematopoietic stem cellsInception – Discovery Stage Research Projects$235,800
Dr Michael A. CaligiuriCity of Hope, Beckman Research InstituteTargeting multiple myeloma with BCMA-CAR NK cells expressing a GPRC5D-NKG2D bispecific antibodyTherapeutic Translational Research Projects$6,036,001
Dr. William J MurphyUniversity of California, DavisOvercoming resistance to standard CD19-targeted CAR T using a novel triple antigen targeted vectorTherapeutic Translational Research Projects$3,869,061
Arun P WiitaUniversity of California, San FranciscoCD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemiaTherapeutic Translational Research Projects$3,330,801
Dr. Lili YangUniversity of California, Los AngelesHSC-Engineered Off-The-Shelf CAR-iNKT Cell Therapy for Multiple MyelomaTherapeutic Translational Research Projects$5,281,199
Yvonne Y. ChenUniversity of California, Los AngelesBCMA/CS1 Bispecific CAR-T Cell Therapy to Prevent Antigen Escape in Multiple MyelomaTherapeutic Translational Research Projects$3,176,805
Dr. Karin Lindgren GaenslerUniversity of California, San FranciscoDeveloping engineered autologous leukemia vaccines to target residual leukemic stem cellsTherapeutic Translational Research Projects$4,171,728
Dan S KaufmanUniversity of California, San DiegoHuman Embryonic Stem Cell-Derived Natural Killer Cells for Cancer TreatmentTherapeutic Translational Research Projects$3,260,000
Catriona JamiesonUniversity of California, San DiegoA Splicing Modulator Targeting Cancer Stem Cells in Acute Myeloid LeukemiaTherapeutic Translational Research Projects$2,511,767
Ezra CohenUniversity of California, San DiegoDevelopment of ROR1 CAR-T cells to target cancer stem cells in advanced malignanciesTherapeutic Translational Research Projects$5,795,584
Dr. Lili YangUniversity of California, Los AngelesStem Cell-Based iNKT Cell Therapy for CancerTherapeutic Translational Research Projects$6,956,775
Elizabeth BuddeCity of Hope, Beckman Research InstitutePhase I Study of Chimeric Antigen Receptor Engineered T Cells targeting CD33 for the Treatment of Relapsed/Refractory Acute Myeloid LeukemiaClinical Trial Stage Projects$11,983,547
Dr. Paul FinneganAngiocrine Bioscience, Inc.AB-205-301 Phase 3 Trial and Related Activities to Support Clinical Development of AB-205Clinical Trial Stage Projects$15,000,000
Dr. Matthew H PorteusStanford UniversityPhase 1/1b study of T-allo10 infusion after HLA-partially matched abdepleted-HSCT in children and young adults with hematologic malignancies.Clinical Trial Stage Projects$10,563,822
Joseph Paul WoodardImmune-Onc TherapeuticsPhase 1 Clinical Development of IO-202, A First-in-Class Antibody Targeting LILRB4, for the Treatment of AML with Monocytic Differentiation and CMMLClinical Trial Stage Projects$6,000,000
Dr. Edward KavalerchikAngiocrine Bioscience, Inc.AB-205-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-205Clinical Trial Stage Projects$6,192,579
Dr. David B. MiklosStanford UniversityPhase 1 Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Adults with Recurrent or Refractory B Cell MalignanciesClinical Trial Stage Projects$11,034,982
Michael A. PulsipherChildren’s Hospital of Los AngelesAntiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES)Clinical Trial Stage Projects$4,825,587
Rajesh Belani MDPoseida Therapeutics, Inc.Clinical Study of T stem cell memory (Tscm)-based CAR-T cells in Patients with Multiple MyelomaClinical Trial Stage Projects$19,813,407
Dr. Edward KavalerchikAngiocrine Bioscience, Inc.AB-110-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-110Clinical Trial Stage Projects$5,000,000
Thomas J KippsUniversity of California, San DiegoA Phase 1b/2a Study of the ROR1-Targeting Monoclonal Antibody, Cirmtuzumab, and the Bruton Tyrosine Kinase Inhibitor, Ibrutinib, in B-Cell CancersClinical Trial Stage Projects$18,292,674
Mark ChaoForty Seven Inc.Phase 1b Trial of Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination with Azacitidine in Patients with Acute Myeloid Leukemia and Myelodysplastic SyndromeClinical Trial Stage Projects$0
Dr. Colleen DelaneyNohla Therapeutics IncA Phase 2 Open-Label, Multi-Center, Randomized, Controlled, Optimal Dose-Finding Study of DCC-UCB in Adults Receiving High Dose Chemotherapy for AMLClinical Trial Stage Projects$4,310,000
Dr Mehrdad AbediUniversity of California, DavisStem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patientsClinical Trial Stage Projects$8,414,265
Catriona JamiesonAspera Biomedicines, Inc.Cancer Stem Cell Interception with Rebecsinib: A First-in-Class ADAR1 InhibitorLate Stage Preclinical Projects$3,200,000
Dr. David R StoverNammi Therapeutics, Inc.IND-Enabling activities for the masked immunocytokine, QXL138AMLate Stage Preclinical Projects$3,999,113
Dr. Karin Lindgren GaenslerUniversity of California, San FranciscoDevelopment of TriLeukeVax, an Engineered Autologous Leukemia Vaccine for Stimulating Cytolytic Immune Responses to Residual Leukemic Stem CellsLate Stage Preclinical Projects$6,000,000
Thomas J KippsUniversity of California, San DiegoLate Stage Pre-Clinical Development of a Cirmtuzumab Based CAR T-cell for the Treatment of ROR1+ Hematological MalignanciesLate Stage Preclinical Projects$4,130,260
Swapna PanugantiCellerant Therapeutics, Inc.Development of CLT030-ADC, a Leukemic Stem Cell Targeting Antibody-Drug-Conjugate, for Treatment of Acute Myeloid LeukemiaLate Stage Preclinical Projects$6,863,755
Dr. Paul W FinneganAngiocrine Bioscience, Inc.Development of AB-110: genetically-modified endothelial cells plus expanded cord blood hematopoietic stem cells as a transplantation therapyLate Stage Preclinical Projects$3,797,117
Dr. Irving L Weissman MDStanford UniversityClinical Investigation of a Humanized Anti-CD47 Antibody in Targeting Cancer Stem Cells in Hematologic Malignancies and Solid TumorsDisease Team Therapy Development III$6,505,568
Thomas J KippsUniversity of California, San DiegoTherapeutic Eradication of Cancer Stem Cells with UC-961 (Cirmtuzumab)Disease Team Therapy Development III$4,179,598
Ann Capela ZoveinUniversity of California, San FranciscoHuman endothelial reprogramming for hematopoietic stem cell therapy.New Faculty Physician Scientist$2,197,683
Michael ClearyStanford UniversityProstaglandin pathway regulation of self-renwal in hematopoietic and leukemia stem cellsBasic Biology IV$1,244,455
Dr Mehrdad AbediUniversity of California, DavisStem Cell Gene Therapy for HIV in AIDS Lymphoma PatientsDisease Team Therapy Planning I$66,880
Dr. Markus MuschenChildren’s Hospital of Los AngelesDual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradicationEarly Translational II$850,769
Markus MuschenUniversity of California, San FranciscoDual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradicationEarly Translational II$2,756,536
Catriona JamiesonUniversity of California, San DiegoPreclinical development of a pan Bcl2 inhibitor for cancer stem cell directed therapyEarly Translational II$3,103,041
Dr. John P ChuteUniversity of California, Los AngelesNiche-Focused Research: Discovery & Development of Hematopoietic Regenerative FactorsResearch Leadership$4,645,297
Dr. Chong-Xian PanUniversity of California, DavisCombinatorial Chemistry Approaches to Develop LIgands against Leukemia Stem CellsNew Faculty I$2,386,409
Dr. Hanna MikkolaUniversity of California, Los AngelesMechanisms of Hematopoietic stem cell Specification and Self-RenewalNew Faculty I$2,286,900
Dr. Emmanuelle PassegueUniversity of California, San FranciscoMechanisms Underlying the Responses of Normal and Cancer Stem Cells to Environmental and Therapeutic InsultsNew Faculty II$2,124,488
Catriona JamiesonUniversity of California, San DiegoDerivation and Characterization of Myeloproliferative Disorder Stem Cells from Human ES CellsNew Faculty II$3,065,572
Catriona JamiesonUniversity of California, San DiegoDerivation and Characterization of Cancer Stem Cells from Human ES CellsSEED Grant$616,305
Dr. Irving L Weissman MDStanford UniversityDevelopment of Therapeutic Antibodies Targeting Human Acute Myeloid Leukemia Stem CellsDisease Team Research I$18,759,276
Dr. Dennis A CarsonUniversity of California, San DiegoDevelopment of Highly Active Anti-Leukemia Stem Cell Therapy (HALT)Disease Team Research I$19,999,826

CIRM Leukemia Videos

Stem Cell Therapies for Leukemia: Marching Toward the Clinic

Anica Sayoc, City of Hope – CIRM Stem Cell #SciencePitch

Catriona Jamieson, UCSD – CIRM Stem Cell #SciencePitch

Irving Weissman, Stanford – CIRM Stem Cell #SciencePitch

Leukemia: Advancing Stem Cell Therapies – 2011 CIRM Grantee Meeting

Spotlight on Basic Research: Irv Weissman

Spotlight on Leukemia: Welcoming Remarks

Spolight on Leukemia: Catriona Jamieson, M.D.

Spotlight on Leukemia: Clinical Trial Participants

Progress and Promise in Leukemia

News and Information


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