Leukemia Fact Sheet

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

Description

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 name Institution Grant Title Grant Type Award Amount
Catriona Jamieson University of California, San Diego A Splicing Modulator Targeting Cancer Stem Cells in Acute Myeloid Leukemia Therapeutic Translational Research Projects $2,511,767
Catriona Jamieson University of California, San Diego Derivation and Characterization of Cancer Stem Cells from Human ES Cells SEED Grant $616,305
Dan Kaufman University of California, San Diego Human Embryonic Stem Cell-Derived Natural Killer Cells for Cancer Treatment Therapeutic Translational Research Projects $4,698,821
Catriona Jamieson University of California, San Diego Preclinical development of a pan Bcl2 inhibitor for cancer stem cell directed therapy Early Translational II $3,103,041
John Zaia City of Hope, Beckman Research Institute The Innovation-Alpha Clinic for Cellular Therapies (I-ACT) – A Program for the Development and Delivery of Innovative Cell-based Treatments and Cures for Life-threatening Diseases. Alpha Stem Cell Clinics $8,000,000
David Baylink Loma Linda University Bone Marrow Targeting of Hematopoietic Stem Cells Engineered to Overexpress 25-OH-VD3 1-α-hydroxylase for Acute Myeloid Leukemia Therapy Inception - Discovery Stage Research Projects $178,967
Markus Muschen University of California, San Francisco Dual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradication Early Translational II $2,756,536
Mehrdad Abedi University of California, Davis Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients Clinical Trial Stage Projects $8,414,265
Jacob Corn University of California, Berkeley Genome editing for causation and reversion of MPN-associated mutations in human hematopoietic stem cells Inception - Discovery Stage Research Projects $235,800
Tannishtha Reya University of California, San Diego Targeting Cancer Stem Cells in Hematologic Malignancies Quest - Discovery Stage Research Projects $1,960,560
Dennis Carson University of California, San Diego Development of Highly Active Anti-Leukemia Stem Cell Therapy (HALT) Disease Team Research I $19,999,826
Dan Kaufman University of California, San Diego Targeted off-the-shelf immunotherapy to treat refractory cancers Quest - Discovery Stage Research Projects $1,936,936
Crystal Mackall Stanford University Phase 1 Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Adults with Recurrent or Refractory B Cell Malignancies Clinical Trial Stage Projects $11,034,982
Irving Weissman Stanford University Development of Therapeutic Antibodies Targeting Human Acute Myeloid Leukemia Stem Cells Disease Team Research I $18,759,276
John Chute University of California, Los Angeles Protein tyrosine phosphatase - sigma inhibitors for hematopoietic regeneration Quest - Discovery Stage Research Projects $2,116,708
Karin Gaensler University of California, San Francisco Developing engineered autologous leukemia vaccines to target residual leukemic stem cells Therapeutic Translational Research Projects $4,171,728
Catriona Jamieson University of California, San Diego Derivation and Characterization of Myeloproliferative Disorder Stem Cells from Human ES Cells New Faculty II $3,065,572
Paul Finnegan Angiocrine Bioscience, Inc. Development of AB-110: genetically-modified endothelial cells plus expanded cord blood hematopoietic stem cells as a transplantation therapy Late Stage Preclinical Projects $3,797,117
Emmanuelle Passegue University of California, San Francisco Mechanisms Underlying the Responses of Normal and Cancer Stem Cells to Environmental and Therapeutic Insults New Faculty II $2,124,488
Markus Müschen City of Hope, Beckman Research Institute Lgr5-mediated self-renewal in B cell selection and leukemia-initiation Quest - Discovery Stage Research Projects $2,186,520
Hanna Mikkola University of California, Los Angeles Mechanisms of Hematopoietic stem cell Specification and Self-Renewal New Faculty I $2,286,900
Jagath Junutula Cellerant Therapeutics, Inc. Development of CLT030-ADC, a Leukemic Stem Cell Targeting Antibody-Drug-Conjugate, for Treatment of Acute Myeloid Leukemia Late Stage Preclinical Projects $6,863,755
Chong-Xian Pan University of California, Davis Combinatorial Chemistry Approaches to Develop LIgands against Leukemia Stem Cells New Faculty I $2,386,409
Paul Finnegan Angiocrine Bioscience, Inc. AB-110-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-110 Clinical Trial Stage Projects $5,000,000
Gay Crooks University of California, Los Angeles Forming the Hematopoietic Niche from Human Pluripotent Stem Cells Basic Biology III $1,252,857
Thomas Kipps University of California, San Diego A Phase 1b/2a Study of the ROR1-Targeting Monoclonal Antibody, Cirmtuzumab, and the Bruton Tyrosine Kinase Inhibitor, Ibrutinib, in B-Cell Cancers Clinical Trial Stage Projects $18,292,674
Mehrdad Abedi University of California, Davis Stem Cell Gene Therapy for HIV in AIDS Lymphoma Patients Disease Team Therapy Planning I $66,880
Colleen Delaney Nohla Therapeutics Inc A Phase 2 Open-Label, Multi-Center, Randomized, Controlled, Optimal Dose-Finding Study of DCC-UCB in Adults Receiving High Dose Chemotherapy for AML Clinical Trial Stage Projects $6,922,109
Michael Cleary Stanford University Prostaglandin pathway regulation of self-renwal in hematopoietic and leukemia stem cells Basic Biology IV $1,244,455
Mark Chao Forty Seven Inc. A Phase 1b Trial of Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination with Azacitidine in Patients with Acute Myeloid Leukemia Clinical Trial Stage Projects $3,200,000
Ann Zovein University of California, San Francisco Human endothelial reprogramming for hematopoietic stem cell therapy. New Faculty Physician Scientist $2,197,683
Mark Walters Children's Hospital of Oakland Research Institute University of California, San Francisco (UCSF) CIRM Alpha Stem Cell Clinic Alpha Stem Cell Clinics Network Expansion $7,999,999
Thomas Kipps University of California, San Diego Therapeutic Eradication of Cancer Stem Cells with UC-961 (Cirmtuzumab) Disease Team Therapy Development III $4,179,598
Michael Pulsipher Children's Hospital of Los Angeles Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES) Clinical Trial Stage Projects $4,825,587
Irving Weissman Stanford University Clinical Investigation of a Humanized Anti-CD47 Antibody in Targeting Cancer Stem Cells in Hematologic Malignancies and Solid Tumors Disease Team Therapy Development III $6,505,568
Matthew Spear Poseida Therapeutics, Inc. Clinical Study of T stem cell memory (Tscm)-based CAR-T cells in Patients with Multiple Myeloma Clinical Trial Stage Projects $19,813,407
Markus Muschen Children's Hospital of Los Angeles Dual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradication Early Translational II $850,769
Total:
$195,557,865.00

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