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.
VIDEO
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
Professor Irving L Weissman MD 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 Thomas J Kipps University of California, San Diego Therapeutic Eradication of Cancer Stem Cells with UC-961 (Cirmtuzumab) Disease Team Therapy Development III $4,179,598 Ann Capela Zovein University of California, San Francisco Human endothelial reprogramming for hematopoietic stem cell therapy. New Faculty Physician Scientist $2,197,683 Michael Cleary Stanford University Prostaglandin pathway regulation of self-renwal in hematopoietic and leukemia stem cells Basic Biology IV $1,244,455 Dr Mehrdad Abedi University of California, Davis Stem Cell Gene Therapy for HIV in AIDS Lymphoma Patients Disease Team Therapy Planning I $66,880 Dr. 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 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 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 Dr. John P Chute University of California, Los Angeles Niche-Focused Research: Discovery & Development of Hematopoietic Regenerative Factors Research Leadership $4,645,297 Dr. Chong-Xian Pan University of California, Davis Combinatorial Chemistry Approaches to Develop LIgands against Leukemia Stem Cells New Faculty I $2,386,409 Dr. Hanna Mikkola University of California, Los Angeles Mechanisms of Hematopoietic stem cell Specification and Self-Renewal New Faculty I $2,286,900 Dr. 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 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 Catriona Jamieson University of California, San Diego Derivation and Characterization of Cancer Stem Cells from Human ES Cells SEED Grant $616,305 Professor Irving L Weissman MD Stanford University Development of Therapeutic Antibodies Targeting Human Acute Myeloid Leukemia Stem Cells Disease Team Research I $18,759,276 Dr. Dennis A Carson University of California, San Diego Development of Highly Active Anti-Leukemia Stem Cell Therapy (HALT) Disease Team Research I $19,999,826 Aniruddha Deshpande Sanford Burnham Prebys Medical Discovery Institute Investigating the SGF29/SAGA complex in regulation of normal and cancer stem cells Foundation – Discovery Stage Research Projects $1,547,999 Dr. Camilla Forsberg University of California, Santa Cruz Determining how age-specific heterogeneity of human hematopoietic stem cells and megakaryocyte progenitors contribute to thrombotic disease upon aging Foundation – Discovery Stage Research Projects $1,336,800 Dr. Robert Negrin MD Stanford University Orthogonal IL2 Receptor Transduced Regulatory T Cells for Clinical Application Quest – Discovery Stage Research Projects $1,920,652 Dr Dinesh S Rao University of California, Los Angeles Novel methods to eliminate cancer stem cells Quest – Discovery Stage Research Projects $1,384,347 Dr Julia Carnevale University of California, San Francisco Generating deeper and more durable BCMA CAR T cell responses in Multiple Myeloma through non-viral knockin/knockout multiplexed genome engineering Quest – Discovery Stage Research Projects $1,463,368 Dr. Tannishtha Reya University of California, San Diego Targeting Cancer Stem Cells in Hematologic Malignancies Quest – Discovery Stage Research Projects $1,960,560 Markus Müschen City of Hope, Beckman Research Institute Lgr5-mediated self-renewal in B cell selection and leukemia-initiation Quest – Discovery Stage Research Projects $2,183,223 Dr. John P Chute University of California, Los Angeles Protein tyrosine phosphatase – sigma inhibitors for hematopoietic regeneration Quest – Discovery Stage Research Projects $2,115,735 Dan S Kaufman University of California, San Diego Targeted off-the-shelf immunotherapy to treat refractory cancers Quest – Discovery Stage Research Projects $1,936,936 Dr. David J 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 Dr. Jacob Ellery Corn Ph.D 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 Dr Michael A. Caligiuri City of Hope, Beckman Research Institute Targeting multiple myeloma with BCMA-CAR NK cells expressing a GPRC5D-NKG2D bispecific antibody Therapeutic Translational Research Projects $6,036,001 Dr. William J Murphy University of California, Davis Overcoming resistance to standard CD19-targeted CAR T using a novel triple antigen targeted vector Therapeutic Translational Research Projects $3,869,061 Arun P Wiita University of California, San Francisco CD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemia Therapeutic Translational Research Projects $3,330,801 Dr. Lili Yang University of California, Los Angeles HSC-Engineered Off-The-Shelf CAR-iNKT Cell Therapy for Multiple Myeloma Therapeutic Translational Research Projects $5,281,199 Yvonne Y. Chen University of California, Los Angeles BCMA/CS1 Bispecific CAR-T Cell Therapy to Prevent Antigen Escape in Multiple Myeloma Therapeutic Translational Research Projects $3,176,805 Dr. Karin Lindgren Gaensler University of California, San Francisco Developing engineered autologous leukemia vaccines to target residual leukemic stem cells Therapeutic Translational Research Projects $4,171,728 Dan S Kaufman University of California, San Diego Human Embryonic Stem Cell-Derived Natural Killer Cells for Cancer Treatment Therapeutic Translational Research Projects $3,260,000 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 Ezra Cohen University of California, San Diego Development of ROR1 CAR-T cells to target cancer stem cells in advanced malignancies Therapeutic Translational Research Projects $5,795,584 Dr. Lili Yang University of California, Los Angeles Stem Cell-Based iNKT Cell Therapy for Cancer Therapeutic Translational Research Projects $6,956,775 Rochelle Emery Senti Biosciences, Inc. Selective, Off-the-Shelf Logic Gated CAR NK Cell Therapy Targeting CD33 and/or FLT3 Expressing Hematologic Malignancies Clinical Trial Stage Projects $8,000,000 Elizabeth Budde City of Hope, Beckman Research Institute Phase I Study of Chimeric Antigen Receptor Engineered T Cells targeting CD33 for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia Clinical Trial Stage Projects $11,983,547 Dr. Paul Finnegan Angiocrine Bioscience, Inc. AB-205-301 Phase 3 Trial and Related Activities to Support Clinical Development of AB-205 Clinical Trial Stage Projects $10,930,014 Dr. Matthew H Porteus Stanford University Phase 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 Woodard Immune-Onc Therapeutics Phase 1 Clinical Development of IO-202, A First-in-Class Antibody Targeting LILRB4, for the Treatment of AML with Monocytic Differentiation and CMML Clinical Trial Stage Projects $6,000,000 Dr. Edward Kavalerchik Angiocrine Bioscience, Inc. AB-205-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-205 Clinical Trial Stage Projects $6,192,579 Dr. David B. Miklos 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 Rajesh Belani MD 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 Michael A. 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 Dr. Edward Kavalerchik 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 Thomas J 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 Mark Chao Forty Seven Inc. Phase 1b Trial of Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination with Azacitidine in Patients with Acute Myeloid Leukemia and Myelodysplastic Syndrome Clinical Trial Stage Projects $0 Dr. 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 $4,310,000 Dr 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 Thomas Martin University of California, San Francisco A 1XX-enhanced and fully non-viral BCMA CAR T cell therapy for Relapsed and Refractory Multiple Myeloma (UCCT-BCMA-1) Late Stage Preclinical Projects $4,585,501 Catriona Jamieson Aspera Biomedicines, Inc. Cancer Stem Cell Interception with Rebecsinib: A First-in-Class ADAR1 Inhibitor Late Stage Preclinical Projects $3,200,000 Dr. David R Stover Nammi Therapeutics, Inc. IND-Enabling activities for the masked immunocytokine, QXL138AM Late Stage Preclinical Projects $3,999,113 Dr. Karin Lindgren Gaensler University of California, San Francisco Development of TriLeukeVax, an Engineered Autologous Leukemia Vaccine for Stimulating Cytolytic Immune Responses to Residual Leukemic Stem Cells Late Stage Preclinical Projects $6,000,000 Thomas J Kipps University of California, San Diego Late Stage Pre-Clinical Development of a Cirmtuzumab Based CAR T-cell for the Treatment of ROR1+ Hematological Malignancies Late Stage Preclinical Projects $2,160,000 Swapna Panuganti 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 Dr. Paul W 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
Total:
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