Human Stem Cell Use: Cancer Stem Cell

Lgr5-mediated self-renewal in B cell selection and leukemia-initiation

Research Objective LGR5-antibody drug conjugate to target LIC in B cell tumors that undergo self-renewal Impact LIC were only defined in myeloid leukemia, while LIC populations in B cell tumors remain elusive. LICs give rise to drug-resistance and relapse and remain unsolved clinical problems in B cell tumors. Major Proposed Activities Proof of concept studies- […]

Targeting cancer stem cells with nanoparticle RNAi delivery to prevent recurrence and metastasis of ovarian cancer

Research Objective Our objective is to develop novel treatments for women with ovarian cancer, specifically treatments that target stem cells to reverse drug resistance. These will treat recurrent metastatic disease. Impact We aim to use nanoparticles to make ovarian cancer stem cells more drug sensitive, less invasive, and less likely to regrow tumors and metastasize. […]

Human Pancreatic Cancer Stem Cells: Developing a Novel Drug for Cancer Eradication

Research Objective We will use human pancreatic cancer (PC) stem cells to show 1 inhibits proliferation, self-renewal and cell viability. This paradigm is transformational for anti-cancer drug discovery for patients. Impact Pancreatic cancer (PC) kills >40,000/yr in the US. PC is a major unmet medical need. Use of PC stem cells in development of 1 […]

Developing engineered autologous leukemia vaccines to target residual leukemic stem cells

Translational Candidate A universally applicable, patient-specific leukemia vaccine engineered to express a novel immune stimulatory combination for post-remission therapy Area of Impact There is a critical and unmet need for new and safe treatment for older acute myelogenous leukemia (AML) patients whose current prognosis is poor Mechanism of Action In older patients with AML, treatment […]

A Splicing Modulator Targeting Cancer Stem Cells in Acute Myeloid Leukemia

Translational Candidate 17S-FD-895 is a potent small molecule splicing modulator that inhibits aberrant splicing in CSCs that have deregulated SF3B1 expression. Area of Impact Development of 17S-FD-895 could address a major bottleneck to reducing AML mortality by targeting splicing deregulated-CSCs that fuel AML relapse. Mechanism of Action 17S-FD-895 will positively impact patients with AML by […]

Development of ROR1 CAR-T cells to target cancer stem cells in advanced malignancies

Translational Candidate Autologous ROR1 CAR-T cell transduced with a lentiviral vector containing scFv (cirmtuzumab) with CD28, CD137, CD3zeta signaling domains Area of Impact ROR1 expressing cancer stem cells in solid tumors and hematologic tumors Mechanism of Action ROR1 CAR is a 3rd generation chimeric construct with an internal endodomain that transmits a CD3 zeta signal […]

2nd Generation Vaccine for the Treatment of Glioblastoma

Translational Candidate It is the peptide LEEKKYNYVVVTDHC conjugated to KLH and used as an anti-cancer vaccine. Area of Impact This is a better optimized, more robust vaccine that aspires to greatly improve glioblastoma patient survival over the current vaccine. Mechanism of Action The vaccine stimulates B cell and T cells. We have found this may […]

Phase 1 Clinical Development of IO-202, A First-in-Class Antibody Targeting LILRB4, for the Treatment of AML with Monocytic Differentiation and CMML

Therapeutic Candidate or Device IO-202, a first-in-class antibody targeting leukocyte immunoglobulin-like receptor B4 (LILRB4), an immune inhibitory receptor Indication Acute myeloid leukemia (AML) with monocytic differentiation and chronic myelomonocytic leukemia (CMML) Therapeutic Mechanism IO-202 is the first T-cell activator for AML. Preclinical studies showed that IO-202 can convert a “don’t kill me” to “kill me” […]

A Phase 1b/2a Study of the ROR1-Targeting Monoclonal Antibody, Cirmtuzumab, and the Bruton Tyrosine Kinase Inhibitor, Ibrutinib, in B-Cell Cancers

Therapeutic Candidate or Device Cirmtuzumab (UC-961) is a therapeutic monoclonal antibody that inhibits ROR1, a tumor-specific protein on the surface of many cancer stem cells Indication Cirmtuzumab will be used with the approved drug, ibrutinib, for patients with chronic lymphocytic leukemia or mantle cell lymphoma Therapeutic Mechanism ROR1 is a cell surface protein which is […]

Phase 1b Trial of Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination with Azacitidine in Patients with Acute Myeloid Leukemia and Myelodysplastic Syndrome

Therapeutic Candidate or Device Hu5F9-G4 is a drug called an antibody that is designed to mobilize the body's immune system to eliminate cancer and cancer stem cells. Indication Patients with relapsed and/or refractory AML or newly diagnosed AML who cannot receive standard high dose chemotherapy. Therapeutic Mechanism This treatment targets cancer stem cells, which are […]