Stage of Program: Clinical Trial, Phase 1
A Phase I, Open-Label Study To Assess The Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells (SB-728MR-HSPC) with Escalating Doses of Busulfan In HIV-1 (R5) Infected Subjects with Suboptimal CD4 Levels on cART
A team at City of Hope and Sangamo Therapeutics is genetically modifying patients’ blood forming stem cells to functionally cure people with HIV. The team is using a technology called zinc finger nucleases – a kind of molecular scissors – to snip out the target gene that codes for the CCR5 receptor. This receptor is the gateway for […]
Therapeutic Eradication of Cancer Stem Cells with UC-961 (Cirmtuzumab)
Cancer is a leading cause of death in California. Many cancers resist current therapies due to therapy-resistant cancer stem cells (CSCs). Scientists at UCSD have discovered a protein, ROR1, that is present on the surface of CSCs, but not on normal, healthy cells. The team has developed an antibody called cirmtuzumab, that specifically targets and attaches to ROR1. […]
Clinical Investigation of a Humanized Anti-CD47 Antibody in Targeting Cancer Stem Cells in Hematologic Malignancies and Solid Tumors
A team at Stanford University is using a molecule known as an antibody to target cancer stem cells. This antibody can recognize and bind to CD47, a protein the cancer stem cells carry on their cell surface. The cancer cells use that protein to evade the component of our immune system that routinely destroys tumors. By disabling […]
In Utero Hematopoietic Stem Cell Transplantation For The Treatment Of Fetuses With Alpha Thalassemia Major
Dr. MacKenzie and her team at UCSF are using hematopoietic stem cells (HSCs) to treat babies in the womb who have alpha thalassemia major, a blood disorder that is almost always fatal. Current treatment requires in utero blood transfusions and monthly blood transfusions for life or a bone marrow transplant if a suitable donor is identified. In this trial, HSCs […]
Clinical Trial of Stem Cell Gene Therapy for Sickle Cell Disease
Sickle cell disease (SCD) is caused by a genetic mutation in the hemoglobin gene which causes red blood cells to “sickle” under conditions of low oxygen. SCD affects 1:500 African-Americans and is also common in Hispanic-Americans. The median survival for patients with SCD is 42 years for males and 48 years for females. A team at UCLA […]
Phase 1 Safety Assessment of CPCB-RPE1, hESC-derived RPE Cell Coated Parylene Membrane Implants, in Patients with Advanced Dry Age Related Macular Degeneration
Age-related macular degeneration is a progressive disease resulting in death of the retinal pigment epithelium (RPE) causing distortion to central vision and eventually to legal blindness. Regenerative Patch Technologies and scientists at the University of Southern California and UC Santa Barbara, are growing specialized cells of the retina (called retinal pigment epithelium) from embryonic stem […]
A Phase 1b Trial of Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination with Azacitidine in Patients with Acute Myeloid Leukemia
Forty Seven Inc. is conducting a Phase 1b clinical trial for acute myeloid leukemia patients. Leukemia stem cells have a protein on their surface that enable them to evade being identified and destroyed by the patient’s own immune system. This protein also helps these leukemia stem cells survive traditional therapies such as chemotherapy, enabling the […]
Genetic Re-programming of Stem Cells to Fight Cancer
There are few options for patients whose cancers have metastasized due to resistance to current therapies. This team is using gene editing technology to modify a patient’s own immune system cells and blood-forming stem cells with the aim of creating a continuous supply of immune cells that can recognize and attack hard-to-treat cancers.
Evaluation of Safety and Preliminary Efficacy of Escalating Doses of GRNOPC1 in Subacute Spinal Cord Injury
Geron carried out a Phase 1 clinical trial to assess the safety and preliminary activity of escalating doses of human embryonic stem cell (hESC) derived oligodendrocyte progenitor cells for treatment of spinal cord injury. Five patients were treated, all safely and without any serious side effects, before the trial was halted by Geron for financial […]
Phase I Study of Chimeric Antigen Receptor Engineered Central Memory T cells for the Treatment of Malignant Glioma
A team at the City of Hope led by Dr. Christine Brown is pursuing a Phase 1 trial targeting an aggressive brain cancer called malignant glioma. City of Hope will re-engineer a patient’s immune system central memory T cells (TCM cells) to express chimeric antigen receptors (CAR). These CAR-T cells will recognize a molecular marker […]