Stage of Program: Clinical Trial, Phase 1/2
Lentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning
St. Jude Children’s Research Hospital is teaming up with UC San Francisco to repair the damaged immune system of children born with SCID. They will genetically modify the patient’s own blood stem cells, with the goal of creating a new blood system and restoring the health of the immune system.
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
This trial proposes to replace SCID patients’ dysfunctional immune cells with healthy ones using a safer form of bone marrow transplant (BMT). Current BMT procedures must use toxic chemotherapy to make space in the bone marrow for the healthy transplanted stem cells to engraft. The Stanford team will instead test a safe, non-toxic protein called […]
A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
X-linked Chronic Granulomatous Disease (X-CGD) is a rare immune disorder that prevents white blood cells from killing foreign invaders. This results in severe, recurrent infections that can impact quality and length of a patient’s life. X-CGD is usually diagnosed before age 5, but without treatment, children die before age 10. A team at UCLA is using the patient’s own genetically […]
Retinal progenitor cells for treatment of retinitis pigmentosa
Retinitis pigmentosa (RP) is a severe form of blindness that runs in families with an incidence of 1:4000. A team at UC Irvine, is using cells called retinal progenitor cells to repair the damage caused by this vision destroying disease. The cells are injected into the back of the eye and it’s hoped they will […]
Progenitor Cells Secreting GDNF for the Treatment of ALS
ALS is a devastating neurodegenerative disease with no cure that specifically affect a patient’s motor neurons in the brain. A team at Cedars-Sinai is transplanting millions of genetically engineered stem cells into patients with ALS. When transplanted into the patient spinal cord, these cells become astrocytes, the support cells that keep nerve cells functioning. Due to […]
MSC engineered to produce BDNF for the treatment of Huntington’s disease
A team at UC Davis plans to use bone marrow derived mesenchymal stem cells to deliver a growth factor, called BDNF, to patients’ brains in order to reduce the death of nerve cells that occurs in Huntington’s Disease (HD). In preparation for such a clinical trial study, the team completed a CIRM-funded observational trial in […]
A Phase I/IIa Dose Escalation Safety Study of AST-OPC1 in Patients with Cervical Sensorimotor Complete Spinal Cord Injury
Up to 12,000 Americans suffer a spinal cord injury (SCI) each year. This condition leads to a high level of permanent disability and decreased life expectancy. Currently, there are no approved therapies for patients with SCI. Asterias Biotherapeutics uses cells derived from embryonic stem cells to heal the spinal cord at the site of injury. They […]
Clinical trial of directly vascularized islet cell replacement therapy for high-risk type 1 diabetes
ViaCyte is developing cell therapies to replace lost beta cells for people with type 1 diabetes (T1D). The therapies are derived from human embryonic stem cells, which are partially matured into becoming pancreatic tissues (the type destroyed in T1D). The cells are inserted into a small pouch that is transplanted under the patient’s skin. The […]
Preclinical and clinical testing of a stem cell-based combination product for insulin-dependent diabetes
ViaCyte is developing cell therapies to replace lost pancreatic beta cells for people with type 1 diabetes (T1D). The therapies are derived from human embryonic stem cells, which are partially matured into becoming pancreatic tissues (the type destroyed in T1D). The cells are inserted into a small pouch that is transplanted under the patient’s skin. The […]
A Phase 1b/2a Study of the ROR1-Targeting Monoclonal Antibody, Cirmtuzumab, and the Bruton Tyrosine Kinase Inhibitor, Ibrutinib, in B-Cell Cancers
Cancer is a leading cause of death in California. Many cancers resist current therapies due to therapy-resistant cancer stem cells (CSCs). A team at UCSD is testing an antibody therapy called cirmtuzumab in a clinical trial study to treat a blood cancer, Chronic Lymphocytic Leukemia (CLL). The antibody recognizes and attaches to a protein on […]