Program Type: Translation
Neuroprotection to treat Alzheimer’s: a new paradigm using human central nervous system cells
Alzheimer’s disease (AD) is an incurable disorder that affects memory, social interaction and the ability to perform everyday activities. In the USA alone, the number of AD patients aged 65 and older has surpassed 5 million and that number may triple by 2050. Annual health care costs have been estimated to exceed 172 billion dollars, […]
Combination therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy
A drug was identified through the use of muscle stem cells that can enhance the effectiveness of exon skipping by antisense oligonucleotides to the DMD gene to restore dystrophin expression and at least partially correct the defect responsible for loss of muscle function in Duchenne. We propose to test the effectiveness of this drug in […]
Preclinical Development and First-In-Human Testing of GRNCM1 in Advanced Heart Failure
This application seeks to bring to the clinic a new treatment for myocardial disease based on human embryonic stem cell (hESC) derived cardiomyocytes. hESC-cardiomyocytes have the unique potential to address the underlying cause of heart disease by repopulating areas of damaged myocardium (heart tissue) with viable cardiac cells. This therapeutic approach represents a potential breakthrough […]
Neural Stem Cell-Based Therapy For Parkinson’s Disease
Ongoing degeneration of dopaminergic (DA) neurons in the midbrain is the hallmark of Parkinson’s disease (PD), a movement disorder that manifests with tremor, bradykinesia and rigidity. One million Americans live with PD and 60,000 are diagnosed with this disease each year. Although the cost is $25 billion per year in the United States alone, existing […]
hESC-derived NPCs Programmed with MEF2C for Cell Transplantation in Parkinson’s Disease
We proposes to use human embryonic stem cells (hESCs) differentiated into neural progenitor/stem cells (NPCs), but modified by transiently programming the cells with the transcription factor MEF2C to drive them more specifically towards dopaminergic (DA) neurons, representing the cells lost in Parkinson’s disease. We will select Parkinson’s patients that no longer respond to L-DOPA and […]
Increasing the endogenous mesenchymal stem cells to the bone surface to treat osteoporosis
Although most individuals are aware that osteoporosis is disease of increased bone fragility that results from estrogen deficiency and aging, most are unaware of the high risk and cost of the disorder. It is estimated that close to 30% of the fractures that occur in the United States each year are due to osteoporosis (Schwartz […]
A CIRM Disease Team to Develop Allopregnanolone for Prevention and Treatment of Alzheimer’s Disease
Alzheimer’s disease (AD) is now a nation-wide epidemic and California is at the epicenter of the epidemic. One-tenth of all people in the United States diagnosed with AD live in California. In the US, 5.4 million people have AD and another American develops AD every 69 seconds. No therapeutic strategies exist to prevent or treat […]
Clinical Development of an osteoinductive therapy to prevent osteoporosis-related fractures
There are over 1.5 million osteoporotic fractures annually in the USA alone, at a cost of approximately $15 billion each year. The majority of these fractures occur in the spine, followed by the hip and wrist. Incidence varies according to age; vertebral fracture rates increase rapidly by the sixth decade of life, whereas the risk […]
Human Embryonic Stem Cell-Derived Cardiomyocytes for Patients with End Stage Heart Failure
Patients with end-stage heart failure (ESHF) have a 2-year survival rate of 50% with conventional medical therapy. This dismal survival rate is actually significantly worse than patients with AIDS, liver cirrhosis, stroke, and other debilitating diseases. Stem cell therapy may be a promising strategy for inducing myocardial regeneration via paracrine activation, prevention of cardiac apoptosis, […]
Airways for Children
The primary goal is to bring a safe and effective therapy to children with severe large airway disease. Our intent is to implement all of the necessary steps for a successful new stem/progenitor cell-derived airway transplant for clinical trials in children within 4 years. Our team builds on first-in-human surgical success with a stem cell-based […]