Neurological Disorders

Coding Dimension ID: 
303
Coding Dimension path name: 
Neurological Disorders
Grant Type: 
Tools and Technologies III
Grant Number: 
RT3-07655
Investigator: 
Type: 
PI
Type: 
Partner-PI
ICOC Funds Committed: 
$1 784 052
Disease Focus: 
Neurological Disorders
Collaborative Funder: 
Germany
Human Stem Cell Use: 
Embryonic Stem Cell
iPS Cell
Public Abstract: 

Three years ago, with help from CIRM funding, we developed an assay. This is a genomics-base diagnostic assay, similar to those now used for diagnosing cancers; but in our case, it is designed to analyze human ES and iPS cells. The assay is very simple to use; researchers use microarrays to profile the genes that are active in their cells. They upload the microarray data to the website, and in a few minutes they find out whether or not their cells are pluripotent. Our assay is replacing the old method for proving pluripotency, which involves producing tumors in animals. Our assay has been extremely popular, with 9,386 samples analyzed by 581 research groups in 29 countries so far. In this proposal, we plan to take the same concept and apply it to translational stem cell applications. Our new assay will allow researchers to easily detect DNA damage in their stem cells, and will enable the detection of undifferentiated or other abnormal cells (which potentially could form a tumor) in populations used for cell replacement therapy. We are also designing specific assays for quality control of neuronal cells to be transplanted to Parkinson's disease patients and for other neurological therapies. Finally, with our European partners, we will develop an assay for ensuring reliability of drug screening assays using stem cells. Our tools will greatly simplify translation of hESCs and iPSCs to the clinic.

Statement of Benefit to California: 

California is at the leading edge of development of stem cell therapies to treat previously untreatable diseases. It is critical at this important stage, when treatments are being transferred from the lab to the clinic, that the cells used for therapy are carefully produced and qualified. Our project combines two of California's best scientific assets: genomics and stem cells. Our quality control assays for stem cell production are based on our long experience in genomic analysis of stem cells and development of genomics-based diagnostic tests. The assays will ensure that stem cells used for therapy are consistently of high quality. This will speed the development of stem cell therapies for Californians.

Grant Type: 
Tools and Technologies III
Grant Number: 
RT3-07616
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$1 308 711
Disease Focus: 
Amyotrophic Lateral Sclerosis
Neurological Disorders
Spinal Cord Injury
Spinal Muscular Atrophy
Human Stem Cell Use: 
Embryonic Stem Cell
Cell Line Generation: 
Embryonic Stem Cell
Public Abstract: 

Motor neurons degenerate and die as a consequence of many conditions, including trauma to the spinal cord and its nerve roots and degenerative diseases such as amyotrophic lateral sclerosis and spinal muscular atrophy. Paralysis and in many cases death may result from a loss of motor neurons. No effective treatments are available for these patients. Most cellular therapy studies for motor neuron disorders are done in rodents. However, because of the dramatic differences between the rodent and human spinal cord, translation of these studies to humans is difficult. In particular, the development of new stem cell based treatments is limited by the lack of large animal models to test promising candidate therapies.
This bottleneck will be addressed by developing a new research tool in which human embryonic stem cell-derived motor neurons are transplanted into the spinal cord of rhesus macaques after injury and surgical repair of motor nerve roots. This injury and repair model mimic many features of motor neuron degeneration in humans. Microscopic studies will determine survival and tissue integration of transplanted human cells in the primate spinal cord tissues. Evaluations of walking, muscle and bladder function, sensation and magnetic resonance imaging (MRI) will test for possible benefits and potential adverse effects. This new research tool will be available for future pre-clinical testing of additional stem cell-based therapies that target motor neuron loss.

Statement of Benefit to California: 

Paralysis resulting from motor neuron loss after cauda equina and conus medullaris forms of spinal cord injury and from neurodegenerative conditions, such as amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), are devastating and affects thousands of patients and their families in California (CA). These conditions also create a significant financial burden on the state of CA. No effective treatments are available for these underserved patients. Development of a clinically relevant research tool is proposed to evaluate emerging stem cell-based motor neuron replacement therapies in translational studies. No such models are presently available to the global research community. As a result, the proposed research tool, which will remain based in CA, may attract interest across the United States and abroad, potentially being able to tap into a global translational research market of stem cell-based therapies and contribute to a positive revenue flow to CA.
Future benefits to people in CA include: 1) Development and translation of a new CA-based research tool to facilitate and expedite clinical realization of emerging stem cell-based therapies for devastating neurological conditions affecting motor neurons; 2) Reduction of health care costs and care giver costs for chronic motor neuron conditions with paralysis; 3) Potential for revenue from intellectual properties related to new cellular treatments entering clinical trials and human use.

Grant Type: 
Conference
Grant Number: 
CG1-99052
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$11 430
Disease Focus: 
Amyotrophic Lateral Sclerosis
Neurological Disorders
Public Abstract: 
Statement of Benefit to California: 
Grant Type: 
Conference
Grant Number: 
CG1-99026
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$17 200
Disease Focus: 
Neurological Disorders
Public Abstract: 
Statement of Benefit to California: 
Grant Type: 
Conference
Grant Number: 
CG1-99024
Investigator: 
Name: 
Type: 
PI
ICOC Funds Committed: 
$18 500
Disease Focus: 
Neurological Disorders
Public Abstract: 
Statement of Benefit to California: 
Grant Type: 
Conference
Grant Number: 
CG1-99008
Investigator: 
ICOC Funds Committed: 
$15 000
Disease Focus: 
Diabetes
Metabolic Disorders
Neurological Disorders
Public Abstract: 
Statement of Benefit to California: 

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