Increased Therapeutic Potential of Migrating Neural Stem Cells and ASC101 Treatment

Award Information
Agency:
Department of Health and Human Services
Branch
n/a
Amount:
$335,440.00
Award Year:
2013
Program:
SBIR
Phase:
Phase I
Contract:
1R43NS079027-01A1
Award Id:
n/a
Agency Tracking Number:
R43NS079027
Solicitation Year:
2013
Solicitation Topic Code:
NINDS
Solicitation Number:
PA12-088
Small Business Information
135 CHINQUAPIN AVE, CARLSBAD, CA, 92008-6277
Hubzone Owned:
N
Minority Owned:
N
Woman Owned:
Y
Duns:
617462234
Principal Investigator:
LEONARDMILLER
(760) 434-8648
miller4atc@adelphia.net
Business Contact:
LYNNETKOH
(760) 434-8648
miller4atc@adelphia.net
Research Institute:
Stub




Abstract
DESCRIPTION (provided by applicant): Multiple sclerosis (MS) affects 1 in 1000 individuals in the US and is characterized by progressive sensory, motor and cognitive impairments. These functional deficits are linked to inflammatory attacks on myelin, oligodendrocytes, and axons. Therapeutic strategies that promote remyelination, protect against inflammation, and protect axons have the potential to prevent and even improve functional declines in MS patients. One therapeutic approach is to use neural stem cells (NSCs), which are able to rescue endangered host neurons and glial cells by providing trophic support, detoxifying the microenvironment, mobilizing endogenous progenitors, triggering self-repair mechanisms, exerting anti-inflammatory and anti-scarring actions, and promoting angiogenesis. Each of these effects has the potential to improve outcomes in MS patients. Intravenous administration of NSCs is an alternative to direct injections into the CNS. This approach is feasible because systemically administered NSCs can circumvent the blood-brain barrier (BBB) and localize to a site of intracranial pathology. However, one limitation of this approach is that NSCs are extremely inefficient (lt5% cells) at homing, even to an injured site in the brain. We have found that the molecules mediating homing in NSCs are defective, which contributes to the poor efficiency of NSC homing. Our preliminary in vitro data show that our proprietary technology, ASC-101, corrects this natural deficiency and improves interactions of NSCs under conditions of physiological shear stress. This Phase I SBIR grant submission is designed to generate a proof-of-principle that ex vivo correction of NSC homing molecules will improve their homing to MS lesions in a mouse model of MS. In Aim 1we will complete the production of research-grade ASC-101 for use in experiments under this grant. In Aim 2, ASC- 101-treated NSCs will be intravenously injected in experimental autoimmune encephalomyelitis (EAE) mice and we will evaluate their migration to the lesions, differentiation and overall efficacy. EAE is a widely accepted and utilized animal model of MS. If successful, this project will provide the basis for further testing ASC-101 technology in additional animal models of MS using NSCs.PUBLIC HEALTH RELEVANCE PUBLIC HEALTH RELEVANCE: Neural stem cells (NSCs) have the potential for treatment of multiple sclerosis (MS). While intravenous (iv) administration of NSCs is preferable over direct injections into the CNS, this approach is limited due to an observed low homing efficiency of NSCs. ASC-101 technology from America Stem Cell improves the interactions of NSCs with vasculature and, thus, has the potential to enhance homing of iv administered NSCs and to improve the overall efficacy ofNSC-based cellular therapy.

* information listed above is at the time of submission.

Agency Micro-sites


SBA logo

Department of Agriculture logo

Department of Commerce logo

Department of Defense logo

Department of Education logo

Department of Energy logo

Department of Health and Human Services logo

Department of Homeland Security logo

Department of Transportation logo

Enviromental Protection Agency logo

National Aeronautics and Space Administration logo

National Science Foundation logo
US Flag An Official Website of the United States Government