A translational research project for defining a stem cell formulation for treating ischemic stroke based on comparison of adult neural stem cells and neural stem cells derived from embryonic stem cells.

Funding Type: 
Disease Team Planning
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

The goal is to advance stem cell therapy into the clinic for the treatment of California residents who have suffered an ischemic stroke and who still are disabled with neurological and movement limitations. Using traditional animal-based testing systems, a variety of neural stem cells will be studied and characterized.

The study will compare the effectiveness for stroke treatment of two-types of neural stem cells. One type is obtained from human neural tissue; the other neural stem cells are derived from human embryonic stem cells.

Both cell types will be studied alone and in combination with stem cells obtained from adult bone marrow. Previous human studies indicate that the bone-marrow derived stem cells support the effectiveness of the neural stem cells for stroke treatment.
The study team members are from collaborating institutions. The team includes experts in growing and manufacturing stem cells, translating medical research into bed-side medical treatment, neurologists and neurosurgeons experienced in the treatment of stroke.

The team will study the neural stem cells at the levels of: (a) molecular genetic and protein expression, (b) the interactions of stem cells with each other, (c) the effects of various biological molecules on differentiation of the stem cells, (d) the movement of stem cells after they are administered to a subject, (e) the combination of stem cell types required for optimal therapeutic outcome in stroke.

Further studies involve administering stem cells either non-invasively by intravenous injections, or invasively by temporary placement of a small tube into the brain through which the stem cells will be delivered.

The safety of the stem cells for treatment of strokes will be extensively studied in toxicology and carcinogenicity tests. Once safety has been established, the team will file an application with the US Food and Drug Administration for a license to begin testing the stem cells in California residents who have had strokes and suffer from on-going neurological and movement limitations.

Statement of Benefit to California: 

The Planning Group seeks to accelerate the development of a safe and efficacious stem cell therapy for treating those afflicted by stroke. The results of this research initiative will provide significant and measurable benefits to the State of California and its health care system by:

1. Restoring the quality of life to thousands of individuals, their families and caretakers.
2. Reducing the short-term and long-term private and public costs associated with treatment, care and rehabilitation.
3. Serving as a model for collaboration between the private and public sectors
4. Making California a ‘magnet’ for world-renowned researchers and grant money to further regenerative medical research.

According to the California Department of Health Services, stroke is the third leading cause of death in America and the number one cause of adult disability. In 2005, an estimated 9.1% of the population aged 65 or older in California was reported to be affected by stroke. The estimated costs for the treatment and prevention programs in the State are estimated to be in excess of $6.84 billion in both direct and indirect costs. It is estimated that IS accounts for approximately seventy-five percent of the overall costs.

These economic costs do not account for the overall time and emotional burden that families face each and every day when someone within their family has been affected by stroke. As our State’s population ages, and as high blood pressure, hypertension, poor diet, lack of exercise and obesity increase in our general population, so too will the incidence of stroke rise. Therefore, the need to find ways to treat individuals who have already had a first stroke is critical to lessening the economic and social burden on California and its citizens.

An increasing number of pre-clinical and clinical studies, (including those already conducted by team members), provide evidence that stem cell therapy improves functional outcome after ischemic brain damage, even in patients who have experienced stroke several years prior to treatment. The depth and breadth of academic and industry experience and contacts of the Planning Team will accelerate a timely, scientifically-supported clinical pathway to FDA approval.

With the full and active support of the CIRM, this Planning Group believes the State of California and its citizens will benefit in a myriad of ways through the advancement of stem cell therapy for those afflicted by ischemic stroke.