Open Competition 5 - Biotechnology

Neural Regeneration with Mesenchymal Stem Cells

Sponsor: Osiris Therapeutics, Inc.

• Project Performance Period: 6/1/2002 - 5/31/2005
• Total project (est.): $3,226,325.00
• Requested ATP funds: $2,000,000.00

Millions of people suffer trauma, diseases, or aging-related afflictions of the central nervous system (CNS). With little capacity to regenerate, the CNS is not very responsive to medical treatment. Existing therapies cannot reduce the substantial medical and economic impacts of acute conditions such as spinal cord injuries and stroke. The estimated cost of treatment and social support for spinal cord injuries is $7.7 billion annually in the United States, and the costs attributed to stroke, which affects the brain, are much higher still. To lay the groundwork for more effective treatments, Osiris Therapeutics proposes to demonstrate the feasibility of using human mesenchymal stem cells (MSCs) from adult bone marrow to regenerate neural tissue in an appropriate environment. Transplanted MSCs -- unspecialized cells that differentiate into functional cells during the body's development -- have a long safety record in animal studies and human trials, do not stimulate an immune response in the recipient, pose no ethical issues, and have been shown to regenerate cells of many connective tissues, bone, and skeletal muscle. Evidence suggests that MSCs also may have the ability to differentiate into neural cells and express the appropriate genes to regenerate tissue. The company hopes to develop "universal" MSCs, collected from volunteers and expanded in a controlled manner, that can be made by low-cost batch production. In the ATP project, Osiris will test the differentiation of MSCs both in culture with a variety of growth and differentiation factors, and in animal models. The company will test the ability of MSCs to engraft and integrate into a spinal cord injury model, slow the progression of the lesion, and improve the animal's abilities and mobility. Similar studies are planned in a stroke model. To visualize the MSCs after implantation, the company will work with the National Institutes of Health (Bethesda, Md.) to develop labeling techniques for real-time cell tracking with magnetic resonance imaging. Among the more challenging aspects of the project, the company plans to identify genes associated with a developmental stage in which MSCs may have expanded potential for differentiation, and to determine whether MSCs can be implanted at the site of injured tissue. Staff at the University of Florida School of Medicine (Gainesville, Fla.) and Henry Ford Hospital (Detroit, Mich.) will be subcontracted to perform animal studies. The ATP funding, which will accelerate the research by three to five years, is needed because private investors will not fund research on novel clinical approaches without promising data from animal studies. If successful, the project would help move this new treatment concept forward to preclinical studies and eventually clinical trials in humans. This approach has the potential to save billions of dollars in healthcare costs; moreover, even a small improvement in a patient's prognosis could mean a vast improvement in quality of life in terms of increased self-sufficiency.

For project information:

Mark F. Pittenger, (410) 522-5005
mpittenger@osiris.com

ATP Project Manager

Mrunal Chapekar, (301) 975-6846
mrunal.chapekar@nist.gov