Problem

Central nervous system (CNS) injury can result in permanent neurological disability and vision loss. There is a dire need to develop novel therapies that overcome barriers to repair within the CNS and promote neuron survival and axon regeneration.

Solution

• Researchers at OSU have developed CD33+ and C33+CD34+ stem cell lines derived from human umbilical cord blood (HUCB) and bone marrow cells, respectively, with neuroprotective/axon-regenerative effects that stimulate the neurite outgrowth of both mouse neurons and human induced pluripotent stem cell-derived neurons, and drive the regeneration of optic nerve axons in vivo upon adoptive transfer into immunodeficient mice with optic nerve crush injury
• The therapeutic effects of CD33⁺ HUCB cell infusions can be enhanced by:
  1. 1. polarizing or genetically engineering primary CD33⁺ HUCB donor cells or CD33⁺CD34⁺ HUCB donor cell lines to expand their repertoire of cytoprotective factors, thereby enhancing their reparative properties or to increase the expression of anti-apoptotic molecules and chemotactic receptors, thereby prolonging their longevity and retention in the eye

1. 1. 2. modifying the CNS or ocular microenvironment by overexpressing polarizing factors and chemoattractants that stabilize reparative HUCB cells and accelerate their local accumulation
2. 1. 3. combining HUCB cell infusion with independent pro-regenerative interventions (ex. blockade of intrinsic axon growth inhibitory pathways), thereby magnifying therapeutic impact via synergistic mechanisms

Applications

• Gene and cell therapy for treating neurological disorders and CNS injuries
• Treatment of vision loss

Advantages

• Versatility: Technology can be adapted to treat different neurological disorders and injuries
• Combination Therapy: Therapeutic effects can be augmented synergistically in combination with other approaches
• Well-tolerated: Early phase clinical trials of autologous/allogeneic HUCB cell infusion in human subjects with neurological disorders have shown a favorable safety profile

Seeking opportunities for out-licensing, collaboration