Our CNS Program
is focused on the development of our lead product candidate HuCNS-SC® human neural stem cells as a treatment for a broad range of neurological diseases and disorders, spanning all three regions of the central nervous system: the brain, the spinal cord and the eye.
The Challenge of CNS Disorders
Degenerative diseases and disorders of the central nervous system (CNS) afflict tens of millions of people worldwide, and often arise due to the failure of CNS tissue due to the loss of functional cells. For many of these conditions, there are no curative treatment options today and effective long-term therapies remain limited. Human neural stem cell therapy offers a promising new approach for the preservation or restoration of cellular function that could fundamentally alter the treatment of neurodegenerative diseases and injuries to the brain or nervous system.
Our Strategy
Our CNS Program initially focused on developing clinical applications in which transplanting HuCNS-SC human neural stem cells would protect the patient’s host cells before such cells are irreversibly damaged or lost due to disease progression. Specifically, our strategy for our first two indications in the clinic has been to use our HuCNS-SC cells to preserve neurological function by providing what the patient's own cells cannot – a missing enzyme in NCL (a fatal lysosomal storage disorder) and myelin in PMD (a fatal myelination disorder).
Although NCL and PMD are rare diseases, these first two indications represent “keyholes” to larger groups of CNS diseases and disorders, which might also be addressed with this “neuroprotection” approach. Demonstration of a therapeutic benefit in NCL, for example, could lead to future treatments for more than 20 CNS-mediated lysosomal storage disorders that cannot be treated with enzyme replacement therapy because enzymes are too large to cross the blood-brain barrier. In the case of PMD, the outcome of our current studies may reveal important information for the treatment of other similar myelination disorders, such as multiple sclerosis, transverse myelitis, and ceratin types of cerebral palsy.
With the March 2011 initiation of our Phase I/II clinical trial in spinal cord injury, we are now broadening our clinical focus to areas of more significant unmet medical need. In addition to our spinal cord injury trial, we are also pursuing the treatment of eye diseases in which retinal degeneration plays a central role, such as age-related macular degeneration (AMD). We plan to file an IND for a Phase I/II trial in AMD by year-end 2011.
Longer term, we believe HuCNS-SC cells have the potential to restore or replace lost cells and cellular function, and could be developed for the treatment of an even broader range of neurological conditions including Alzheimer’s disease and stroke.
