Therapeutic Programs

StemCells, Inc. is the first and only company to complete a US clinical trial using human neural stem cells, tackling central nervous system (CNS) disorders with our HuCNS-SC® product candidate.

Our liver program is also among the first of its kind, targeting the treatment of liver disease with our proprietary hLEC human liver engrafting cells.

Learn more about Our Approach…

Broad Pipeline

  • Research
  • Preclinical
  • Clinical Development
CNS (HuCNS-SC)
Spinal Cord Injury 
 
PMD
 
Retinal Disorders (AMD)
 
Alzheimers
 
Stroke
 

Current estimates indicate that degenerative conditions of the central nervous system (CNS) and the liver affect more than 35 million people in the United States1 alone. Many of these conditions lack effective treatment options. We believe that stem cell-based therapy holds the potential to treat, and in some cases possibly cure, a wide range of such diseases that continue to defy conventional medicine.

StemCells therapeutic product development programs include:

CNS Program

The StemCells CNS Program is focused on development of our lead product candidate, HuCNS-SC human neural stem cells, which we believe will have broad therapeutic application for many diseases and disorders of the CNS. Our industry-leading program addresses all three major components of the CNS: the brain, the spinal cord and the eye.

StemCells HuCNS-SC product candidate is currently in clinical development for spinal cord injury and for Pelizaeus-Merzbacher Disease (PMD), a fatal myelination disorder in children.  We are also in preclinical development with our human neural stem cells for retinal disorders such as age-related macular degeneration (AMD), and plan to file an IND for a Phase I/II clinical trial in AMD by year-end 2011.  Other research and preclinical studies are underway in Alzheimer's disease and stroke.  Previous clinical development activities include the completion of a Phase I trial in neuronal ceroid lipofuscinosis (NCL), also often referred to as Batten disease, a fatal neurodegenerative disorder that primarily affects young children.


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  1. Based on information from the Alzheimer’s Association, the Alzheimer’s Disease Education & Referral Center (National Institute on Aging), the National Parkinson Foundation, the National Institutes of Health’s National Institute on Neurological Disorders and Stroke, the Foundation for Spinal Cord Injury Prevention, Care & Cure, the Travis Roy Foundation, the Centers for Disease Control and Prevention, the Wisconsin Chapter of the Huntington’s Disease Society of America, the American Liver Foundation, and the Cincinnati Children’s Hospital Medical Center.

The Therapeutic Potential of Stem Cells

Because of the self-renewal property of stem cells, a single transplantation treatment may have the potential to return an impaired organ to proper function for the life of the patient.

Learn more…

  • CNS Program achievements and clinical development plans for HuCNS-SC® cells

    Martin McGlynn
    (Run Time: 00:03:03)

  • Evolution of stem cell research and its impact on the CNS field

    Irving Weissman, MD
    (Run Time: 00:01:23)

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