Clinical Development Programs
We are currently conducting or have completed clinical trials of our proprietary HuCNS-SC® product candidate (purified human neural stem cells) in spinal cord injury and in two fatal neurodegenerative disorders:
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Spinal Cord Injury
In December 2010, we received authorization from Swissmedic, the Swiss regulatory agency for therapeutic products, to initiate a Phase I/II clinical trial of our HuCNS-SC cells in chronic spinal cord injury. The trial initiated in March 2011 and is currently open for enrollment. The trial is being conducted in Switzerland at the Balgrist University Hospital, University of Zurich. Learn more…
Spinal Cord Injury Clinical Trial
The trial is designed to assess both safety and preliminary efficacy in patients with varying degrees of paralysis who are three to 12 months post-injury, and will progressively enroll patients based upon the severity of injury. The trial will enroll 12 patients in Europe with thoracic (chest-level) spinal cord injury, and will include both complete and incomplete injuries as classified by the American Spinal Injury Association (ASIA) Impairment Scale. The first cohort will include patients classified as ASIA A. These patients have what is considered to be a "complete" injury, or no movement or feeling below the level of the injury. The second cohort will progress to patients classified as ASIA B, or patients with some degree of feeling below the injury. The third cohort will consist of patients classified as ASIA C, or patients with some degree of movement below the injury. In addition to assessing safety, the trial will evaluate preliminary efficacy using defined clinical endpoints, such as changes in sensation, motor, and bowel/bladder function.
All patients will receive HuCNS-SC cells through direct transplantation into the spinal cord, and will be temporarily immunosuppressed. Following transplantation, the patients will be evaluated regularly over a 12-month period in order to monitor and evaluate the safety and tolerability of the HuCNS-SC cells, the surgery and the immunosuppression, and to measure any recovery of neurological function below the injury site. As we intend to follow the effects of this therapy long-term, a separate four-year observational study will be initiated at the conclusion of this trial.
For enrollment inquiries, please submit our contact us form.
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Pelizaeus-Merzbacher Disease (PMD)
In November 2009, we initiated a Phase I trial of our HuCNS-SC cells in PMD, a myelination disorder that primarily affects young children. The trial is being conducted at the University of California, San Francisco (UCSF) Benioff Children's Hospital. Learn more…
PMD Clinical Trial
In December 2008 the US Food and Drug Administration (FDA) allowed our Investigational New Drug (IND) application to conduct a clinical trial of our HuCNS-SC product candidate in patients with PMD, and in November 2009 we initiated the trial at UCSF Benioff Children’s Hospital. In February 2010 our cells were used to treat the first patient enrolled in the trial, marking the first time that neural stem cells have been transplanted as a potential treatment for a myelination disorder. In February 2011, the fourth and final patient was enrolled and dosed. Results of the trial will be reported in early 2012.
This Phase I trial is designed to assess the safety and preliminary efficacy of our HuCNS-SC cells as a potential treatment for PMD. While the primary focus in this first trial is safety, we will also be looking for evidence of new myelin formation in the patients’ brains following the transplantation of our cells, as well as any signs of improved neurological function. We have enrolled four patients with connatal PMD, the most severe form of the disease. All patients were transplanted with the HuCNS-SC cells and are being evaluated regularly over a 12-month period in order to monitor and evaluate the safety and tolerability of the HuCNS-SC cells, the surgery and the immunosuppression. In addition, MRI examination of the brain post-transplant may enable the measurement of new myelin formation. We are planning to follow the effects of this therapy long-term, so, as with our Phase I NCL trial, a separate, four-year observational study will be initiated at the conclusion of this trial.
For more information on our work in this area, download our Fact Sheet on our HuCNS-SC Therapeutic Development Program in PMD.
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Neuronal Ceroid Lipofuscinosis (NCL or Batten Disease)
In January 2009, we completed a Phase I clinical trial of our HuCNS-SC cells in NCL, a lysosomal storage disorder in children. The trial was conducted at Oregon Health & Science University (OHSU) Doernbecher Children’s Hospital, a leading medical center with nationally recognized programs in pediatric neurology and neurosurgery. Learn more…
NCL Clinical Trials
Phase I Trial – Completed January 2009
Our Phase I trial in NCL, initiated in 2006, was the first ever FDA-authorized clinical trial of human neural stem cells. This landmark trial primarily focused on assessing the safety of our HuCNS-SC product candidate as a potential treatment for NCL. Data from this study, reported in June 2009, demonstrated the clinical safety and tolerability of the cells, and included evidence of engraftment and long-term survival of the donor cells. We continue to follow patients who completed this trial, some of whom are now more than three years post-transplant.
Phase Ib Trial in NCL – Initiated October 2010 / Discontinued April 2011
Based on the favorable safety data from our first NCL trial, we initiated in October 2010 a second clinical trial at OHSU to further assess the safety and preliminary efficacy of HuCNS-SC cells as a potential treatment for NCL. This Phase Ib trial was designed to enroll patients with less advanced stages of the disease than those enrolled in our first NCL trial, with the goal of enhancing the prospect for clinical benefit.
In April 2011, we discontinued the Phase Ib trial study and shelved our NCL program due to a lack of eligible patients identified within a reasonable timeframe.
For more information on our work in this area, download our Fact Sheet on our HuCNS-SC Therapeutic Development Program in NCL.
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We believe that the human safety data we are accumulating for our HuCNS-SC cells through these clinical trials will facilitate the pathway for future clinical testing in other central nervous system (CNS) disorders, including retinal degenerative diseases such as age-related macular degeneration and retinitis pigmentosa.
Additional information about our clinical trials can be found at the US National Institutes of Health Clinical Trials website.
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