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, dry age-related macular deneneration, and in two fatal neurodegenerative disorders:
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.
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.
Dry Age-Related Macular Degeneration (AMD)
In June, 2012 StemCells, Inc. initiated of a Phase I/II clinical trial of the Company's proprietary HuCNS-SC® product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD) referred to as Geographic Atrophy. There are no approved treatments for dry AMD. The trial is being conducted at the Retina Foundation of the Southwest (RFSW) in Dallas, Texas, one of the leading independent vision research centers in the United States. David G. Birch, Ph.D., Chief Scientific and Executive Officer of the RFSW and Director of the Rose-Silverthorne Retinal Degenerations Laboratory, is the principal investigator of the study. Learn more…
AMD Clinical Trial
Age-related macular degeneration refers to a loss of photoreceptors (rods and cones) from the macula, the central part of the retina. AMD is a degenerative retinal disease that typically strikes adults in their 50s or early 60s, and progresses painlessly, gradually destroying central vision., There are approximately 1.75 million Americans age 40 years and older with some form of age-related macular degeneration, and with more than seven million at risk of developing AMD, the disease continues to be the number one cause of irreversible vision loss among senior citizens in the US.
The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a potential treatment for the dry form of AMD. The trial will be an open-label, dose-escalation study, and is expected to enroll a total of 16 male or female patients 50 years and older. Patients will be enrolled based on specific inclusion/exclusion criteria. The investigation will be divided into two sequential cohorts. Patients will be enrolled into each cohort based on best-corrected visual acuity (BCVA) as determined by the Electronic Early Treatment Diabetic Retinopathy Study (E-ETDRS) acuity test. BCVA of less than or equal to 20/400 in both eyes will be required for enrollment in Cohort I. Patients with BCVA of 20/200 to 20/100 will be eligible for enrollment in Cohort II. An independent Data Monitoring Committee (DMC) will review accruing safety data for all patients.
The HuCNS-SC cells will be administered by a single injection into the space beneath the retina in the most affected eye. Oral immunosuppressive agents will be administered for a period of three months after surgery. Vision will be evaluated using both conventional and state-of-the-art ophthalmological assessments. Evaluations will be performed at routine intervals over a one-year period to assess safety and signs of visual benefit. Patients will then be followed for an additional four years in a separate observational study. Patients interested in participating in the clinical trial please submit our contact us form or contact the RFSW site directly at 214-363-3911.
Additional information regarding our clinical trial can be found at www.clinicaltrials.gov. and using its ClinicalTrials.gov identifier: NCT01632527
United States, Texas
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.