Stem cells have set the scientific world agog because it has been proposed as candidates to treat a myriad of diseases ranging from alzheimer's to arthritis, blindness, burns, cancer, diabetes, heart disease, liver disorders, multiple sclerosis, parkinson's, spinal cord injury and stroke.
Engaged in the development of novel stem cell therapeutics targeting diseases of the central nervous system and liver is clinical-stage company StemCells Inc. (STEM).
For readers who are new to this Palo Alto, California-based company, here's what to expect in the coming months...
StemCells' lead product candidate is HuCNS-SC cells, a highly purified composition of human neural stem cells, currently in clinical development for spinal cord injury and for Pelizaeus-Merzbacher Disease, or PMD, a fatal myelination disorder in children.
A phase I/II clinical trial of HuCNS-SC cells in chronic spinal cord injury was initiated by the company last March. The trial, which is the world's first neural stem cell trial in spinal cord injury, is designed to enroll patients with thoracic (chest-level) neurological injuries with progressively decreasing severity of injury in three sequential cohorts.
The first patient in the trial was successfully transplanted with the company's proprietary HuCNS-SC adult neural stem cells last September, and enrollment in the first cohort of the spinal cord injury trial was completed last December. Following transplantation, the patients 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 trial, which is currently open for enrollment for the remaining cohorts, is being conducted in Switzerland at the Balgrist University Hospital, University of Zurich.
In November 2011, Geron Corp. (GERN), the first company to get FDA approval for a clinical trial of an embryonic stem cell-based therapy, abandoned its phase I stem cell trial in patients paralyzed by spinal cord injuries - largely because of financial reasons.
The difference between the spinal cord injury trials of StemCells and Geron lies in the type of stem cells being evaluated. While Geron used human embryonic stem cells to treat spinal cord injuries in its trial, StemCells is using tissue-derived "adult" (non-embryonic) stem cells in its trials.
Yet another trial of StemCells that is underway is a phase I trial evaluating the safety and preliminary efficacy of HuCNS-SC cells as a treatment for Pelizaeus-Merzbacher Disease that primarily affects infants and young children.
Pelizaeus-Merzbacher Disease, or PMD, is a myelination disorder in which there is insufficient myelination of nerve cells in the brain, leading to loss of neurological function and eventually death in the most severe forms of the disease.
Some of the clinical features of PMD are nystagmus (involuntary oscillatory movements of the eyes), stridor (noisy breathing), hypotonia (lack of muscle tone; floppiness) or spasticity (a type of increased muscle tone or stiffness of the muscles and joints).
The company completed patient accrual in the phase I clinical trial in PMD in February 2011. According to the interim data of the PMD trial, announced last November, the company remains cautiously optimistic that changes in patient MRIs are consistent with early development of new myelin in the regions in which the cells were transplanted.
If things go as planned, the company expects to report final study results in the next quarter.(2Q).
Last month, the company received FDA authorization to initiate a phase I/II clinical trial of HuCNS-SC in dry age-related macular degeneration, the most common form of AMD.
The trial is expected to enroll a total of 16 patients and they will be administered HuCNS-SC cells as a single injection into the space beneath the retina. The patients' vision will be evaluated using conventional methods of ophthalmological assessment at predetermined intervals over a one-year period. Patients will then be followed for an additional four years in a separate observational study.
The phase I/II clinical trial in dry AMD is expected to be initiated in the near future. In addition, the company is pursuing preclinical studies of its HuCNS-SC cells in Alzheimer's disease.
StemCells is also developing hLEC, a purified population of human liver engrafting cells isolated from primary human tissue, as a potential therapy for liver disease. The human liver engrafting cells are under preclinical studies.
A quick look at the company's balance sheet...
StemCells derives revenue by licensing certain of its patented cells and sales of cell culture products for use in research. The company markets stem cell research products, including media and reagents, under the SC Proven brand. As of September 30, 2011, the company had an accumulated deficit of $325 million.
In the third quarter of 2011, the company narrowed its net loss to $4.33 million or $0.31 per share from $5.55 million or $0.44 per share in the year-ago comparable quarter.
Total revenue, which included product sales and license revenue, during the third quarter of 2011 declined to $224,000 from $254,000 in the same period of 2010.
Revenue from product sales in the third quarter of 2011 rose 40% to $182,000, driven by both increased unit volumes and new product launches in the company's SC Proven media and reagents business. However, revenue from licensing agreements and grants dropped 67 percent to $41,000, due to the completion and termination of several projects funded by grants.
Shares of StemCells have gained nearly 24% year-to-date. STEM has thus far hit a 52-week low of $0.70 and a 52-week high of $9.78. The stock underwent a 1-for-10 reverse stock split on July 6, 2011. STEM closed Tuesday's trading at $1.02, down 1.92%.
For comments and feedback: editorial@rttnews.com