Purified human neural stem cells (HuCNS-SC) are being trialled to see if they can help treat neurodegenerative disorders that primarily afflict children.
A Phase I clinical trial for patients with Pelizaeus-Merzbacher Disease (PMD) is being conducted at the University of California, San Francisco (UCSF) Children's Hospital.
PMD results from a defective gene that results in a lack of myelin, a substance that surrounds and insulates nerve cells' communications fibres. These fibres function much like electrical wires, and without sufficient myelination are unable to properly transmit nerve impulses, leading to the loss of neurological function and eventually death in the most severe forms of PMD. Currently, there are no effective treatment options for patients with PMD.
HuCNS-SCs are highly purified human neural stem cells that are expanded and stored as banks of cells. Preclinical research has shown that when directly transplanted into the central nervous system, they behave like normal stem cells, suggesting they may continually replenish normal human neural cells.
StemCells, the company that developed HuCNS-SCs, is investigating a cell therapy approach to assess whether HuCNS-SCs can produce the myelin needed for nerve cells to communicate.
The company hopes that their discoveries will lead to a viable treatment option for children with PMD, and possibly also for patients suffering from other myelination disorders, such as multiple sclerosis, transverse myelitis and certain types of cerebral palsy.
The Phase I trial will assess the safety and preliminary effectiveness of HuCNS-SC cells as a treatment for PMD. It will involve four patients with connatal PMD, which is the most severe form of the disease. All patients will be transplanted with HuCNS-SC cells, and will be immunosuppressed for nine months.
Following transplantation, the patients will be evaluated regularly over a 12-month period to monitor and evaluate the safety and tolerability of the HuCNS-SC cells, the surgery, and the immunosuppression. In addition, magnetic resonance imaging (MRI) of the brain post-transplant may enable the measurement of new myelin formation.
After the first phase of the trial, an observational study lasting 4 years will be initiated to study the long-term effects of this therapy.