STEM provided an update on its ongoing Phase I clinical trial in Pelizeaus-Merzbacher Disease (NASDAQ:PMD), a fatal myelination disorder that afflicts male children.
2 of 4 planned patients for this trial have now been enrolled and transplanted with HuCNS-SC® human neural stem cells with a 3rd patient before year-end and the 4th shortly thereafter.
- This clinical trial is the 1st to evaluate purified neural stem cells as a potential treatment for a myelination disorder. The trial is being conducted at UCSF Benioff Children’s Hospital.
The Phase I trial is designed to assess the safety and preliminary effectiveness of HuCNS-SC cells as a potential treatment for PMD.
- The trial as stated enrolls 4 patients with connatal PMD;
- All patients will be transplanted with HuCNS-SC cells, and will be immunosuppressed for 9 months;
- 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;
- In addition, magnetic resonance imaging (MRI) of the brain post-transplant may enable the measurement of new myelin formation;
- STEM intends to follow the effects of this therapy long-term with a separate 4 year observational study initiated at the conclusion of this trial.
Patients with PMD are born with a defective gene, which leads to insufficient myelin in the brain.
- Myelin is the substance that surrounds and insulates nerve cells’ communications fibers (also known as axons);
- Without sufficient myelination, these fibers are unable to properly transmit nerve impulses, leading to a progressive loss of neurological function;
- Those with the most severe form of the disease, connatal PMD, lose the ability to walk and talk and eventually die, often before the age of 10;
- Currently, there are no effective treatments for PMD.