The first patient in the landmark Pilot Investigation of Stem Cells in Stroke (PISCES) trial has been treated with neural stem cells. The study is the first fully regulated clinical trial of neural stem cell therapy for stroke. Investigators will evaluate the safety of ReNeuron cells in disabled ischemic stroke patients.
“This is a first in man safety study,” lead investigator Keith Muir, MD, from the University of Glasgow‘s Institute of Neuroscience and Psychology in Scotland, explained to Medscape Medical News. “We are looking at neurological effects — clinical and imaging — only as secondary goals.”
The stem cells are from a genetically modified immortalized cell line derived from a tissue sample from 12-week fetal cortex.
“Critics of stem cell development generally have various reasons for their ethical objections,” Dr. Muir said. “For most, it relates to embryo use — not relevant in this case,” he argues. “Others might object to termination of pregnancy, but these cells are derived from material donated to a tissue bank from a single legal termination many years ago, which was done for social reasons and was unrelated to the subsequent use.”
This study was approved by the United Kingdom Medicines and Healthcare Products Regulatory Agency. ReNeuron is the first company to receive regulatory approval for any stem cell–based clinical trial in the United Kingdom.
The first patient in this pilot investigation was treated in Glasgow. The male patient was injected with cells to the affected region of the brain and has since been discharged from the hospital. He will be monitored closely for 2 years with longer-term follow-up procedures afterward.
Investigators are still recruiting for the trial and plan for 12 patients to take part. They will be given the ReN001 therapy between 6 and 24 months after stroke.
PISCES is an open-label study with no control group. “We feel this is an appropriate design for a first in man study,” Dr. Muir said, “and we are not seeking to draw conclusions about efficacy at this stage.”
Trial investigators suggest that because of the nature of the procedure and the characteristics of the stem cells, immunosuppression will not be necessary for patients taking part in the study. This, they say, will eliminate the safety risks typically associated with immunosuppression regimens.
Michael Hunt, chief executive officer of ReNeuron, said in a news release, “Our thanks and best wishes go to the first patient and his family for their participation in this important and groundbreaking clinical trial.”