ReNeuron confirmed that the first patient has been treated in what it claims is the world’s first fully regulated clinical trial to evaluate neural stem cell therapy in stroke patients. The Pisces (pilot investigation of stem cells in stroke) study is evaluating ReNeuron’s lead ReN001 cell therapy in patients who have been disabled by ischemic stroke. The firm says the first individual to receive the implanted cells was discharged from the Institute of Neurological Sciences at the Southern General Hospital in Glasgow two days after the "straightforward neurosurgical procedure" was carried out to implant the cells. His progress will be reviewed in December by the trial’s data safety monitoring board, before the remainder of the first cohort of patients are treated.
The Pisces trial will evaluate the safety of ReN001 at a range of cell doses, although measures of efficacy will also be collected. Patients will be monitored for two years initially. “The initiation of the Pisces clinical trial is a major and hard-won milestone for ReNeuron and a significant milestone in the development of therapies to address the severely disabling effects of ischemic stroke,” comments Michael Hunt, ReNeuron CEO.
The company was granted U.K. Gene Therapy Advisory Committee (GTAC) approval to start the ReN001 trial back in February. GTAC acts as the national research ethics committee for gene therapy and stem cell therapy clinical trials in the U.K. The committee’s clearance represented the final national regulatory step in taking the stem cell therapy into patients in the U.K. The Medicines and Healthcare Products Regulatory Agency (MHRA) had already approved the study in 2009.
ReNeuron is dedicated to the development of potentially off-the-shelf stem cell therapies for a range of diseases. A preclinical-stage stem cell therapy, ReN009, is in development for the treatment of peripheral arterial disease. Stem cell-based treatments for other conditions, including blindness-causing diseases of the retina, are also in the pipeline. The resulting products utilize either ReNeuron’s CTX cortical cell line, which has already been scaled up to cGMP manufacturing standards, or cell lines generated specifically from the relevant region of the brain or body.
The firm says its stem cell therapy pipeline hinges on its stem cell expansion technology that enables the growth of selected human stem cells into banks of quality-assured stem cell lines, starting from a single tissue sample. This capability is allowing the development of nonpatient-specific, allogeneic stem cell treatments capable of addressing diseases with large patient populations, ReNeuron claims. The expansion platform also allows the scale-up of stem cell lines for clinical and commercial use without the need to rederive cell lines from early prototypes. The firm says this gives it a significant competitive advantage in terms of the time and expense in moving a potential stem cell therapy through a clinical development program.
ReNeuron is separately commercializing its ReNcell® cell lines for nontherapeutic research applications. ReNcell®VM is a neural cell line derived from the ventral mesencephalon region of the brain, and ReNcell®CX is derived from the cerebral cortex. Both cell lines have been licensed exclusively to Millipore for manufacture and worldwide distribution through their research reagent catalog.
The start of first-in-man testing with ReNeuron’s ReN001 therapy follows on from FDA approval in July, for Geron to start what it claimed at the time would be the world’s first human trial with a human embryonic stem cell (hESC)-based therapy, GRNOPC1. This Phase I trial is designed to evaluate the hESC-derived oligodendrocyte progenitor cell therapy in patients with complete American Spinal Injury Association Impairment Scale grade A subacute thoracic spinal cord injuries.