The first patient has now been treated in a Phase I clinical trial of ReN001, the UK-based ReNeuron Group’s stem cell therapy for stroke.
The positive news on the PISCES (Pilot Investigation of Stem Cells in Stroke) study, which ReNeuron described as the world’s first fully regulated clinical trial of a neural stem cell therapy for disabled stroke patients, pushed up the company’s share price to close at 7.85 pence.
ReNeuron was also the first company to secure regulatory approval for any stem cell-based clinical trial in the UK. The Medicines and Healthcare products Regulatory Agency cleared the PISCES study in January 2009 but additional preclinical data were requested by the Gene Therapy Advisory Committee (GTAC), which acts as the national research ethics committee for clinical trials with gene therapies and stem cell therapies in the UK.
The GTAC gave its final approval in February, at which point ReNeuron said it expected to start patient recruitment and evaluation for the PISCES first-in-human trial in April 2010.
The first patient was treated at the Institute of Neurological Sciences at Southern General Hospital in Glasgow, Scotland. The patient was safely discharged following the “straightforward neurosurgical procedure” used to administer the ReN001 cells, ReNeuron reported.
Led by Professor Keith Muir, SINAPSE Professor of Clinical Imaging in the Division of Clinical Neurosciences at the University of Glasgow, the PISCES study is designed primarily to test the safety of ReN001 in ischaemic stroke patients at a range of doses –although a number of efficacy measures will also be evaluated over the course of the trial.
The 12 patients taking part in the trial, who will be given ReN001 therapy between six and 24 months after their stroke, will be monitored for two years, with longer-term follow-up procedures in place after that.
Assuming a satisfactory independent Data Safety Monitoring Board review of the first patient’s progress in December, the remainder of the first-dose cohort in the PISCES trial will be treated shortly afterwards, ReNeuron noted.
The company is hoping to pursue an accelerated clinical development pathway with ReN001, focusing on particular groups of stroke patients who are expected to benefit most from the therapy.
Michael Hunt, chief executive officer of ReNeuron, said the start of the PISCES trial was “a major and hard-won milestone for ReNeuron and a significant milestone in the development of therapies to address the severely disabling effects of ischaemic stroke”. The BioIndustry Association hailed a “ground-breaking achievement” that “highlights the UK‘s world-leading expertise in stem cell research”.
Geron Corporation launched a Phase I clinical trial of GRNOPC1, its treatment for acute spinal cord injuries based on human embryonic stem cells, in the US last month. ReN001 consists of a neural stem cell line, derived from non-embryonic human tissue and developed using ReNeuron’s proprietary cell expansion and cell selection technologies.
The UK company has also provided an update on progress with ReN009, its stem cell therapy for peripheral arterial disease (PAD). Data from preclinical efficacy testing of ReN009 at the Bristol Heart Institute in the UK are being presented at the American Heart Association Scientific Sessions 2010 in Chicago, US.
In this study, ReNeuron’s lead CTX stem cell line was evaluated in a recognised diabetic mouse model of hind limb ischaemia. The treated diabetic mice “exhibited a significant and dose-dependent recovery of blood flow to the ischaemic limb, with significantly increased re-vascularisation of the damaged tissue”, the company said.
ReNeuron is developing ReN009 as a non-patient specific stem cell treatment for late-stage PAD, or critical limb ischaemia, in diabetic patients where PAD is a side-effect of their diabetes. A multicentre first-in-human clinical trial is planned for early 2012.