UK-based stem cell company ReNeuron has been given the thumbs up by UK regulators to start clinical trials using stem cell therapy.
The first-of-its-kind trial using expanded neural stem cells will be used as a treatment for patients who have been disabled by an ischaemic stroke.
The Medicines and Healthcare products Regulatory Agency approval for the trial, using ReNeuron’s ReN001 stem cell therapy, has been given subject to provision of data both from an ongoing pre-clinical study and from the long-term follow-up of trial participants.
“This regulatory approval marks the first step in the process of testing the safety and potency of our lead ReN001 stroke therapy at a clinical level,” said Michael Hunt, chief executive of ReNeuron.
“It is the most important milestone in ReNeuron’s history thus far and also represents a significant development in the wider field as regards the translation of exciting stem cell science into clinical stage therapies. In many ways, ReNeuron has set the regulatory pathway in the UK for cell therapy trials of this type, and we are delighted to have been given the opportunity to move ReN001 into its clinical phase on home territory in the UK.”
The initial trial will test the safety profile of ReN001 in ischaemic stroke patients at a range of cell doses, but a number of efficacy measures will also be evaluated over the course of the trial. Investigators hope, that if successful the trial will lead to larger studies looking at the effects of the treatment on patient recovery.
ReN001 has been shown to reverse the functional deficits associated with stroke disability when administered several weeks after the stroke event in pre-clinical models. Other pre-clinical testing has indicated the therapy is safe, with ReN001 cells eventually cleared from the body with no adverse safety effects.
In the Phase I trial, 12 patients will receive the therapy between six and 24 months after their stroke. The ReN001 cells will be administered by direct injection into the affected region of the brain.
The procedure and characteristics of the stem cells mean patients will not require immunosuppression following treatment, thereby eliminating the safety risks associated with immunosuppression regimens. Patients in the trial will be monitored for one year, with longer-term follow-up procedures after that.
The therapy consists of a neural stem cell line, designated CTX, which has been generated using the company’s proprietary cell expansion and cell selection technologies. Patient recruitment is expected to start in the second quarter of this year.
Unsurprisingly shares in ReNeuron soared on the news, rising five pence to 7.88p.
