Regenerative Medicine Takes Off: En Route to Reality?
November 24, 2010
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A month or so after Geron Corp. initiated the world’s first embryonic stem cell-based clinical trial, UK-based ReNeuron has announced that it has treated its first stroke patient with expanded populations of neural stem cells at the Institute of Neurological Sciences, Southern General Hospital, in Glasgow. The PISCES (Pilot Investigation of Stem Cells in Stroke) study is the first stem cell-based clinical trial to be given the go-ahead by the UK government. ReNeuron’s phase 1 study of ReN001 will involve transplanting neural stem cells into the brains of patients that have been left disabled by stroke. Assessing the safety of the implantation technique will be top priority in the study, however ReNeuron hopes to collect at least some preliminary efficacy data. Patients will be watched closely for two years following surgery, after which a longer-term monitoring process will begin. Contingent on a positive safety review of the first patient in December, a larger cohort of patients will be treated shortly thereafter. Keith Muir, the principal investigator overlooking the trial said:
“We are pleased that the first patient in the PISCES trial has undergone surgery successfully. Stroke is a common and serious condition that leaves a large number of people with significant disability. In this trial, we are seeking to establish the safety and feasibility of stem cell implantation, which will require careful follow-up of the patients who take part. We hope that in future it will lead on to larger studies to determine the effects of stem cells on the disabilities that result from stroke.”
There are three typical treatment modalities for stroke: prevention, acute-phase stroke treatment, and post-stroke rehabilitation. It is the final of these that ReNeuron aims to provide with ReN001 treatment. Contrary to Geron’s oligodendrocyte progenitors being used to treat spinal cord injury, ReNeuron’s ReN001 therapeutic cells are generated from adult sources. Neural stem cells are selected from human tissue samples and expanded in vitro under cGMP manufacturing standards. ReNeuron has also developed quality control measures to select cells that have the proper phenotype, the ability to expand rapidly, and a propensity to engraft well with little immune rejection.
On our side of the ocean California-based StemCells Inc. has filed for Swiss regulatory approval to begin testing its neural stem cells in patients with non-acute spinal cord injury. This contrasts Geron’s approach in its ongoing study which is directed at acute-phase injury. A comparison of these clinical approaches down the line should yield useful information as to when stem cell-based biological intervention is most helpful to patients with spinal cord injury. Massachusetts-based Advanced Cell Technology (ACT) has also announced FDA approval of the world’s second embryonic stem cell-based clinical trial in patients suffering from Stargardt’s Macular Degeneration. The disease, characterized by degeneration of the retinal pigment epithelium, manifests itself early on in life. Twelve patients are expected to participate in the study where retinal pigment epithelial (RPE) cells derived from embryonic stem cells will be transplanted into the eye. The FDA has granted ACT’s RPE cells orphan status. As a result, the technology will be eligible for accelerated FDA approval and ACT may receive grant money from the government to fund clinical trials.