ReNeuron Files Application with FDA to Begin Phase I Study of Stem Cell Treatment for Stroke Patients.

HD Lighthouse Contributing Editor's Comment: ReNeuron, a UK based company, has filed an application with the FDA to conduct a Phase I (safety) study of stem cell treatment for stroke patients. This is exciting news for the Huntington's Disease community since ReNeuron has also developed a cell line for Huntington's Disease and the clinical research with stroke patients will shorten the learning curve.

ReNeuron has published its stroke research in peer reviewed academic journals and it looks very promising but I cannot find any journal articles about its stem cell line for Huntington's Disease. The company reports success in animal models of Huntington's Disease but no details are provided. From information on their website, the cell line was produced from fetal stem cells in the striatum and it has a chemical 'safety switch' which turns off cell division.

ReNeuron's graphic representation of its patented stem cell technology is used by permission.

-- Marsha L. Miller, Ph.D.
Posted to the HDL: 09 Dec 2006

First such application to use neural (brain) stem cells to treat a major neurological condition

Guildford, UK, 6 December 2006:
ReNeuron Group plc (LSE: RENE.L) today announces the filing of its first Investigational New Drug (IND) application with the US Food and Drug Administration (FDA) to commence initial clinical studies in the US with ReN001, its stem cell therapy for stroke.

This filing is a key milestone in ReNeuron's history and, the Company believes, the world's first such application concerning a neural stem cell treatment for a major neurological disorder. Stroke is the single largest cause of adult disability in the developed world. Over 130,000 people suffer a stroke each year in the UK, and over 700,000 people in the US.

ReNeuron's ReN001 stem cell therapy seeks to treat those patients who have suffered a stroke and have been left disabled by it. These patients constitute approximately one third of the total stroke patient population. There are currently no approved treatments available to address the causes of their disability. Existing treatments for stroke in the chronic phase are primarily focused on the prevention of recurrent strokes. The annual health and social costs of caring for disabled stroke patients is estimated to be in excess of £5 billion in the UK, with stroke patients occupying 25 per cent of long term hospital beds. In the US, the annual direct and indirect costs of stroke are estimated to be in excess of US$50 billion.

Following approval of the IND, the initial clinical study will be conducted under the direction of Professor Douglas Kondziolka MD, MSc, FRCS, FACS at the University of Pittsburgh Medical Center. The initial Phase I study with ReN001 will be an open label study, with two dose levels, in disabled stroke patients. The primary objective of the study will be to monitor the safety profile of the treatment. Preliminary efficacy measures will also be recorded, however, to provide an indication of therapeutic potential ahead of further efficacy studies in a larger cohort of patients once the safety of the ReN001 therapy has been confirmed.

Michael Hunt, Chief Executive Officer of ReNeuron, said, "Today's announcement is a significant milestone for ReNeuron and for the wider stem cell field. We have been greatly encouraged by the pre-clinical data generated in support of the IND application, and we now look forward to taking ReN001 into its clinical phase. The IND filing represents the first step towards determining the safety and efficacy of our ReN001 therapy in man, in order to ultimately bring this treatment to the many patients who could benefit from it. Importantly, approval of this IND will also provide validation for our proprietary c-mycERTAM cell expansion technology, thereby adding impetus to our other therapeutic programmes to which this technology has been applied."

Professor Kondziolka at the University of Pittsburgh Medical Center, commented, "Our team has participated in the design of ReNeuron's first clinical study and we are excited about the potential this research offers in the field of brain repair. This study seeks to address the immense problem of disability following stroke, for which there are few treatment options."

ReNeuron is a leading, UK-based adult stem cell therapy business. It is applying its novel stem cell platform technologies in the development of ground-breaking stem cell therapies to serve significant and unmet or poorly-met clinical needs.

ReNeuron has used its c-mycERTAM technology to generate genetically stable neural stem cell lines. This technology platform has multi-national patent protection and is fully regulated by means of a chemically-induced safety switch. Cell growth can therefore be completely arrested prior to in vivo implantation. ReNeuron has filed for approval to commence initial clinical studies in the US with its lead ReN001 stem cell therapy for chronic stroke disability. This represents the world's first such filing concerning a neural stem cell treatment for a major neurological disorder. There are an estimated 50 million stroke survivors worldwide, approximately one half of which are left with permanent disabilities.

ReNeuron has also generated pre-clinical efficacy data with its ReN005 stem cell therapy for Huntington's disease, a genetic and fatal neurodegenerative disorder that affects around 1 in 10,000 people. This programme is in pre-clinical development.

In addition to its stroke and Huntington's disease programmes, ReNeuron is developing stem cell therapies for Parkinson's disease, Type 1 diabetes and diseases of the retina.

ReNeuron has also leveraged its stem cell technologies into non-therapeutic areas – its ReNcell range of cell lines for use in drug discovery applications in the pharmaceutical industry.

ReNeuron's shares are traded on the London AIM market under the symbol RENE.L, and its warrants are traded under the symbol RENW.L.

Further information on ReNeuron and its products can be found at www.reneuron.com.

Source: ReNeuron

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