ReN001 for stroke

A stroke occurs when blood flow leading to, or in, the brain is blocked (ischaemic stroke) or a blood vessel in the brain ruptures (haemorrhagic stroke), which can result in damage to the nerve cells in the brain and a loss of bodily functions. Stroke is the single largest cause of adult disability in the developed world.Over 150,000 people suffer a stroke each year in the UK, and over 700,000 people in the US. Approximately 80% of these strokes are ischaemic in nature. Our ReN001 stem cell therapy seeks initially to treat those patients who have suffered an ischaemic stroke and have been left disabled by it. These patients constitute approximately one half of stroke survivors.

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. The type of stroke treatment a patient should receive depends on the stage of disease. Generally there are three treatment stages of stroke:

Prevention - treatments to prevent a first or recurrent stroke are based on treating associated risk factors, e.g. high cholesterol, smoking and diabetes;

Treatment immediately after the stroke; acute-phase stroke treatments attempt to arrest a stroke whilst it is happening by dissolving the blood clot that has caused the infarct; and

Post stroke rehabilitation - the aim of post stroke rehabilitation is to improve both functional and cognitive recovery in the patient some weeks or months after the stroke event.

It is this third treatment stage that our ReN001 stem cell therapy will address in initial clinical trials. A number of treatments exist or are in development to treat stroke patients in the acute phase. However, there are currently no therapies available for patients who have a stable and fixed neurological deficit following a stroke. Our ReN001 cell therapy for stroke consists of a neural cell line, designated CTX, which has been generated using our proprietary cell expansion and cell selection technologies and then taken through a full manufacturing scale-up and quality-testing process. As such, ReN001 is a standardised, clinical and commercial-grade cell therapy product capable of treating all eligible patients presenting.

ReN001 has been shown to reverse the functional deficits associated with stroke disability when administered several weeks after the stroke event in relevant pre-clinical models. Extensive pre-clinical testing also indicates that the therapy is safe, with the ReN001 cells eventually cleared from the body with no adverse safety effects arising.

We have received regulatory approval to commence a Phase I clinical trial in the UK with ReN001. Patient recruitment for this trial will commence shortly.

The aim of the initial Phase I clinical trial with ReN001 is to evaluate the safety of the implantation technique and to establish the side effect profile associated with the implantation of ReN001 stem cells in patients who have suffered an ischaemic stroke.Additionally, we aim to collect preliminary efficacy data by evaluating any improvement reported in stroke-specific assessments that will be made regarding neurological function on an on-going basis throughout the study.

The actual surgical technique that will be used in the ReN001 clinical trials known as stereotactic injection, is a well-established and relatively straightforward procedure in neurosurgery. With the aid of a stereotactic co-ordinate frame in place around the patient's head, the neurosurgeon will use a special cannula to implant the ReN001 cells directly into the target brain region through a single, small craniostomy burr hole in the skull. The implantation procedure takes between one and two hours, depending on the cell dose administered. The patient will remain in the hospital overnight and will normally be discharged the morning after surgery.

Patients in the Phase I trial will be followed up over a two year period to evaluate the safety of the ReN001 therapy and any potential improvement in neurological function. These clinical assessments will involve scoring against several stroke-specific scales as well as a battery of other tests designed to evaluate both motor and cognitive function over time. Ongoing monitoring of the patients will also continue in the longer term following the two year end-point.

Data sources: UK Stroke Association ; American Stroke Association.

Top of page