A UK company has received the go-ahead to start the world鈥檚 first trial using stem cells to repair brain damage in stroke victims.
, based in Guildford, hopes to start the trials within the next six months on 12 patients. Although six children with a rare, inherited brain condition called Batten鈥檚 disease were treated with stem cells in 2006, the newly approved trial will be the first to treat a common killer.
鈥淲e鈥檙e absolutely delighted,鈥� says John Sinden, the company鈥檚 chief scientific officer. 鈥淚t鈥檚 a milestone internationally, and sets the road map for what you have to do to get a stem-cell therapy into the clinic.鈥�
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Stroke victims will receive injections of neural stem cells through holes drilled into the skull to allow access to the worst-affected regions of their brains with a fine syringe.
鈥淯ltimately, we hope these cells will be able to differentiate into brain tissue, neurons and supporting tissue, and will re-institute connections lost through the stroke,鈥� says Keith Muir, a neurologist at the Southern General Hospital in Glasgow, where he is principle investigator for the trial.
鈥淚t may be that the cells themselves, or something they produce, will help to trigger repairs.鈥�
Close monitoring
Such effects have been seen in experiments on mice, enabling animals to recover memory and cognitive function. 鈥淲e鈥檙e hoping to do the same for people,鈥� says Muir.
Muir admits that it will be virtually impossible to track what happens to the cells, as they have no radioactive or fluorescent labels that can distinguish them from native cells in brain scans. Nor will it be easy to track physical changes in the brain, although patients will have regular scans to see if brain activity and function alters after the treatment.
To make it easier to spot any clinical improvements, Muir and his colleagues will select 鈥渟table鈥� patients who have had no signs of improvement, and no prospect of improvement, six months after their stroke.
They will also monitor the patients closely to check that there are no serious side effects, such as tumours forming from the stem cells. 鈥淔rom brain-imaging studies, we will be looking for anything that might go amiss,鈥� says Muir. This monitoring will continue indefinitely to guard against unforeseen effects.
US hopes
Approval for the trial was given on 19 January by the .
ReNeuron hopes the UK approval will help it win over the US Food and Drug Administration so that a trial can begin in the US. 鈥淲e submitted our application two years ago, and we don鈥檛 want it to be on hold forever, so we鈥檙e informally discussing with them how to take it forward,鈥� says Sinden.
The trial in children with Batten鈥檚 disease is due to finish this month, but so far there have been from StemCells, the company in Palo Alto, California, that is conducting the work.
The company is also hoping to use its stem cells to treat Huntington鈥檚 disease, and recently won FDA approval to conduct a trial in another . Infants with Pelizaeus-Merzbacher disease die young because their nerve and brain cells are poorly coated in myelin 鈥� a protective protein.
Both companies took embryonic stem cells and 鈥渞eprogrammed鈥� them into neural stem cells. These stem cells can grow into all types of brain and nerve cells. 鈥淲e received our tissue in 2003, and now have enough material to treat one million patients,鈥� says Sinden.
