The encouraging results of the Phase I PISCES trial have featured prominently in the media in past two days, but results of this stroke trial aren't the only interesting and exciting stem cell discoveries to have been reported in the neurological field in the past week. Here's my pick of three of them:
ReNeuron, a UK-based clinical-stage stem cell company, announced some encouraging results from their Pilot Investigation of Stem Cells in Stroke (PISCES) study. The clinical trial, which ReNeuron say is the world's first fully regulated clinical trial of a neural stem cell therapy for disabled stroke patients, tests ReNeuron's ReN001 stem cell therapy in 12 patients left disabled by an ischaemic stroke. While the aim of the Phase I study is to evaluate the safety of the implantation technique and establish the side effect profile of the therapy, the PharmaTimes reports that data from the first 9 patients shows most of the patients experienced sustained modest reductions in neurological impairment and an improved ability to undertake routine tasks.
However, as the BBC reports, it might still be too soon to tell whether the effect is due to the treatment they are receiving or whether it's due to the closer medical attention the patients are receiving. "The evidence of functional improvement requires further investigation in a suitably designed Phase II efficacy study and we look forward to being a principal clinical site in that study when it commences," said Professor Keith Muir, of Glasgow University, who is leading the trial. “It seems odd that it should all just be chance and a placebo effect,” he told BBC News. “We are seeing things that are interesting and somewhat surprising. We’ve seen people who now have the ability to move their fingers where they have had several years of complete paralysis. We have seen some people that have been able to walk around their house whereas previously they had been dependent on assistance and we have had improvements that have enabled people to recognise what is happening around them.”
Researchers at the Waisman Center, University of Wisconsin-Madison, transformed skin cells taken from individuals with Down syndrome into induced pluripotent stem cells, and then manipulated these cells to grow into neurons. They then studied the connections that formed among the neurons, and found that Down syndrome neurons had only about 60% of the usual number of synapses and synaptic activity. “They communicate less, are quieter," said Anita Bhattacharyya, a neuroscientist working on the study. "This is new, but it fits with what little we know about the Down syndrome brain.”
Another difference that the researchers noted was that the output of genes that respond to oxidative stress. “We definitely found a high level of oxidative stress in the Down syndrome neurons,” says Bhattacharyya. “This has been suggested before from other studies, but we were pleased to find more evidence for that. We now have a system we can manipulate to study the effects of oxidative stress and possibly prevent them.” Bhattacharyya suggests that the high levels of oxidative stress may contribute to some of the symptoms of Down syndrome, including an increased susceptibility to Alzheimer's. The research will be published in the week of May 27 2013 in the Proceedings of the National Academy of Sciences. Potentially, the cells could be used to test or design drugs to treat the symptoms of Down syndrome.
A team of researchers at the University of California, San Diego School of Medicine, reported that rats transplanted with a single injection of spinal cord-derived human neural stem cells three days after spinal cord injury showed improvement in both motor and sensory deficits. Neuralstem reported that the cells used in the study were their NSI-566 cells, which have also been used in rat models of ALS and human Phase I trials in ALS. The research was published online in the May 28 2013 issue of Stem Cell Research & Therapy. “The primary benefits were improvement in the positioning and control of paws during walking tests and suppression of muscle spasticity,” said Martin Marsala, MD. “In all cell-grafted animals, there was robust engraftment, and neuronal maturation of grafted human neurons was noted.” According to Marsala, the grafted stem cells appeared to both stimulate host neuron regeneration and partially replace the function of lost neurons.
Richard Garr, Director & CEO of Neuralstem recently gave a presentation on ‘Understanding the Practicalities of Using Innovative Therapies on Patients’ at the World Stem Cells & Regenerative Medicine Congress 2013. You can watch his fascinating presentation in full here:
What do you think? What other stem cell stories have excited you this week? Why not join our discussion on LinkedIn, or leave a comment below. Want more from Total BioPharma? Sign up to our newsletter – it doesn't cost anything and only takes a minute.
Did you miss World Stem Cells & Regenerative Medicine Congress 2013? Total BioPharma invited several guest bloggers to the event, and you can read some reports of the conference here: