ReNeuron stem cell test helps mobility in stroke patients but misses endpoint

Do they have any clue if the stem cells survived? That should be an endpoint. Can't tell from this if spontaneous recovery and placebo could account for all of the gains.
http://www.fiercebiotech.com/biotech/reneuron-stem-cell-test-helps-mobility-stroke-patients-but-missed-endpoint
British biotech ReNeuron has posted generally encouraging midstage data which showed their stem cell therapy could help certain stroke patients move their arms better after being left disabled by their condition. Despite not hitting its primary endpoint, it is still plotting a pivotal test starting next year.
Its CTX cell therapy candidate was being tested in a small phase 2 trial, known as Pisces II, that worked as a single-arm, open-label study in patients living with disability resulting from ischemic stroke.
The study’s primary endpoint was relatively modest: It needed two patients out of 21 to reach a minimum two-point improvement in what is known as the grasping and lifting test, a part of the Action Research Arm Test (ARAT), at three months after treatment.
In the end, three of the 21 patients achieved this at three, six or 12 months respectively after treatment, and were within a group of four responders who also showed “clinically relevant improvements” on the total ARAT score of arm motor performance.
But it missed its target as some responses came later than the three-month target. The biotech however still said that the result was “nonetheless highly encouraging,” and the market tended to agree, with its shares jumping over 22% on the news today.
This was also because “strongly positive results” were also reported in the other endpoints of the study, with seven patients (33%) showing a “clinically relevant improvement” on the Modified Rankin Scale (a measure of disability and dependence) and eight patients (38%) showing a clinically relevant improvement on the Barthel Index (a measure of performance in activities of daily living).
In all, 15 out of 21 patients had a clinically significant response on at least one efficacy measure, with these improvements kept up throughout the follow-up period.
The company says that it will now seek a chat with the FDA and EMA about starting a randomized, placebo-controlled, pivotal trial in patients who are living with disability poststroke.
Olav Hellebø, CEO of ReNeuron, said: “We are delighted that the Pisces II clinical trial has shown our CTX cell therapy candidate has the potential to become a treatment option for patients living with chronic consequences following stroke. We are particularly excited by the response rate seen on the measures relating to disability and activities of daily living, given that these are the most important for patients and their carers.”
Hellebø noted that these measures are also the ones viewed by regulatory authorities as most relevant for late-stage clinical development.
He added that the co has a “strong balance sheet” to fund its planned pivotal study.
Stem cell therapies, once seen as a great hope in the research field for a variety of diseases, have in recent years been beset with testing setbacks, with progress in getting new therapies to market much slower than originally hoped for.
Back in the summer, Teva returned the full rights to a phase 3 experimental heart drug to Mesoblast as it walked away from development. And in the spring, CA-based BioCardia delayed its plans to raise $50 million for its stem cell research

ReNeuron: Stem-cell therapy helped stroke patients

Impossible to tell if this really works, not enough information is given to analyze. If this truly worked they would have enough detail for researchers to duplicate the results. Because they don't and are a profit making company I think this is puffery.
http://www.marketwatch.com/story/reneuron-stem-cell-therapy-helped-stroke-patients-2016-12-05
ReNeuron Group PLC (RENE.LN) said its stem-cell therapy aided the rehabilitation of patients who had been disabled by a stroke, adding to a small but growing body of evidence that such an approach holds promise.
U.K.-based ReNeuron said 15 patients in its 21-person study had a "clinically significant response" on at least one of the three commonly-used scales for assessing improvements following treatment.
Chief Executive Olav Hellebø said he was "particularly excited" that the treatment seemed to help patients perform daily tasks as this was the measure most prized by regulators in Europe and the U.S. The treatment, dubbed CTX, involves having stem cells injected into the brain near the site affected by the stroke.
The company said it would now apply to regulators in Europe and the U.S. to start a larger trial.

Stem-cell therapy, if proven successful, would constitute a rare breakthrough for stroke medicine. Blood-clot busting treatments given in the immediate aftermath of a stroke can minimize damage, but there are currently no other therapies available for stroke, according to Alessandra Granata, a researcher at the University of Cambridge.
Other groups are making progress. Earlier this year, a small clinical trials by researchers at Japanese biotech SanBio Co. suggested that stem-cell therapy had potential to aid stroke patients' rehabilitation. Another small study using SanBio's treatment, by researchers at Stanford University, had positive results.
Athersys Inc., a Cleveland, Ohio-based company using stem-cell therapy in stroke patients, plans to start enrolling patients into a phase-three clinical trial next year. Its therapy--given within 36 hours of a stroke--is aimed at preventing damage rather than rehabilitation.
-Write to Denise Rolande at denise.rolande@wsj.com

BBC Breakfast interviews patient in ReNeuron’s Phase II stroke clinical trial

The unasked question. 'What proof have your doctors given you that these stem cells caused your improvement?' Without that answer, this is all just placebo and magic. I could ascribe the results to the needle traveling thru the brain or the trepanation.
http://www.reneuron.com/news-list/bbc-breakfast-news-interview-stem-cells-used-in-stroke-treatment/

Without further proof this is all still quackery.

First stem cell trial for stroke shows lasting benefits

Will your doctor tell you about this before you tell them? Why doesn't your doctor know a damn thing?
http://www.newscientist.com/article/dn25541-first-stem-cell-trial-for-stroke-shows-lasting-benefits.html#.U21tc1dvB0o

People who received the world's first stem cell treatment for strokes have shown measurable reductions in disability and handicap a year after the injection into their damaged brains.

Some can move limbs and manage everyday tasks that were impossible before they received an injection of neural progenitor stem cells, which were clones of cells originally taken from the cortex of a donated fetus.

Apart from physical rehabilitation, there are few treatments for people left severely disabled by a stroke. Demand for more options is high, with 800,000 new cases each year in the US and 150,000 in the UK.

"We're encouraged, and it's a nice progressive piece of news," says Michael Hunt, the chief executive officer of ReNeuron, the company in Guildford, UK, that developed the treatment. "We must be circumspect, but we are seeing what seems to be a general trend towards improvement in a disparate group of patients," he says.

ReNeuron presented its latest results on the first 11 patients on 7 May in Nice, France, at the 23rd European Stroke Conference. They build on interim findings released last year.

More at link.

Encouraging data from stem cell trial in stroke patients as plans for Phase II progress

I am somewhat concerned that they talk about enrolling stroke patients, all of whom will have suffered a stroke within a few weeks. That means there is no way to split off spontaneous recovery from any boost provided by the stem cells. Bad science.
http://medicalxpress.com/news/2013-05-stem-cell-trial-patients-phase.html

Updated interim data from the PISCES trial, which has seen the brains of ischaemic stroke patients injected with neural stem cells to test the safety and tolerability of the treatment, was presented to the 22nd European Stroke Conference in London today. Professor Keith Muir of the University of Glasgow, who is heading the trial of ReNeuron Group plc's ReN001 stem cell therapy at the Southern General Hospital, Glasgow reported that data from the first nine patients treated has shown no cell-related or immunological adverse affects. He added that most patients had experienced sustained modest reductions in neurological impairment compared to their pre-treatment baseline performance, accompanied by improvement in abilities to undertake day to day tasks. A further two patients have been treated since the data were collated and the trial is now drawing to a close, with full results due to be published next year. Meanwhile, plans are proceeding for a Phase II trial which will examine the efficacy of stem cell treatment in stroke patients and an application is expected to be submitted to the UK regulatory authorities in early July. If approved the Phase II trial is scheduled to commence later this year. The Phase II trial will be a controlled multi-centre trial involving around 20 patients initially, all of whom will have suffered a stroke within a few weeks. Professor Muir said: "We remain pleased and encouraged by the data emerging from the PISCES study. The data to date identify no safety issues with the ReN001 treatment – which is the primary focus of this Phase I trial. "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." Michael Hunt, Chief Executive Officer of ReNeuron, said: "The PISCES study continues to yield encouraging results. Assuming the remaining required short-term follow up data confirm the good safety profile of the treatment, we will be able to move the ReN001 therapy confidently into Phase II clinical development, as planned, later this year." The Phase II study plan has been adopted by the NHS National Institute for Health Research Stroke Research Network (SRN). This important endorsement will enable ReNeuron to work closely with the SRN to optimise performance against defined targets regarding site set-up, patient recruitment and monitoring activities across the various sites participating in the study. ReNeuron will seek final regulatory and ethical approvals for the Phase II stroke study by submitting a data package including three month follow-up data on the final dose cohort in the PISCES study to the UK regulatory authorities in early July and, assuming approvals are granted, expects to commence recruitment into the Phase II study shortly thereafter. The PISCES study is the world's first fully-regulated clinical trial of a neural stem cell therapy for disabled stroke patients. Stroke is the third largest cause of death and the single largest cause of adult disability in the developed world. The trial is being conducted at the Institute of Neurological Sciences, Southern General Hospital, Greater Glasgow and Clyde NHS Board.

Read more at: http://medicalxpress.com/news/2013-05-stem-cell-trial-patients-phase.html#jCp

Updated interim data from the PISCES trial, which has seen the brains of ischaemic stroke patients injected with neural stem cells to test the safety and tolerability of the treatment, was presented to the 22nd European Stroke Conference in London today. Professor Keith Muir of the University of Glasgow, who is heading the trial of ReNeuron Group plc's ReN001 stem cell therapy at the Southern General Hospital, Glasgow reported that data from the first nine patients treated has shown no cell-related or immunological adverse affects. He added that most patients had experienced sustained modest reductions in neurological impairment compared to their pre-treatment baseline performance, accompanied by improvement in abilities to undertake day to day tasks. A further two patients have been treated since the data were collated and the trial is now drawing to a close, with full results due to be published next year. Meanwhile, plans are proceeding for a Phase II trial which will examine the efficacy of stem cell treatment in stroke patients and an application is expected to be submitted to the UK regulatory authorities in early July. If approved the Phase II trial is scheduled to commence later this year. The Phase II trial will be a controlled multi-centre trial involving around 20 patients initially, all of whom will have suffered a stroke within a few weeks. Professor Muir said: "We remain pleased and encouraged by the data emerging from the PISCES study. The data to date identify no safety issues with the ReN001 treatment – which is the primary focus of this Phase I trial. "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." Michael Hunt, Chief Executive Officer of ReNeuron, said: "The PISCES study continues to yield encouraging results. Assuming the remaining required short-term follow up data confirm the good safety profile of the treatment, we will be able to move the ReN001 therapy confidently into Phase II clinical development, as planned, later this year." The Phase II study plan has been adopted by the NHS National Institute for Health Research Stroke Research Network (SRN). This important endorsement will enable ReNeuron to work closely with the SRN to optimise performance against defined targets regarding site set-up, patient recruitment and monitoring activities across the various sites participating in the study. ReNeuron will seek final regulatory and ethical approvals for the Phase II stroke study by submitting a data package including three month follow-up data on the final dose cohort in the PISCES study to the UK regulatory authorities in early July and, assuming approvals are granted, expects to commence recruitment into the Phase II study shortly thereafter. The PISCES study is the world's first fully-regulated clinical trial of a neural stem cell therapy for disabled stroke patients. Stroke is the third largest cause of death and the single largest cause of adult disability in the developed world. The trial is being conducted at the Institute of Neurological Sciences, Southern General Hospital, Greater Glasgow and Clyde NHS Board.

Read more at: http://medicalxpress.com/news/2013-05-stem-cell-trial-patients-phase.html#jCp

Updated interim data from the PISCES trial, which has seen the brains of ischaemic stroke patients injected with neural stem cells to test the safety and tolerability of the treatment, was presented to the 22nd European Stroke Conference in London today. Professor Keith Muir of the University of Glasgow, who is heading the trial of ReNeuron Group plc's ReN001 stem cell therapy at the Southern General Hospital, Glasgow reported that data from the first nine patients treated has shown no cell-related or immunological adverse affects. He added that most patients had experienced sustained modest reductions in neurological impairment compared to their pre-treatment baseline performance, accompanied by improvement in abilities to undertake day to day tasks. A further two patients have been treated since the data were collated and the trial is now drawing to a close, with full results due to be published next year. Meanwhile, plans are proceeding for a Phase II trial which will examine the efficacy of stem cell treatment in stroke patients and an application is expected to be submitted to the UK regulatory authorities in early July. If approved the Phase II trial is scheduled to commence later this year. The Phase II trial will be a controlled multi-centre trial involving around 20 patients initially, all of whom will have suffered a stroke within a few weeks. Professor Muir said: "We remain pleased and encouraged by the data emerging from the PISCES study. The data to date identify no safety issues with the ReN001 treatment – which is the primary focus of this Phase I trial. "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." Michael Hunt, Chief Executive Officer of ReNeuron, said: "The PISCES study continues to yield encouraging results. Assuming the remaining required short-term follow up data confirm the good safety profile of the treatment, we will be able to move the ReN001 therapy confidently into Phase II clinical development, as planned, later this year." The Phase II study plan has been adopted by the NHS National Institute for Health Research Stroke Research Network (SRN). This important endorsement will enable ReNeuron to work closely with the SRN to optimise performance against defined targets regarding site set-up, patient recruitment and monitoring activities across the various sites participating in the study. ReNeuron will seek final regulatory and ethical approvals for the Phase II stroke study by submitting a data package including three month follow-up data on the final dose cohort in the PISCES study to the UK regulatory authorities in early July and, assuming approvals are granted, expects to commence recruitment into the Phase II study shortly thereafter. The PISCES study is the world's first fully-regulated clinical trial of a neural stem cell therapy for disabled stroke patients. Stroke is the third largest cause of death and the single largest cause of adult disability in the developed world. The trial is being conducted at the Institute of Neurological Sciences, Southern General Hospital, Greater Glasgow and Clyde NHS Board.

Stroke victims respond positively to stem cell treatment - UK

No self-medication here. Ask your doctor first.
http://zeenews.india.com/news/health/diseases/stroke-victims-respond-positively-to-stem-cell-treatment_17449.html
The first set of stroke patients who took part in pioneering stem cell treatment trials have shown signs of improvement, doctors have disclosed.

Six patients, who had human stem cells inserted close to the damaged part of their brain, have since witnessed improvements in the limb weakness that they suffered as a result of their stroke.

In one case, a man who underwent the treatment regained the power of speech after the stem cells of an aborted 12-week-old baby were injected into his brain.

However, doctors have cautioned against reading too much into the early results of the clinical trial, a world first for neural stem cell therapy for stroke victims.

The trial led by Glasgow University neurologist Professor Keith Muir, is being conducted at the Institute of Neurological Sciences at the Southern General Hospital in Glasgow.

“So far we`ve seen no evidence of any harmful effects. We`re dealing with a group of people a long time after a stroke with significant disability and we don`t really expect these patients to show any change over time,” the Telegraph quoted Muir as telling the BBC.

“So it`s interesting to see that in all the patients so far they have improved slightly over the course of their involvement in the study,” he said.

The six patients had suffered strokes between six months and five years before they were treated, and all had been left with limb weakness.

The patients were assessed using the National Institutes of Health Stroke Scale, which ranked the first five patients with a median score of eight before the treatment and four points three months afterwards.

The sixth patient was treated less than three months ago. Six further patients will be treated as part of this Phase 1 trial.

Professor Muir said that he was “intrigued” by the early results of the research.

“We know that if you`re involved in a trial you are going to see patients change in behaviour, particularly if you`re doing something invasive, so we need to be very cautious indeed in interpreting these results,” he said.

“However, that said, it is not something we`d anticipated seeing in this group of patients,” he said.

Further trials are needed to establish whether stem cells actually help the brain repair damaged tissue.

Michael Hunt, chief executive officer of the company developing the treatment, ReNeuron, said that the clinical trial should be considered with caution at this stage.

“The clinical trial is primarily a safety study and we must therefore treat any of the observed early indications of functional benefit with considerable caution at this stage,” he said.

“That said, we remain encouraged by the results seen in the study to date and we look forward to providing further updates,” he added.