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Engineered stem cell advance points toward treatment for ALS

Public release date: 28-May-2013 [ | E-mail | Share ]

Contact: Masatoshi Suzuki msuzuki@vetmed.wisc.edu University of Wisconsin-Madison

MADISON, Wis. Transplantation of human stem cells in an experiment conducted at the University of Wisconsin-Madison improved survival and muscle function in rats used to model ALS, a nerve disease that destroys nerve control of muscles, causing death by respiratory failure.

ALS (amyotrophic lateral sclerosis) is sometimes called "Lou Gehrig's disease." According to the ALS Association, the condition strikes about 5,600 Americans each year. Only about half of patients are alive three years after diagnosis.

In work recently completed at the UW School of Veterinary Medicine, Masatoshi Suzuki, an assistant professor of comparative biosciences, and his colleagues used adult stem cells from human bone marrow and genetically engineered the cells to produce compounds called growth factors that can support damaged nerve cells.

The researchers then implanted the cells directly into the muscles of rats that were genetically modified to have symptoms and nerve damage resembling ALS.

In people, the motor neurons that trigger contraction of leg muscles are up to three feet long. These nerve cells are often the first to suffer damage in ALS, but it's unclear where the deterioration begins. Many scientists have focused on the closer end of the neuron, at the spinal cord, but Suzuki observes that the distant end, where the nerve touches and activates the muscle, is often damaged early in the disease.

The connection between the neuron and the muscle, called the neuro-muscular junction, is where Suzuki focuses his attention. "This is one of our primary differences," Suzuki says. "We know that the neuro-muscular junction is a site of early deterioration, and we suspected that it might be the villain in causing the nerve cell to die. It might not be an innocent victim of damage that starts elsewhere."

Previously, Suzuki found that injecting glial cell line-derived neurotropic factor (GDNF) at the junction helped the neurons survive. The new study, published in the journal Molecular Therapy on May 28, expands the research to show a similar effect from a second compound, called vascular endothelial growth factor.

In the study, Suzuki found that using stem cells to deliver vascular endothelial growth factor alone improved survival and delayed the onset of disease and the decline in muscle function. That result mirrored his earlier study with GDNF.

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Engineered stem cell advance points toward treatment for ALS

Stem Cell Trial Leads To 'Mild To Moderate' Improvement In Stroke Patients

May 28, 2013

redOrbit Staff & Wire Reports Your Universe Online

More than half of the seriously disabled stroke victims participating in preliminary clinical trials of a new stem cell procedure have shown modest signs of recovery, the scientists carrying out the research reported Monday.

According to Sam Marsden of The Telegraph, five out of the nine ischemic stroke patients taking part in the PISCES trial were able to regain some movement in their hands and legs after undergoing the procedure, in which stem cells were injected directly into the damaged parts of their brains.

The patients showed mild to moderate improvement in their conditions after receiving injections of the ReNeuron Groups ReN001 stem cell therapy, Marsden explained. Some of the patients were able to move their fingers again after several years of complete paralysis, while others found that they could walk around their houses by themselves without assistance, he added.

Glasgow University professor Keith Muir, who is treating the patients, told BBC News Science Correspondent Pallab Ghosh that he was surprised by the amount of recovery in the patients. However, he also emphasized that it was too soon to determine whether or not the stem cell treatment is responsible for their partial recovery.

The results, which were presented by Muir at the European Stroke Conference in London on Monday, demonstrate that there have been no adverse side effects observed in the patients to this point. The beneficial effects, though, could be attributed to the close medical attention being given to the patients and not the treatment itself, Ghosh said.

It is well documented that the feeling of wellbeing resulting from such attention, known as the placebo effect, can have a positive effect on peoples health, the BBC News reporter said. But it is thought that stroke patients do not recover after the first six months of their stroke. All the patients involved in the trial had their strokes between six months and five years before they received the treatment.

The PISCES trial is nearing its end, the university said, and full results will be published sometime in 2014. In the meantime, plans are underway for a Phase II trial designed to investigate the efficacy of stem cell treatment in stroke patients.

The Phase II trial would be a controlled multi-facility trial involving approximately 20 patients, all of whom would have suffered a stroke just a few weeks before starting treatment. An application is expected to be submitted to UK regulatory agencies in early July, and if approved, the trial would begin later this year.

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Stem Cell Trial Leads To 'Mild To Moderate' Improvement In Stroke Patients

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Brodie Before After Stemlogix Stem Cell Therapy
Brodie a 3 year old rottweiler had a complete cranial cruciate ligament tear of his left rear leg. Dr. Stephanie Meyer at the Creatures Great Small Vet Hos...

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Stroke victims show signs of recovery following pioneering stem cell therapy

Five stroke victims have shown small signs of recovery following pioneering stem cell therapy.

Prof Keith Muir, of Glasgow University, said the results were "not what we would have expected" from the group of patients who had previously shown no indications of their conditions improving.

The trial involved injecting stem cells directly into the damaged parts of the patients' brains, with the hope that they would turn into healthy tissue or "kick-start" the body's own repair processes.

Frank Marsh, 80, one of the nine patients taking part in the trial at Glasgow's Southern General Hospital, told the BBC he had seen improvements in the use of his left hand.

"I can grip certain things that I never gripped before, like the hand rail at the baths, with my left hand as well as my right," he said."It still feels fairly weak and it's still a wee bit difficult to co-ordinate but it's much better than it was."He added: "I'd like to get back to playing my piano."

His wife Claire said: "He had reached a plateau and wasn't really improving (after his stroke). But following the operation he is able to do things he couldn't do before, such as make coffee, dressing, and holding on to things."

The study involved patients who suffered strokes some time ago and had shown no signs of making any further spontaneous improvement.

Prof Muir said the results were "at the present time not what we would have expected in this group but far from being able to say whether it's something specifically related to the cells".

He told BBC Radio 4's Today programme: "We know that some of the cells will survive and potentially turn into relevant tissue. We also suspect that a large part of what we do is kick-starting repair processes that are already present in the body.

"So there's probably a mixture of things going on. Quite what it is that's happening in the patients, we won't know for some time to come."

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Stroke victims show signs of recovery following pioneering stem cell therapy

UK & World News: Stem cell therapy 'shows results'

May 27 2013

Five stroke victims have shown small signs of recovery following pioneering stem cell therapy.

Prof Keith Muir, of Glasgow University, said the results were "not what we would have expected" from the group of patients who had previously shown no indications of their conditions improving.

The trial involved injecting stem cells directly into the damaged parts of the patients' brains, with the hope that they would turn into healthy tissue or "kick-start" the body's own repair processes.

Frank Marsh, 80, one of the nine patients taking part in the trial at Glasgow's Southern General Hospital, told the BBC he had seen improvements in the use of his left hand.

"I can grip certain things that I never gripped before, like the hand rail at the baths, with my left hand as well as my right," he said."It still feels fairly weak and it's still a wee bit difficult to co-ordinate, but it's much better than it was." He added: "I'd like to get back to playing my piano."

His wife Claire said: "He had reached a plateau and wasn't really improving (after his stroke). But following the operation he is able to do things he couldn't do before, such as make coffee, dressing and holding on to things."

The study involved patients who suffered strokes some time ago and had shown no signs of making any further spontaneous improvement.

Prof Muir said the results were "at the present time not what we would have expected in this group but far from being able to say whether it's something specifically related to the cells".

He told BBC Radio 4's Today programme: "We know that some of the cells will survive and potentially turn into relevant tissue. We also suspect that a large part of what we do is kick-starting repair processes that are already present in the body. So there's probably a mixture of things going on. Quite what it is that's happening in the patients, we won't know for some time to come."

Original post:
UK & World News: Stem cell therapy 'shows results'

Stem cell therapy 'shows results'

Five stroke victims have shown small signs of recovery following pioneering stem cell therapy.

Prof Keith Muir, of Glasgow University, said the results were "not what we would have expected" from the group of patients who had previously shown no indications of their conditions improving.

The trial involved injecting stem cells directly into the damaged parts of the patients' brains, with the hope that they would turn into healthy tissue or "kick-start" the body's own repair processes.

Frank Marsh, 80, one of the nine patients taking part in the trial at Glasgow's Southern General Hospital, told the BBC he had seen improvements in the use of his left hand.

"I can grip certain things that I never gripped before, like the hand rail at the baths, with my left hand as well as my right," he said."It still feels fairly weak and it's still a wee bit difficult to co-ordinate, but it's much better than it was." He added: "I'd like to get back to playing my piano."

His wife Claire said: "He had reached a plateau and wasn't really improving (after his stroke). But following the operation he is able to do things he couldn't do before, such as make coffee, dressing and holding on to things."

The study involved patients who suffered strokes some time ago and had shown no signs of making any further spontaneous improvement.

Prof Muir said the results were "at the present time not what we would have expected in this group but far from being able to say whether it's something specifically related to the cells".

He told BBC Radio 4's Today programme: "We know that some of the cells will survive and potentially turn into relevant tissue. We also suspect that a large part of what we do is kick-starting repair processes that are already present in the body. So there's probably a mixture of things going on. Quite what it is that's happening in the patients, we won't know for some time to come."

The stem cells were created 10 years ago from one sample of nerve tissue taken from a foetus.

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Stem cell therapy 'shows results'

Non-white patients suffer from lack of marrow, stem cell donors

There are concerns a dramatic shortage of stem cell and bone marrow donors from non-Caucasian ethnic groups is putting lives in jeopardy.

And experts are trying to fix the problem by tackling the biggest issue lack of awareness.

For Angela Yee Hamshaw, news of her sister's leukemia diagnosis was devastating.

Doctors could not find a bone marrow match in the family for 37-year-old Audrey Popowich. So as here chemotherapy began, so too did the search for a donor.

But because of her Chinese heritage, Popowich was warned that finding a match could prove extremely difficult.

"It can be very difficult. Within specific ethnic groups we do struggle to find donors, said Cassandra DeLuca, a coordinator with Canadian Blood Services.

About 75 per cent of the 330,000 registered Canadian donors are Caucasian. Chinese Canadians make up about six per cent, while South Asian donors account for less than three per cent of the supply.

"It comes from general awareness. Within certain communities there is more awareness than others, DeLuca said.

The huge gap is frustrating, said Dr. Mona Shafey, who works at the Alberta Bone Marrow Transplant program.

They're facing life and death here, she said.

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Non-white patients suffer from lack of marrow, stem cell donors

Stroke patients show signs of recovery in stem cell treatment trial

The studies so far have not been able to rule out completely the possibility that the patients benefited from the placebo effect of receiving close medical attention.

However, Prof Muir told the BBC: "It seems odd that it should all just be chance and a placebo effect. We are seeing things that are interesting and somewhat surprising.

He added: "My expectation had been that we would see very little change and if we did see change it would be a relatively short-lived temporary change. [But] we have seen changes that have been maintained over time.

The patients in the study, which took place at Glasgow's Southern General Hospital, were aged in their 60s, 70s and 80s, and all had suffered their stroke between six months and five years earlier.

Frank Marsh, 80, a retired teacher, was once a keen piano player, but a stroke five years ago left him with poor strength and co-ordination in his left hand.

Describing the improvement he experienced after receiving the stem cell treatment, he told the BBC: "I can grip certain things that I never gripped before, like the hand rail at the baths, with my left hand as well as my right.

"It still feels fairly weak and it's still a wee bit difficult to co-ordinate but it's much better than it was."

He added: "I'd like to get back to my piano. I'd like to walk a bit steadier and further."

The small-scale study, whose results will be presented to the European Stroke Conference in London tomorrow, is the worlds first clinical trial examining the safety of neural stem cell treatment in stroke patients.

The first phase of the research was carried out to check that the procedure is safe. Now Prof Muir and his team are planning a larger phase two trial later this year to explore further how effective the treatment is.

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Stroke patients show signs of recovery in stem cell treatment trial

Stroke stem-cell trial shows promise

27 May 2013 Last updated at 00:01 ET By Pallab Ghosh Science correspondent, BBC News

Five seriously disabled stroke patients have shown small signs of recovery following the injection of stem cells into their brain.

Prof Keith Muir, of Glasgow University, who is treating them, says he is "surprised" by the mild to moderate improvements in the five patients.

He stresses it is too soon to tell whether the effect is due to the treatment they are receiving.

The results will be presented at the European Stroke Conference in London.

BBC News has had the first exclusive interview with one of the patients involved.

They are taking part in a small clinical trial involving nine patients in their 60s, 70s and 80s at Glasgow's Southern General Hospital to assess the safety of the procedure which involves injecting stem cells into the damaged brain part.

It is one of the first trials in the world to test the use of stem cells in patients.

Results to be presented on Tuesday show that there have been no adverse effects on the patients so far and there have been improvements to more than half participating in the trial.

However, at this stage it is not possible to say whether the improvements are due to the close medical attention the patients are receiving. It is well documented that the feeling of wellbeing resulting from such attention, known as the placebo effect, can have a positive effect on people's health.

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Stroke stem-cell trial shows promise