April 17, 2017 by Hannah L. Robbins SMN protein (red) is necessary for the survival of spinal cord neurons (motor neurons) responsible for breathing and all movement. Harvard researchers have found a compound that stabilized this protein in mouse and human motor neurons. This may lead to the development of new treatments for motor neuron diseases including Spinal Muscular Atrophy and Lou Gehrigs disease. Credit: Natalia Rodriguez-Muela

Harvard Stem Cell Institute (HSCI) researchers have identified a compound that helps protect the cells destroyed by spinal muscular atrophy (SMA), the most frequent fatal genetic disease in children under 2 years of age.

SMA is a neurodegenerative disease targeting motor neurons, the long nerve cells that relay messages from the brain to the muscles and that are, consequently, responsible for bodily movements, including walking, swallowing, and even breathing. Patients with milder forms of SMA experience muscle wasting that may confine them to a wheelchair, while the more severe forms cause paralysis and death before the second birthday.

About one in 50 people are genetic carriers of the disease.

Because of a dysfunctional gene, many motor neurons in SMA patients are unable to produce adequate amounts of a protein called survival of motor neuron (SMN). The deficiency causes cellular stress and eventually cell death. Rather than fixing the gene, which has been the strategy of many labs looking to develop SMA therapies, the Harvard team has identified a compound that helps stabilize the SMN protein both in human neurons in a dish and in mouse models.

The findings were published in the journal Cell Reports.

"This discovery opens up new lines of drug interrogation," said Lee Rubin, HSCI principal faculty member and the senior author on the study. Rubin's lab, which operates out of in Harvard's Department of Stem Cell and Regenerative Biology, uses induced pluripotent stem cells (iPS cells) to make human models of neurological diseases.

In 2015, Rubin made a variety of neuronal types from the iPS cells of SMA patients in order to determine why motor neurons in particular were targeted, and found they experienced a fatal stress response similar to motor neurons affected by amyotrophic lateral sclerosis (ALS), the late-onset neurodegenerative disease more commonly known as Lou Gehrig's disease.

Additionally, some SMA-affected motor neurons were dying before others, though all of the neurons had the same genetic mutations and were experiencing the same stressful environment.

"Clearly, some motor neurons were surviving, so the next question was whether this is random or if there is a molecular explanation," Rubin said.

Early on in their most recent study, the researchers found that within a single petri dish of motor neurons derived from an SMA patient, some produced up to four times as much SMN protein as their neighbors. Over time, those motor neurons with higher levels of SMN were more likely to survive after exposure to toxic environments and stressors.

When the team analyzed motor neurons derived from ALS patients, they found similar results: Motor neurons with higher levels of SMN were likelier to survive than those with lower levels.

"The surprise was when we looked in a control culture and also saw differences between the individual neurons," Rubin said.

"It is clear that the SMN protein is necessary for all motor neuron survival, not just motor neurons targeted by ALS or SMA," said Natalia Rodrguez-Muela, a postdoctoral fellow in Rubin's lab and co-first author on the paper. The results suggest that if the team could increase the amount of SMN protein in any single motor neuron, they would be able to rescue the cell.

During a cell's life span, proteins are constantly being made and degraded, made and degraded again. To interrupt the process of breaking down the SMN protein, the researchers looked at a family of proteins called Cullins, which act as a part of the cell machinery that regulates protein degradation.

In 2011, the Rubin lab had determined that an enzyme called GSK3b helps control SMN stability. Nearly all proteins degraded by GSK3b are flagged for degradation by a pathway that involves a specific member of the Cullin family. Rubin said the researchers hypothesized that if they could block that Cullin-mediated process, the SMN proteins would not be flagged for degradation and would remain stable longer.

The researchers, led by co-first author Nadia Litterman, then dosed human and murine motor neurons with a compound known to block the specific Cullin and found that exposure to the compound made SMN proteins more stable and more abundant. As a consequence, the compound promoted survival of all motor neurons, both in human cells in the dish and in mouse models.

Additionally, mice with SMA, even the more severe forms of the disease, had some of their symptoms improve after exposure to the compound.

"This process points to an unexplored therapeutic direction that could benefit patients of not one, but two separate diseases," Rubin said.

Explore further: Hope against disease targeting children

More information: Natalia Rodriguez-Muela et al. Single-Cell Analysis of SMN Reveals Its Broader Role in Neuromuscular Disease, Cell Reports (2017). DOI: 10.1016/j.celrep.2017.01.035

Journal reference: Cell Reports

Provided by: Harvard University

This story is published courtesy of the Harvard Gazette, Harvard University's official newspaper. For additional university news, visit Harvard.edu.

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Scientists find evidence that ALS and SMA could be treated with a ... - Medical Xpress

Spinal Cord Stem Cells Comments Off on Scientists find evidence that ALS and SMA could be treated with a … – Medical Xpress | April 17th, 2017

A quick glance at your calendar will reveal that we're now in 2017. 2017, you may recall, is the year when contraversial surgeon Sergio Canavero has promised to perform the world's first human head transplant.

But just how feasible is a human head transplant? Is it the stuff of science fiction, or does it have a basis in current sceintific thinking? Read on for everything you need to know about 2017 most alarming scientific development.

A human head transplant is exactly what it sounds like taking one living head and putting it onto a new body.

But actually, thats a little misleading. In real terms, its a body transplant, as the head will be gaining a new body to control. However, as the term whole body transplant is already used to mean transferring the brain between bodies, calling it a head transplant makes it clear that the whole head is to be switched, brain included.

Until recently, a head transplant seemed totally implausible, but the Italian scientist Dr Sergio Canavero believes its possible, and intends to conduct the first surgery in 2017.

Canavero outlines the procedure in detail here, but these are the basics of the process. Remember: dont try this at home, kids.

The donor body and the head to be attached are first cooled down to 12-15C to ensure that the cells last longer than a few minutes without oxygen. The tissue around the neck is then cut, with the major blood vessels linked with tiny tubes. The spinal cord on each party is then severed cleanly with an extremely sharp blade.

"Post coma, Canavero believes the patient would immediately be able to move, feel their face and even speak with the same voice."

At this point, the head is ready to be moved, and the two ends of the spinal cord are fused using a chemical called polyethylene glycol, encouraging the cells to mesh. This chemical has been shown to prompt the growth of spinal cord nerves in animals, although Canavero suggests that introducing stem cells or olfactory ensheathing cells into the spinal cord could also be tried.

After the muscles and blood supply are successfully connected, the patient is kept in a coma for a month to limit movement of the newly fused neck, while electrodes stimulate the spinal cord to strengthen its new connections.

Following the coma, Canavero anticipates that the patient would immediately be able to move, feel their face and even speak with the same voice. He believes physiotherapy would allow the patient to walk within a year.

He explains his suggested methods in the TED talk below.

Sceptical would be a nice way of putting it. Horrified would, in most cases, be more accurate.

Dr Hunt Batjer has attracted headlines for being particularly blunt: I would not wish this on anyone. I would not allow anyone to do it to me as there are a lot of things worse than death.

Dr Jerry Silver witnessed the 1970s monkey head transplant experiment more on which later and describes the procedure as bad science, adding that just to do the experiments is unethical. This is a particular blow to Canavero, as he states that Silvers own work in reconnecting rats spinal cords should give hope to the human head transplant. Silver dismisses this: To sever a head and even contemplate the possibility of gluing axons back properly across the lesion to their neighbours is pure and utter fantasy in my opinion.

Dr Chad Gordon, professor of plastic and reconstructive surgery and neurological surgery at Johns Hopkins University, agrees that Canaveros claims are scientifically implausible. He told BuzzFeed: Theres no way hes going to hook up somebodys brain to someones spinal cord and have them be functional.

On the conservative side, were about 100 years away from being able to figure this out, he continued. If hes saying two, and hes promising a living, breathing, talking, moving human being? Hes lying.

Dr Paul Myers, associate professor of biology at the University of Minnesota at Morris, puts it even more explicitly: This procedure will not work... Try it with monkeys first. But he cant: the result would be, at best, a shambling horror, an animal driven mad with pain and terror, crippled and whimpering, and a poor advertisement for his experiment. And most likely what hed have is a collection of corpses that suffered briefly before expiring.

Others wonder whether Canavero might simply be enjoying the limelight with a PR stunt, including Dr Arthur Caplan, director of ethics at the NYU Langone Medical Centre. Describing the doctor as nuts, he explained to CNN: Their bodies would end up being overwhelmed with different pathways and chemistry than theyre used to, and theyd go crazy.

"We'll probably see a head on a robot before we see it on [another] body," he told Live Science.

Dr John Adler of Stanford University's school of medicine is slightly more optimistic... but not much more. "Conceptually, much of this could work, but the most favourable outcome will be little more than a Christopher Reeve level of function," he told Newsweek.

Canavero is aware of this criticism, claiming that silently hes received a lot of support from the medical community. Of Dr Batjers comments that the surgery would be a fate worse than death, Canavero is scathing. Hes a vascular surgeon. A vascular surgeon of the brain, yes, but he knows nothing, he argued. How can you say such a thing? Its incredible.

"The world is moving, the critics are dwindling. Of course, there will always be critics. Science teaches us that when you propose something groundbreaking, you must be confronted by criticism. If no critics really step forward, you are saying nothing special," he told Medical News Today.

No-one has ever attempted a human head transplant before, and attempts on animals have to put it charitably had limited success.

Image: from Motherboard, uploaded under fair use from a 1959 issue of Life

The photo above really does show a dog with two heads and its not a fake. This was the work of Soviet scientist Vladimir Demikhov, and for four days the hybrid of two dogs lived as normally as such a scientific horror could be expected to. Then they died.

Demikhov tried the experiment more than 24 times, but was unable to find a way of avoiding the dogs dying shortly after surgery. Although the results are horrifying to see, Demikhovs research did pave the way for human organ transplants.

"For four days this hybrid of two dogs lived as normally as such a scientific horror could be expected to. Then they died."

But back to the topic of head transplants. The first time a straight swap was successful, was by Dr Robert White, in an experiment on a rhesus monkey in 1970. I feel the need to qualify the word successful with quotation marks, because although the monkey did live, he didnt live very long. Eight days, to be exact, and as the spinal cord wasnt attached to its new body, the monkey was paralysed for its remaining days. However, it could indeed see, hear, smell and taste before the body rejected the foreign head.

According to Canavero in his paper on human head transplants, the monkey lived eight days and was, by all measures, normal, having suffered no complications. However, Dr Jerry Silver who worked in the same lab as Dr White has more haunting memories. He toldCBS: I remember that the head would wake up, the facial expressions looked like terrible pain and confusion and anxiety in the animal. The head will stay alive, but not very long. It was just awful. I dont think it should ever be done again.

More recently, Chinese doctor Xiaoping Ren claims to have conducted head transplants on more than 1,000 mice. The Wall Street Journal reports to have witnessed a mouse with a new head moving, breathing, looking around and drinking. But, crucially, none of these mice have lived longer than a few minutes.

Still, Dr Rens studies continue, and the latest reports are said to be promising, offering a possible answer to the risk of severe blood loss (or brain ischemia) during transplantation. The experimental method that we have described can allow for long-term survival, and thus assessment of transplant rejection and central nervous system recovery, bringing us one step closer to AHBR in man, the researchers wrote.

Ren himself has not ruled out taking part in the first human head transplant operation, according to the Daily Mail. "A human head transplant will be a new frontier in science. Some people say it is the last frontier in medicine. It is a very sensitive and very controversial subject but if we can translate it to clinical practice, we can save a lot of lives," he said.

"Many people say a head transplant is not ethical. But what is the essence of a person? A person is the brain not the body. The body is just an organ," he added.

In January 2016, Canavero told New Scientist that a head transplant had been successfully completed on a monkey in China, although details were sparse. "The monkey fully survived the procedure without any neurological injury of whatever kind," he said, although the article notes that the monkey only kept alive for 20 hours after the surgery for "ethical reasons," limiting its use as a comparison somewhat.

In September 2016, Canavero revealeda further trial of the head transplant on dogs.New Scientisthas seen video footage of a dog appearing to walk three weeks after its spinal cord was severed, with Canavero claiming that the outcome is the result of the same techniques he plans to use on Spiridonov next year.

However, speaking to a number of scientists for their view on the new evidence, New Scientistcould find few sceptics converted. "These papers do not support moving forward in humans," said Jerry Silver a neuroscientist at Cape Western Reserve University in Ohio.

"The dog is a case report, and you cant learn very much from a single animal without controls. They claim they cut the cervical cord 90 per cent but theres no evidence of that in the paper, just some crude pictures," added Silver.

You could say so, though Canavero doesn't see it quite like that. In fact, controversially he sees it more as a failure of other types of medicine, telling Medical News Today, "It will be about curing incurable neurological disorders for which other treatments have failed big time, so gene therapy,stem cells- they all just came to nothing. We have failed despite billions of dollars being poured into this sort of research."

"So actually, head transplant or body transplant, whatever your angle is, is actually a failure of medicine. It is not a brilliant success, a brilliant advancement to medical science. When you just haven't tackled biology, you don't know how to treat genes, you don't really understand, and you really need to resort to a body transplant, it means that you've failed. So this must not be construed as a success of medical research," he added.

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Human head transplant: Sergio Canavero considers the UK as the venue for 2017's most talked about operation - Alphr

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A desperate mum-of-two says she is losing her memory so fast she may soon be unable to recognise her young daughters.

Chantelle Fox was diagnosed with multiple sclerosis (MS) last May after suffering fatigue and "a little numbness" in her arm.

Since the devastating diagnosis, the 41-year-old's condition has quickly deteriorated, leaving her fearful for the future.

She has 79 lesions on her brain and her short-term memory is fading, meaning she often forgets where she has put things.

She also forgets promising her "beautiful" daughters, Lilly, five, and Edie, three, that she will take them somewhere special.

Her worst fear is that in just four years, she may not even remember the little girls at all.

Now, she faces a race against time to fund radical stem cell treatment in Russia - which she believes could halt the progress of her disease.

MS is a chronic condition, for which there is currently no cure.

The disease is caused by the immune system malfunctioning and mistakenly attacking nerve cells in the brain and spinal cord.

It can lead to patients suffering from a range of mild or severe symptoms.

In a bid to stop the progress of the "monster" condition, Chantelle, from Australia, plans to undergo surgery in Moscow.

MS Australia has deemed the treatment risky, while one neurologist told the mum there is no evidence it works and it could be dangerous.

However, another neurologist reportedly told her she would be a great candidate for a haematopoietic stem cell transplant (HSCT) trial.

But Chantelle, who lives in Melbourne, said waiting a long time for the chance to take part in a clinical trial in her home country wasn't an option.

Instead, she plans to travel abroad in June to undergo surgery.

I have two beautiful kids and I might not remember them in four years time if I dont go to Russia," she told the Herald Sun .

She added: I have to fight for my kids. I want to help them study, to see them married, to be a grandparent."

Chantelle has been accepted into an autologous haematopoietic stem cell transplant programme in Moscow.

She claimed the treatment has an "86 per cent success rate" in halting the progress of the neurological condition.

However, her family said the costs involved are "crippling".

They are trying to raise $150,000 (120,000) to cover the price of the treatment and transport to and from Russia.

Chantelle's sister, Maxine Parker, has set up dedicated GoFundMe and Facebook pages to help raise money for the surgery.

On the GoFundMe page, she describes how her younger sibling was "devastated" when she was diagnosed with MS.

She writes: "Chantelle was first diagnosed in May 2016. What started out as fatigue and a little numbness in her left arm, she put it down to just being tired from being a full time working mum of two young girls...

"A trip to her doctor one Sunday afternoon changed her and her family life forever. She was told to head straight to hospital, the dr believes she may have had a minor stroke.

"24 hours later, sitting in the hospital bed the neurologist suggests its either a brain tumour, motor neurone disease or MS and the only way to confirm is with a lumbar puncture and full MRI of her brain & spine.

"I will never forget sitting there holding my baby sister's hand as she lays on the bed with the nurse injecting a large needle into her spine to obtain spinal fluid. Almost an hour and half goes by and they confirm its been unsuccessful and they will need to try again.

"Next is the MRI and after two hours my sister returns to her hospital bed waiting for the news that will change her life forever."

She adds: "That neurologist returns to deliver the news, Chantelle you have multiple sclerosis... Chantelle is devastated, all she can think about is her two young girls and if she will be around to watch them grow up."

In a post on the page, Chantelle herself pays tribute to her sister, her husband Dara O'Donoghue and her two little girls.

Addressing Lilly and Edie, she writes: "You are the reason, I will never give up fighting this terrible disease, MS.

"You are my world and I will love you for eternity."

She also expresses her gratitude to her other relatives and friends.

It is estimated that around 100,000 people in the UK have MS.

HSCT involves the intravenous infusion of stem cells derived from peripheral blood, bone marrow or umbilical cord blood.

In autologous cases, the patient's own stem cells are used.

Their immune system is usually wiped out with chemotherapy treatment before it is regrown using their stem cells.

To visit Chantelle's GoFundMe page, click here .

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Desperate mum's race against time to fund treatment before she forgets her two little girls - Mirror.co.uk

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GERMANTOWN, Md., April 12, 2017 (GLOBE NEWSWIRE) -- Neuralstem, Inc. (CUR), a biopharmaceutical company focused on the development of nervous system therapies based on its neural stem cell technology, announced that a new cohort of four patients will be added to its ongoing Phase 1 human clinical trial evaluating the safety and feasibility of using NSI-566 spinal cord-derived neural stem cells to repair chronic spinal cord injury (cSCI). The amended protocol was approved by the U.S. Food and Drug Administration and the Institutional Review Board at the study site, University of California San Diego (UCSD). NSI-566 is Neuralstems lead stem cell therapy candidate.

Under the amended protocol, updated on clinicaltrials.gov (NCT01772810), four qualifying patients with AIS-A complete, quadriplegic, cervical injuries involving C5-C7 cord will be added to the study. The injury must have occurred 1-2 years prior to the time of stem cell treatment, which is a one-time surgery involving six injections of NSI-566 into the affected area of the cord. The study has begun active recruitment of patients.

About 250,000 Americans are living with cSCI, and approximately 11,000 new injuries are reported each year1. Roughly 52% of these individuals will be considered paraplegic and 47% will be considered quadriplegic1. cSCI is a permanent and disabling condition with few to no treatments. Its devastating effect can be measured from social, healthcare, and economic perspectives.

This expansion of the study to cervical injuries builds on the results demonstrating that the implantation of NSI-566 stem cells in the first four patients with AIS-A complete thoracic cSCI was safe and feasible with no serious adverse events, said Karl Johe, Ph.D., Chief Scientific Officer, Neuralstem. There is a tremendous unmet need in the treatment of cSCI and we are privileged to have the experts at UCSD School of Medicine and the Sanford Stem Cell Clinical Center at UC San Diego Health conducting the research. We look forward to further evaluating NSI-566 neural stem cells in chronic complete cervical injuries."

Long-term safety data from the first cohort of chronic complete thoracic injuries is currently being analyzed by the study team at UCSD School of Medicine.

About Neuralstem Neuralstems patented technology enables the commercial-scale production of multiple types of central nervous system stem cells, which are being developed as potential therapies for multiple central nervous system diseases and conditions.

Neuralstems technology also enables the discovery of small molecule compounds by systematic screening chemical compounds against its proprietary human hippocampal stem cell line. The screening process has led to the discovery and patenting of molecules that Neuralstem believes may stimulate the brains capacity to generate new neurons, potentially reversing pathophysiologies associated with certain central nervous system (CNS) conditions.

The company has completed Phase 1a and 1b trials evaluating NSI-189, a novel neurogenic small molecule product candidate, for the treatment of major depressive disorder or MDD, and is currently conducting a Phase 2 efficacy study for MDD.

Neuralstems stem cell therapy product candidate, NSI-566, is a spinal cord-derived neural stem cell line. Neuralstem is currently evaluating NSI-566 in three indications: stroke, chronic spinal cord injury (cSCI), and Amyotrophic Lateral Sclerosis (ALS).

Neuralstem is conducting a Phase 1 safety study for the treatment of paralysis from chronic motor stroke at the BaYi Brain Hospital in Beijing, China. In addition, NSI-566 was evaluated in a Phase 1 safety study to treat paralysis due to chronic spinal cord injury as well as a Phase 1 and Phase 2a risk escalation, safety trials for ALS. Subjects from all three indications are currently in long-term observational follow-up periods to continue to monitor safety and possible therapeutic benefits.

Cautionary Statement Regarding Forward-Looking Information This news release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements relate to future, not past, events and may often be identified by words such as expect, anticipate, intend, plan, believe, seek or will. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Specific risks and uncertainties that could cause our actual results to differ materially from those expressed in our forward-looking statements include risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstems periodic reports, including the Annual Report on Form 10-K for the year ended December 31, 2015, and Form 10-Q for the nine months ended September 30, 2016, filed with the Securities and Exchange Commission (SEC), and in other reports filed with the SEC. We do not assume any obligation to update any forward-looking statements.

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Neuralstem Expands Phase 1 Safety Trial of NSI-566 Neural Stem Cells in Spinal Injury - Yahoo Finance

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Germantown, Md.-based Neuralstem is involved in a Phase 1 human clinical trial testing the safety and feasibility of using NSI-566 spinal cord-derived neural stem cells to repair chronic spinal cord injury.

A biopharmaceutical company, Neuralstem develops nervous system therapies derived from neural stem cell technology.

Here are six takeaways:

1. NSI-566 represents the company's lead stem cell therapy candidate.

2. University of California San Diego serves as the study site, and just added a new cohort of four patients.

3. The four new patients all have AIS-A complete, quadriplegic, cervical injuries involving C5-C7 cord.

4. The patients suffered the injury one to two years before undergoing stem cell treatment.

5. The treatment involves six injections of NSI-566 into the spinal cord's affected area.

6. UCSD researchers are analyzing long-term safety data from the study's first cohort on chronic complete thoracic injuries.

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UC San Diego adds 4 patients to Neuralstem's neural stem cell study for spinal cord injury: 6 takeaways - Becker's Orthopedic & Spine

Spinal Cord Stem Cells Comments Off on UC San Diego adds 4 patients to Neuralstem’s neural stem cell study for spinal cord injury: 6 takeaways – Becker’s Orthopedic & Spine | April 14th, 2017

Alan Mackay-Sim, the 2017 Australian of the Year, told Tuesday's GE:Decoding Industry conference that too much university medical research languished for want of a different financing model.

Alan Mackay-Sim, the biomedical scientist who is 2017 Australian of the Year, has called for a new model of public-private partnership to fill the void left by major pharmaceutical companies withdrawing from neuroscience research.

Mr Mackay-Sim, a Griffith University researcher who led a team famed for proving the safety of using nasal cells to repair spinal cord damage, told a GE conference on Tuesday that all of the major pharmaceutical companies had closed or scaled back their neuroscience research units this decade because of the expense and risk in proving that drugs worked in the general population.

"Both Pfizer and Eli Lilly had treatments for Alzenheimer's Disease that failed at that finalstage of the [US Food & Drug Administration] approval process, and it had cost them each $US600million to getthere," he said.

With private enterprise less willing to solve complex neurological problems, Mr Mackay-Sim said it fell to "chumps like me" in the publicly-funded research sector.However, a fundamental mismatch between thebusiness model of universities and corporations had to be solved first.

"The problem is that for a university researcher today, the currency is to get your research published, that's how you get the next grant," he told the conference.

"But once it's published, it's no longer novel, it's not patentable and therefore private enterprise has no interest.Uni researchers are in it for the public good but unfortunately none of that good gets to the public without the commercial imperative."

Mr Mackay-Sim said universities could only afford to patent a fraction of the research they produced, and even then it too often languished for a lack of investors able to fund clinical trials. This happened to Mr Mackay-Sim's own 2001 patent for making stem cells from olfactory sheathing cells.

A new model was required which recognised the "value of future costs saved" in medical research and broadened the pool of potential financial backers, he told The Australian Financial Review on the sidelines of the GE conference.

"Our trial to prove that transplanting olfactory sheathing cells into the spinal cord was safe cost us $1 million, a second trial proving it works might costs us $20 million and the third trial to prove it works broadly might be $100 million or more - but we spend $2 billion a year in Australia caring for those with a spinal cord injury, so surely that's a good investment," Mr Mackay-Simsaid.

Insurance companies were a relatively untapped source of funding for medical research, he added, given their commercial interest in reducing the cost of medical care.

Patentable drugs had a hard enough time being commercialised, but it was even more difficult to fund trials for improved procedures.

"A friend of mine [University of WA Professor Sarah Dunlop] is trying to get up a clinical trial where people with spinal cord injuries are cooled as soon as the first responders get there, like what happens with heart attack patients," Mr Mackay-Sim said.

The slowing of the metabolism through cooling is thought to provide an opportunity for interventions that could increase the mobility of someone with a spinal cord injury.

"But it's a process, not a drug, it's not really patentable so it's proving a struggle to get funding despite this maybe meaning the difference between quadraplegiaand just having an arm immobilised."

The potential value of university-generated medical research couldbe recognised and supported by business earlier if a new kind of public-private partnership was supported and incentivised by government, spreading the risks and rewards, according to Mr Mackay-Sim.

"But it will take unis, government, investors, insurers, pharmaceutical companies and perhaps CSIRO coming together, to rethink this system where the patent and guarding all your IP is the model," he said.

Mr Mackay-Sim stressed he was "not a business guy", but said a benefit of being Australian of the Year was the access he was getting to people who could collaborate on a new funding model for medical research.

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Australian of the Year Alan Mackay-Sim calls for medical research funding change - The Australian Financial Review

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Eight young Otago people have won awards in the 2017 New Zealand Youth Awards.

The awards recognise young New Zealanders who have achieved outstanding results and given back to their communities, as well as those who have made a significant contribution to the support of young people.

Kelly Young (20) and Jo Mohan (19), both of Dunedin, won a Change Maker Cultural Award for co-founding the University of Otago Students Without Borders Club, which helps refugees integrate into the Dunedin community.

Damon Lillis (21), of Dunedin, won a Working for Youth Award for his work on the board of the Playhouse Children's Theatre and has directed several plays which helps young people to increase their confidence and self-esteem.

He also co-ordinates the Aspire programme which helps young people from low decile schools find out more about University life.

Bokyong Mun (20), of Dunedin, also won a Working for Youth Award for her support and development of the United Nations Youth Council.

Fawzan Dinnunhan (24), of Dunedin, won a Giving Back Award for his contribution to research and improving a number of key IT platforms for the Spinal Cord Society - a non-profit organisation that studies the use of stem cells as a cure for type-1 diabetes.

Leo Munro-Heward (16), of Wanaka, also won a Giving Back Award for his establishment of the Queer Straight Alliance in Wanaka, which aims to raise awareness and support.

Holly Robinson (22), of Dunedin, won a Youth with Disability Award for representing New Zealand as the flag bearer in the opening ceremony for the Rio 2016 Paralympic Games.

She also broke the world record for the F46 javelin, and placed second overall.

Casey Davies-Bell (23), of Dunedin, won a Leadership Award for establishing Global Energy Impact Assessment (Geia) Ltd - a New Zealand based start-up company which aims to accelerate New Zealand's transition towards a sustainable future.

Youth Minister Nikki Kaye said about 190 nominations from around the country were received, and 50 were given awards at a ceremony in Parliament last night.

''The calibre of the award winners in the new Youth Enterprise category was outstanding, with the recipients including a number of under-20-year-olds who have founded successful businesses which have achieved significant social or business impact, both locally and internationally,'' she said.

''It's particularly encouraging to see the way these young leaders and entrepreneurs have embraced new technology and social media, to develop innovative new approaches to achieve their business or social vision.

''When you look at the drive, skills, compassion and integrity evident in the winners across all the categories, it's clear that our young people have enormous talent and potential, and the future of our country is in safe hands.''

john.lewis@odt.co.nz

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Eight from Otago receive NZ youth awards - Otago Daily Times

Spinal Cord Stem Cells Comments Off on Eight from Otago receive NZ youth awards – Otago Daily Times | April 11th, 2017

Suspended from a tree in the wilds of Tennessee, the remains of his hang-glider entangled in the branches above, his lower left leg pulverised and his chest badly bruised from his dramatic fall into the forest canopy, Alan Mackay-Sim felt hyper-alert from the electricity of adrenalin, the clarity of shock. Only the wind was audible, softly rustling the branches around him as he sucked in the forest air, perfumed with poplar and sweet-gum.

Knowing that the adrenalin coursing through his veins would soon give way to an agonising and possibly debilitating pain, the 28-year-old used these precious minutes to assess his predicament, to figure it out coolly like a man of science.

A broken leg, no doubt shattered in multiple places. Possibly hours before his fellow hang-gliding friends would be able to locate him; if they didn't reach him by nightfall, he could be dangling here until the next morning. Unfastening his harness and climbing down to the ground five metres below was not an option, at least, not without incurring further injury. To prevent blood from pooling and to save his leg, he quickly concluded, he'd have to carefully oh-so carefully free the hang-glider's stirrup bar and one of the ropes from his harness, create a splint for his injured left leg, secure it to his right leg and hoist up both limbs while hanging there like a gammy fruit bat.

Mackay-Sim had only arrived in the US a few weeks before, a post-doctoral researcher from the University of Sydney eager to extend his studies into the olfactory system specifically, what the nose tells the brain at the University of Philadelphia. But on that blustery October day back in 1979, when a freak wind gust whooshing around Lookout Mountain near Chattanooga sent a promising young Australian scientist nosediving into the forest, before a rescue team found himhanging in the tree just before sunset, both legs securely elevated, Mackay-Sim was set to gain some useful insights that would become valuable to him in his later life. Insights that would be peculiarly relevant to his work as a pioneering stem cell researcher specialising in the treatment of spinal cord injuries.

So badly broken was his leg that Mackay-Sim spent more than six months in a wheelchair, and many more months afterwards receiving intensive physiotherapy.

"It gave me some insight into what life's like in a wheelchair, and it stayed with me," says Mackay-Sim, settling into a chair in his office at the Institute for Drug Discovery at Griffith University, just down the corridor from the laboratory where he spent years toiling over petri dishes of nasal stem cells, in his life's mission to treat spinal injuries, hereditary spastic paraplegia and diseases like Parkinson's.

A photo of the late actor Christopher Reeve is pinned on a noticeboard behind him. "I met Christopher in 2003 when he came out for a conference; he was interested in our clinical trials," Mackay-Sim says, looking at the photo. "Then in the following year I spent some time at his home in New York, and we talked a lot about spinal cord injury repair, and his own personal story."

As Mackay-Sim explains, the higher up the spinal cord an injury is, the more severe the effects. "As we know, Christopher fell off a horse and became a full paraplegic on a respirator, but in fact he suffered only a small injury; the problem was that the bleed went straight into his spinal cord. It only takes a very small injury to stop transmission; you can have large injuries to the chest and not suffer long-term repercussions but here, in the neck, a small event can change your life."

Back in the late 1980s, after he started at Griffith University, Mackay-Sim became interested in a set of extraordinary busy-bee cells in the human nose called olfactory ensheathing cells nerve cells that regenerate every single day to recreate our sense of smell. If these wonder cells are continually regenerating, he kept asking himself, could they not be transplanted to another part of the body where cells don't regenerate, like the spinal cord?

Years of scientific slog followed until 2002, when Mackay-Sim was the first researcher in the world to remove cells from the nose of a patient paralysed in a car accident, grow them in a cell culture and then, with the help of surgeons at Brisbane's Princess Alexandra Hospital, implant them in the same patient's spinal cord. "By the time Christopher died in 2006, we'd transferred stem cells from the nose into three patients and shown it was safe to do so," he says. "One of the patients recovered some sensation above the injury, which was hopeful, but one person does not make real scientific evidence."

For Mackay-Sim, the importance of scientific breakthroughs in the treatment of life-threatening illnesses is deeply personal. In 2014, he was diagnosed with multiple myeloma, an incurable form of leukaemia. As a result of the illness, which breaks down bones in an advanced form of osteoporosis, and the punishing series of treatments that followed his diagnosis, involving radiation, chemotherapy and stem cell therapy (albeit a very different form from the one the scientist was researching), Mackay-Sim lost nine centimetres in height and shed more than 15 kilograms of body weight. "I became extremely sick from the chemotherapy just prior to the bone marrow transplant," the 65-year-old recalls. "It was the worst experience of my life."

There was also the initial shock of the diagnosis, and grief for the loss of his health after a highly active life, from football and rowing in his teens to distance cycling, scuba diving and hang-gliding, which he took up while atuniversity. "Both my parents lived into their 80s and 90s and I'd been cycling up to 200 kilometres a week for decades, so I wasn't anticipating something like this."

Still, as a scientist he couldn't help but observe the trajectory of his illness with stricken fascination. "I had some good conversations with my oncologist," he smiles. "As a biologist examining my own biology, it did demystify lots of things. One minute I was a grieving patient, the next an interested scientist."

Above all, Mackay-Sim refuses to sentimentalise his battle with the illness and asks that I don't embroider it in this story by turning it into some kind of triumph of personal will power over disease. "My survival is determined by the vagaries of the particular cancer I've got," he says matter-of-factly. "Some people have nasty genetic diseases that mean they die earlier. For the moment, I feel very healthy."

Surely his extreme fitness at least helped him to survive the ravages of chemo? "I think being fit and active all my life has given me a higher quality of life after treatment," he acknowledges. "But one doctor put it to me that I probably would have sought out treatment earlier if I wasn't so fit, because I dismissed the symptoms as simple back pain from the cycling. It took two years after the chemo and radiation for the pain to go away. 2016 was a year of normality for me my back became stable enough for me to get on a road bike again."

The diagnosis added poignancy to the evening in Canberra in late January when Mackay-Sim, out of 3000- plus nominations, was crowned Australian of the Year. Sitting alongside him were his American-born wife of nearly 34 years, Lisa Peine, a retired primary school teacher, their 28-year-old daughter Matilda, a trainee psychiatrist, and 25-year-old son Callum, an engineer.

Mackay-Sim with wife Lisa Peine in North Queensland in 1983. Photo: Courtesy of Alan Mackay-Sim

Perhaps no Australian of the Year is better placed to recognise just how precious a year can be, and more determined to seize the moment to put science and innovation at the top of the national conversation. A former Queenslander of the Year, Mackay-Sim sees science as vital to our future national wellbeing, especially after the recent wake-up call in international school education rankings, which placed Australia behind Kazakhstan and Slovenia in maths and science.

Mackay-Sim agrees unequivocally with Michelle Simmons, professor of quantum physics at the University of NSW, who drew headlines recently when she declared that the "feminised" nature of Australia's high school physics curriculum (emphasising the sociology of science with essays and theory instead of rigorous lab experiments and mathematical problem-solving) had been an unmitigated failure. Introduced in the 1980s, the approach had resulted in a long, slow decline in standards.

"Scientific understanding comes from learning the processes; it can be hard work but is absolutely essential," Mackay-Sim insists. "The key to a good science education in schools is to get well-trained teachers." (Mackay-Sim has been deeply encouraged by some of the science teachers he's met since winning the award.)

The choice of Mackay-Sim the first scientist honoured as Australian of the Year since immunologist Ian Frazer in 2006 was met with near-universal applause by Australia's scientific community, who no doubt feel dispirited in this post-truth world of climate-change denial, cuts to the CSIRO and the growing view by government agencies that basic research isn't worth it.

"We need to invest in young scientists," Mackay-Sim declared in his acceptance speech, adding that the discovery of new medical treatments can reduce the strain on health budgets. "More than 10,000 Australians live with a spinal cord injury a new person is added to this tally every day." But politicians need to take a long-term view of the benefits of basic research, he tells me, "a view much longer than the political horizon".

The announcement also gave the image of the Australian of the Year awards a much-needed polish. The 2016 winner, Lieutenant-General David Morrison, drew criticism for charging up to $15,000 a pop forpublic speaking engagements, as well as grandstanding about sexism in the military despite his own handling of the army's "Jedi Council" sex scandal, in which demeaning sex videos of women were distributed among a group of soldiers. (It was revealed that Morrison's office knew of the scandal 11 months prior to the former Chief of Army releasing a now-famous condemnation on YouTube of those involved.)

Will Mackay-Sim accept speakers' fees? "I knew nothing about speakers' fees when I accepted the award," he says crisply. "I'm not pursuing money after all, I've spent my life doing public research."

Although he hasn't received any fees to date, Mackay-Sim insists that if they are offered, the funds will be donated to the Hereditary Spastic Paraplegia Research Foundation, his charity of choice.

Mackay-Sim only had a day or so to bask in the glow of being named Australian of the Year before there was a claim his scientific achievements had beenoverstated in the application. A Polish scientist, Professor Pawel Tabakow, after being approached by an Australian journalist in Europe, declared that Mackay-Sim had nothing to do with the world-first surgery using olfactory stem cells that enabled a Polish paraplegic, Darek Fidyka, to walk again. "It is not our business who should be Australian of the Year," Tabakow told The Weekend Australian. "But it is our business when his work is being linked to the surgery of Fidyka. He has no link whatsoever."

The scientific hullaballoo arose from the submission to the Australia Day Council (ADC), which states that Mackay-Sim's research "helped play a central role in proving the safety of science that was a precursor to Dr Tabokow in Poland undertaking the first successful restoration of mobility in a quadriplegic man".

Although Mackay-Sim didn't write the submission to the ADC, doesn't know who did, and never claimed to be involved in Tabokow's work, an artificial straight line was drawn between the two scientists, especially when the word "precursor" was dropped from condensed versions of the ADC's quote in multiple news stories (we'll examine the fallout from the controversy a little later).

Padding amiably about his large, multi-room laboratory, past refrigerator-sized storage cabinets containing cell cultures, past white-coated scientists peering into microscopes, Mackay-Sim seems to be in his element, with every second person saying "Hi", "Hello", or "How are you?" If stem cells are indeedthe microscopic building blocks of the world, this is the tiny universe the scientist feels most comfortable in. But it's a laboratory that now has to hum along without him Mackay-Sim retired late last year, his duties now limited to popping into the university once a week as an emeritus professor.

Later in the day, Professor George D. Mellick, head of Clinical Neurosciences at Griffith, tells me that Mackay-Sim has always set aside time to mentor younger scientists, and to explain sometimes hideously complicated science to a lay audience, but would be the last person to crow about his own scientific achievements.

"One of the things that isn't highlighted very much about Alan's work is his research into Parkinson's. We've been able to learn a lot about Parkinson's by studying cells from people with the disease, and the information coming out of this research will hopefully lead to better treatments."

Back in his office, Mackay-Sim gives me a quick rundown, 101-style, on the human nose. No, the human sense of smell doesn't necessarily decline with age, unless illness or disease set in, and it is astonishingly adept at distinguishing hundreds of thousands of different odours. Yes, women do have a superior sense of smell to men, but the difference is surprisingly only slight. Yes, the first symptom of Parkinson's, before the typical tremors set in, is a reduced sense of smell, as it is with those sufferers who will go on to develop dementia. And yes paws down dogs do have a vastly more powerful sense of smell than humans, although it's impossible to quantify by exactly how much (Mackay-Sim has been known to hide from his spoodle Henry, to measure how long it takes for the dog to find him).

As he relays all this, Mackay-Sim's eyes twinkle and a smile lights up his face: it's easy to see how he'd be the perfect academic for Griffith to call on to schmooze a government minister or potential philanthropist and secure desperately sought-after funding. I ask him about his trademark moustache, which he's had since the early 1990s, when he shaved off a beard. "My wife wouldn't recognise me without it," he jokes. "She says that a small mammal could roost beneath my mouth."

Mackay-Sim, whose double-barrelled surname comes from his paternal grandfather, grew up in middle-class Roseville, on Sydney's leafy North Shore, the third of four brothers. His mother Lois was a nurse during World War II and later a full-time mum while his father Malcolm ran a hardware importing and distributing business, Macsim Distributors (now Macsim Fasteners, owned by Alan's eldest brother, Fraser). At North Sydney Boys' High he was "the opposite of a shit-stirrer. I was vice captain, head of the cadets, played football, was in the rowing team, had a shot at athletics, sang in the choir I did it all."

With wife, Lisa Peine, in Sulawesi, Indonesia, 2007. Photo: Courtesy of Alan Mackay-Sim

After graduating with honours in science from Macquarie University, Mackay-Sim picked up tutoring work in the department of physiology at the University of Sydney, where he completed a PhD on the brain's visual system. Two academic stints in the US followed, first at the University of Pennsylvania from 1979 until 1981, followed by two years at the University of Wyoming, during which time he met his wife Lisa, then living in northern Colorado.

The pair married in 1984, by which time Mackay-Sim had been offered a research role in the department of physiology at the University of Adelaide. He started at Griffith University in 1987, where his research concentrated on the biology of nasal cells.

At the height of the heated moral debate over the use of embryonic stem cells whether the therapeutic potential of stem cells could justify destroying human embryos to extract them Mackay-Sim met Pope Benedict XVI at a Vatican conference in 2005. The Pope congratulated him on his exclusive use of adult stem cells.

"I wasn't avoiding embryonic stem cells for religious reasons," Mackay-Sim explains. "It just so happenedthat I was working with adult stem cells at the time and the conference was looking at alternatives to using embryonic stem cells. But it was a scientific conference and I was impressed with its calibre; the only difference was that men in purple robes were sitting at the back asking questions."

Later in the same trip, Mackay-Sim was invited, along with a host of others, to the Apostolic Palace at Castel Gandolfo the Vatican summer palace. "You feel the history of the Roman Catholic Church, with the Pope coming in with his cardinals and the Swiss Guards," he says. "I'm not a believer, but it was a very powerful experience."

In 2006, the debate over embryonic stem cells virtually vanished when scientist Shinya Yamanaka from Japan's Kyoto University stunned the world by proving that stem cells needn't come from human embryos adult cells can be reprogrammed to act like stem cells, to be returned to an embryo-like state (Yamanaka's discovery won him the Nobel Prize in 2012). "Yamanaka worked out how to genetically engineer any cells so that they had the properties of embryonic stem cells," says Mackay-Sim, who nonetheless continued to focus on adult stem cells only.

Mackay-Sim accomplished his own world first in 2002 when, with the assistance of doctors at Brisbane's Princess Alexandra Hospital, he transplanted olfactory stem cells into the spinal cord of a man crippled in a car accident. The procedure was repeated with two other paraplegic patients at the same hospital and the study wrapped up in 2007.

While the procedures didn't result in any of the patients regaining useful movement in their legs, the results of Mackay-Sim's clinical trials, published in 2005 and 2008, paved the way for further development of olfactory stem cell transplantation.

One researcher who followed Mackay-Sim's trials closely was Geoffrey Raisman from University College London, who visited the Australian team shortly after the first operation in Brisbane to study their work. Raisman later led the British team who worked with Polish surgeon Tabakow on Darek Fidyka in 2012.

Tabakow deployed 100 separate micro-injections of olfactory sheathing cells above and below Fidyka's spinal injury, with the hope these cells would provide a skeleton for nerve fibres to grow and reconnect. A former volunteer firefighter, Fidyka had become paralysed in 2010 after a severe knife attack by the jealous ex-husband of his girlfriend. The repeated stab wounds to Fidyka's back severed his spinal cord, paralysing from the waistdown. (Fidyka's attacker, a fellow firefighter, committed suicide shortly afterwards.)

There's no doubt Tabakow's work was a major advance on Mackay-Sim's research. Tabakow's strategy was to extract ensheathing cells specifically from the olfactory bulbs in Fidyka's nose, grow them in a culture, while also extracting nerve cells from his ankle in a multi-pronged attempt at spinal cord reconstruction. After a series of operations, Fidyka can walk with the assistance of a frame, has regained some bladder control and sexual function, and can ride a tricycle.

Raisman described their new stem cell procedure as "more impressive than man walking on the moon", but it will have be tested on other paraplegics, including those with more severe injuries than Fidyka's, such as car accident victims who have had more of their spinal cord damaged, before it can be declared a reliable method of restoring mobility. As impressive as Tabakow's achievement is, it has still only worked on one patient.

Nobody, however, disputes Mackay-Sim's immense contribution to stem cell transplantation; his work is unimpeachable. If nothing else, he was at the forefront of the science showing that restoring the ability to walk to paraplegics is no longer science fiction. "What I've always said is that we did the first phase of clinicaltrials with olfactory stem cells, and the aim of those trials was to show they were safe," says Mackay-Sim. "That was the first important step."

Mackay-Sim wrote to Tabakow shortly after the controversy blew up, explaining that he didn't write the submission to the Australia Day Council, and was in no way claiming credit for Fidyka's remarkable recovery. "He wrote back a very nice email," says Mackay-Sim. "I believe I've given credit to other scientists in every interview I've given to journalists. I feel comfortable in my behaviour and ethics."

With Prime Minister Turnbull in January this year. Photo: Elesa Kurtz

Mackay-Sim can remember the day when he felt something was wrong terribly wrong. He'd been suffering back pain for months, but dismissed it as old age, or strain from bending over on his bicycle on long rides, and stocked up his pantry with painkillers. "I was in Colorado with Lisa visiting her family, and the pain became so bad I couldn't walk very far. I found the pain eased when I got on my bicycle. I flew home a week before she did; the plane trip back was absolute hell."

What followed was a swift diagnostic journey from his GP to specialists at Brisbane's Wesley Hospital, resulting in a devastating diagnosis. "They suspected something cancerous quite quickly. I didn't realise how ill I was; by this stage, my kidneys weren't coping at all with the antibodies released from my white blood cells, which were going berserk trying to fight the disease. I was at risk of kidney failure and my bones were becoming very fragile. I started therapy almost immediately, in June 2014. Then began the cycles of chemotherapy and stem cell treatment in December."

Since the beginning of last year, however, Mackay-Sim's health has dramatically improved, and even though he's retired to his beachside home in Currimundi on the Sunshine Coast, he is still active in university affairs. He concedes that his health may prevent him from being as active as Rosie Batty, perhaps our most vigorous Australian of the Year to date. But he's already spoken at functions in Brisbane, Sydney and Perth, and will be attending the national March for Science on April 22, which coincides with Earth Day. He moves with the speed and fluidity of a man 10 or 15 years younger.

"I feel very healthy, very energised at the moment," says Mackay-Sim, who is planning a bicycle ride in Italy's Dolomites in July with a couple of mates. (Last year he and his wife went on the Great Victorian Bike Ride, a seven-day ride averaging 85 kilometres a day.)

"I do need to be selective with the number of invitations around Australian of the Year," he concedes, "but I'll do everything I can. After all, what more exciting time could you have to talk about science?"

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Australian of the Year Alan Mackay-Sim on the advantage of being 'an interested scientist' - The Sydney Morning Herald

Spinal Cord Stem Cells Comments Off on Australian of the Year Alan Mackay-Sim on the advantage of being ‘an interested scientist’ – The Sydney Morning Herald | April 9th, 2017

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Spinal Cord Injury (SCI) Stem Cell Treatment

Spinal Cord Stem Cells Comments Off on Spinal Cord Injury (SCI) Stem Cell Treatment | April 8th, 2017

Suspended from a tree in the wilds of Tennessee, the remains of his hang-glider entangled in the branches above, his lower left leg pulverised and his chest badly bruised from his dramatic fall into the forest canopy, Alan Mackay-Sim felt hyper-alert from the electricity of adrenalin, the clarity of shock. Only the wind was audible, softly rustling the branches around him as he sucked in the forest air, perfumed with poplar and sweet-gum.

Knowing that the adrenalin coursing through his veins would soon give way to an agonising and possibly debilitating pain, the 28-year-old used these precious minutes to assess his predicament, to figure it out coolly like a man of science.

A broken leg, no doubt shattered in multiple places. Possibly hours before his fellow hang-gliding friends would be able to locate him; if they didn't reach him by nightfall, he could be dangling here until the next morning. Unfastening his harness and climbing down to the ground five metres below was not an option, at least, not without incurring further injury. To prevent blood from pooling and to save his leg, he quickly concluded, he'd have to carefully oh-so carefully free the hang-glider's stirrup bar and one of the ropes from his harness, create a splint for his injured left leg, secure it to his right leg and hoist up both limbs while hanging there like a gammy fruit bat.

Mackay-Sim had only arrived in the US a few weeks before, a post-doctoral researcher from the University of Sydney eager to extend his studies into the olfactory system specifically, what the nose tells the brain at the University of Philadelphia. But on that blustery October day back in 1979, when a freak wind gust whooshing around Lookout Mountain near Chattanooga sent a promising young Australian scientist nosediving into the forest, before a rescue team found himhanging in the tree just before sunset, both legs securely elevated, Mackay-Sim was set to gain some useful insights that would become valuable to him in his later life. Insights that would be peculiarly relevant to his work as a pioneering stem cell researcher specialising in the treatment of spinal cord injuries.

So badly broken was his leg that Mackay-Sim spent more than six months in a wheelchair, and many more months afterwards receiving intensive physiotherapy.

"It gave me some insight into what life's like in a wheelchair, and it stayed with me," says Mackay-Sim, settling into a chair in his office at the Institute for Drug Discovery at Griffith University, just down the corridor from the laboratory where he spent years toiling over petri dishes of nasal stem cells, in his life's mission to treat spinal injuries, hereditary spastic paraplegia and diseases like Parkinson's.

A photo of the late actor Christopher Reeve is pinned on a noticeboard behind him. "I met Christopher in 2003 when he came out for a conference; he was interested in our clinical trials," Mackay-Sim says, looking at the photo. "Then in the following year I spent some time at his home in New York, and we talked a lot about spinal cord injury repair, and his own personal story."

As Mackay-Sim explains, the higher up the spinal cord an injury is, the more severe the effects. "As we know, Christopher fell off a horse and became a full paraplegic on a respirator, but in fact he suffered only a small injury; the problem was that the bleed went straight into his spinal cord. It only takes a very small injury to stop transmission; you can have large injuries to the chest and not suffer long-term repercussions but here, in the neck, a small event can change your life."

Back in the late 1980s, after he started at Griffith University, Mackay-Sim became interested in a set of extraordinary busy-bee cells in the human nose called olfactory ensheathing cells nerve cells that regenerate every single day to recreate our sense of smell. If these wonder cells are continually regenerating, he kept asking himself, could they not be transplanted to another part of the body where cells don't regenerate, like the spinal cord?

Years of scientific slog followed until 2002, when Mackay-Sim was the first researcher in the world to remove cells from the nose of a patient paralysed in a car accident, grow them in a cell culture and then, with the help of surgeons at Brisbane's Princess Alexandra Hospital, implant them in the same patient's spinal cord. "By the time Christopher died in 2006, we'd transferred stem cells from the nose into three patients and shown it was safe to do so," he says. "One of the patients recovered some sensation above the injury, which was hopeful, but one person does not make real scientific evidence."

For Mackay-Sim, the importance of scientific breakthroughs in the treatment of life-threatening illnesses is deeply personal. In 2014, he was diagnosed with multiple myeloma, an incurable form of leukaemia. As a result of the illness, which breaks down bones in an advanced form of osteoporosis, and the punishing series of treatments that followed his diagnosis, involving radiation, chemotherapy and stem cell therapy (albeit a very different form from the one the scientist was researching), Mackay-Sim lost nine centimetres in height and shed more than 15 kilograms of body weight. "I became extremely sick from the chemotherapy just prior to the bone marrow transplant," the 65-year-old recalls. "It was the worst experience of my life."

There was also the initial shock of the diagnosis, and grief for the loss of his health after a highly active life, from football and rowing in his teens to distance cycling, scuba diving and hang-gliding, which he took up while atuniversity. "Both my parents lived into their 80s and 90s and I'd been cycling up to 200 kilometres a week for decades, so I wasn't anticipating something like this."

Still, as a scientist he couldn't help but observe the trajectory of his illness with stricken fascination. "I had some good conversations with my oncologist," he smiles. "As a biologist examining my own biology, it did demystify lots of things. One minute I was a grieving patient, the next an interested scientist."

Above all, Mackay-Sim refuses to sentimentalise his battle with the illness and asks that I don't embroider it in this story by turning it into some kind of triumph of personal will power over disease. "My survival is determined by the vagaries of the particular cancer I've got," he says matter-of-factly. "Some people have nasty genetic diseases that mean they die earlier. For the moment, I feel very healthy."

Surely his extreme fitness at least helped him to survive the ravages of chemo? "I think being fit and active all my life has given me a higher quality of life after treatment," he acknowledges. "But one doctor put it to me that I probably would have sought out treatment earlier if I wasn't so fit, because I dismissed the symptoms as simple back pain from the cycling. It took two years after the chemo and radiation for the pain to go away. 2016 was a year of normality for me my back became stable enough for me to get on a road bike again."

The diagnosis added poignancy to the evening in Canberra in late January when Mackay-Sim, out of 3000- plus nominations, was crowned Australian of the Year. Sitting alongside him were his American-born wife of nearly 34 years, Lisa Peine, a retired primary school teacher, their 28-year-old daughter Matilda, a trainee psychiatrist, and 25-year-old son Callum, an engineer.

Mackay-Sim with wife Lisa Peine in North Queensland in 1983. Photo: Courtesy of Alan Mackay-Sim

Perhaps no Australian of the Year is better placed to recognise just how precious a year can be, and more determined to seize the moment to put science and innovation at the top of the national conversation. A former Queenslander of the Year, Mackay-Sim sees science as vital to our future national wellbeing, especially after the recent wake-up call in international school education rankings, which placed Australia behind Kazakhstan and Slovenia in maths and science.

Mackay-Sim agrees unequivocally with Michelle Simmons, professor of quantum physics at the University of NSW, who drew headlines recently when she declared that the "feminised" nature of Australia's high school physics curriculum (emphasising the sociology of science with essays and theory instead of rigorous lab experiments and mathematical problem-solving) had been an unmitigated failure. Introduced in the 1980s, the approach had resulted in a long, slow decline in standards.

"Scientific understanding comes from learning the processes; it can be hard work but is absolutely essential," Mackay-Sim insists. "The key to a good science education in schools is to get well-trained teachers." (Mackay-Sim has been deeply encouraged by some of the science teachers he's met since winning the award.)

The choice of Mackay-Sim the first scientist honoured as Australian of the Year since immunologist Ian Frazer in 2006 was met with near-universal applause by Australia's scientific community, who no doubt feel dispirited in this post-truth world of climate-change denial, cuts to the CSIRO and the growing view by government agencies that basic research isn't worth it.

"We need to invest in young scientists," Mackay-Sim declared in his acceptance speech, adding that the discovery of new medical treatments can reduce the strain on health budgets. "More than 10,000 Australians live with a spinal cord injury a new person is added to this tally every day." But politicians need to take a long-term view of the benefits of basic research, he tells me, "a view much longer than the political horizon".

The announcement also gave the image of the Australian of the Year awards a much-needed polish. The 2016 winner, Lieutenant-General David Morrison, drew criticism for charging up to $15,000 a pop forpublic speaking engagements, as well as grandstanding about sexism in the military despite his own handling of the army's "Jedi Council" sex scandal, in which demeaning sex videos of women were distributed among a group of soldiers. (It was revealed that Morrison's office knew of the scandal 11 months prior to the former Chief of Army releasing a now-famous condemnation on YouTube of those involved.)

Will Mackay-Sim accept speakers' fees? "I knew nothing about speakers' fees when I accepted the award," he says crisply. "I'm not pursuing money after all, I've spent my life doing public research."

Although he hasn't received any fees to date, Mackay-Sim insists that if they are offered, the funds will be donated to the Hereditary Spastic Paraplegia Research Foundation, his charity of choice.

Mackay-Sim only had a day or so to bask in the glow of being named Australian of the Year before there was a claim his scientific achievements had beenoverstated in the application. A Polish scientist, Professor Pawel Tabakow, after being approached by an Australian journalist in Europe, declared that Mackay-Sim had nothing to do with the world-first surgery using olfactory stem cells that enabled a Polish paraplegic, Darek Fidyka, to walk again. "It is not our business who should be Australian of the Year," Tabakow told The Weekend Australian. "But it is our business when his work is being linked to the surgery of Fidyka. He has no link whatsoever."

The scientific hullaballoo arose from the submission to the Australia Day Council (ADC), which states that Mackay-Sim's research "helped play a central role in proving the safety of science that was a precursor to Dr Tabokow in Poland undertaking the first successful restoration of mobility in a quadriplegic man".

Although Mackay-Sim didn't write the submission to the ADC, doesn't know who did, and never claimed to be involved in Tabokow's work, an artificial straight line was drawn between the two scientists, especially when the word "precursor" was dropped from condensed versions of the ADC's quote in multiple news stories (we'll examine the fallout from the controversy a little later).

Padding amiably about his large, multi-room laboratory, past refrigerator-sized storage cabinets containing cell cultures, past white-coated scientists peering into microscopes, Mackay-Sim seems to be in his element, with every second person saying "Hi", "Hello", or "How are you?" If stem cells are indeedthe microscopic building blocks of the world, this is the tiny universe the scientist feels most comfortable in. But it's a laboratory that now has to hum along without him Mackay-Sim retired late last year, his duties now limited to popping into the university once a week as an emeritus professor.

Later in the day, Professor George D. Mellick, head of Clinical Neurosciences at Griffith, tells me that Mackay-Sim has always set aside time to mentor younger scientists, and to explain sometimes hideously complicated science to a lay audience, but would be the last person to crow about his own scientific achievements.

"One of the things that isn't highlighted very much about Alan's work is his research into Parkinson's. We've been able to learn a lot about Parkinson's by studying cells from people with the disease, and the information coming out of this research will hopefully lead to better treatments."

Back in his office, Mackay-Sim gives me a quick rundown, 101-style, on the human nose. No, the human sense of smell doesn't necessarily decline with age, unless illness or disease set in, and it is astonishingly adept at distinguishing hundreds of thousands of different odours. Yes, women do have a superior sense of smell to men, but the difference is surprisingly only slight. Yes, the first symptom of Parkinson's, before the typical tremors set in, is a reduced sense of smell, as it is with those sufferers who will go on to develop dementia. And yes paws down dogs do have a vastly more powerful sense of smell than humans, although it's impossible to quantify by exactly how much (Mackay-Sim has been known to hide from his spoodle Henry, to measure how long it takes for the dog to find him).

As he relays all this, Mackay-Sim's eyes twinkle and a smile lights up his face: it's easy to see how he'd be the perfect academic for Griffith to call on to schmooze a government minister or potential philanthropist and secure desperately sought-after funding. I ask him about his trademark moustache, which he's had since the early 1990s, when he shaved off a beard. "My wife wouldn't recognise me without it," he jokes. "She says that a small mammal could roost beneath my mouth."

Mackay-Sim, whose double-barrelled surname comes from his paternal grandfather, grew up in middle-class Roseville, on Sydney's leafy North Shore, the third of four brothers. His mother Lois was a nurse during World War II and later a full-time mum while his father Malcolm ran a hardware importing and distributing business, Macsim Distributors (now Macsim Fasteners, owned by Alan's eldest brother, Fraser). At North Sydney Boys' High he was "the opposite of a shit-stirrer. I was vice captain, head of the cadets, played football, was in the rowing team, had a shot at athletics, sang in the choir I did it all."

With wife, Lisa Peine, in Sulawesi, Indonesia, 2007. Photo: Courtesy of Alan Mackay-Sim

After graduating with honours in science from Macquarie University, Mackay-Sim picked up tutoring work in the department of physiology at the University of Sydney, where he completed a PhD on the brain's visual system. Two academic stints in the US followed, first at the University of Pennsylvania from 1979 until 1981, followed by two years at the University of Wyoming, during which time he met his wife Lisa, then living in northern Colorado.

The pair married in 1984, by which time Mackay-Sim had been offered a research role in the department of physiology at the University of Adelaide. He started at Griffith University in 1987, where his research concentrated on the biology of nasal cells.

At the height of the heated moral debate over the use of embryonic stem cells whether the therapeutic potential of stem cells could justify destroying human embryos to extract them Mackay-Sim met Pope Benedict XVI at a Vatican conference in 2005. The Pope congratulated him on his exclusive use of adult stem cells.

"I wasn't avoiding embryonic stem cells for religious reasons," Mackay-Sim explains. "It just so happenedthat I was working with adult stem cells at the time and the conference was looking at alternatives to using embryonic stem cells. But it was a scientific conference and I was impressed with its calibre; the only difference was that men in purple robes were sitting at the back asking questions."

Later in the same trip, Mackay-Sim was invited, along with a host of others, to the Apostolic Palace at Castel Gandolfo the Vatican summer palace. "You feel the history of the Roman Catholic Church, with the Pope coming in with his cardinals and the Swiss Guards," he says. "I'm not a believer, but it was a very powerful experience."

In 2006, the debate over embryonic stem cells virtually vanished when scientist Shinya Yamanaka from Japan's Kyoto University stunned the world by proving that stem cells needn't come from human embryos adult cells can be reprogrammed to act like stem cells, to be returned to an embryo-like state (Yamanaka's discovery won him the Nobel Prize in 2012). "Yamanaka worked out how to genetically engineer any cells so that they had the properties of embryonic stem cells," says Mackay-Sim, who nonetheless continued to focus on adult stem cells only.

Mackay-Sim accomplished his own world first in 2002 when, with the assistance of doctors at Brisbane's Princess Alexandra Hospital, he transplanted olfactory stem cells into the spinal cord of a man crippled in a car accident. The procedure was repeated with two other paraplegic patients at the same hospital and the study wrapped up in 2007.

While the procedures didn't result in any of the patients regaining useful movement in their legs, the results of Mackay-Sim's clinical trials, published in 2005 and 2008, paved the way for further development of olfactory stem cell transplantation.

One researcher who followed Mackay-Sim's trials closely was Geoffrey Raisman from University College London, who visited the Australian team shortly after the first operation in Brisbane to study their work. Raisman later led the British team who worked with Polish surgeon Tabakow on Darek Fidyka in 2012.

Tabakow deployed 100 separate micro-injections of olfactory sheathing cells above and below Fidyka's spinal injury, with the hope these cells would provide a skeleton for nerve fibres to grow and reconnect. A former volunteer firefighter, Fidyka had become paralysed in 2010 after a severe knife attack by the jealous ex-husband of his girlfriend. The repeated stab wounds to Fidyka's back severed his spinal cord, paralysing from the waistdown. (Fidyka's attacker, a fellow firefighter, committed suicide shortly afterwards.)

There's no doubt Tabakow's work was a major advance on Mackay-Sim's research. Tabakow's strategy was to extract ensheathing cells specifically from the olfactory bulbs in Fidyka's nose, grow them in a culture, while also extracting nerve cells from his ankle in a multi-pronged attempt at spinal cord reconstruction. After a series of operations, Fidyka can walk with the assistance of a frame, has regained some bladder control and sexual function, and can ride a tricycle.

Raisman described their new stem cell procedure as "more impressive than man walking on the moon", but it will have be tested on other paraplegics, including those with more severe injuries than Fidyka's, such as car accident victims who have had more of their spinal cord damaged, before it can be declared a reliable method of restoring mobility. As impressive as Tabakow's achievement is, it has still only worked on one patient.

Nobody, however, disputes Mackay-Sim's immense contribution to stem cell transplantation; his work is unimpeachable. If nothing else, he was at the forefront of the science showing that restoring the ability to walk to paraplegics is no longer science fiction. "What I've always said is that we did the first phase of clinicaltrials with olfactory stem cells, and the aim of those trials was to show they were safe," says Mackay-Sim. "That was the first important step."

Mackay-Sim wrote to Tabakow shortly after the controversy blew up, explaining that he didn't write the submission to the Australia Day Council, and was in no way claiming credit for Fidyka's remarkable recovery. "He wrote back a very nice email," says Mackay-Sim. "I believe I've given credit to other scientists in every interview I've given to journalists. I feel comfortable in my behaviour and ethics."

With Prime Minister Turnbull in January this year. Photo: Elesa Kurtz

Mackay-Sim can remember the day when he felt something was wrong terribly wrong. He'd been suffering back pain for months, but dismissed it as old age, or strain from bending over on his bicycle on long rides, and stocked up his pantry with painkillers. "I was in Colorado with Lisa visiting her family, and the pain became so bad I couldn't walk very far. I found the pain eased when I got on my bicycle. I flew home a week before she did; the plane trip back was absolute hell."

What followed was a swift diagnostic journey from his GP to specialists at Brisbane's Wesley Hospital, resulting in a devastating diagnosis. "They suspected something cancerous quite quickly. I didn't realise how ill I was; by this stage, my kidneys weren't coping at all with the antibodies released from my white blood cells, which were going berserk trying to fight the disease. I was at risk of kidney failure and my bones were becoming very fragile. I started therapy almost immediately, in June 2014. Then began the cycles of chemotherapy and stem cell treatment in December."

Since the beginning of last year, however, Mackay-Sim's health has dramatically improved, and even though he's retired to his beachside home in Currimundi on the Sunshine Coast, he is still active in university affairs. He concedes that his health may prevent him from being as active as Rosie Batty, perhaps our most vigorous Australian of the Year to date. But he's already spoken at functions in Brisbane, Sydney and Perth, and will be attending the national March for Science on April 22, which coincides with Earth Day. He moves with the speed and fluidity of a man 10 or 15 years younger.

"I feel very healthy, very energised at the moment," says Mackay-Sim, who is planning a bicycle ride in Italy's Dolomites in July with a couple of mates. (Last year he and his wife went on the Great Victorian Bike Ride, a seven-day ride averaging 85 kilometres a day.)

"I do need to be selective with the number of invitations around Australian of the Year," he concedes, "but I'll do everything I can. After all, what more exciting time could you have to talk about science?"

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Australian of the Year Alan Mackay-Sim on the advantage of being 'an interested scientist' - The Age

Spinal Cord Stem Cells Comments Off on Australian of the Year Alan Mackay-Sim on the advantage of being ‘an interested scientist’ – The Age | April 8th, 2017

Yesterday we highlighted the GTCbio Stem Cell Summit in Boston. Another company we've been closely following is also represented at the conference, Asterias Biotherapeutics (NYSE: AST). Asterias is developing therapies based on pluripotent stem cells to address significant unmet medical needs in neurology and oncology. According to Boston Children's Hospital, pluripotent stem cells are the body's master cells, potentially able to produce any cell or tissue the body needs to repair itself. Like all stem cells, pluripotent stem cells are also able to self-renew, meaning they can perpetually create more copies of themselves.

The company draws its name from the common starfish, Asterias rubens, which has the ability to regenerate lost limbs even in adulthood. The company's lead product candidate, AST-OPC1, is currently in a Phase 1/2a dose ranging trial for spinal cord injury. As Patrick Cox discussed two months ago, Asterias' clinical trial has been generating significant interest.

In the company's earnings call last week, CEO Steve Cartt highlighted the ongoing clinical and corporate progress:

Source: Asterias Biotherapeutics Corporate Presentation, Jan. 24, 2017

We note that the company's cash position is much better than it was a year ago. As shown in the chart above, Asterias' spinal cord trial is funded by a grant from the California Institute of Regenerative Medicine (CIRM), which has certainly eased the burden. As of the end of February 2017, the company's cash and cash equivalents totaled $19.9 million and combined with its available-for-sale securities (mostly BioTime stock - see below*) totaled $33.5 million. Ryan Chavez, CFO, said, "We believe we currently have sufficient capital to fund operations through at least the first quarter of 2018."

* Readers may recall that Asterias was spun out of BioTime (NYSE: BTX) in 2014. BioTime remains the dominant shareholder, controlling 47% of the outstanding common and three out of the nine board seats (with a fourth board seat occupied by Broadwood Capital, BioTime's largest shareholder). Also, as mentioned above, Asterias holds about $13 million worth of BioTime stock. While the kids all seem to be playing well in the sandbox, this close relationship merits watching.

Jane Lebkowski, Asterias' President of Research and Development, was part of an immunotherapy roundtable at the Stem Cell Summit yesterday, discussing the company's research in therapeutics for lung cancer. Today, April 6th, at 10:40am ET, Dr. Michael West, CEO of BioTime, will deliver the conference keynote address entitled "The Promise of Pluripotency in the Manufacture of Advanced Cell-Based Therapeutics" - which sounds like a direct advertorial for a key BioTime asset - Asterias Biotherapeutics.

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Asterias (AST): Blazing the Stem Cell Trail for Spinal Cord Injury - Equities.com

Spinal Cord Stem Cells Comments Off on Asterias (AST): Blazing the Stem Cell Trail for Spinal Cord Injury – Equities.com | April 8th, 2017

Newswise Launched in 2014 with the initial phase I study, this first-in-human clinical trial is evaluating the safety of neural stem cell transplantation in patients with chronic spinal cord injuries. The trial is a collaboration between researchers at UC San Diego School of Medicine, the Sanford Stem Cell Clinical Center at UC San Diego Health and Neuralstem, a Maryland-based biotechnology company.

The trial has been expanded to add four more qualifying participants with chronic cervical injuries involving C5-C7 vertebrae. Due to the intensive follow-up schedule, participants should consider their geographical distance from San Diego. Living within a 500-mile radius of San Diego is recommended. The primary objective is to determine the safety and toxicity of treatment, which involves a surgical intervention with six stem cell injections and a follow-up period of 60 months. Researchers will be using a line of human stem cells approved by the FDA for human trials in patients with chronic traumatic spinal injuries. The stem cells have previously been tested for safety in patients with amyotrophic lateral sclerosis.

The ultimate goal is development of an effective treatment for paralyzing spinal cord injuries, said Joseph Ciacci, MD, principal investigator and neurosurgeon at UC San Diego Health. The immediate goal is to determine whether injecting these neural stem cells into the spines of patients with injuries is safe.

For more information on the trial or participation, contact Ciaccis research office at nksidhu@ucsd.edu or 619-471-3698.

###

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First-in-Human Stem Cell Clinical Trial for Spinal Injury Expands - Newswise (press release)

Spinal Cord Stem Cells Comments Off on First-in-Human Stem Cell Clinical Trial for Spinal Injury Expands – Newswise (press release) | April 4th, 2017

An Italian neurosurgeon is saying he plans on transplanting a head onto a donor body, not in some distant future, but by the end of 2017.

When Dr. Sergio Canavero first announced his plans a couple of years ago, most people thought he was either crazy, or it was a publicity stunt. Now Canavero says he will put the head of 30-year-old Russian Valery Spiridonov on a donor body in December. Spiridonov suffers from Werdnig-Hoffman disease, which is a form of spinal muscular atrophy.

The surgeon said the procedure would take humanity closer to extending life indefinitely.

Although Canavero insists everything is ready to go, a lot of the details remain murky, and it might still be more fantasy than reality.

Dr. David Albert Jones, the director of the Oxford-based Anscombe Bioethics Centre, says the risks associated with such an attempt are not justifiable.

The center is a Catholic academic institute that studies the moral issues surrounding medicine.

The current scientific and medical consensus is that this experiment has very little chance of success, Jones told Crux, adding the most likely outcome is either death during the operation or survival in a paralyzed state for a few hours or days.

Similar experiments have been done with small animals, to little success. No animal has ever come out of the procedure without being paralyzed, and they all have died soon after.

Jones said the studies are not even advanced enough to attempt the procedure on primates such as monkeys or chimpanzees, let alone a human subject.

There is nothing to suggest that the current proposal for a head transplant is realistic, Jones said, adding even if it were, it would not put mankind on a path to immortality.

People who have received donor organs live longer than they would have done, but they do not live longer, on average, than the average life expectancy of the general population, Jones said.

We will all die.

Jones did warn that if immortality became the goal of a society, this could be a real concern because the quest for unachievable goals can detract from the achievable goals of society, the realistic goals of healthcare, education and social solidarity.

Jones responded to some questions from Crux by email, and told us the scientific and ethical concerns about the proposed procedure.

Crux: Is this even possible with todays technology?

Jones: The idea of a head transplant (or a neck down body-transplant) has been attempted in animals but most animals have either died or have been completely paralyzed and none have lived more than a few days. Given the very poor outcome with mice at the present time it is very difficult to justify attempting this with primates, let alone with humans.

A key challenge is reconnecting the spinal cord. Only if we could finally overcome this problem in patients suffering from spinal cord injury (for example, by the use of gene therapy, stem cells and/or growth factors) would it be realistic to deliberately severe the spinal cord and reconnect the head to a different body.

Thought must also be given to the consequences if the body were to reject the new head. Could the head be kept alive apart from the body, and what kind of existence would this be?

Is such a transplant ethically permitted?

The current scientific and medical consensus is that this experiment has very little chance of success. The most likely outcome is either death during the operation or survival in a paralyzed state for a few hours or days.

The risks are such that it is not justifiable even with consent, but there is an added concern in that it seems likely that the patient has been given misinformation about the realistic prospects for success, and in these circumstances it seems doubtful that consent is properly informed.

It should also be noticed that the operation would not only take great financial and human resources but would also require a donor whose heart, lungs, liver, and/or kidneys could have given real benefits to several patients on the organ transplant waiting list. The opportunity costs would, at the very least, involve extending the suffering of these patients and could involve the death of a patient who might otherwise have been saved.

Many are saying that if such a surgery is successful, it puts humanity on the path to immortality. Should such a goal concern us?

There is nothing to suggest that the current proposal for a head transplant is realistic. If some time in the future the technical problems were overcome, it would not be the path to immortality any more than current, very successful, transplant medicine puts people on a path to immortality. People who have received donor organs live longer than they would have done, but they do not live longer, on average, than the average life expectancy of the general population. We will all die.

How can the Church do more to help people assess the morality of new biotechnologies and medical (or pseudo-medical) procedures?

The goal of immortality is unachievable. There is no need to be concerned therefore about the achievement of this goal. On the other hand if (virtual) immortality became the goal of a society, this could be a real concern because the quest for unachievable goals can detract from the achievable goals of society, the realistic goals of healthcare, education and social solidarity.

The virtue of temperateness is needed if society is to avoid such vain and destructive desires. The Church could do more to promote the virtues of temperateness and humility, which are necessary not only in relation to this issue but in the wider context of the care of creation.

How should the governments involved handle such things, both on a national and international level? I mean, it seems odd that this doctor is even being allowed to attempt this procedure, given the objections from many that the technology has not even been tested properly.

Governments should ensure that experimental surgery is subject to the same level of ethical scrutiny as the clinical trials of drugs or of medical devices. Unfortunately surgery is sometimes given a degree of latitude that leaves patients vulnerable to exploitation. Experimental procedures should not be permitted by a hospital unless and until it has been subject to scientific peer review and has satisfied a clinical ethics committee. It is difficult to see how the current proposal could fulfill such criteria.

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Should a head transplant be allowed to happen? - Crux: Covering all things Catholic

Spinal Cord Stem Cells Comments Off on Should a head transplant be allowed to happen? – Crux: Covering all things Catholic | March 31st, 2017

It offers hope for sufferers of diseases hitherto thought incurable

Adult Stem Cell Therapy has gained popularity in developed countries as an alternative to the conventional treatment of many diseases. There are several studies and clinical trials conducted in the United States to support this.

Some current line of treatments are not typically effective, and some can cause detrimental side effects. Medicine is evolving to a more natural and more effective means with the use of stem cells. Due to the numerous religious and ethical issues that comes with the use of embryonic stem cells, todays medicine is moving towards the application of Adult Stem Cell Therapy. This article highlights some new applications of adult stem cells in conditions and diseases that has posed a problem in our society for far too long.

Sickle Cell Anemia

Tissue-based treatments is already evident in the United States of America. In 2012, a patient was successfully cured of sickle cell disease after receiving achemotherapy-free stem cell transplant for sickle cell disease. Additional patients have been successfully treated since then. Two studies conducted in 2014 and 2015 have shown that the use of adult stem cell therapy can greatly reduce complications or even stop the progression of the diseases by providing stem cells to the needed areas. This reduces the need for surgeries for many of these patients.

Diabetes

According to International Diabetes Federation (IDF), there were over 40,000 deaths due to diabetes documented in Nigeria in 2015. Treatment for diabetes has been a focal point for medical research for many years. Consequently, some studies and clinical trials conducted have shown that Adult Adipose (fat) Stem Cell Transplantation can lower and regulate sugar levels resulting in reducing or eliminating the amount of medication or insulin that patients need to take.

In a recently conducted clinical trial, some of the patients achieved insulin independence that remained stable for a median time of 29 months, and another patient for 43 months ongoing. In fact, all the patients studied showed substantial improvement in their dependence on insulin and overall diabetic condition.

Sexual Dysfunction

The emergence of Regenerative Medicine (which includes Adult Stem Cell and Platelet Rich Plasma therapy) has positively impacted the sexual life of both women (O-Shot) and men (P-Shot), and the treatment is also being used for urinary incontinence, etc. There is now ample evidence to show that O-Shot helps women increase their sexual responses, the ability to have Vaginal Orgasm, arousal from clitoral stimulation, sexual desire and natural lubrication, arousal from G-spot stimulation, as well as decrease pain during intercourse and tighten vaginal opening.

Furthermore, P-Shotin men regenerates damaged penile tissues faster and stronger than most traditional treatments. In most cases, treated men see increase in length up to 1 inch or more and girth up to 3/4 inch or more while also increasing their sexual stamina.

Arthritis

Adult stem cells transplantationhas also been studied in arthritis, and there has been some positive reports about its efficacy. In 2014, the effects of stem cells for articular cartilage regeneration was studied.They studied the effect of stem cells injection in treating osteoarthritis of the knee, and the results showed significant improvement.

Neurological Disorders (e.g. Spinal Cord Injury)

The usefulness of stem cell therapy in neurological disorders like Multiple Sclerosis, Cerebral Palsy, Spinal Cord Injury, etc. has been shown in different studies and clinical trials. The prognosis for spinal cord injuries is generally believed to be poor. However, recent researches and case studies are changing this ideology as the value of adult stem cell therapy for patients with spinal cord injuries is emerging.

An example of this can be seen in a case study published in 2015. In this case study, a patient with functional loss below the lesion level due to a motor vehicle accident failed standard therapy but saw clinically meaningful improvements after multiple adult stem cell treatments. Stem cell transplantations over a period of months led to the restoration of the patients ability to move lower extremities against gravity, control the body trunk, and the ability to control the bladder. The patient was also able to stand as well as walk with the aid of hip and knee ortheses. The sensation level also increased.

Conclusion

Regenerative medicine involving adult stem cells is continually being studied and researched to gather more evidence to enable harnessing its clinical potentials. The use of adult stem cells for clinical therapy is now a reality for many patients who were not able to shed the yoke of many diseases that conventional medicine provided very little hope of permanent relief for.

One new innovation is the Umbilical Cord Stem Cells transplantation that is now done without the need for HLA matching. This allows anyone to be treated for conditions or diseases where applicable. In case of sickle cell disease, case studies have been published on the use of Umbilical Cord Stem Cells from HLA matching donors to remedy sickle cell disease; however, case studies are yet to be published on the efficacy of the Umbilical Cord Stem Cells not requiring HLA matching for the treatment of sickle cell disease. I am cautious to note that the results may differ from case to case, so not every sickle cell patient will be a good candidate for the treatment.

Currently, Adult Stem Cell Therapy is now seen as a viable therapeutic alternative for joint and back pain, sexual dysfunction, diabetes, End Stage Renal Disease on hemodialysis, arthritis, etc. The treatments are gaining popularity among patients and doctors because it is natural and can help repair and regenerate most parts of the human tissues.

Ikudaiyisi is the Medical Director of Glory Wellness and Regenerative Centre in USA, Lagos and Abuja and can be reached on info@glorywellness.org

Excerpt from:
How Adult Stem Cell Therapy Is Breaking Glass Ceiling Of Modern Medicine - Leadership Newspapers

Spinal Cord Stem Cells Comments Off on How Adult Stem Cell Therapy Is Breaking Glass Ceiling Of Modern Medicine – Leadership Newspapers | March 31st, 2017

OSAKA -- Researchers transplanted induced pluripotent stem cells derived from donors into a patient in Kobe Tuesday, in the first such trial using cells not from the patient -- an advance with hopeful implications for Japanese regenerative medicine.

Research institute Riken worked with the Kobe City Medical Center General Hospital, as well as Kyoto University and other partners, to make the procedure a reality.

The patient, a Hyogo Prefecture man in his 60s, suffered from age-related macular degeneration, an intractable eye disease that can cause blindness. He received in his right eye an injection of a solution containing 250,000 retinal cells grown from iPS cells. The procedure began before 2 p.m. at the Kobe hospital and lasted about an hour.

The operation went smoothly, Yasuo Kurimoto, director of ophthalmology at the hospital, told reporters. Masayo Takahashi, head of the project at Riken, said the researchers had reached about the halfway point in their pursuit of the practical adoption of iPS cells.

The institutions hope to perform the transplants for five patients in all to determine the safety of the process. The transplants are not expected to improve their eyesight much, but should eliminate the need to take medicine regularly and keep vision from deteriorating further.

Riken performed the world's first such procedure in 2014, but used iPS cells derived from the patient. The process drew notice for its cost and lengthiness, taking roughly 100 million yen ($905,600 at current rates) including inspections to make sure the cells would not become cancerous, and around 10 months.

The patient appeared to be doing well two years later, and none of the injected cells appeared to have turned cancerous or otherwise behaved abnormally. Her disease appeared to have halted, with no noteworthy side effects.

In Tuesday's procedure, using stock iPS cells carefully tested for safety, Riken and its partners expect costs of just several million yen -- less than a tenth those of the first trial. The patient also reportedly waited just about six weeks for the procedure after agreeing to it.

Researchers seek to use iPS cells to treat conditions including spinal cord injuries as well. Donor iPS cells may make this possible for such injuries and other urgent operations for which gathering cells from patients would take too long.

Riken and the other institutions intend to carefully observe the new patient to ensure the procedure's long-term safety. It is possible the patient's body could reject the transplanted cells. The results should be announced two or three years down the road, as it will take time to see the case through fully, said Takahashi.

Using donor cells requires thorough oversight. Kyoto University stopped supplying iPS cells grown from umbilical cord blood to outside institutions in January due to the possibility that an incorrect reagent was used in the cells' creation. The university then announced a research partnership with the biomedical company Takara Bio dedicated to ensuring the quality of iPS cells.

(Nikkei)

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Japan performs first transplant of donor iPS cells - Nikkei Asian Review

Spinal Cord Stem Cells Comments Off on Japan performs first transplant of donor iPS cells – Nikkei Asian Review | March 29th, 2017

Dr. David Ikudayisi is the Medical Director, Glory Wellness & Regenerative Centre, a multi-specialty health care center, in Lagos and Abuja. Ikudayisi who is based in the United States said he is excited about the prospects of coming back home to help put Nigeria on the continental and global medical map through regenerative medicine. He also spoke on the controversy surrounding embroyonic stem cell therapy among others.

What is regenerative medicine allabout?

Regenerative Medicine is a branch of medicine that aims to restore normal function by repairing or replacing damaged or malfunctioning cells and tissues in patients who have lost tissue or organ function due to age, disease, or congenital defects. It comprises different components including, Platelet Rich Plasma (PRP) Therapy and Adult Stem Cell (ASC) Therapy, etc.

I was inspired into this branch of medicine when I was looking for alternative ways to alleviate my patients pain in USA without using addictive pain medications and frequent steroid injections. I discovered the benefits of the Platelet Rich Plasma and/or Adult Stem Cell Therapy for patients who were in need of joint pain relief, youthful appearance, or a restored sexual function.

In medical school, we were taught that the central nervous system rarely regenerates, that there is little or no hope for paralyzed patients, and that damaged brain tissue may be a permanent condition, just to name a few.

Nowadays, the re-growth of brain cells and improvements of neurological function in spinal cord injured patients have been documented. When applicable, adult stem cell treatment is basically a medical time machine. The results doctors see in medical practice every day is what keeps my drive for the advancement of regenerative medicine.

How will regenerative medicine help address health care challenges in Nigeria?

Nigeria has a lot of ways to go when it comes to health, as we are recording very poor health indices in recent times. This can be traced to not enough emphasizes on preventive medicine or regenerative medicine. We tend to lose hope at different stages of degrading health rather than come up with solutions that can be attainable no matter the initial cost. If taken without levity, Regenerative Medicine, as in Platelet Rich Plasma (PRP) Therapy & Adult Stem Cell (ASC) Therapy, can help reduce our mortality rate in Nigeria, thereby recording better health indices.

This branch of medicine holds answers to many questions and problems that we doctors used to believe had no solutions. Many medical conditions that we thought were not treatable are now treatable. We have ample evidence to show the potentials of regenerative medicines for ailments that have domineered our people. The use of these techniques will propel Nigerian healthcare to boundless heights if provided the opportunity.

Regenerative medicine has a vast amount of uses especially for people who seek to stop being dependent on taking medications daily, avoid surgery, feel younger and more energized, perform their marital enjoyment at older ages, and prevent the manifestation of some complications of diseases.

Many countries around the world are taking advantage of these therapies, and Nigeria should not be left behind. I know this is not an undertaking with quick payoff nationally, but I believe that with these innovations, Nigeria can be the centre for medical tourism in Africa. This is not a cheap option, as most novel innovations never are, but it will pay dividends in the long run, and we have seen proofs of that.

Some doctors are already offering regenerative medicine (PRP therapy and ASCT) in Nigeria. So, people dont have to travel abroad to benefit from regenerative medicine. Just ask your doctor if someone in your area is offering ASCT and PRP Therapy.

What illnesses does ASCT and PRP therapy treat?

The applications of ASCT are enormous, and there is much more to be discovered. To determine if this can be of use in a particular medical problem, just ask if there is a need for repair and/regeneration of any part of the human tissue/organ: if yes, then the ASCT with/without PRP therapy may be an option that will help. The exceptions are non-hematological cancer treatments, as these treatments with stem cells are under investigations/research using tissue engineering.

More specifically, these therapies can be used for issues like multiple joint pain, back pain, meniscal tears, ligament tears, avascular necrosis of the hip joints, facet arthropathy, hair thinning, erectile dysfunction, female sexual dysfunction, female urinary incontinence, cosmetic/aesthetic applications (vampire facial, vampire facelift, vampire breast and nipple lift); diabetes, hypertension, anti-aging (generalized treatment), chronic kidney disease, multiple sclerosis, cerebral palsy, spinal cord injury, COPD (lung disease) and infertility (helps to increase fertility chances and not serve as a cure), to mention a few.

Why is there controversy around embryonic stem cell therapy?

The ethical, religious and moral arguments for and against Embryonic Stem Cell use has been stressed for many years now. Stem cells possess the ability to make new cells needed in the body. Embryonic stem cells are an example of this. In the body, they are what we use to develop the cells we later go on to have.

Many scientists prefer it because of its endless possibilities to recreate virtually any type of cell in the body. However, it involves the use of embryos from day 6 to day 14; that is, a baby-to-be in the first two weeks of pregnancy or after In vitro fertilization (IVF). As you can see, this involves tampering with potential or future babies that are yet to be born. This has both religious and ethical issues, leading to the controversy around it.

What is the difference between adult stem cell therapy and cloning?

It is important to understand that there are three main types of stem therapies. In addition to Embryonic Stem Cell (ESC) therapy, there are also induced Pluripotent Stem Cells (iPSCs) therapy and Adult Stem Cells (ASC)therapy. iPSCs are produced in the lab by reprogramming adult cells to express embryonic stem cells characteristics, and Adult Stem Cells (ASC) are retrieved from individuals bone marrow, adipose (fat) or umbilical cord. Although ASCs can be used for a vast number of therapies, they do not possess the same capabilities as ESCs, but the downside of limited growth and differentiation makes ASCs applicable in medicine today.

Cloning on the other hand is the process of producing similar populations of genetically identical organisms to sometimes replace damaged or lost tissues or organs. Every single bit of their DNA is identical to the original specimen. Clones can happen naturally, like identical twins or triplets, or they can be made in the lab, like Dolly the sheep. Reproductive cloning for humans has been banned in several countries, and mainstream scientists consider it unethical.

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'Nigeria should harness potentials of regenerative medicine' - Daily Trust

Spinal Cord Stem Cells Comments Off on ‘Nigeria should harness potentials of regenerative medicine’ – Daily Trust | March 27th, 2017

Malia

March is Multiple Sclerosis Month and Hadassah leads the world in the stunning success and implementation of the research at Hadassah Hospital in Jerusalem. Malia is just one of the many stories of triumph in Prof. Dimitrios Karussis program.

Malia was a top Dallas trial attorney-a senior partner in a law firm and the mother of three children, the youngest, age five. Her energy level was high and her life was full. And then, 18 years ago, she was diagnosed with multiple sclerosis (MS).

In the beginning, her symptoms were mild. For example, she was a little unsteady in her walking. She began taking a weekly injection of Avonex because, as her doctor told her, "You wouldn't want me to treat you for high blood pressure only after you had a heart attack."

Malia took Avonex for about 10 years. Still, her life became a journey of declining energy and mobility. Her biggest fear was that she would lose her vision and not be able to drive her children around. Fortunately, she didn't experience any problems with her vision, but her gait did get worse, as did the fatigue.

Ultimately, she could only walk a very short distance with a walker. Her balance became more unsteady. Her speech was harder to understand. And the fatigue got so bad that even taking a shower was so exhausting that she needed a nap afterward.

Malia tried four or five different medicines, but none seemed to help. As she explained, however, the promise of these medicines is only to slow the progression of the disease, not to improve upon one's symptoms.

At one point on this odyssey of decline, Malia fell and broke her leg and found herself in a wheelchair for six weeks. She began to research other treatment options, surfing the Internet extensively. It was how she came upon the innovative stem-cell work of Prof. Dimitrios Karussis, senior neurologist at Hadassah Hospital Ein Kerem and head of its Multiple Sclerosis Center. She learned that Prof. Karussis was conducting a unique MS clinical trial involving the injection of a patient's own bone marrow-derived stem cells directly into the spinal cord.

When she met Prof. Karussis for the first time, she was walking with a walker, but couldn't walk any real distance. It was December 2014. In the spring of 2015, Malia, who was accepted into one of Prof. Karussis' clinical trials, traveled back to Hadassah to have a bone marrow extraction, which would be the basis for her stem-cell transplant. As she explains, just a syringe of cells is extracted, but then these cells are enhanced in culture for six months. In the fall of 2016, Malia received her first infusion. The actual infusion, itself, Malia relates, took only five minutes!

"I got the infusion at 2 p.m.," Malia recalled, "and eight hours later, I did something I had not done in two to three years." She explained that before the infusion, whenever she got out of bed, to get back in, she would have to lift her right leg with her hands. This time, post infusion, after she got up to go to the bathroom, she was able to get back into bed by just lifting her right leg onto the bedwithout holding it! She called the kids; her husband, David, made a video of her new ability. When her doctors came in that next morning to see how she was doing, she showed them her new skill. "Everyone was sobbing," she recalled; "we were all so excited by how amazing it was."

Malia said: "The most important thing of all was that the level of my fatigue was so improved."

Taking herself back to the night she returned home from that first infusion, Malia recalled: "We arrived back home at midnight. The next morning at 8, I was up writing my blog. Then I unpacked, did laundry, and made a five-course dinner for my husband, after which he went to bed, exhausted from the jet lag. I, however, stayed up to crochet a new blanket until midnight."

That renewed energy has never disappeared since Malia started getting stem-cell infusions at Hadassah. She received two more in 2016 and the most recent in February 2017. The kind of improvements varied with each infusion-sometimes the improvement in her gait was most noticeable; other times, it was the clarity of her speech. With the 2017 infusion, it was her balance. A year or so ago, she could only balance herself on a "whole body vibration machine" for about a minute and a half. Then she worked this up to five minutes. After this fourth infusion, she could stay on the machine for 12 minutes and even let go of the bar with one hand.

In addition, before every infusion, a baseline measurement of her abilities is taken. The most recent baseline, Malia reported, was higher than ever before. By the same token, her overall physical improvements are the best they've been.

Malia's recovery is not without its setbacks. As the stem-cell treatment wears off, she does experience some backsliding in her improvements-perhaps in her speech or the way she walks. Nevertheless, after she has a treatment, the improvements return or surpass the previous ones.

"I had forgotten what it was like to feel like a regular person, like someone with a normal level of energy; I had forgotten what it was like to feel good," Malia said.

When she is out and about with her walker, she sees people looking at her with sympathy. But she doesn't feel sorry for herself, she notes, because she has gotten her life back. Malia feels blessed that she has come this far. "Until you've been there, you can't really understand just how important it is to feel like a regular person."

Malia had stopped practicing law when she was diagnosed with MS. But just recently she reactivated her law license. She took on a case, with a colleague. And in the beginning of April, she will be going to trial.

"When you feel like you can do things you used to do, life is as it should be," Malia said.

Malia no longer takes any medication for MS and being an MS patient is no longer a central focus of her life. But, she is now a stem-cell activist and fundraiser on behalf of Hadassah because "I feel I have been given a gift and I want to ensure that more people with MS can be helped."

While she doesn't know the other participants in the clinical trial, she did meet one man from London last time she was at the hospital for her infusion. As she relates, this man was "giddy with excitement, lifting his cane in the air with one hand." They began to talk and he explained that he just had his first stem-cell infusion and, overnight, he was able to lift his cane up in the air with his right hand when for 15 years he had been unable to even hold anything in that hand.

"What I would like Hadassah supporters around the world to understand," Malia related, "is that yes, I get the wonderful benefits of Prof. Karussis' stem-cell treatments (and, of course, she does not minimize his trailblazing hard work and miraculous success), "but I also was surprised to learn that I have this large Hadassah cheering squad that gives me hope and supports me-making me believe in possibilities for improvement. As Malia recalled, when she sits with a visitor from Hadassah and sees the look of awe on the face of this Hadassah supporter because of the leap in her improvement, it gives her renewed strength. "There are many things I want to do with the rest of my life," Malia said, "and raising money for Hadassah is at the top of the list."

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Hadassah is winning the battle with MS - Heritage Florida Jewish News

Spinal Cord Stem Cells Comments Off on Hadassah is winning the battle with MS – Heritage Florida Jewish News | March 24th, 2017

A small stem cell trial in which patients with severe spinal injuriesappeared to make remarkable progress is still showing excellent results, according to the company conductingthe research.

One of the patients in the trial is 21-year-old Kris Boesen, from Bakersfield, California, whose story we reported on last year.A car crash had left theBakersfield, California native with three crushed vertebrae, almost no feeling below his neck, and a grimprognosis. Doctors believed he would live the rest of his life as a paraplegic.

Enter stem cell therapy. Most treatments for serious spinal injuries concentrate on physical therapy to expand the range of the patients remainingmotor skills and to limit further injury, not to reverse the actual damage. But last April, as part of an experimental phase 2 clinical trial called SCiStar, researchers injected Boesen with 10 million stem cells. By July, hehad recovered use of his hands to the point where he could use a wheelchair, a computer and a cellphone, and could take care of most of his daily living needs.In recent months his progress has continued, says his father.

Boesen is not the only patient to have improved in the trial, according toAsterias Biotherapeutics, which is conducting the research. Boesen is part of a cohort of six patients who were experiencing various levels of paralysis and were injected with the 10 million stem cell dose. In a Jan. 24update, the company saidfive of those patientshad improved either one or twolevels on a widely used scale to measuremotor function in spinal injury patients.

On Tuesday, Asterias issued a newupdate, announcingthat the sixth patient in the cohort has experienced a similar improvement.

While spontaneous recovery for spinal injury patients does occur,the likelihood of all six patients recovering to the degree they haveis less likely, researchers say.

This is as good as you could hope at this point, said Charles Liu, Boesens neurosurgeon and director of the USC Neurorestoration Center. So far all the evidence is pointing in the right direction.

To measure improvement in spinal injury patients, researchers use two yardsticks: the Upper Extremity Motor Scale, or UEMS, and the International Standards for Neurological Classification of Spinal Cord Injury, or ISNCSCI. On the UEMS scale,patients are scored from 0 to 5 on theirability to use five key muscles in the wrists, elbows and fingers. The ISNCSCI scale assesses where damage has occurred along the different levels of the cervical vertebrae, which generally determines the scope of impairment to the body and the level of care needed.

For instance, if a patient has sustained damage at the fourth cervical vertebra down, known as C-4, at the base of the neck, it generally means that person is paralyzed from the neck down, requiring round-the-clock care and a ventilator to breathe.A patient with a C-5 injury may not be able to move her arms or hands, requiring about 6 to 12 hours per day of assisted care; and at the C-6 level, better motor function mayallow a patient to take care of most of herdaily living needs on her own.

Which is all to say that even one level of recovery could substantially improve the daily life ofa spinal injury patient.

According to Asterias, all six patients in the 10million-cell cohort have improved their general UEMS scores, and jumped at least one motor level on the ISNCSCI scale on one or both sides of their body.

Two patients have improvedtwo motor levels on one side; and one patient,Boesen, has improved two motor levels on both sides.

Steve Cartt, president and CEO of Asterias, said anotherpatient, Jake Javier of Danville, California, has gonefrom partial paralysis to being able to use his hands well enough to considerpursuing a computer science career.

Throws Like a Regular Throw

In September, Boesens father, Rod Boesen, told us how excited he wasthat his son had regained some feeling in one of his feet. Last week, at11 months post-injection, the elder Boesensaid Kris has continued to improve.

Now he can move his toe and his knee together at the same time, Boesen said. Theyre about to give him a manual wheelchair now [instead of a motorized one]. He can grip with his hands enough to use a manual one.

Boesen said the movement in his sons arms and hands has greatlyimproved since September.Kris, a formerhigh school pitcher, had beenflinging a ball to his dog like people throw hand grenades, Boesen said. They kind of cradle them and thats how Kris would do it. But now he throws like a regular throw, tosses that ball down the hall, has that release point down, and just wings it.

Asterias is currently recruiting patients for a trial in which theyll receive 20 million stem cells, the optimal dose, according to company researchers. Two patients have already started the 20 million stem cell therapy, and six-month results from those patients will be released in the fall, Cartt said.

Patients who received 2 million stem cells in an earlier phase of the study have not shown much change in their condition, according to the Jan. 24 update.

Guarded Optimism

While Boesens father is impressed with the results, the optimism of researchers inside and outside the studyhas been guarded.The trial is still in its early stages, and the sample size is small, said Paul Knoepfler, a cell biology professor and stem cell researcher at UC Davis, who is not involved in the SCiStar study.

As a scientist, I still would want to wait for more data, Knoepfler said. Its certainly interesting, but its still early. Its a phase 2 trial.

To address the issue of small sample size, Asterias islooking at historical data to determinethe level of improvement for patients in similar circumstances who did not receive stem cell therapy. The company has said it found a meaningful difference in the recovery of its study patients compared to the norm.

Liu said one of the most importantresults is the lack of significant side effects or other negative outcomes resulting from the treatment to date.

Thats very significant to me, Liu said. Thats the first thing you look for, is anyone hurt from this therapy.

There was also a concern, he said, that some patients might regress over time, once the initial injection of stem cells wore off. Thathasyet to occur.

No one has lost anything theyve gained, Liu said. We were very happy to see that. This is all very promising.

The next step for the SCiStartrial will be to establish a control group, Cartt said.

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Strong Progress for Paralyzed Patients After Stem Cell Therapy, Company Says - KQED

Spinal Cord Stem Cells Comments Off on Strong Progress for Paralyzed Patients After Stem Cell Therapy, Company Says – KQED | March 23rd, 2017

For a soldier who suffered a spinal cord injury on the battlefield, the promise of regenerative medicine is to fully repair the resulting limb paralysis. But that hope is still years from reality.

Not only powerful, but efficient. Studying diseases in lab-created tissue may help reduce the price tag now roughly $1.8 billion for bringing a new drug to market, which is one of the reasons Ashton received a National Science Foundation CAREER Award for advancing tissue engineering of the human spinal cord. During the projects five-year funding period, his lab in the Wisconsin Institute for Discovery will fine-tune the technology for growing a neural tube, the developmental predecessor of the spinal cord, from scratch.

As the neural tube matures and diversifies during the development of a human embryo, it gives rise to the two core parts of our central nervous system: the brain and spinal cord. By replicating this process in a dish, Ashton hopes to develop a platform for research that is highly reproducible and can be broadly disseminated. Biologists could simply add their cells to Ashtons starter tissue to build a model of whichever spinal cord disease they desire.

By starting with cells from an individual patient, researchers will be able to target disease therapies to a particular genetic background a concept known as personalized medicine. Drug tests in engineered spinal cords may become an intermediate step between animal models and clinical trials of patients affected by Lou Gehrigs disease, multiple sclerosis or spinal cord injuries, helping to bridge the differences between a human and rodent spinal cord.

Weve cured spinal cord diseases in a lot of rodents over the years, but only a small percentage of those drugs work in humans, Ashton says. If we can make the engineered tissue as close as possible to whats in our body, this will eventually translate to better drugs.

The recipe Ashton and fellow UWMadison engineering professors Lih-Sheng (Tom) Turng and David Beebe plan to follow to coax stem cells into forming a neural tube goes something like this: First, they use water-soluble Lego-type molds to create microscale cavities within a jelly-like substance. Then they add human neural stem cells into these cavities, and let the cells coalesce as they do naturally to form neural tube-resembling tissue.

Next, they add signaling molecules whose variable concentration instructs the stem cells to turn into different types of neurons and neuron-supporting cells. Last comes the greatest challenge: getting these specialized cells to connect to one another and form electric circuits that give the spinal cord its function.

The complexity of the central nervous system exists because specific circuits have to form over very long distances. If any part of a circuit goes awry, you lose function, Ashton explains. The biggest open question is whether the tissue we create in vitro will have the proper wiring of different cell types to yield circuits similar to those in our body.

Ashtons CAREER Award will also fund educational activities that range from expanding outreach programs for underrepresented minority K-12 students, to creating a website and exhibit for the public, to educating Madison-area high school teachers about tissue engineering.

Ashton, who is African-American, comes from a socially active family background his grandfather was a minister and president of the Virginia chapter of the NAACP. He has assisted the nonprofit group 100 Black Men of Madison with its K-12 mentoring programs for years.

He plans to use the new grant to develop virtual interactive lab experiments and matching teaching modules.

I hope to attract more students to the fast-growing field of regenerative medicine, Ashton says, and to motivate people to continue to fund this kind of research so that we can develop therapies to cure diseases, instead of just treating their symptoms.

This article has been republished frommaterialsprovided by University of Wisconsin-Madison. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Growing a Neural Tube in a Dish | Technology Networks - Technology Networks

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Men's Fitness

Want more stem cells and a higher pain tolerance? Try shocking yourself. Men's Fitness The acupuncture stimulus we're giving these animals has a rapid effect on neuroanatomical pathways that connect the stimulus point in the arm to responsive neurons in the spinal cord and into a region in the brain called the hypothalamus. In turn, the ...

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Want more stem cells and a higher pain tolerance? Try shocking yourself. - Men's Fitness

Spinal Cord Stem Cells Comments Off on Want more stem cells and a higher pain tolerance? Try shocking yourself. – Men’s Fitness | March 22nd, 2017