Stem Cell Research

12
Dec 2016
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bayer

A major boost for Canada’s stem cell sector: Bayer/Versant Ventures invest $225 million

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The announcement by Bayer AG and Versant Ventures that they will invest $225 million U.S. to create a Toronto-based cell-therapy company shows the massive opportunities to be realized in the next wave of stem cell R&D and illustrates how visibility and support for the sector is soaring.…

The announcement by Bayer AG and Versant Ventures that they will invest $225 million U.S. to create a Toronto-based cell-therapy company shows the massive opportunities to be realized in the next wave of stem cell R&D and illustrates how visibility and support for the sector is soaring.

“We think we’re on the cutting edge of the next generation of stem-cell therapies,” said Brad Bolzon, Managing Director of Versant Ventures in a report by the Globe and Mail.

According to Bayer’s press release, the new company, called BlueRock Therapeutics, will advance breakthrough treatments based on latest stem cell technology with an initial focus on finding treatments for cardiovascular diseases and neurodegenerative disorders like Parkinson’s disease, two areas where Canadian research is particularly strong.  The investment, one of the largest-ever first-round financings for a biotech company, gives BlueRock Therapeutics at least four years to get a number of programs into the clinic.

It comes in the wake of several developments including the Stem Cell Network’s announcement of $9 million in funding for projects to turn research into new treatments and the Canada First Research Excellence Fund’s $114-million grant to the University of Toronto’s Medicine by Design program.  As well, the Centre for Commercialization of Regenerative Medicine announced earlier this year that it will receive $20 million in federal funds, matched by GE Healthcare, to develop cell manufacturing capacity in Canada.

“All of these announcements align with the Canadian Stem Cell Strategy’s goals of mobilizing private capital and attracting investment,” said James Price, President and CEO of the Canadian Stem Cell Foundation.  “They are strong indications that Canada is well positioned to lead in the next wave of stem cell advances.  That’s why we’re seeking a commitment from the Liberal Government to make stem cell research and development a national priority.”

The Canadian Stem Cell Strategy is designed to deliver 10 new therapies to the clinic within 10 years, create 12,000 jobs and position Canada as a global leader in the field.  It calls on the Government to provide one-third of the total investment, about $50 million annually over 10 years for a total of $500 million, to be doubled by $1 billion in private and philanthropic investments.

An indication of the economic power of stem cell R&D is exemplified by BC’s STEMCELL Technologies Inc., which, with 900 workers and $150 million in annual revenues, has become Canada’s largest biotech by selling high-quality “Made in Canada” stem cell products worldwide.

“We have a huge opportunity in front of us, an opportunity we don’t want to miss,” said Mr.  Price.  “Visibility and support for the sector has never been stronger.  We just need to take the next big step forward with the Strategy.”

 

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23
Aug 2016
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Dr. Michael Rudnicki

Dr. Michael Rudnicki

Ottawa researcher receives $4.9 million grant

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Congratulations to Dr. Michael Rudnicki at The Ottawa Hospital who has been awarded $4.9 million to further his research into stem cells and muscle regeneration.…

Congratulations to Dr. Michael Rudnicki at The Ottawa Hospital who has been awarded $4.9 million to further his research into stem cells and muscle regeneration.

Dr. Rudnicki, who is also Scientific Director of the Stem Cell Network and a University of Ottawa professor, received the grant from the Canadian Institutes of Health Research (CIHR) as part of its Foundation funding competition to provide the country’s top health researchers with stable, long-term support.

His colleague Dr. Lynn Megeney also received funding, under CIHR’s Project competition, to study heart muscle regeneration and remodeling.

Dr. Rudnicki is one of the world’s leading researchers in stem cells and muscle regeneration. Last fall, his lab published a paper in Nature Medicine that could completely alter perceptions on how Duchenne muscular dystrophy happens — linking it to intrinsic defects in the function of muscle stem cells

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09
Aug 2016
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Dr. Molly Shoichet

Dr. Molly Shoichet

Breakthroughs can produce a lot of hope, but also frustration

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You read about a stem cell breakthrough that could lead to a whole new way of treating — maybe even curing — a disease. …

You read about a stem cell breakthrough that could lead to a whole new way of treating — maybe even curing — a disease. Then … nothing happens.

The gap in time between a medical science discovery and actual clinical application can seem like a millennium, especially if you are living with the disease in question.

This week in the Toronto Star, Dr. Molly Shoichet articulately — and compassionately — explains why it takes so long to move from the “Eureka!” moment in the research lab to a “We can treat this” scenario at the hospital bed.

She knows first-hand, having worked with a University of Toronto team including Drs. Derek van der Kooy,  Cindi Morshead, Brian Ballios and Michael Cooke that created a hydrogel to help transplanted stem cells thrive in the brain and eye. By injecting photoreceptor cells, the team was able to restore vision by approximately 15% in blind mice.

Their 2015 discovery garnered plenty of media attention, sparking hope that people with macular degeneration — a currently incurable condition — could have a new treatment. More than a year later, no such treatment is available or even on the horizon.

“For many people with macular degeneration, or any degenerative disease for that matter, studies like mine often produce a lot of hope, but also frustration that the ‘product’ is not available to them,” she writes in the Star.

As Dr. Shoichet explains, it can easily take 17 years or more to move a discovery from the lab to the patient.  It takes lots of testing with animals before a new treatment can safely move to the three stages of human clinical trials, with all the attendant regulatory controls and approvals.  It involves plenty of hard, painstaking work. And it costs lots of money.

But Dr. Shoichet remains optimistic. The opportunity to improve the lives of people with deadly diseases is what drives scientists to get up in the morning and get to work.  As she writes, “I am optimistic we will deliver on the promise of regenerative medicine — but it won’t happen right away.”

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06
May 2016
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Potent cells could spare preemies from long-term lung troubles

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Dr. Bernard Thébaud

When babies are born prematurely — especially at fewer than 28 weeks in the womb — their lungs are not fully formed and they need help to breathe. …

Dr. Bernard Thébaud

Dr. Bernard Thébaud

When babies are born prematurely — especially at fewer than 28 weeks in the womb — their lungs are not fully formed and they need help to breathe.  At neonatal units in hospitals around the world, doctors put these babies on ventilators to give them additional oxygen.

Unfortunately, the very device that keeps these tiny babies alive can create life-long consequences by triggering bronchopulmonary dysplasia (BPD).

“Approximately 40% of extreme pre-term babies will get BPD,” says Dr. Bernard Thébaud, a researcher and clinician with The Ottawa Hospital and the Children’s Hospital of Eastern Ontario. “They will have a higher risk of developing asthma and of being re-hospitalized after being sent home. They may be at risk of early onset aging of their lungs — something similar to smoker’s lung.”

There is no treatment for BPD.  Steroids that decrease the inflammation it causes can also inhibit brain development.  “So we’re very cautious in administering steroids,” says Dr. Thébaud, who is working on a stem cell solution to the ventilator/BPD conundrum.

In mesenchymal stem cells extracted from Wharton’s jelly, a gelatinous substance within the umbilical cord, Dr. Thébaud hopes to find a BPD treatment that will also help other organs thrive.

“These cells will do several things.  They will attenuate the inflammation, but also promote lung growth. In addition, we believe their benefits will not be limited to the lung.  They will also have some beneficial effects on the brain, the gut and other organs in preterm babies that still need to grow and need to be protected from injury. “

So far, Dr. Thébaud, who is also a professor of pediatrics at the University of Ottawa where his holds the uOttawa Partnership Research Chair in Regenerative Medicine, has only tested the mesenchymal stem cells on animals. But the results have been encouraging, with the stem cells protecting the lungs from oxygen induced injuries. “We still have to do further tests with the cells made in a Good Manufacturing Process facility for a clinical grade product.”

He looks forward to conducting a Phase 1 clinical trial in the near future to test the feasibility and safety in newborns.  “If it’s safe, we’ll move to Phase 2 and enroll more babies.”

As a researcher who cares for these fragile babies every day, Dr. Thébaud is both frustrated and hopeful.

“When we see these babies in our unit and we discuss what we can offer, we can see how the disease evolves and we know that we will be stuck at one point. Sometimes a nurse will come up to me and say, ‘Oh, Bernard, this baby needs your stem cells. Can you bring some over from our lab?’  On one hand it’s frustrating. On the other, it’s very rewarding to see that everyone recognizes the potential of this therapy and is waiting for it.”

The beauty of these mesenchymal stem cells, also called stromal cells, is that they are easily harvested from umbilical cords that would otherwise be discarded as medical waste. They are also easy to expand in a lab, generating the millions of cells required for transplant.  Because no genetic match is required between donor and recipient, they can be supplied off-the-shelf. And they are powerful.

“Because the cells are young, they might be more potent than bone-marrow-derived mesenchymal stem cells,” says Dr. Thébaud.  “So it could be that the product we are developing for the babies could also be beneficial in treating adult diseases.” and superior to current bone marrow-derived stem cells.”

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25
Apr 2016
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Dr. Arezu Jahani-Asl

Dr. Arezu Jahani-Asl

Researchers take aim at protein in fight against brain tumours

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Researchers believe they have found a key piece to the puzzle of brain tumour formation.

A study published today in Nature Neuroscience shows that glioblastoma tumours need a protein called OSMR (Oncostatin M Receptor) to form.…

Researchers believe they have found a key piece to the puzzle of brain tumour formation.

A study published today in Nature Neuroscience shows that glioblastoma tumours need a protein called OSMR (Oncostatin M Receptor) to form. Glioblastoma is one of the most deadly cancers, resistant to radiation, chemotherapy and difficult to remove with surgery.

“The fact that most patients with these brain tumours live only 16 months is just heartbreaking,” said Dr. Arezu Jahani-Asl, lead author of the study. Dr. Jahani-Asl, an assistant professor at McGill University and a principal investigator at the Jewish General Hospital, did much of the research while she was a postdoctoral fellow co supervised by Dr. Michael Rudnicki at The Ottawa Hospital and the University of Ottawa and by Dr. Azad Bonni from Harvard Medical School and Washington University School of Medicine.

“Right now there is no effective treatment, and that’s what drives me to study this disease,” said Dr. Jahani-Asl.

The research team studied human brain tumour stem cells taken from glioblastoma patients. While it was previously believed that any cancer cell could reproduce to form a whole tumour, researchers have since learned that in brain cancer only a few kinds of cells have this ability. If a single one of these brain tumour stem cells is left behind after surgery, it can create a whole new tumour.  Working with mice, the research team found that blocking OSMR activity in these cells prevented them from forming brain tumours.

“Being able to stop tumour formation entirely was a dramatic and stunning result,” said Dr. Rudnicki, senior co corresponding author of the study. “It means that this protein is a key piece of the puzzle, and could be a possible target for future treatments.”

Dr. Bonni, senior co-corresponding author, said that while the results are exciting, there is much more work to be done.  “The next step is to find small molecules or antibodies that can shut down the protein OSMR or stop it from interacting with EGFR (the epidermal growth factor receptor that drives tumour formation in glioblastoma).  But any human treatment targeting this protein is years away.”

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14
Apr 2016
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Stem Cells 101

Stem Cells 101: Forums offer hype-free facts

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If everything you’ve ever heard was true, cure-all stem cells would soon be available at your corner drug store or clinic.…

If everything you’ve ever heard was true, cure-all stem cells would soon be available at your corner drug store or clinic.

But it’s not that simple.

Which is why the Ontario Institute for Regenerative Medicine is co-sponsoring Stem Cells 101 — a series of free public events in four Ontario cities beginning next week.

The sessions, held in partnership with Western University, McMaster University, SickKids Research Institute, the University of Ottawa and The Ottawa Hospital, are geared to provide the general public with hype-free, up-to-date, science-based information on stem cells and regenerative medicine: Such as:

  • What are these incredible cells?
  • What stem cell therapies are currently available and what’s on the horizon?
  • How are we getting to the next big breakthrough and what are the societal considerations for this research?

The sessions feature top Canadian researchers and clinicians explaining their work and talking about the potential and possibilities. Here’s the lineup:

Tuesday, April 19: London
7-9 pm at Western University More information

Stem cells: Powerful tools for regenerative medicine. Dr. David Hess, scientist in the Molecular Medicine Research Group and Krembil Centre for Stem Cell Biology at Robarts Research Institute.

Can I please have a stem cell injection doc? Dr. Alan Getgood, orthopedic surgeon at the Fowler Kennedy Sport Medicine Clinic in London.

The ethics of stem cell research. Dr. Carolyn McLeod, professor of philosophy and an affiliate member of Women’s Studies and Feminist Research at Western University.

Thursday, April 21: Hamilton 
6-9 pm at the Art Gallery of Hamilton More information

Stem cells: Where they live and how they work. Dr Jonathan Draper, associate professor in McMaster University’s Department of Pathology and Molecular Medicine.

Being patient: How stem cells are impacting treatment options. Dr. Sheila Singh, associate professor of surgery and biochemistry at McMaster University.

Hype vs reality. Dr. Mick Bhatia, Scientific Director and Senior Scientist of the Stem Cell and Cancer Research Institute at McMaster University.

Tuesday, April 26: Toronto
6-8 pm at the Peter Gilgan Centre (SickKids) More information

Stem cells: What they are and what they can do. Dr. Cindi  Morshead, Chair of the Division of Anatomy at the University of Toronto.

Stem cells for the critically iIl: Promise and potential. Dr. John Laffey, Anesthesiologist-in-Chief at St Michael’s Hospital, Toronto.

Introduction to the ethical, legal and social issues surrounding stem cells. Dr. Ubaka Ogbogu, assistant professor in the Faculties of Law and Pharmacy & Pharmaceutical Sciences at the University of Alberta.

Tuesday, May 10: Ottawa
7-8:30 pm at The Ottawa Hospital (Civic Campus) More information

A crash course in stem cell biology. Dr. William (Bill) L. Stanford, senior scientist at the Sprott Centre for Stem Cell Research at the Ottawa Hospital.

Stem cells in medicine: Today and tomorrow. Dr. Harold Atkins, physician with The Ottawa Hospital’s Blood and Marrow Transplant Program.

Ethical issues in stem cell research and application. Dr. Jeff Blackmer, Vice President, Medical Professionalism at the Canadian Medical Association.

 

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31
Mar 2016
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Dr.Lauralyn McIntyre

Dr.Lauralyn McIntyre

Man makes remarkable recovery from septic shock after stem cell transplant

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A 73-year-old man from Hawkesbury, Ontario, survived a deadly infection after receiving millions of mesenchymal stem cells in a world-first trial at The Ottawa Hospital.…

A 73-year-old man from Hawkesbury, Ontario, survived a deadly infection after receiving millions of mesenchymal stem cells in a world-first trial at The Ottawa Hospital.

Charles Berniqué developed severe septic shock in June of last year after his esophagus burst, likely from food poisoning. Septic shock is a deadly condition in which rampant infection triggers hyper-activation of the immune system, causing the cardiovascular system and organs to fail.

Mr. Berniqué was first treated by thoracic surgeons, who restored his fluids, repaired his esophagus and started antibiotic therapy. He was placed into a coma in the intensive care unit where mechanical ventilation and dialysis supported his heart, lungs and kidneys.

During this time, his wife Maureen consented to his participation in the clinical trial led by Drs. Duncan Stewart and Lauralyn McIntyre.

Within 24 hours, Mr. Berniqué received an intravenous infusion of 30 million mesenchymal stem cells originally extracted from the bone marrow of a healthy Ottawa volunteer.

“Our laboratory studies that showed that mesenchymal stem cell therapy tripled survival in a mouse model of septic shock,” Dr. Stewart, Executive Vice-President of Research and senior scientist at The Ottawa Hospital, said in a media release. “The cells also reduced damaging inflammation and helped the mice eliminate the bacteria.”

Dr. McIntyre, an intensive care physician and senior scientist at The Ottawa Hospital, was impressed by the results. “Researchers around the world have spent decades trying to find a therapy that will treat the root causes of septic shock rather than just the symptoms, but so far, none of these therapies have improved survival,” she said. “We don’t know whether the cell therapy played any role in Mr. Berniqué’ s remarkable recovery, but the cells were very well tolerated and we are excited to continue to study this promising therapy in more patients.”

Mesenchymal stem cells have been studied extensively in human clinical trials for other conditions, but The Ottawa Hospital trial is the first in the world to evaluate the cells specifically for the treatment of septic shock. The main goal of the Phase I trial is to evaluate the tolerability and feasibility of the cells. However, the researchers have already received funding from the Ontario Institute for Regenerative Medicine to begin scaling up their cell bank for a larger Phase II trial, which will help determine if the therapy is effective against septic shock.

As for Mr. Berniqué, he’s grateful to be alive and happy that the care he received may do the same for others. “It is tremendous what The Ottawa Hospital did for me,” he said. “I was so close to death, but I received the best care in the world and got to participate in this study which could help many people.”

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14
Mar 2016
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Clinical Journal of Sports Medicine

Stem cell treatment for bad knees lacks high-quality clinical proof

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Recently we blogged about the proliferation of clinics offering an expensive, unproven stem cell treatment for arthritic and damaged knees.

In the current edition of the Clinical Journal of Sport Medicine, the Australasian College of Sports Physicians (ACSP) published a position paper on the use of mesenchymal stem cell therapies in sports and exercise medicine.…

Recently we blogged about the proliferation of clinics offering an expensive, unproven stem cell treatment for arthritic and damaged knees.

In the current edition of the Clinical Journal of Sport Medicine, the Australasian College of Sports Physicians (ACSP) published a position paper on the use of mesenchymal stem cell therapies in sports and exercise medicine. You can find the full paper here. It’s well worth reading if you are thinking about spending thousands of dollars for such a treatment or know someone who is.

Mesenchymal stem cells are commonly extracted from adipose (fat) tissue, skeletal muscle, umbilical cord blood, placenta, and bone marrow. In our blog post we dealt mainly with a same-day treatment known as the BMAC (bone marrow aspirate concentrate) procedure. It involves extracting the mesenchymal stem cells from a patient’s pelvis, running them through a centrifuge to concentrate them, and then re-injecting them into the bad knee to reduce inflammation and repair damage. The ACSP, which represents sports physicians in Australia and New Zealand, also considered a treatment in which cells are extracted via liposuction from fat tissue and then put through the centrifuge — a procedure the U.S. Food and Drug Administration is cracking down on.

The paper suggests technology is running ahead of scientific knowledge:  “A contemporary development in manufacturing techniques uses far shorter preparation times (often less than two hours) at far less cost, producing a non-expanded mixed cell population. However, mesenchymal stem cell numbers are low in these preparations and the presence of multiple cell types raises questions about the efficacy of these techniques. Although the progress in biotechnology has resulted in commercially attractive outcomes, the science substantiating their effectiveness has lagged behind … No published randomized controlled trials for ‘same-day bone marrow stem cell concentrates’ are available as of 2015.”

This is not to say such treatments don’t work, but that much more study is needed. And it needs to meet the gold standard of a double-blind, randomized, placebo-controlled clinical trial in which some patients get the mesenchymal stem cells and others get a placebo treatment. The ACSP found only one such trial has been done and it did not involve BMAC. Rather, it demonstrated no difference in outcomes between the treatment group who received adipose-derived mesenchymal stem cells and those who got a saline injection. The full study has yet to be published.

According to the ACSP, using stem cells to treat damaged knees should be initiated under only two conditions:

  • As part of a rigorous clinical research trial.
  • As an individualized innovative therapy where there is a commitment to the transparent collection of data for analysis by a research body external to the clinic.

“Innovative therapy” refers to a treatment undertaken by a physician in the hope it will help the patient, though its effectiveness has yet to be proven.  The upside is that this is how medical practice improves: doctors try something and it works.  The downside, as the ACSP notes, is that such treatments may become adopted practices with no proof they actually do any good. The worst case scenario is that an unproven treatment actually does harm.

Randomized, placebo-controlled clinical trials can take years and cost hundreds of thousands of dollars. To someone with arthritic or damaged knees — or hips or shoulders for that matter — it’s cold comfort to hear that more study is needed before such stem cell treatments get the thumbs up or thumbs down.   In the meantime, we don’t know.

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07
Mar 2016
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Re$earch Money

Coordinated approach to stem cell R&D can mean more than cures

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The latest edition of Re$earch Money magazine asks an intriguing question:

“Is half a billion dollars too big a budget ask to cure chronic conditions such as diabetes, heart disease, multiple sclerosis and Parkinson’s that account for the bulk of Canada’s health care costs?”

The article, available here, profiles the Canadian Stem Cell Foundation’s  Pre-Budget Submission to the House of Commons Finance Committee calling on the federal government to commit $250 million in 2016 and an equal amount five years later to support the $1.5 billion Canadian Stem Cell Strategy. …

The latest edition of Re$earch Money magazine asks an intriguing question:

“Is half a billion dollars too big a budget ask to cure chronic conditions such as diabetes, heart disease, multiple sclerosis and Parkinson’s that account for the bulk of Canada’s health care costs?”

The article, available here, profiles the Canadian Stem Cell Foundation’s  Pre-Budget Submission to the House of Commons Finance Committee calling on the federal government to commit $250 million in 2016 and an equal amount five years later to support the $1.5 billion Canadian Stem Cell Strategy.  The federal outlay will be doubled with more than $1 billion from other sources, including the private sector, philanthropists, health charities and other partners.

Foundation President & CEO James Price told Re$earch Money that the government has been receptive to the case that he and coalition members have been making for the Strategy in the lead-up to March 22 tabling of Budget 2016.  “Our proposal is strongly aligned with what we’re hearing in terms of the government’s proposed innovation agenda looking at investments that target key growth sectors for Canada that have the ability to attract investment, grow competitive export-oriented companies and diversify the economy.”

The article points out that the lion’s share of private funds would come from STEMCELL Technologies Inc., a Vancouver-based firm that plans to invest  $350 million to $500 million in R&D over the Strategy’s 10-year time span. “If you want faster results (from stem cell research) you need a coordinated approach across Canada and that requires money,” says Dr. Allen Eaves, STEMCELL’s President and CEO.

 

 

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17
Nov 2015
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From right, Will Wang, Caroline Brun, Dr. Michael Rudnicki and Dr. Nicolas Dumont

From right, Will Wang, Caroline Brun, Dr. Michael Rudnicki and Dr. Nicolas Dumont

Canadian reseachers deliver another game-changer

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Within the space of two weeks, two Canadian scientists have unveiled game-changing research into stem cells — providing further proof of Canada’s prominent position in the field.…

Within the space of two weeks, two Canadian scientists have unveiled game-changing research into stem cells — providing further proof of Canada’s prominent position in the field.

On November 5th, the University of Toronto’s Dr. John Dick published a paper in Science that has researchers around the world rethinking how human blood gets made.  Dr. Dick’s team showed that the traditional understanding of blood production is wrong and that stem cells drive production of different kinds of blood cells much earlier than previously thought. This has huge implications for future treatments for blood-based cancers. We blogged about it here.

Yesterday came news that a University of Ottawa team led by Dr. Michael Rudnicki published a paper in Nature Medicine that could completely alter perceptions on how Duchenne muscular dystrophy happens — linking it to intrinsic defects in the function of muscle stem cells.

Affecting about one in about 3,600 boys, Duchenne muscular dystrophy occurs when genetic mutations deplete production of dystrophin protein, causing muscles to deteriorate.

According to an Ottawa Hospital Research Institute release, dystrophin was thought to be a simple structural protein found only in muscle fibres. The Ottawa team discovered that muscle stem cells also express the dystrophin protein. Without it they can produce only one-tenth the number of muscle precursor cells needed to generate functional muscle fibre.

Dr. Nicolas A. Dumont and Yu Xin (Will) Wang are co-lead authors on the paper. that also showed that dystrophin is a key piece of the molecular machinery that enables muscle stem cells to function.

“Muscle stem cells that lack dystrophin cannot tell which way is up and which way is down,” said Dr. Rudnicki. “This is crucial because muscle stem cells need to sense their environment to decide whether to produce more stem cells or to form new muscle fibres. Without this information, muscle stem cells cannot divide properly and cannot properly repair damaged muscle.”

Dr. Rudnicki was featured in many news reports about the discovery, including this feature by CBC.

 

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