We recently featured the story of Tina Ceroni, a Burlington Ontario athlete whose life was sidelined by a rare, insidious disorder called Stiff Person Syndrome.…
We recently featured the story of Tina Ceroni, a Burlington Ontario athlete whose life was sidelined by a rare, insidious disorder called Stiff Person Syndrome. Thanks to a stem cell transplant, Tina’s fitness career — and her life — is back on track. To show her gratitude, Tina raised almost $37,000 for the Ottawa Hospital’s Stem Cell and Bone Marrow Transplant Program. When she was presented the cheque to Dr. Harry Atkins on Dec. 4th, Tina met with two other young women whose lives have been rescued by stem cell treatments: Jelissa Morgan and Jennifer Molson. Today we share Jelissa’s story.
From nurse to patient and (soon) back again
When Jelissa Morgan’s neuromyelitis optica flared up again in early 2012, she was devastated.
The rare immunological disorder, similar to Multiple Sclerosis, inflames the optic nerve and the spinal cord. Jelissa was losing her vision — she couldn’t read or watch movies and had trouble identifying who was talking to her. Walking was difficult. She had serious bladder and bowel problems and her skin itched. ‘My body was in pain,” she says, “but didn’t know how to express it.”
That flare-up, the worst of a series the young woman had experienced since finishing high school, transformed Jelissa from hospital nurse to hospital patient.
Until then, her doctors had kept the condition under control with Prednisone, a synthetic corticosteroid that comes with a slew of potential side-effects such as weight gain, sleep difficulties and mood swings. Some patients develop a humped back and a moon face. Jelissa had all of those symptoms and more.
A switch to a different drug, called Imuran, proved futile: she was on it when the worst flare-up struck.
“They were running out of medication options. My neurologist said, ‘What do you think about a stem cell bone marrow transplant?’ She knew about Dr. Harry Atkins and the work he was doing with MS patients.”
For the past several years, the Ottawa Hospital’s Dr. Atkins, working with neurologist Dr. Mark Freedman, has been treating MS patients with stem cell bone marrow transplants. In essence, he takes stem cells from patients with the most severe cases of the immunological disease and purifies and fortifies them. The patient then undergoes an extreme course of chemotherapy treatments that virtually annihilates their diseased immune system. The robust stem cells are then returned to them to rebuild a new — hopefully disease-free — immune system.
The process has worked well with about 30 MS patients whose disease progression has been stopped. In some cases, symptoms of the disease have diminished significantly and — in at least one case — disappeared entirely.
Dr. Atkins is also using the procedure for people who have other autoimmune disorders such as Crohn’s disease, Stiff Person Syndrome — and neuromyelitis optica.
Jelissa decided to have the experimental stem cell treatment.
“It’s kind of one of those decisions where you don’t want to wait,” she say. “You don’t know when the next flare-up will be. That last flare-up was so bad. Luckily, I was able to regain a lot of the functions I’d lost. But it’s like rolling the dice. You don’t know what could happen with the next flare-up.”
Jelissa received her stem cell transplant in the fall of 2012. She has been free of neuromyelitis optica symptoms ever since.
“I feel better than I did even before the disease began. I have much more energy. I’m off all my pills for the immune disorder and the transplant meds. I’m looking forward to getting my life back on track. That includes buying a car, moving out of my parent’s house and traveling.”
She is excited about returning to nursing in the next few months, after receiving a series of vaccinations to replace the ones lost during the chemotherapy treatment.
“From a patient perspective, a transplant is a hard thing to go through. But I survived. I feel like it’s given me a second chance at life. ”
She strongly supports stem cell research to help more people with diseases like hers.
“Stem cell research opens up so many possibilities. It’s like opening a door and entering a room and you don’t even know what’s in the room yet. It could mean treatments for so many things. There is so much hope there.”
Leaders from 18 national health-based charities are gathering in Toronto today as part of a national dialogue to define a bold vision and strategy for advancing stem cell research toward new therapies and treatments.…
Leaders from 18 national health-based charities are gathering in Toronto today as part of a national dialogue to define a bold vision and strategy for advancing stem cell research toward new therapies and treatments.
Participants at the Health Charities Forum for a Canadian Stem Cell Strategy — whose organizations speak for millions of Canadians dealing with conditions that range from Alzheimer’s disease to spinal cord injury — will consider how to build on Canada’s tremendous strength in stem cell research to achieve better health outcomes for Canadians.
“The idea,” says James Price, President & CEO of the Canadian Stem Cell Foundation, “is to get Canada’s leading charities in one place and hear what they think should be included in a Canadian Strategy. Because stem cells have the potential to make an impact on almost every disease, we need the health charities’ input.”
The Foundation, in partnership with the Stem Cell Network, the Centre for Commercialization of Regenerative Medicine, the Ontario Stem Cell Initiative and the Health Charities Coalition of Canada is leading the campaign for a Canadian Stem Cell Strategy.
“We believe that stem cell science could soon lead to new therapies for a wide range of devastating diseases,” says Deirdre Freiheit, Executive Director of the HCCC, which helped put the Forum together. “That’s why our Coalition supports the dialogue to investigate creating a Canadian strategy that can bring together all the players to make these new treatments and cures a reality.”
Ideas generated at the Forum will help in crafting the White Paper that will form the basis of the Strategy. Similar ongoing consultations with leading stem cell researchers, business and community leaders, industry and other stakeholders will also help shape the Strategy.
At the Forum, health charities leaders will discuss the outcomes they expect a Canadian Stem Cell Strategy to deliver and the guiding principles they would like to see put in place. “This is a major first step in the consultation process with the charities,” says the Foundation’s Price. “But it won’t be the last. We are building a critically important dialogue.”
Who’s at the table?
• Alzheimer Society of Canada
• Canadian Breast Cancer Foundation
• Canadian Cancer Society
• Canadian Diabetes Association
• Canadian Liver Foundation
• Crohn’s and Colitis Foundation of Canada
• Cystic Fibrosis Canada
• Health Charities Coalition of Canada
• Heart and Stroke Foundation
• JDRF Canada
• Kidney Cancer Canada
• Lymphoma Foundation Canada
• Multiple Sclerosis Society of Canada
• Muscular Dystrophy Canada
• Prostate Cancer Canada
• The Foundation Fighting Blindness – Canada
• The Kidney Foundation of Canada
• The Rick Hansen Institute
The news broke Monday that three American scientists – Dr. Thomas Südhof of Stanford, Dr. Randy Schekman of University of California at Berkeley and Dr.…
The news broke Monday that three American scientists – Dr. Thomas Südhof of Stanford, Dr. Randy Schekman of University of California at Berkeley and Dr. James Rothman of Yale University have won the 2013 Nobel Prize in Physiology or Medicine.
As the San Jose Mercury explained, the three scientists, working separately, figured out how a cell organizes its internal transportation system, controlling the creation and release of important hormones and enzymes. They showed how this cellular cargo is delivered to the right place at the right time. Breakdowns in this trafficking system lead to neurological diseases, diabetes, immunological disorders and ultimately death.
While the three researchers’ amazing discoveries are to be celebrated as Nobel-worthy, the announcement likely is final proof that the selection committee will never right the grievous wrong of not awarding the most prestigious prize to Canada’s Dr. James Till and Dr. Ernest McCulloch who, in the early 1960s, proved the existence of stem cells – a truly paradigm-shifting moment in medical research.
Every October many Canadian medical scientists hope to see this mistake corrected. Every year they are disappointed.
“Bun” McCulloch’s chance to win is gone: he died two years ago and the Nobel is not awarded posthumously. However, his research partner, Jim Till, is alive and well – no doubt due in large part to the fact he is life-long avid curler – in Toronto.
Many thought that the Till oversight would have been corrected in 2012 when Stockholm decided to award the Nobel to Japan’s Dr. Shinya Yamanaka, who showed how to create induced pluripotent stem (iPS) cells in 2006 (with mice) and 2007 (with humans). However, Dr. Yamanaka’s co-winner last year was the United Kingdom’s Sir John Bertrand Gurdon, for his pioneering work in the late 1950s in nuclear transplantation and cloning. Dr. Till, who could have been the third laureate in a cell-based triple play, was once again left out.
Canada, in fact, has not won a Nobel Prize in Medicine since Sir Frederick Banting captured the prize (with Scotland’s J.J.R. Macleod) in 1923 for discovering insulin. Canadian-born medical researchers who did their work elsewhere have won during that nine-decade Nobel drought, but no native son or daughter who did their groundbreaking work in Canada has made the trip to Sweden for the acceptance ceremony.
We have, of course, won in other categories. For example, the late Dr. Michael Smith, a British-born Canadian, won the Nobel for Chemistry in 1993 for his efforts in developing site-directed mutagenesis. But the Physiology and Medicine prize? Nothing in 90 years.
It is not merely Canadians who, every October, can’t comprehend this oversight. Dr. David Scadden, who co-founded and co-directs the Harvard Stem Cell Institute, has said he can’t fathom why Till and McCulloch were overlooked, noting that, “Till and McCulloch clearly are giants. They clearly paved the way and made this whole field (of stem cells).”
But while it would be enough to give the True North an inferiority complex, the simple truth is many giants in medical research have been overlooked by the Nobel selection committee. A book due for publication later this year or in early 2014 tells the stories of more than a dozen such titans from around the world who got the cold shoulder. Pioneers of Medicine Without a Nobel Prize, to be published by Imperial College Press, includes chapters on Sir Archibald E. Garrod, the founding father of biochemical genetics, Sir William Richard Shaboe Doll, who linked smoking and lung cancer, Dr. Albert Sabin, who developed an oral polio vaccine, and heart transplant pioneers Drs. Christiaan Barnard and Norman Shumway. Also featured are Drs. Inge Edler and Hellmuth Hertz, for their development of ultrasound for clinical use, Drs. Herbert Boyer and Stanley Cohen, who came up with recombinant DNA, and Dr. Akira Endo, who discovered statins.
It will, of course, include a chapter on Till and McCulloch.
The unveiling of a clinical trial to test the use of genetically enhanced stem cells to rebuild badly damaged hearts captured major media attention in early September.…
The unveiling of a clinical trial to test the use of genetically enhanced stem cells to rebuild badly damaged hearts captured major media attention in early September.
CBC News, the Sun and Postmedia newspapers all called the trial “groundbreaking” and gave it prominent play. CTV called it a “world-first clinical trial.” The Globe and Mail coverage was somewhat more restrained but went into fine detail to explain how the patient’s own blood stem cells are enhanced with a gene called endothelial nitric oxide synthase (eNOS) that is then infused into the heart at the site of the damage.
It didn’t hurt that the Ottawa Hospital Research Institute, where Principal Investigator Dr. Duncan Stewart is CEO and Scientific Director, was able to put a gentle human face on complicated science by presenting Patient No. 1. Sixty-eight-year-old Harriet Garrow of Cornwall, Ontario suffered a major, heart-stopping myocardial infarction in July. Holding hands with her husband Peter Garrow, she was amiable, articulate and authentic. Reporting the news Sept. 10, the Aboriginal Peoples Television Network highlighted her heritage, headlining its story “Mohawk woman in centre of ground breaking medical treatment.”
This is a double-blind study – meaning neither the investigators nor the 100 participants in Ottawa, Montreal and Toronto will know who received the souped-up stem cells or the comparative controls of ordinary stem cells or placebos until after the results are in. That doesn’t matter to Mrs. Garrow, who, at the very least, is getting the best of current cardiac care. “I am thrilled to play a part in this research that could help people like me in the future and, who knows, perhaps even my children and grandchildren,” she said in the OHRI’s media release.
A decade of work
Investigators will start analyzing the results after Patient No. 100 has been enrolled and treated – more than two years from now. By that time, Dr. Stewart will have put 10 years into the project he originally started at St. Michael’s Hospital in Toronto.
But things could happen quickly after that.
“Most major medical centres in Canada and the United States have the capacity to do this kind of cell manufacturing, “ says Dr. Stewart. “So, if we allow ourselves to dream a bit, that this really is a very positive trial with major improvements, I think it could be adopted quite quickly.”
The implications – in terms of thousands of lives that could be saved and millions of dollars in health care costs avoided – are immense. About 70,000 Canadians have heart attacks every year. Dr. Stewart estimates that about one-third of those, around 23,000, suffer damage severe enough to require this level of intervention.
“This segment of the infarct population is one that’s going to cost an awful lot of money because before they die they are going to develop heart failure. They’re going to be having multiple prolonged hospital admissions and require implantation of defibrillators and are going to have all kinds of other treatments that cost the system a lot of money.”
Other trials using ordinary stem cells have shown some positive effect on repairing heart tissue, but nothing that would spark widespread change in how heart attack patients are treated. The difference here is these stem cells have rejuvenated with eNOS to do a more effective repair job.
“It’s always been our view that getting the most robust therapy requires some manipulation of the cells, particularly when you’re using the patient’s own cells,” says Dr. Stewart. “The cells are the same age as the patient, which is usually 60 or 70 years old, and they have been exposed to the same factors that produced heart disease in the first place. We know they don’t work well. But if we can recover the activity of these cells we’re going to get more benefit.”
It all depends, of course, on The Big If: If it works.
Given the rigorous controls and criteria included in the clinical trial, the answer should be obvious within three years.
Welcome to the first instalment of the Stem Cell NewsDesk, the Foundation’s attempt to help Canadians better understand where a “breakthrough” fits on the research lab-to-clinic continuum.…
Welcome to the first instalment of the Stem Cell NewsDesk, the Foundation’s attempt to help Canadians better understand where a “breakthrough” fits on the research lab-to-clinic continuum.
Essentially, the aim of NewsDesk is to try to answer one question: how does [insert news-making development/discovery/breakthrough here] contribute to finding a treatment or a cure for a currently untreatable or incurable disease? The idea is not to hype stem cell science but to provide realistic reports on developments as they occur.
It won’t be easy. Stem cell science is complicated and it can be hard to decipher whether a discovery represents a monumental leap forward or is just an incremental improvement in understanding how stem cells function. Sometimes it is obvious, as with Dr. Shinya Yamanaka’s 2006 Nobel-winning discovery of how to make embryonic-like stem cells from almost any cell in the body – cells we now call induced pluripotent stem cells. Sometimes it’s not. Remember that the first demonstration of the unique properties of stem cells 50 years ago flew in under the radar.
In email correspondence, Dr. Connie Eaves, a Vancouver-based researcher whose team was the first to isolate breast stem cells, shared her thoughts on why this is such a challenge:
- Every ‘new’ piece of information about how cells work and how their behaviour can be predictably manipulated is potentially a breakthrough – but it may take years to understand whether/when/where/or for what that will be true. So, making a fast judgment is rarely possible.
- Current efforts use unknowns (new molecules with an experimental rationale) to treat unknowns (human tumours we don’t understand).
- Clinically, an improvement of long-term survival from 5% to 15% would be considered a big advance. But if you were an affected patient, you might not see it that way, as overall your survival chances would still be pretty bad.
- What is useful clinically requires a controlled trial and this usually takes a long time (10 years) and the result may appear sort of boring by the time the answers are all in.
Case in point: the ‘sharpshooter’ story
Dr. Eaves is part of the 100-person team led by Princess Margaret Cancer Centre’s Dr. Tak Mak and Dr. Denis Slamon, (pictured at right) of the University of California, Los Angeles that made headlines in mid-June by announcing they had developed a new kind of “sharpshooter” anti-cancer drug. Given the excellent track record of the two scientists – Dr. Mak revolutionized how scientists think about the human immune system by cloning the T-Cell receptor and Dr. Slamon developed the breast cancer drug Herceptin – it’s not surprising the announcement garnered major media attention.
As the Toronto Star explained, the new drug, which has been tested on mice for ovarian, breast, pancreas, lung and colon cancer is called a sharpshooter because it goes after a specific enzyme to shut down cancer. Unlike chemotherapy, which can kill healthy, quick-replicating cells, the drug, called CFI-400945, takes aim only at the cancer cells.
On CTV’s Canada AM, host Bev Thompson described it as “being hailed as a major breakthrough in cancer research” and said while “we’ve talked about breakthroughs before … this seems like a cut above.”
In the Globe and Mail, however, Canada’s leading health writer André Picard, pointed out that CFI-400945, has “not been tested on a single person” and that “even in a best-case scenario” a new cancer drug “is at least a decade away.”
As excited as they were, the Princess Margaret researchers also urged patience. On that Canada AM segment, Dr. Philippe Bedard explained that the three-phase clinical trial process is a marathon, not a sprint, stressing that there is a long road ahead and it “can take many years.”
So where does that leave cancer patients?
Officials at Princess Margaret say there has been lots of interest from people who want the new drug. That will take some time: Health Canada approved CFI-400945 for use in human trials in mid-July. Next, it goes before the University Health Network’s Research Ethics Board for approval. A trial involving a small number of patients to see if CFI-400945 is safe – likely will begin in November.
So why did Princess Margaret bang the drum so loudly at such an early stage? The sharpshooter announcement actually came from The Princess Margaret Cancer Foundation to make donors aware of the potential advances that are critically dependent on the funding support that their donations provide. Makes sense: Canadians support medical research through their charitable donations as well as through their taxes and want to know how their investments are doing.
No quick fixes
But the reaction shows that there is a real and growing need for a resource to help people understand how a treatment may have an impact on them. As stem cell research moves closer to providing new treatments, people will want to know more.
NewsDesk hopes to help in this. Again, not an easy task. And there will be lots of cautions and caveats attached to our discussions of breakthroughs. Because the reality is: there are no quick fixes or magic bullets. But progress is being made – almost every day.
So let’s go back to Dr. Eaves, who is a member of the Foundation’s Science Leadership Council, and her thoughts on the sharpshooter announcement:
“The Tak Mak result looks very exciting in the experimental models studied to date. But there is not much history yet to know how these will correlate with patient outcomes. I can’t say much more than that and don’t think anyone can at this early stage.”
When we set about revamping our website here at the Canadian Stem Cell Foundation, we realized that a subject as exciting and loaded with potential as stem cells ought to have a website that captured that sense of excitement and explored that potential.…
When we set about revamping our website here at the Canadian Stem Cell Foundation, we realized that a subject as exciting and loaded with potential as stem cells ought to have a website that captured that sense of excitement and explored that potential.
Not that our previous version didn’t. Our original site was innovative enough to win a Webby Award – the online equivalent to an Oscar – in 2010. But it was created in our early days and, just like the field of stem cell science, we have moved ahead, taking on bold new challenges our old site could no longer encompass.
The first step was to simplify the message. As the national voice and champion, our essential role is to unite scientists, business and community leaders behind accelerating the process of turning stem cell research into clinical applications to treat or cure currently untreatable and incurable diseases. We were able to distil that role into the seven words trumpeted on our home page: Help Us Help Stem Cells Help You.
Everything found on the new site fans out from there. The Toward Treatments section, for example, is there to explain – in language everyone can understand – how far along stem cell research is in addressing diseases that have perplexed medical science for centuries. The idea is to provide a resource where patients, their families and friends can go to get the straight goods on whether there is a stem cell treatment available or where a potential treatment is in the research-to-clinic pipeline.
‘Watch this space’
Currently, we have 14 diseases/conditions covered, ranging from ALS to Wound Healing. We hope to have a total of 18 prepared by year’s end. Which speaks to the evolving nature of our website. It is a work in progress. For example, our Stem Cell NewsDesk is in “watch this space” mode. The idea with the NewsDesk is to provide regular updates on progress in stem cell research, assess the importance of advances that the mainstream media are highlighting and shine a light on work that may be going unnoticed. It will be the go-to place for fair and balanced reportage of the progress that stem cell science is making. We plan to test run it over the summer and have it operational for the fall.
As for the Canadian Stem Cell Strategy, it represents the Foundation’s number one priority for the next two years. Canada needs a properly funded, publicly supported research and development strategy to stay at the forefront of this exciting field of medical research and to move further, faster toward the types of treatments everyone is hoping for. We are in the formative stages of creating the Strategy, but Canadians will be hearing a lot more about it over the coming months.
New features, old favourites
One of the new features on the site is our powerful Legacy Video that outlines Canada’s role in founding and developing stem cell science. Watching it, you can’t help but be impressed by Canada’s remarkable contributions to founding and developing this exciting and important field of research, and to fully appreciate the notion that, ‘If hockey is Canada’s game, its science is stem cells.”
For those who enjoyed the old website, do not despair. We have maintained its most-visited and best-loved elements. The Rock Star Scientists Video that features several of Canada’s leading scientists talking about their work continues to inspire. We encourage you to visit the Stem Cell Charter page and, if you haven’t already, add your name to the thousands of Canadians who believe in the therapeutic potential of stem cells and who have committed to becoming more informed about and engaged in advancing stem cell science.
We built this site to evolve and move forward as the science we champion evolves and moves forward. To explore the potential. Now, that’s exciting.
The Canadian Stem Cell Foundation is on the move – in more ways than one.
After two years of operating out of the Stem Cell Network offices at the Ottawa Hospital, the Foundation is now in new quarters at 6 Gurdwara Road in South Ottawa and a satellite office on Spadina Road in the heart of Toronto.…
The Canadian Stem Cell Foundation is on the move – in more ways than one.
After two years of operating out of the Stem Cell Network offices at the Ottawa Hospital, the Foundation is now in new quarters at 6 Gurdwara Road in South Ottawa and a satellite office on Spadina Road in the heart of Toronto.
The new space is necessary to accommodate exciting new projects the Foundation is rolling out First up is a completely redesigned website with a bold theme: Help Us, Help Stem Cells Help You. Its centrepiece attraction is Toward Treatments – patient-focused summaries of what stem cell researchers are doing in the battle against 14 currently incurable diseases, such as Multiple Sclerosis and Parkinson’s disease. Prepared in partnership with the Stem Cell Network, each summary is as authoritative as it is accessible, written in reader-friendly language that makes complicated science easily comprehensible.
Stem Cell NewsDesk
We expect to have the site up and running in the summer and will be adding to it as we build for a major fall launch when another much-needed feature will be ready to go: Stem Cell NewsDesk will provide timely and realistic assessments of advances in research that the mainstream media are highlighting, and shine a light on important work that may be going overlooked.
The idea is to give Canadians a clear understanding of the difference between an incremental advance and a major accomplishment. We anticipate the NewDesk will be the go-to place for fair and balanced reportage of the progress stem cells science is making. Watch for it this fall.
A Canadian Strategy
But that is just a starting point.
The Foundation’s major undertaking over the next two years will be the crafting and implementation of the Canadian Stem Cell Strategy to guide the progress of research and development over the next 15 to 20 years. Wheels are already turning. The Foundation’s formidable Science Leadership Council is the framework for the strategy. We are now recruiting some of the world’s leading thinkers in stem cell science and its application to join a blue-ribbon panel of international experts to provide the global context required to shape the strategy. And we have begun consulting with Canada’s entrepreneurial and philanthropic leaders to get their input— and commitment – to help move the science safely and quickly forward.
The potential of stem cell science to treat diseases and ease suffering is immense. We believe stem cell science is now at a tipping point – a time when potential solutions are on the verge of becoming real ones. The Canadian Stem Cell Foundation is moving forward to help make that happen.