stem cell transplants
Last fall we told the story of Dan Muscat, an Ontario jeweler whose combination chemo/stem cell transplant treatment helped him recover from scleroderma, an extremely painful, life-threatening autoimmune disease that hardens the skin and attacks internal organs.…
Last fall we told the story of Dan Muscat, an Ontario jeweler whose combination chemo/stem cell transplant treatment helped him recover from scleroderma, an extremely painful, life-threatening autoimmune disease that hardens the skin and attacks internal organs.
We believed then that his transplant, which took place at The Ottawa Hospital over the spring and summer of 2016, was the first time such a therapy had been tried in Canada. It even made news: CTV’s Avis Favaro profiled Dan early this year.
It turns out that other Canadian hospitals are trying — and have been using — the innovative therapy, which involves first extracting bone marrow stem cells from a patient to purify them, then wiping out their diseased immune system with chemotherapy, and finally returning the robust stem cells to the patient. The goal is to have these fortified stem cells rebuild a disease-free immune system.
Calgary’s Dr. Jan Storek has employed the chemo/stem cell treatment on “four or five” patients since 2011 and has contributed to the international Scleroderma: Cyclophosphamide Or Transplantation (SCOT) study, the results of which have yet to be published. He began working with the treatment in the 1990s at the Fred Hutchinson Cancer Center in Seattle.
“I was part of the development of transplants for autoimmune diseases,” says Dr. Storek. “Then the University of Calgary hired me to start a research program in bone marrow transplantation including transplantation for autoimmune diseases.”
He points to the work of Dr. Sharon LeClercq, a Calgary rheumatologist and expert in systemic sclerosis, without whose involvement there would have been no Canadian participation in a randomized study. “Patients participating in studies, particularly in the three randomized studies leading to this success should also be greatly acknowledged,” says Dr. Storek.
One of his transplant recipients is Miaya Killips, pictured above shortly after her 2016 transplant and as she is today. Miaya, who lives in Spruce Grove, Alberta, was diagnosed with scleroderma three years ago. She suffered lung damage, along with hardening of the skin. “I had limited mobility because my joints were tight and I was not able to swallow well — I would choke on food.”
These days, things are much better. “Scleroderma-wise, I’m feeling great. My mobility is much better and I don’t have the pain I had before. I am not choking when I eat and my lungs have stabilized, which is good because before the transplant my doctor had me on a standby list for a lung transplant.”
An electrician by trade, she wants to go back to school as part of a career change. And she and her husband plan to start a family soon, using embryos harvested through in vitro fertilization before she underwent chemotherapy.
We also heard from Shane Knihnitski, a 28-year-old heavy equipment mechanic who underwent a bone marrow stem cell transplant at Saskatoon’s Royal University Hospital in February to treat his scleroderma, which had caused large calcium deposits to form at his joints, resulting in considerable pain and restricted mobility.
“Things are turning around,” Shane says, “and the skin is starting to loosen up. I wasn’t able to see my elbow for two years — it was a big lump like a soft ball — but I can see the bone again.”
Dr. Keltie Anderson, Shane’s rheumatologist, says that while “it’s still very early” Shane has regained “a fairly significant amount of mobility in his hands already.
“When I first started seeing him, he could sort of cup his hands but couldn’t make a fist. Now he can bring the fingers all the way to the palm — almost make a full fist. To have such change already is very impressive.”
That the stem cell transplant procedure is having good results comes as great news to rheumatologists like Dr. Anderson who, until now, had few weapons with which to fight this painful, life-stealing disease. “Basically, you would do cyclophosphamide (a chemotherapy medication to suppress the immune system) and then cross your fingers.
“In Shane’s case he not only has extensive skin involvement, he also had extensive lung involvement. His outlook was quite bleak. And he’s a young guy and (he and his wife) just had their first child.”
Dr. Mohamed Elemary, who directs the hospital’s stem cell transplant program, says Shane was originally referred to Dr. Storek for inclusion in the Calgary study but it had already been completed. “So we started to review the data and see if it was something feasible for him here in Saskatoon. The stem cell transplant unit is run by the cancer centre and by the health region with the University Hospital. We can barely accommodate our patients with malignancies.”
After receiving permission to try the treatment with Shane, making sure he was a good candidate for the procedure, and consulting with Dr. Storek, Dr. Elemary went ahead with the transplant two months ago. “Our concerns vanished because he did tremendously well and was out of the hospital almost two weeks after his transplant. I just saw him a couple of weeks ago and I was amazed at the way that he is responding.”
The treatment does not come without risks. In Shane’s case, he was told there was a 20% chance the treatment could kill him. “But he came through it with flying colours,” says Dr. Anderson. “The last time I saw him he looked great. He didn’t look like someone who’d just gone through ablative chemotherapy with a stem cell transplant. And he was more upbeat than I’d seen him in a long time.”
Dr. Elemary is hoping to get approval to treat another scleroderma patient with stem cells and “with the success of Shane’s story” is optimistic that will happen.
In May, Calgary’s Dr. Storek, with co-authors Drs. LeClercq and Andrew Daly, will publish a report in the Canadian Medical Association Journal, reviewing the challenges of integrating the treatment in Canadian hospitals, given that stem cell transplantation programs are set up to treat patients with malignant cancers. Dr, Storek also
Dan Muscat, whose recovery continues, is delighted that more scleroderma patients are getting the treatment. “I’m trying to get the word out. This should be an option for people when they are diagnosed.”
Q&A with Dr. Heidi Elmoazzen
One thousand Canadians need a stem cell donation on any given day, according to Canadian Blood Services (CBS). …
Q&A with Dr. Heidi Elmoazzen
One thousand Canadians need a stem cell donation on any given day, according to Canadian Blood Services (CBS). About 250 usually can find a match in their own family. The other 750 must look elsewhere. For years, CBS has operated its OneMatch Stem Cell and Marrow Network to find and match volunteer donors to patients. Last year, CBS added to the much needed supply of donor stems cells by officially launching its Cord Blood Bank where new mothers can donate umbilical cord blood — a plentiful source of stem cells that are more easily matched than adult stem cells. The bank is supported by nine provinces and the three territories (Québec has its own cord blood bank and registry). Five collection sites now operate in four cities. Dr. Heidi Elmoazzen is the Director of the Cord Blood Bank.
Q: The official launch was last year, but how long has the national public cord blood bank been operating?
A: We started collecting in Ottawa in 2013, then rolled out to Brampton in 2014. We started collections in Edmonton and Vancouver in 2015. We did the official launch in June of 2015 to say, “We’re open.”
Q: Canada was the last G7 country to establish a public cord blood bank. Why were we so late to the party?
A: Part of it is just the way funding is structured. We needed agreement from the provinces and territories. But there are actually advantages of us coming late to the party. We’ve been able to learn from other cord blood banks and we’re fortunate to now be in a position where we’ve set up one of the best in the world in terms of quality of inventory.
Q: What stage are you at now?
A: Our biggest goal is building up an inventory that’s reflective of the unique population we have here in Canada. We have a lot of ethnic groups that you don’t find in other parts of the world, including our Aboriginal — First Nations, Métis and Inuit — populations. We have a lot of mixed race patients here. It’s building up that inventory that reflects that unique population. And we’re really going for quality units — large units with lots of stem cells in them so there are enough stem cells for a transplant. As you can appreciate, we need an inventory of a certain size before we see uptake. We’re in that phase now. We currently have 1,145 units listed and available to Canadian and international transplant centres. We’ve had inquiries, but nothing has been shipped out yet. So that’s our next big milestone. I’m expecting we will probably see that soon.
Q: So, for example, a Canadian patient with a blood-based cancer who is looking for a stem cell transplant, they would look at what your bank has available and look internationally as well?
A: It goes through our OneMatch Stem Cell and Marrow Network, where we search all the adult donors and cord blood units in Canada as well as internationally. We have access to over 27 million adult donors around the world. In Canada there are 345,000 adult donors. And then you have 684,000 publicly banked (cord blood) units, with our own units in there as well.
Q: Are most people aware that the public bank is there and there’s an option to donate cord blood?
A: There are always opportunities to increase awareness. We really try to make moms who are delivering at one of our designated collection hospitals aware. We have conversations with all of the physicians who have delivering rights at those hospitals. We try to do outreach so that moms do know this is available to them.
Q: What’s their reaction been?
A: It has been great. It’s a very easy sell to mothers because they know the alternative is the cord blood will be discarded as medical waste. We don’t get push-back.
Q: The collection service is only available in four centres. What happens if you don’t happen to live in Ottawa, Brampton, Edmonton and Vancouver?
A: Unfortunately, then you can’t participate in the public bank. But it’s important to realize these units are available to anyone in Canada and around the world who needs them. We selectively chose the cities we are in and what hospitals we partner with. We wanted large urban centres with a large ethnic diversity. We wanted hospitals that had at least 4,000 births (per year) with a minimum 20% ethnic diversity. We’re trying to build a bank that reflects the ethnic diversity in Canada.
Q: Will there be more collection centres opening?
A: We’re always looking at whether we need to do that. At this time there are no plans to open any more sites.
Q: The total price tag for the bank is $48 million, with $12.5 million raised in a fundraising campaign and the rest coming from the provinces and territories. Why did one-quarter of the funds come from fundraising?
A: It was to show the governments that we had skin in the game and that Canadian Blood Services was committed to this.
Q: Does this save money? If a Canadian gets a stem cell transplant from here in Canada, does that save money compared to going to Europe to have the stem cells harvested and shipped over here?
A: Absolutely. It costs about $40,000 when we use either an adult donor or a cord blood unit from overseas. It’s a huge cost savings to the health care system, especially when you’re talking about adults because they’re using two cord blood units per transplant. The fact that these are available through our transplant centres at no cost represents a huge savings.
Q: Can the cord blood also be used for research?
A: Yes; we have a cord blood for research program. Moms who donate in Ottawa can indicate on their permission to collect form that in the event that their baby’s cord blood is not bankable it can go to research. We make those research units available to scientists across Canada. We have shipped out hundreds of units for research purposes. There’s been a big uptake because it’s such a valuable resource.
(For more information on the Cord Blood Bank, click here.)
It can take millions of cells to do the most simple stem cell transplant. Coming up with ways to produce huge volumes of pure, safe cells is a challenge.…
It can take millions of cells to do the most simple stem cell transplant. Coming up with ways to produce huge volumes of pure, safe cells is a challenge. In mid-January, Prime Minister Justin Trudeau made an appearance at the MaRS Discovery District building in downtown Toronto to announce $20 million in federal funding for advanced therapeutic cell manufacturing to be managed by the Centre for Commercialization of Regenerative Medicine (CCRM), with GE Healthcare also committing $20 million. CCRM’s CEO Dr. Michael May talks about plans for the centre.
Q: How did this come about?
A: This has been evolving over five years as we developed very strong relationships with industry leaders, of which GE HealthcCare is one. As the industry evolves, manufacturing issues are maybe the most talked about bottleneck in cell and gene therapy.
Q: What is your centre going to do?
A: This centre is a business unit of the overall CCRM operation. It has two main activities. One is targeting particular bottlenecks — strategic gaps in the industry — around particular cell types and specific operations and then inventing solutions that can be commercialized as tools and devices. The second thing is the centre will utilize the expertise in the team and the new solutions that will be invented, or technologies that will be integrated, to tackle company problems on a fee-for-service basis.
Q: Can you give a hypothetical example?
A: Any company, large or small, or an academic who is making cells likely has not addressed scale-up and manufacturing in an appropriate way. They are a potential client for not only the solutions we will come up with but also the fee-for-service optimization and scale-up that we will do.
Q: So, if I’m a scientist and I want to do some testing using a particular cell type, but I need millions and millions of cells, I come to you?
A: Yes. We might help in lots of different ways. If there is scale-up that’s needed, we would advise on what needs to get done and create a project around that. And we could hopefully help fund it. It could be fee-for-service or a co-development or any combination.
Q: The space that you have, will scientists be using it themselves or will they be hiring your team to do something for them?
A: It’s not that we would advise them what to do and they would go off to their labs and do it. This will be an advanced manufacturing centre. Our focus with this funding will be our new Good Manufacturing Practice (GMP) facility to make cells for clinical trials. We are going to hire 30 to 40 people to conduct these projects and do this fee-for-service work.
Q: You’ve got GE Healthcare as a major partner, but do you want more businesses to come in?
A: This is a consortium model. GE Healthcare is an anchor partner but we want to bring together and integrate technologies from other sources as well. So, we’re looking for technology partners. But we’re also looking for clients — cell therapy companies that need these solutions. An advantage of the site is it is in such a rich clinical environment in Toronto and, more broadly, Canada. That was a great attraction for GE because it enables them to engage with their customers in clinical trials and projects where the technologies can be stress-tested. This is real-time engagement of the community and the market.
Q: What’s the facility itself?
A: The facility is in MaRS (a not-for-profit corporation founded in downtown Toronto in 2000). It’s going to be part of an entire floor of activities focused on regenerative medicine. There will be the GMP facility there. Our CCRM employees will be there. It will also be the headquarters for the Ontario Institute for Regenerative Medicine and the University of Toronto’s new Medicine by Design project. So when people enter the 10th floor of MaRS, they are going to see a very integrated, co-ordinated ecosystem in and around cell therapy and regenerative medicine.
Q: Will you be working with other cell manufacturing centres like the one opening in Edmonton, and centres in Laval, Montreal and Ottawa?
A: We’re working with those through CellCAN, that’s one point of contact. I was just in Montreal and promoting the idea of the centre being the process development arm. If we need to optimize or scale up manufacturing we can do it in our centre and transfer those solutions back to manufacturing facilities as needed across the country.
Q: So it’s the Canadian model of working collaboratively, not competitively?
A: Absolutely. Although this will be, within MaRS, a very unique ecosystem, it has to be integrated with other activities across the country. It has to be integrated with other activities across the globe. Trying to achieve the proper scale is impossible with one centre or even one country.
Q: What are the anticipated outcomes?
A: The outcomes will be new technologies and tools to enable clinical- and commercial-scale manufacturing of cells — so there will be very tangible widgets that come out. Over the medium term, there will be a blueprint for cell manufacturing of the future,with integration of a number of technologies. We believe this centre will attract clinical trials to Canada and accelerate the development of technologies and clinical translation so more patients will be receiving cell therapies. It will support companies created in Canada but, as I mentioned, attract companies to Canada. We can’t just talk about scientific leadership anymore. We have to be leading commercialization. We need to be leading translation, through clinical trials. And this piece is manufacturing. Because with manufacturing comes stickiness and companies that are sustainable in Canada.
Q: What do you mean by ‘stickiness?’
A: This is advanced manufacturing; it’s not like making a car that you can diffuse production to the cheapest site in the world. This is an area where the leading edge is still being developed. If we are the leaders in manufacturing cells and those cells get manufactured here, then the companies that are here and the jobs we create here will be sticky.
The National Public Cord Blood Bank will have an impact in cities beyond those doing the collection.
The first cord blood collecting facility was opened in September 2013 in Ottawa, followed by Brampton, Edmonton and Vancouver, where a collection facility was launched in January at BC Women’s Hospital and Health Centre.…
The National Public Cord Blood Bank will have an impact in cities beyond those doing the collection.
The first cord blood collecting facility was opened in September 2013 in Ottawa, followed by Brampton, Edmonton and Vancouver, where a collection facility was launched in January at BC Women’s Hospital and Health Centre.
The Calgary Herald reported yesterday that the collection of umbilical cord blood will benefit the Southern Alberta city’s hospitals making it possible to perform stem cell transplants at the Alberta Children’s Hospital and the Tom Baker Cancer Centre later this year.
“There’s a good chance we may find donors for Canadian children in the Canadian cord bank,” says Dr. Victor Lewis, a pediatric oncologist at the Alberta Children’s Hospital.
Cord blood cells are a rich source of stem cells, which can be transplanted to treat diseases such as leukemia and lymphoma, which account for almost half of all cancers occurring in children between the ages of 0 and 14.
The existence of a national bank will reduce the costs of importing cord blood units from abroad and will increase the chances of finding a better match for Canadians in need of a transplant.
Toronto Life has profiled 30 of “Toronto’s Best Doctors,” with about 1,000 of the city’s physicians participating in a poll to nominate the best MDs based on skills, reputation and their contributions to their field of specialty.…
Toronto Life has profiled 30 of “Toronto’s Best Doctors,” with about 1,000 of the city’s physicians participating in a poll to nominate the best MDs based on skills, reputation and their contributions to their field of specialty.
Dr. Allan R. Slomovic, the Research Director of the Cornea/External Disease Service at the Toronto Western Hospital, University Health Network (UHN), has been profiled as a top eye surgeon.
We recently featured the story of Taylor Binns, who suffered from limbal stem cell deficiency, a rare condition that occurs when the stem cells in a narrow band of tissue around the cornea break down, causing blindness and extreme pain. Dr. Slomovic performed first stem cell transplant in Canada to treat the condition and Taylor is now free of pain and back to his normal life.
Dr. Slomovic has done about eight limbal stem cell transplants in the last two years and is hoping to make UHN the leader in the field of ocular regenerative program.
Taylor Binns’ transformation from blindness to “as close to 20/20 as can be with corrective lenses” may seem like story book stuff, but more than three years after his limbal stem cell transplant, he continues to write new chapters.…
Taylor Binns’ transformation from blindness to “as close to 20/20 as can be with corrective lenses” may seem like story book stuff, but more than three years after his limbal stem cell transplant, he continues to write new chapters.
“I am working for a consulting firm, but I am also in a process of getting into medical school,” says the 26-year-old. “The hope is that within a year-and-a-half I will be in medical school somewhere.”
He is also back on the road, driving a car. Back playing his beloved sport of rugby. And free of the piercing pain that staggered him for almost four years while he was working on his commerce degree at Queen’s University in Kingston.
“It was excruciating,” says Taylor, who grew up in Orillia, Ontario. “Imagine the worst time you ever had with something in your eye. And there was a constant burning sensation.”
Limbal stem cell deficiency (LSCD), a rare condition that occurs when the stem cells in a narrow band of tissue around the cornea break down, produced Taylor’s blindness and eye agony. Common causes are chemical damage or burns, but sometimes the condition is congenital. Contact lenses, which Taylor wore, have also been implicated.
As the video above dramatically shows, it was limbal stem cells harvested from his sister Tori that returned Taylor’s sight and banished his pain. Beyond undergoing the procedure, donating her cells posed no problems for Tori. “She’s doing great,” says Taylor. “She’s living in Vancouver where she does hair and makeup for movies and TV shows.”
Taylor, whose LSCD struck during a summer volunteer work stint in Haiti, can’t say enough about Dr Allan R. Slomovic, who performed his four operations at Toronto Western Hospital, beginning in November of 2010. “I was sent to see many doctors around North America and there is no one I would recommend more than him. Professionally and personally, he’s the best.”
Dr. Slomovic, who has done about eight limbal stem cell transplants in the last two years, says the success rate has been good. “But not everyone’s like Taylor. He was very fortunate: when we removed the scar tissue from his cornea, the underlying cornea itself was healthy.”
Dr. Slomovic credits Dr. Edward Cole and the staff at University Health Network’s Renal Transplant Program for making limbal stem cell transplants such a success. Their experience in arranging living and deceased donor kidney transplants gives them the expertise to ensure the most compatible donor is found and that the recipient is put on the most appropriate immunosuppression regime after receiving the donated cells. “It’s a team effort,” says Dr. Slomovic.
As for Taylor, the world is no longer a dark and painful place. His checkups are down to one every six months. Other than “a little bit” of immunosuppressant drugs, he is no longer on medication.
(For more on stem cells and eye diseases click here.)