Archives des Stem Cell | The Ottawa Hospital Foundation

Published: May 2024

Emmy Cogan was extremely tiny when she arrived in this world, but the impact of her birth was big. Born at 23 weeks gestation, she weighed only 515 grams — that’s just over one pound. Emmy was one of nine babies enrolled in a world-first cell therapy trial to heal the lungs of preemies and was the first in North America to receive the therapy. Now, that promising trial is ready for its next phase.

Her early arrival happened not long after first-time parents Alicia Racine and Mike Cogan returned from a trip to Hawaii. Alicia was back at work as a 911 operator for the Ottawa Police when her water broke.

“My sister works with me, and she brought me to The Ottawa Hospital’s General Campus. I was in a lot of pain, and I wasn’t too sure what was going on. And then we found out that it was contractions, and I started dilating,” explains Alicia.

The baby would hold on for another six days before being born on February 20, 2023. Those few extra days in the womb were critical to give Emmy a chance at life. “It changed the game entirely for us and her. She was able to be intubated, and she just started fighting from that moment on,” explains Mike.

Health challenges lie ahead

Initially, Emmy was cared for in The Ottawa Hospital’s Neonatal Intensive Care Unit (NICU), followed by 10 days at CHEO before returning to our hospital.

Emmy’s first month of life faced many challenges, including a duct between her heart and lungs that wouldn’t close, gastro-intestinal issues causing her to become septic, and concerns of a blood infection. Once Emmy got through those life-threatening issues, she was extubated and put on a high-flow oxygen. “We got to hold her for the first time at that point and my parents were able to be there for that, which was really nice,” says Mike.

Emmy also developed bronchopulmonary dysplasia (BPD). This is a condition known to affect many preemies. Because these infants are born so prematurely, their tiny lungs are underdeveloped and require extra oxygen to help them breathe properly. But giving this oxygen — critical for survival — can damage their tiny lungs. It’s like starting life with emphysema.

The devastating impact of BPD

In Canada, 1,000 babies are diagnosed with BPD every year. That number jumps to approximately 150,000 worldwide. Often, babies with BPD develop other chronic lung diseases, such as asthma, and may require prolonged oxygen and ventilation.

Additionally, they have a high rate of hospital readmissions in the first two years of life. Babies with BPD often have problems in other organs as well, such as the brain or eyes. There is currently no cure, but this world-first clinical trial led by Dr. Bernard Thébaud, a senior scientist and neonatologist, hopes to change that.

Two decades ago, Dr. Thébaud’s team discovered that stem cells from the umbilical cord — known as mesenchymal stromal cells (MSCs) — could heal lung injury and prevent BPD in newborn rodents. Since then, the team has worked tirelessly, here at home and collaborating with other scientists around the world, to bring this novel therapy to babies and their families through clinical trials. While other trials have tested MSCs for treatment of BPD in premature babies, no other group has used MSCs taken from the whole umbilical cord and processed them the way that Dr. Thébaud’s team has. 

What is bronchopulmonary dysplasia?

Bronchopulmonary dysplasia — or BPD — is a chronic lung disease that most often occurs in premature or low-weight babies who have received supplemental oxygen or received mechanical ventilation for long periods.

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“In our rodent research, we’ve used stem cells isolated from the umbilical cords of healthy newborns to prevent lung injury or even to some degree regenerate the damaged lung,” says Dr. Thébaud. “We foresee that these stem cells, given during a certain time during the hospital stay of these babies, could prevent the progression of the disease.”

Shortly after Emmy’s birth, her parents met Chantal Horth, a clinical trial coordinator, and were introduced to Dr. Thébaud. “Chantal came to us and said Emmy qualified for the trial,” remembers Mike. “It sounded like a great opportunity.”

"Being a preemie, she’s going to have some health issues, and anything that could help her, we wanted to give her that extra shot."

— Alicia Racine

Saying ‘yes’ to a world-first clinical trial

The couple met with Dr. Thébaud, and he answered a long list of questions they had about the trial. “He’s a very personable guy, and it was very easy to talk to him. We trusted him. Being a preemie, she’s going to have some health issues, and anything that could help her, we wanted to give her that extra shot,” says Alicia.

To qualify for the trial, the premature babies — born at 23- or 24-weeks’ gestation at The Ottawa Hospital — had to be seven to 21 days old and treated in the NICU. They also had to require 35% oxygen. This level of oxygen puts them at 60-70% risk of developing BPD. Sunnybrook Health Sciences Centre recruited one baby, becoming the second site involved.

On March 3, 2023, at 11 days old, Emmy received an IV infusion of umbilical cord tissue grown from the donated cords of healthy newborns. It was a special moment for everyone involved. She was the first baby in North America to receive this kind of therapy.

"This is the first trial of its kind in the world, and what could be more rewarding than helping preemies?"

– Dr. Bernard Thébaud

“Dr. Thébaud administered the stem cells, and everyone clapped,” says Mike. “She will have follow-up appointments at different stages for two years, and then she’s going to be followed up by phone for 10 years.”

For Dr. Thébaud, it was a moment he and his team had dreamed about. “It was an exciting and huge milestone when that day arrived — after 20 years of work we were able to test this therapy for the first time in a patient. This is the first trial of its kind in the world and what could be more rewarding than helping preemies?”

The next step for this stem cell trial

Thanks to those nine tiny patients, including Emmy, recruitment for the Phase 1 trial is now complete. The purpose of this trial is to test the feasibility and safety of the stem cell therapy. The next phase will test safety as well as how effective it is.

"All the stars lined up to have her be a part of that little piece of history — something that could impact babies like her in the future.”

– Alicia Racine

“Now we can determine if this therapy will make a difference in patients,” explains Dr. Thébaud. “There will be two groups in the next phase — one that will receive cells and one that will receive the placebo — it’s a randomized controlled trial. We’ll need 168 patients to determine if these stem cells make a difference.”

While babies for the first phase were recruited from NICUs at The Ottawa Hospital and Sunnybrook Health Sciences Centre, the next phase will be a multi-centre trial across the country. Dr. Thébaud hopes it will begin by the end of 2024 and it will take two years.

“Working with babies is, I think, the most beautiful job on Earth. Because they’re born, and they have all their life and all their potential in front of them. Our task is to give them a great jumpstart,” says Dr. Thébaud.

As for Emmy, she left the hospital five months after she was born, and while Mike and Alicia don’t know if the stem cells impacted her health, Emmy is doing well. “We don’t know what she would be like without it, but she’s awesome right now,” says Mike. “We felt very fortunate to be in the right place at the right time for our little girl.”

It’s something Alicia feels makes Emmy all the more unique. “All the stars lined up to have her be a part of that little piece of history — something that could impact babies like her in the future,” explains Alicia.

That’s certainly what Dr. Thébaud is hoping for. “It would change the way we care for premature babies. It’s my hope that these tiny patients have a chance to thrive, grow up, and have an impact on the world around them.”

Emmy doesn’t know she’s made history, but that’s ok. For now, she’s keeping her parents busy. She’s pulling herself up and will be walking in no time. She’s also been off oxygen since November 2023, giving her even more mobility. “It was really fun to have her free. We had a cordless baby for the first time! That was a big step when she didn’t need to rely on the oxygen anymore,” smiles Mike.

This Phase 1 trial is funded by the Stem Cell Network with in-kind matching funds from MDTB Cells GmbH. Dr. Thébaud’s research is also possible because of funding from the Ontario Institute for Regenerative Medicine, the Canadian Institutes of Health Research, The Ottawa Hospital Foundation, and the CHEO Foundation. 

Categories: NICU, Patient Care

Published: March 2023

The search for the silver bullet for sepsis has been decades in the making. However, The Ottawa Hospital is taking a big step forward in the next phase of a world-first clinical trial using stem cells in patients with septic shock — not so much a silver bullet, but a seed that could lead to future innovative treatment options and impact millions of patients. The hope is to not only save more lives but also improve the quality of life of those who do survive this devastating illness.

Sepsis is caused by our own body’s response to infection. When that infection spreads through the blood stream and over-activates the immune and coagulation systems, it can cause the heart and other organs to fail. Sepsis is associated with a death rate from 20% to 40% and upwards from that, depending on the patient. Survivors of this devastating condition often have their quality of life impacted and often for the long term. Sepsis knows no borders and impacts people globally.

World-Leading Research

Each year, sepsis affects 49 million people worldwide. But researchers here at The Ottawa Hospital are working to make an impact with a world-first clinical trial exploring the use of mesenchymal stem cells to treat septic shock.

What is Sepsis?

Sepsis occurs when the body has an extreme, life-threatening response to an infection. The infection includes bacteria that enter the blood stream, triggering a chain reaction during which the patient’s immune system response damages its own tissues, potentially leading to organ failure and death.

Learn More

Dr. Lauralyn McIntyre is an intensive care unit (ICU) physician and senior scientist at The Ottawa Hospital, and it’s her care of critically ill patients that has motivated her research into sepsis. Over the years, she’s witnessed the debilitating impact it can have on patients and their families. “It’s why I’m doing this research. As researchers, we love science. We love posing questions and the thinking that goes with these questions, and we love the answering those scientific questions. But the main reason we’re doing research is to help patients,” says Dr. McIntyre. “If there’s some way we can just move that needle to help these patients and their families, that just means so much.”

The global impact of sepsis

Sepsis is recognized as a global health priority. It’s estimated there are 48.9 million cases of sepsis annually and 11 million sepsis-related deaths — those account for almost 20% of global deaths. It is also the leading cause of death among COVID-19 patients.

To put that in perspective, a study published in 2021 led by researchers at The Ottawa Hospital and ICES (Institute for Clinical Evaluative Sciences) showed that severe sepsis is linked with higher mortality, increased hospital readmission, and higher healthcare costs. In Ontario alone, sepsis related costs are estimated at $1 billion per year.

“It’s the complexity of the infection and the challenge that drew me to the research, but also knowing the potential to really help patients and see if we can make them better.”

– Dr. Lauralyn McIntyre

According to Dr. McIntyre, sepsis is the most common reason why patients are admitted to ICUs. “They account for about 20% of the cases in the ICU at our hospital. From a provincial glance, over a four-year period, there were 270,000 cases of patients that were admitted to hospitals in Ontario for infection — about 30% had the more severe form of sepsis, with infection plus organ failure which amounts to about 67,500 patients a year in Ontario alone – it’s staggering,” explains Dr. McIntyre.

These data are a key motivator to learn more about sepsis and how to treat it. “It’s the complexity of the infection and the challenge that drew me to the research, but also knowing the potential to help patients and see if we can make them better,” says Dr. McIntyre.

Putting a face to the impact of the infection

Ten years ago, sepsis changed the life of Christine Caron — a single, working mother with four children who, at the time, ranged in age from 15 to 24. Throughout the winter and spring of 2013, she hadn’t been feeling well. Then in late May, while playing tug-of-war with her four dogs, her left hand was accidentally nipped. “It wasn’t a serious bite, just a break in the skin. I had no redness or pain, so I washed it out and disinfected the area,” recalls Christine.

Four days later when Christine was at work, she realized she hadn’t gone to the bathroom all day — eventually she learned this was because her kidneys were shutting down. The following day, she set out for a morning run. “I was winded and had to walk home but felt better after a shower. Later that day, I had terrible stomach pain — like someone had punched me in the stomach — and felt disoriented. I went home and slept. My son woke me up at one point to say I was breathing funny, but I assured him I was fine and fell back to sleep. I was shocked when I woke up and realized how long I had been asleep,” says Christine.

Christine Caron is a survivor of sepsis.

She remembers feeling agitated and more symptoms developed, including sweating despite feeling cold and becoming very thirsty. She went to a local urgent care centre, but it was closed. “I had no idea how sick I was, and the thought of sitting in an emergency department was overwhelming. I decided if I wasn’t feeling better in the morning that I would go to the hospital then.” 

Later that night, while her children slept, she became very sick — flu-like symptoms as she describes it. “I lay on the bathroom floor, probably ‘till three in the morning. I thought about calling an ambulance, but I didn’t want to wake up my family,” says Christine. “I wasn’t thinking clearly. I now know this was delirium.”

The next morning, a friend took Christine to a local hospital. “I was dizzy, I could barely breathe. I handed my health card to the attending nurse and then I collapsed,” explains Christine.

Christine wouldn’t regain consciousness for a month. On June 13, she woke up at the Civic Campus of The Ottawa Hospital to learn the devastating news of what sepsis had done to her body. This was when she heard about septic shock for the first time. “I had bronchitis that progressed to walking pneumonia. It was this condition that compromised my immune system resulting in the reaction to the bacteria when I was nipped by my dog. It quickly escalated to septic shock.”

As it would turn out, the sepsis infection had caused irreparable damage. By June 22, Christine began a series of surgeries to amputate her legs, her left arm, and remove dead tissue from her remaining limb and her face — changing her life forever. Little did she know at the time, but this set her on a path of becoming a voice for sepsis survivors. By early July, she was released from the hospital and would learn a new way of life at our Rehabilitation Centre, where she learned to walk again and received support for PTSD. Today, Christine is an active advocate for sepsis survivors, awareness, and for research.

Christine with her dogs.

Christine with her family.

Moving the needle for sepsis treatment

For decades, there has been little progress in advancing specific treatment for sepsis, but world-first research at our hospital shows that a specific type of stem cells may be the key to helping balance out the body’s immune system to improve its response to sepsis. Laboratory studies and early clinical trial results were so promising that Dr. McIntyre’s research was awarded $2.3 million from the Canadian Institutes of Health Research and the Stem Cell Network to begin a larger trial. “Researchers around the world have spent decades trying to find new therapies for septic shock, but so far nothing has improved survival, nor the quality of life for survivors of this devastating illness,” says Dr. McIntyre. “We urgently need new treatments for septic shock and to test them in randomized controlled trials like this one.”

This injection of funds will allow the team to expand the trial to 10 centres across Canada to see whether the stem cells can reduce patients’ needs for organ support in the ICU.

For Dr. McIntyre, this research, which is a huge collaboration among hospital colleagues, including Drs. Duncan Stewart, Dean Fergusson, and Shirley Mei, as well as colleagues throughout Canada and abroad. It provides hope that years of dedication to this mysterious illness may finally move the needle forward for sepsis treatment. “These stem cells hold, in my opinion, immense therapeutic promise for the treatment of sepsis, because these cells act through many mechanisms that relate to sepsis. Not only do they recognize and ultimately kill the bugs causing the infection, but they also calm the immune and blood-clotting responses that our body has to the infection,” explains Dr. McIntyre.

“I see this trial as the very first beginning — it’s a little bud, and we’re just going to grow from it.”

– Dr. Lauralyn McIntyre

And while Dr. McIntyre says her research has shown these cells have other benefits, such as restoring energy to the tissues, and reducing vessel leakiness and the swelling that goes with it, treating sepsis is still an enormously complex problem. “We can’t expect that there’s a silver bullet that’s going to completely cure sepsis, but from what we have learned so far, these cells have the potential to make a real dent in the immense death from sepsis, and we hope will improve the quality of life for survivors of this devastating illness.”

The “little bud” that will grow into future sepsis research

This clinical trial is just the starting point to learn more about this deadly infection, and the results will help inform future trials. As the research advances, and more is learned about how the body responds to these cells during sepsis, it will help identify future patients that may have the most to benefit. “So, I see this trial as the very beginning — it’s a little bud and we’re just going to grow from it,” explains Dr. McIntyre.

The growth of this research has been cultivated by what Dr. McIntyre describes as a major collaborative team approach. It includes researchers, both basic and clinical, cell manufacturing experts, trainees, project managers, clinicians, and nurses, as well as patient and family partners, and sepsis survivors, like Christine, who is the lead patient partner. “Working with these patient partners has just been illuminating about post-sepsis survivorship. People like Christine have been so helpful in enabling us to understand the need to study more about the survivorship of these patients and their families, and the quality of that survival,” explains Dr. McIntyre.

“Sepsis took so much from me — it scarred me in so many ways. We need to advocate and educate because sepsis does not discriminate.”

– Christine Caron

There’s a mutual admiration between the two women, who have each seen sepsis through a very different lens. Christine is thrilled to have her voice heard and to see that needle move forward. “Dr. McIntyre’s research is phenomenal because a lot of patients come out with organ damage, and stem cell research could save so much for so many people. Wouldn’t it be so wonderful if it did?” Christine adds, “Sepsis took so much from me — it scarred me in so many ways. We need to advocate and educate because sepsis does not discriminate.”

“If there's something that we can do to reduce death and help how patients survive this immense illness, we’ve just got to go there.”

– Dr. Lauralyn McIntyre

And so, for Dr. McIntyre, it’s those faces she sees in the ICU and those like Christine, who work alongside her, that continue to motivate her with each step forward in the search for answers in this challenging puzzle of sepsis. “If there’s something that we can do to reduce death and help how patients survive this immense illness, we’ve just got to go there.”

The Ottawa Hospital is a leading academic health, research, and learning hospital proudly affiliated with the University of Ottawa.

Categories: New Campus Development, World-Leading Research

Published: September 2022

When Geneviève Bétournay reflects on the past decade, she thinks about the adversity she has overcome. As the owner of the Art House, a coffee house/art gallery, she’s endured keeping her business alive during the pandemic. However, an even bigger challenge was her diagnosis of multiple sclerosis (MS) in 2010. Today, thanks to a life-changing stem cell transplant at The Ottawa Hospital, she has a whole new perspective on the possibilities that lie ahead for her.

Find out how the Campaign to Create Tomorrow will take research to unprecendented heights.

Geneviève is no stranger to adapting to change. She was in her early 20s and going to university when she started to have issues with her vision. Especially during stressful times, her vision would become blurry, but she didn’t associate it with anything more serious. That changed when she was 23 and living in Japan — new symptoms emerged. “I had more issues with my vision. It was getting worse and that was scary. Also, my foot would drop — it would drag when I tried to move it and there were other issues related to movement,” recalls Geneviève.

News of MS diagnosis hits hard

When she returned home from Japan, she started to seek answers, but it was some time before anyone would connect the dots and uncover the problem. “Ultimately, it was the vision issues that ended up getting me to see a neurologist because basically, my optometrist was able to get my prescription right, but my vision was still blurred,” explains Geneviève.

It was Geneviève’s neuro-ophthalmologist that first revealed the severity of what she was facing. Early indications suggested her symptoms could be the result of a brain tumour or MS. “I remember that day sitting in the office. I was extremely emotional because I didn’t know what that meant to be honest. When you grow up, in our society as it is, chronic illness is not something that’s talked about all that often.”

An MRI finally provided Geneviève with answers. But along with those answers came the shock of an MS diagnosis.

“What is life with MS? What would life even look like? It just sounded scary. I didn’t know what was going to happen to me.”

– Geneviève Bétournay

MS is a condition that can affect the brain and spinal cord, causing a wide range of potential symptoms, including problems with vision, arm or leg movement, sensation, and balance. For Geneviève, there were so many unknowns and even more questions. “What is life with MS? What would life even look like? It just sounded scary. I didn’t know what was going to happen to me,” she says.

Effects of MS on the body

Fatigue, cognitive disruption, mood changes
Vision issues such as vision loss, blurred vision, and double vision
Difficulty with speech and swallowing
Incontinence and digestive problems
Difficulty with hand-eye coordination and numbness/tingling in hands
Sexual dysfunction
Loss of power in a limb or numbness
Walking difficulties and balance problems

Groundbreaking MS treatment pioneered in Ottawa

Once Geneviève and her family processed the news of her diagnosis, she learned everything she could about the illness. A family friend, who happens to be a nurse, had heard a lot about Dr. Mark Freedman and his transformational MS stem cell transplant research in collaboration with Dr. Harold Atkins and encouraged Geneviève to get in touch with The Ottawa Hospital’s MS Clinic. Meeting Dr. Freedman was a pivotal day that would shed new light on her future. “I see it as one of those very lucky life-changing moments when I made that call,” explains Geneviève.

Twenty-four years ago, many were skeptical when Drs. Atkins and Freedman first proposed the idea of using stem cells to reprogram the immune system to halt the progression of MS. Today, they are known for pioneering this groundbreaking treatment which is now being used in many countries around the world. In fact, it was serendipity that led Dr. Atkins, a hematologist, and Dr. Freedman, a neurologist, to meet while they were working on a different project. The two started to discuss stem cell transplants and that would ultimately lead to the transformation of MS treatment.

While this was a time of uncertainty for Geneviève, she felt she was in the right hands to handle the complexity of her case. “Dr. Freedman was very kind from the beginning. It was very comforting to know that there were multiple treatment options if something didn’t work.”

Initially, Geneviève received the first line of treatment for a year that included injections every two days; however, it didn’t provide positive results. Next in line was a form of chemotherapy that targeted her immune cells. She remained on that treatment for two years, but once again, the results didn’t have the impact her medical team had hoped for, so, in January 2013, Dr. Freedman recommended the MS stem cell transplant.

MS symptoms were progressing quickly

At this point, Geneviève’s MS was progressing quickly. Both of her eyes were now affected, and increasingly her mobility was impacted. “I felt numbness below the waist. It was very difficult for me to walk unassisted. I would need something to hold on to — either a person or a wall or something like that. There were also issues with muscle spasms and dizziness,” explains Geneviève.

Headaches were also a challenge, and life was becoming more difficult for this young woman who was working on her Master’s degree at UOttawa. Her degree was in organic chemistry, and lab work was challenging because of blind spots in her vision and a lack of dexterity. When it came time to decide whether to participate in the transplant, she explains there was no decision to make. “I knew about this treatment from the beginning. It was always in the back of my mind. I had already processed it. I do remember being sad that nothing else had worked and I had to do this or else my condition would continue to go downhill rapidly. It gave me hope but it was a very emotional day,” recalls Geneviève.

“I felt numbness below the waist. It was very difficult for me to walk unassisted. I would need something to hold on to.”

– Geneviève Bétournay

She remembers the compassionate support she received from Dr. Freedman when it was time to consider the transplant. He explained it would be one of the hardest decisions she’d ever make and encouraged her to take the time to make sure it was right for her.

Geneviève learned that she could become infertile because of the procedure, so in the few months before her transplant, she had some of her eggs harvested and she also received a shot that could allow her eggs to go into “hiding” during the treatment.

Time for the MS stem cell transplant

In July 2013, Geneviève received her stem cell transplant. The process begins with purifying and freezing the patient’s stem cells which will later be “cleaned” in the lab. The next step is a strong chemotherapy treatment to destroy the patient’s immune system. The final step is the transplant of the clean stem cells back into the patient — that’s when a new immune system starts to develop.

It’s not an easy process, but Geneviève was focused on what the result might give her. “Mentally and physically, it was challenging, but to be honest, I’m a different person today because I went through that.”

How did a stem cell transplant halt MS?

Geneviève underwent an immunoablation and autologous hematopoietic stem cell transplantation (IAHST) to treat her MS. It’s a groundbreaking treatment pioneered by researchers at The Ottawa Hospital for patients with certain forms of multiple sclerosis. It involves harvesting and treating a patient’s own stem cells to remove traces of disease, eliminating their immune system, and then creating a new one using their newly “cleaned” stem cells. IAHSCT is currently being used to treat two other rare autoimmune disorders: myasthenia gravis and stiff person syndrome.

Learn more about stem cells.

“Those moments where something happened that I wasn’t able to do before – it’s like pure euphoria. It was joy. It was gratitude for something that you never thought you’d ever feel again.”

– Geneviève Bétournay

Geneviève says she was in the hospital for eight days before she was able to go home because she didn’t have any major infections or require constant monitoring. Also at that point, her cell counts started to go back up — her new immune system was starting to grow. She returned to the hospital as an out-patient for several months.

The next step was to determine the impact the transplant would have on her overall health. It took between eight and 12 months for Geneviève to recover, and then she started to notice tiny improvements. “That’s when I first noticed I could do something that I couldn’t do before, or I had lost the ability to do. The first thing I noticed was I could lift my right leg, because my right side was more affected than the left.”

As Geneviève saw these small improvements begin, she tried to temper her expectations. She didn’t want to let her hopes get too high, but she admits sometimes she couldn’t resist. “Those moments where something happened that I wasn’t able to do before — it was like pure euphoria. It was a joy. It was gratitude for something that I never thought I’d ever feel again.”

Was it all worth it?

Over the past decade, Geneviève has seen every single symptom get better. From lifting a single toe to moving a foot to being able to jump again, and then walking several kilometres without tripping and falling to the ground — these are big milestones on her road to recovery. The signs of MS are disappearing. “To date, it would seem I have not had any new relapses. No new disease activity. My vision improved, and I continue to recover, albeit very slowly as it takes a while to heal.”

When Geneviève reflects on how far she’s come, had she not undergone the stem cell transplant, she believes she would likely be in a wheelchair today. “It was 100% worth it. Just the fact that I can move blows my mind. I have a great deal of gratitude for the doctors and everything the hospital had done for me.”

And today, with only a slight limp, she walks up the steps into the Art House and appreciates each moment she has to celebrate the creative artists in our city and transformational treatment she received at our hospital. “It’s priceless what I have gained. Aside from developing MS, nothing has had a more extensive impact on my life than undergoing this stem cell treatment. Simply put, it saved my life, or perhaps you could say it gave me a second one.”

“It’s priceless what I have gained. Aside from developing MS, nothing has had a more extensive impact on my life than undergoing this stem cell treatment.”

– Geneviève Bétournay

Listen to Geneviève share her journey with MS in episode 66 of Pulse Podcast.

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Categories: Clinical Trials, Clinical Trials, Clinical Trials, Clinical Trials, Clinical Trials, Creating Tomorrow, Neuro, Patient Care, Patient Care

Vittorio Petrin has never seen his grandchildren’s faces. The Italian draftsman started to lose his peripheral vision in the early 1980’s after his second son was born, forcing him to leave work and take an early pension. He was diagnosed with retinitis pigmentosa, a genetic disorder that causes the cells in the retina to break down. There is no cure. His vision steadily got worse until he couldn’t see any light at all.

Before his vision went dark, Vittorio spent six years building a model of St. Mark’s Basilica in Venice, using over 3,000 copper pieces. “It was the most beautiful place I’d seen, and I wanted to replicate it. Working on it kept my mind away from what was going to happen,” he says.

An image of Vittorio Petrin with a replica of St. Mark’s Basilica he built while losing his vision to retinitis pigmentosa. — Vittorio Petrin with a replica of St. Mark’s Basilica he built while losing his vision to retinitis pigmentosa.

“My dad was an artist. He was able to draw phenomenally, he liked taking videos. Sight was important to him,” says Vittorio’s son Dino Petrin. “He never complained about going blind, we never saw it as children. He always had a sense of humour and a strong character. He never asked for any pity, he just took it in stride.”

Millions of people in North America live with retinal diseases like retinitis pigmentosa, glaucoma, retinal ischemia and age-related macular degeneration. These diseases are poorly understood, progressive, and often untreatable.

But thanks to promising gene and cell therapies in development, Dino hopes that one day people like his father won’t have to lose their vision.

Dr. Catherine Tsilfidis' research is aimed at developing a gene therapy strategy that blocks apoptosis and slows down retinal disease progression. — Dr. Catherine Tsilfidis

“Soon we’ll be able to do what our lab has been trying to do all along – bring XIAP gene therapy into the clinic.”

– Dr. Catherine Tsilfidis

A discovery with game-changing potential

Dr. Catherine Tsilfidis can imagine the day when the first patient is treated with the retinal disease gene therapy her lab has worked on for the past 20 years. While it won’t happen tomorrow, that day is not far off.

“XIAP gene therapy is exciting because it keeps cells in the back of the eye from dying,” said Dr. Tsilfidis, a senior scientist at The Ottawa Hospital and associate professor at the University of Ottawa. “It could slow or stop vision loss caused by many different retinal diseases.”

Dr. Tsilfidis is leading a world-class team of researchers that recently received $2.4 M from the Ontario Research Fund to develop gene and cell therapies for retinal diseases. One of their goals is to do the work needed to bring XIAP gene therapy into clinical trials, which could start in the next few years.

The time is right for gene and cell therapy

The promise of replacing defective genes and cells in the eye with healthy ones is undeniable. While these fields are still in their infancy, they are expected to grow exponentially over the next decade. Gene therapy for the eyes has particularly taken off, with Health Canada approval of the first gene therapy for a rare genetic form of vision loss in 2020.

“This research program could make Ontario a leader in the fields of both gene and stem cell therapy

– Dr. Pierre Mattar

When it comes to cell therapies, Ottawa and Toronto are major hubs in the growing area of stem cell research. As partners in the retinal research program led by Dr. Tsilfidis, UHN scientist Dr. Valerie Wallace will work on increasing the survival of transplanted stem cells in the eye, while The Ottawa Hospital’s Dr. Pierre Mattar aims to develop stem cell therapies for retinal ganglion cell diseases such as glaucoma. “This research program could make Ontario a leader in the fields of both gene and stem cell therapy,” said Dr. Mattar. “By learning the best way to mass produce and integrate stem cells for retinal disease, we can advance stem cell research in other fields.”

The Ottawa Hospital's Dr. Pierre Mattar aims to develop stem cell therapies for retinal ganglion cell diseases such as glaucoma. — Dr. Pierre Mattar

Collaboration between lab researchers and clinicians key to success

The incredible challenge of bringing a basic science discovery to clinical trials requires an exceptional team. For this research program, Dr. Tsilfidis assembled a “dream team” of long-time collaborators and new partners.

As a basic scientist, Dr. Tsilfidis has always worked closely with clinicians to help ensure her research reflects patient needs.

“Ophthalmologists help us identify the most important questions to ask,” said Dr. Tsilfidis. “Our lab started working on diseases like Leber hereditary optic neuropathy and glaucoma because clinicians told us how much of a problem they were.”

Two of Dr. Tsilfidis’ long-time clinical collaborators, Drs. Stuart Coupland and Brian Leonard, are part of this new retinal research program. They are joined by retina specialists Drs. Bernard Hurley and Michael Dollin, who will assist in developing clinical trial protocols.

“Our researchers have an incredible track record of taking discoveries from the lab to the bedside,”

– Dr. Duncan Stewart

Dr. Tsilfidis’ lab and office are just down the hall from the ophthalmologists’ offices and clinics, which makes collaboration easier. This kind of co-location of scientists and clinicians has been key to The Ottawa Hospital’s success in translating discoveries from the lab bench to the patient bedside.

The highly skilled team at The Ottawa Hospital's Biotherapeutics Manufacturing Centre will make the clinical-grade virus to deliver gene therapy into the eye. — The highly skilled team at our Biotherapeutics Manufacturing Centre will make the clinical-grade virus to deliver gene therapy into the eye.

Leveraging our biomanufacturing expertise at The Ottawa Hospital

In addition to clinical experts, the team knew they needed new resources and partners to be successful.

“We’ve been very much a basic science lab in the past,” said Dr. Tsilfidis. “Now that we’re at the stage that we want to get XIAP to the clinic, we need all the help we can get.”

One missing piece was a special clinical-grade virus used to deliver the XIAP gene into the eye, known as an adeno-associated virus (AAV). Finding cost-effective sources of AAVs has been a major bottleneck for getting gene therapy trials and treatments off the ground.

Thankfully, The Ottawa Hospital is home to the Biotherapeutics Manufacturing Centre (BMC), a world-class facility that has manufactured more than a dozen different virus- and cell-based products for human clinical trials on four continents. Experts at the BMC were already starting to expand into AAV manufacturing when Dr. Tsilfidis approached them about collaborating on the retinal research program.

The BMC has since been working with Dr. Tsilfidis and her team to develop a process to manufacture the AAVs the team will need for Health Canada approval of the XIAP gene therapy for clinical trials.

The BMC is on track to become the first facility in in Canada to make clinical-grade AAV vectors for gene therapy studies. This new expertise will help them support other gene therapy trials with a focus on rare disease.

Learn more about our Biotherapeutics Manufacturing Centre.

How to plan a world-class clinical trial

In addition to the clinical-grade virus, the retinal research team needed help planning a future clinical trial of XIAP gene therapy. Fortunately, there are no shortage of clinical trial experts at The Ottawa Hospital.

“I’ve never planned a clinical trial before,” said Dr. Tsilfidis “But I knew someone who had – Dr. Dean Fergusson. I’ve always been impressed by the rigorous trails he’s helped develop. When I asked for his advice, he referred me to the Ottawa Methods Centre.”

The Ottawa Methods Centre is The Ottawa Hospital’s one-stop shop for research expertise and support. Their goal is to help all clinicians, staff and researchers at the hospital conduct the highest quality research, using the best methods. They support over 200 research projects a year, led by clinical and basic researchers alike.

“The Ottawa Methods Centre has been amazing to work with,” said Dr. Tsilfidis. “Their research methodology expertise has strengthened this research program and our funding applications.”

Drs. Manoj Lalu and Dean Fergusson along with other experts at the Ottawa Methods Centre are helping to plan a future clinical trial of gene therapy for retinal disease.

At the Ottawa Methods Centre, the team is leveraging the Blueprint Translational Research Group’s Excelerator program, designed to enable efficient translation of basic research discoveries to the clinic through rigorous methods and approaches. Co-led by Dr. Dean Fergusson and Dr. Manoj Lalu, the program will help design the clinical trial protocol, and support the clinical trial application to Health Canada through systematic reviews of available pre-clinical and clinical data.

Research program holds enormous promise

Tackling retinal disease will be a big challenge, but Dr. Tsilfidis has assembled an excellent team of partners both old and new to move this research program forward.

“These therapies could be life-changing. If we could cure or slow down the progression of vision loss, that would be amazing.”

– Dino Petrin

“Our researchers have an incredible track record of taking discoveries from the lab to the bedside, but it can only be done through team efforts like this one,” said Dr. Duncan Stewart, Executive Vice-President of Research at The Ottawa Hospital and professor of medicine at the University of Ottawa. “Fully leveraging our basic and clinical expertise, as well as our world-class core research resources is the key to getting new treatments to the patients who need them.”

For Dr. Tsilfidis, the excitement is palpable. “Soon we’ll be able to do what our lab has been trying to do all along – bring XIAP gene therapy into the clinic.”

Dino, a former graduate student in Dr. Tsilfidis’ lab, sees the potential of gene therapies to help people like his father. “These therapies could be life-changing,” he said, “If we could cure or slow down the progression of vision loss, that would be amazing.”

Vittorio Petrin pictured with his wife Maria Petrin — Vittorio Petrin with his wife Maria Petrin

The Ottawa Hospital is a leading academic health, research, and learning hospital proudly affiliated with the University of Ottawa.

Categories: Biotherapeutics, Innovation and Technology, Technology

What are stem cells?

Every tissue and organ in the body is made up of billions of cells, each with a specialized function. Stem cells are the blank-slates from which all other types of cells originate. They have the ability to divide and make exact copies of themselves or produce specialized cells that can then become new heart muscle cells, brain cells, or other types of cells that can repair damaged organs and tissues.

Two types of stem cells are most commonly used in research:

Pluripotent stem cells

Pluripotent Stem Cells, like embryonic stem cells, are able to produce all the different kinds of cells of the body. Because of recent advances, this powerful stem cell can now be made in the laboratory by genetically reprogramming regular adult cells to make them behave like embryonic stem cells. These ‘induced pluripotent stem cells’ (iPSCs) are commonly used to investigate disease onset and progression, to speed up drug development, and to create new therapies for cell and organ transplant applications.

Somatic or adult stem cells

Somatic or adult stem cells are found in most mature tissues and organs. These cells are already somewhat specialized and can only make the kinds of cells found in the original tissue. They may also release biological factors into surrounding tissue, to encourage healing in other ways.

How are stem cells used?

Researchers have found that stem cells can be used to treat injury and disease by stimulating the body to repair itself. An early and common example of a stem cell treatment is a bone marrow transplant. A cutting-edge technique when it was introduced about 60 years ago, there are now more than 50,000 performed worldwide, every year.

Bone marrow transplants replace diseased stem cells, incapable of making normal blood cells, with stem cells derived from healthy bone marrow. These new stem cells grow and reproduce, allowing the marrow to make healthy, new blood cells. These procedures have revolutionized the way we treat blood disorders and cancers of the blood like leukemia or lymphoma.

Today, researchers are investigating new ways to use stem cells to rebuild damaged tissue throughout the body, and to treat a wide range of debilitating diseases. In fact, at The Ottawa Hospital our researchers are exploring stem cell therapies for every major system in the human body and we are now using stem cells as a standard treatment for certain kinds of multiple sclerosis.

We are also leading several groundbreaking clinical trials of stem cell treatments for conditions like heart attack, septic shock, COVID-19 and lung damage in premature babies.

How can I access stem cell therapy?

Stem cell therapy is still considered experimental in most cases. If you are interested in stem cell research, you should speak with your healthcare provider to find out if there are any clinical trials that you may be eligible for. You should also be skeptical of anyone who offers stem cell therapies for a fee.

Microscopic image of stem cells

Update: In early 2023, Dr. Thébaud’s team treated the first baby in their clinical trial that aims to prevent BPD in premature infants. This is the first trial of its kind in in the world. Read Dr. Thébaud’s Q&A to learn more about his critical work and what motivates him.

Published: August 2020

Little Olivia Eberts had oxygen tubes in her nose until after her first birthday. Because she was born prematurely, her tiny lungs were underdeveloped, and she couldn’t breathe without oxygen. Ironically for Olivia, and many premature babies like her, the oxygen that saved her life also damaged her lungs, causing bronchopulmonary dysplasia (BPD), which is like starting out life with emphysema. But a clinical trial at The Ottawa Hospital led by Dr. Bernard Thébaud, which uses stem cells to heal the lungs of premature babies, may be a game changer.

An unexpected early birth

Jamie Eberts was 22 weeks pregnant with twins when she started feeling some discomfort. She arrived at the General Campus of The Ottawa Hospital and was admitted — she was going into early labour.

Thankfully, the doctors and nurses at The Ottawa Hospital were ready for any scenario Jamie, her husband Tim, and the babies faced. Each day, there were gentle conversations about how the babies were doing, the process of delivering them, and the chances of survival. Every hour counted. Then, one of the babies developed an infection and all three lives were at risk — the babies had to be delivered.

Baby in NICU — Baby Olivia in the neonatal intensive care unit (NICU).

“Our babies, Liam and Olivia, were born at 5 a.m. on January 29, 2017, at 23-and-half-week’s gestation. Liam was born first. He was small, red, and didn’t make a sound,” remembers Jamie. Olivia weighed one pound, two ounces and Liam weighed only a few ounces more than she did. Both babies required oxygen and mechanical ventilation to keep them alive. As a result, both developed BPD — the most common cause of death in premature babies.

Sadly, baby Liam passed away a few weeks after he was born while Olivia remained in the Neonatal Intensive Care Unit (NICU) at The Ottawa Hospital for nine long months.

BPD in Canada

In Canada, 1,000 babies are diagnosed with BPD every year. Often, babies with BPD develop other chronic lung diseases, such as asthma, and many require prolonged oxygen and ventilation. Additionally, they have a high incidence of hospital readmissions in the first two years of life. Babies with BPD often have problems in other organs as well, such as the brain or the eyes.

When Olivia was finally discharged, she went home with an oxygen tank. During the first year of her life, Olivia spent more time in hospital than out.

“Even now, a simple flu that put me in bed for a couple of days put her in hospital and turns into pneumonia. It’s scary,” says Jamie. The doctor told her that with Olivia’s respiratory issues, she may require hospital intervention for the rest of her life.

Lack of treatment options

“Currently there is no treatment for this disease,” says Dr. Bernard Thébaud, a neonatologist and senior scientist at The Ottawa Hospital. With that being said, he’s determined to improve the outcome for babies, like Olivia, who have BPD.

 “In the laboratory, we discovered that a particular type of stem cell can prevent BPD or regenerate newborn lungs.”

— Dr. Bernard Thébaud

Read our Q&A with Dr. Bernard Thébaud

Dr. Bernard Thebaud, neonatal intensive care unit, The Ottawa Hospital — Dr. Bernard Thebaud in the neonatal intensive care unit (NICU) at The Ottawa Hospital.

“Our research uses stem cells, isolated from the umbilical cords of healthy newborns, to prevent the lung injury or even to some degree regenerate a damaged lung in the laboratory. We foresee that these stem cells, given during a certain time during the hospital stay of these babies, could prevent the progression of the lung disease.”

Unlike traditional stem cells that can directly replace damaged cells and tissues, the stem cells that Dr. Thébaud is studying work by producing healing factors that promote regeneration and repair.

Clinical trial offers hope

Dr. Thébaud and his research team have launched a phase I clinical trial to test the feasibility and safety of the stem cell treatment in premature babies. The team is doing everything in their power to make this clinical trial a success, including consulting with healthcare providers and parents of premature babies.

One thing they’ve learned from these consultations is that many parents don’t feel like they know enough about stem cells and clinical trials to decide if they want to enroll their child in the trial. So, Dr. Thébaud and his team created an animated video to explain these concepts and help parents make an informed decision. Parents can share the video with family members if they’d like a second opinion.

Jamie was involved in these consultations and her firsthand experience provided valuable insight to the research team as they planned this project. This is just one example of how researchers at The Ottawa Hospital are partnering with patients and caregivers to improve the quality and success of their research.

Dr. Bernard Thébaud looks at a premature baby in an isolette.

Dr. Thébaud and his team held a “dress rehearsal” for the clinical trial. “The dress rehearsal lets us test and tweak our tools for approaching parents, including the video, so we know what works best once we’re ready to begin offering the experimental treatment.”

If this initial trial is successful, Dr. Thébaud and his team will launch a larger Canadian clinical trial. “This is a critical step towards providing a potential breakthrough therapy that could help premature babies in Canada and around the world,” says Dr. Thébaud.

The made-in-Ottawa clinical trial is supported by The Ottawa Hospital’s Ottawa Methods Centre and Biotherapeutics Manufacturing Centre, and the Blueprint Translational Research Group.

First trial of its kind in Canada

“Stem cell research is incredibly innovative. Here, we have a very promising, emerging therapy that could prevent lung injury but also improve brain development and eyesight,” says Janet Brintnell, Clinical Manager of the NICU who has seen dozens of premature babies with BPD.

“It’s amazing when you think of what it may be able to do for the quality of life for the child, for their family, and for our healthcare system. It could reduce length of stay, hospital admissions, and improve long-term outcomes. It could help these little ones lead healthier lives.”

— Janet Brintnell

“We are the only ones doing this kind of stem cell research in Canada, and there are only a few other teams in the world that are doing this.”

— Dr. Bernard Thébaud

Yet, three years ago when Olivia was in the NICU, this treatment wasn’t yet available. Now, Jamie and Tim are self-described “cheerleaders” of Dr. Thébaud’s research and are hopeful for what it might mean for future preemies and their families.

“I believe this is our future,” says Jamie. “When I think about what this could have done for our family, I wonder if Liam could have possibly survived. Olivia may not be facing the delays she’s experiencing today. Even to this day, if we are asked to put Olivia in the trial as an older candidate, we will.”

Jamie also adds the there was an impact from a mental health and financial perspective. “Our oldest child, Jacob, has had a very unusual first four years of his life because of how adaptable he has had to be during these difficult times. As a family, this entire experience has been very challenging financially due to a variety of therapies for Olivia and having to get used to becoming a single income family for several years in order to manage Olivia’s complicated schedule. All of this could have potentially been avoided if Dr. Thébaud’s research were available to our twins.”

A new beginning

Olivia is now a happy, active toddler who loves copying what her older brother Jacob does. Although, she still has BPD, it is increasingly manageable, and she no longer requires supplemental oxygen. While Olivia may suffer respiratory illness her entire life, one day a stem cell treatment developed here in Ottawa could mean that the next generation of babies with BPD won’t.

Listen to Pulse Podcast and learn how one day stem cells could heal the lungs of premature babies with Dr. Bernard Thébaud and what it could mean for parents like Jamie Eberts.

The Ottawa Hospital is a leading academic health, research, and learning hospital proudly affiliated with the University of Ottawa.

Categories: Biotherapeutics, Creating Tomorrow, NICU

Published: June 2019

Heather Harris was driving her fiancé to a golf tournament one morning in 2001 when her right foot went numb. By the end of the day, the numbness had spread up the entire right side of her body.

The then-24-year-old Thunder Bay resident had an MRI, which showed signs of multiple sclerosis (MS). The numbness was her first MS attack.

MS is a devastating disease that occurs when the immune system—which protects against foreign organisms such as viruses or bacteria—mistakenly attacks the body’s own central nervous system, which includes the brain, spinal cord, and optic nerve.

Heather Harris preparing herself prior to her transplant operation.

Heather met with neurologist and MS specialist Dr. Mark Freedman just a few weeks before her wedding. Heather’s disease was progressing rapidly. Dr. Freedman told her she would be in a wheelchair within five years.

Dr. Freedman and hematologist and scientist Dr. Harold Atkins were leading a world-first clinical trial, investigating whether patients with early, aggressive MS would benefit if their immune system was wiped out with high-dose chemotherapy and then regenerated with blood stem cells.

The stem cell treatment seemed her only hope. Heather and her husband moved to Ottawa for a year while she took part in the trial. She had the stem cell transplant in November 2006.

“It’s now 12 years since my stem cell transplant. I really feel like I’m cured,” said Heather who has no symptoms of the disease. She works full-time as a school principal, and is back to camping, skiing, running and driving a manual shift car.

Heather and her husband wanted to have a baby. With the help of in vitro fertilization, Heather had a baby girl in 2016. She said her little Zoe is the second miracle in her life.

In June 2016, Drs. Freedman and Atkins published the results of their successful clinical trial in The Lancet, a top medical journal. To date, more than 50 MS patients, like Heather, from all over Canada have undergone this treatment, which eliminated all signs of damaging active brain inflammation.

Three years ago, Sandy Patenaude was given the devastating news that she had stage 4 colorectal cancer. It had spread to her liver and lungs, and was inoperable. Sandy’s oncologist asked if she would like to go on a clinical trial, testing a new cancer stem cell inhibitor drug along with her chemotherapy.

“Cancer stem cell inhibitors, why not?” said Sandy who agreed to be part of the trial.

Dr. Derek Jonker, Medical Oncologist at The Ottawa Hospital, is leading the international trial for people with colorectal cancer, with the experimental drug napabucasin. He explained that cancer stem cells are the rare, immature cells in a tumour, which are often resistant to chemotherapy. They can give rise to the more mature cancer cells that make up the bulk of a tumour. Cancer stem cells are not the same as the normal stem cells that live in many healthy adult tissues and help with healing and repair.

“With chemotherapy, we can deliver treatment that can shrink the vast part of the cancer,” said Dr. Jonker, who is also an associate professor at the University of Ottawa. “Often the bulk of the tumour disappears, but what’s left is a small tumour with lots of these chemo-resistant cancer stem cells, which are able to spread and seed other places in the body. Often, we keep giving the same chemotherapy and find the tumour has regrown, but it’s not the same tumour it was when we started.”

Dr. Jonker is switching up the treatment to target the  cancer stem  cells that aren’t affected by standard chemo. In a previous randomized  clinical  trial he led , patients either  received a placebo or  napabucasin  to test its effectiveness at  inhibiting, or preventing,  the growth of the  cancer stem cells. The trial was carried out at  40  sites in Canada, Australia, New Zealand, and Japan. The  562  patients enrolled had advanced colorectal cancer  and chemotherapy no longer worked for them.

Looking at the results of the trial, Dr. Jonker said  they didn’t see much benefit in the group overall. “But when we looked at patients who had a  tumour  that  had characteristics of a high cancer stem cell (phospho-STAT3) over expression there was very significant improvement in their survival.”

Dr. Jonker presented his findings in October 2016 at the European Society for Medical Oncology, showing that where the cancer stem cell inhibitor didn’t work in all patients, there was an improvement in the survival of the 22 percent of patients who had  tumours  with high phospho-STAT3.  He said it’s “proof of principle that stem cells are an important target for cancer patients.” Napabucasin is now being combined in the  current trial  with chemotherapy to attack the cancer on two fronts  at the same time.

“We know  with results of the clinical trial that  the majority of  patients did not respond to it, but we have two patients here in Ottawa who  have responded and definitely developed benefit from the clinical agent,” said  medical oncologist Dr. Christine Cripps.

I thought I’d be part of the trial, because I thought well, it’s new.”

Sandy is one of those patients who benefited.  Her tumours shrank,  and the surgeons were able to remove spots in her liver and the primary  tumour in her rectum.  Dr. Cripps said she believes that part of the success in keeping Sandy’s cancer at bay is the napabucasin she is taking as part of  the  clinical trial.

“A stem cell inhibitor works differently than traditional chemotherapy, in that it prevents new disease from  appearing,” said  Saara  Ali, research coordinator for clinical trials in gastrointestinal cancers. “The hope is that the pill [napabucasin] will prevent new disease from showing. And in Sandy’s case there hasn’t been new disease  since her treatment. Everything was there before, so it may be doing its job.”

Next steps: Dr. Jonker hopes to start  another clinical trial with the cancer stem cell inhibitor that will be used specifically for patients who have lots of phospho-STAT3 in their  tumour. These patients could be identified for the clinical trial with molecular testing, using The Ottawa Hospital’s Molecular Oncology Diagnostics lab.  This would target the patients presumed to be the most likely to benefit most from the drug.

“We would repeat our study, randomize those patients with  napabucasin  and a placebo, and if we can prove that  napabucasin is effective for them, then it would be an option for patients who have run out of all other treatment options,” said Dr. Jonker.

Dr. Cripps said that Sandy is a candidate for this next trial,  and her tumours  will be analyzed by the molecular lab to see  whether she has high phospho-STAT3 cancer stem cell expression. Regardless, Sandy will continue using the trial drug as long as it is working for her. And it is working. The mother of three adult children said  she’s busy doing a million things, playing euchre, the ukulele, skiing, hiking, biking, and enjoying life.

The Ottawa Hospital is a leading academic health, research, and learning hospital proudly affiliated with the University of Ottawa.

Categories: Cancer, Clinical Trials, World-Leading Research, World-Leading Research, World-Leading Research, World-Leading Research, World-Leading Research, World-Leading Research, World-Leading Research

A strange thing happened before John Chafe started working in Kenora in 1993. His eyes crossed. He didn’t know it at the time, but it was the first sign of a debilitating disease that would change the course of his life forever.

His family doctor told him he had the flu and prescribed antibiotics. But after a week, when his eyes remained crossed, he bought an eye patch and drove five hours from Thunder Bay to fill the temporary posting at a bank in Kenora. A week later, his eyes straightened and returned to normal. But then other symptoms started appearing, he was losing his balance and couldn’t walk in a straight line.

“I then started have difficulties walking straight. I completely failed a simple balance beam experiment at the Ontario Science Centre,” said John. “I mentioned these symptoms to a friend, who mentioned them to a friend, who fortunately happened to be Dr. Heather MacLean, a neurologist at The Ottawa Hospital.”

To Dr. MacLean, John’s symptoms sounded like multiple sclerosis (MS), an autoimmune disease where the body’s immune system attacks its own central nervous system, brain, and spinal cord. John needed an MRI and spinal tap to properly diagnose his symptoms. The results were analyzed by Dr. Mark Freedman, Director, Multiple Sclerosis Research Unit, Neurology, who confirmed his diagnosis. John had an aggressive form of multiple sclerosis.

*John Chafe skiing at Blackcomb just after diagnosis in 1994.*

A different life after MS diagnosis

Incredibly interested in rock climbing and skiing, John didn’t give up his active lifestyle after his diagnosis, despite the fact that he was experiencing MS exacerbations – an attack that causes new MS symptoms, or worsens old symptoms – every eight months. He returned to Thunder Bay and opened a rock-climbing gym, thinking, “MS is not going to affect me.”

But it did. It completely sidetracked his life.

After suffering another MS exacerbation, John realized it was becoming more difficult for him to get out to see clients for financial planning sessions.

“I was stumbling along and thought, ‘How can I ask them to trust me with their money?’ My MS was getting worse and worse,” said John. “I needed a desk job, so I went into computer programming.”

His treatments weren’t helping. He needed a miracle. So he moved to Ottawa to be close to The Ottawa Hospital where we could receive the very best treatment.

Leading-edge clinical trial in Ottawa

*Dr. Harold Atkins and Dr. Mark Freedman conducted an innovative stem cell treatment for MS that has halted the disease in over 50 patients*

One day, John heard Dr. Freedman on the radio talking about an innovative stem cell transplant study that he described as akin to pressing reboot on the immune system. Dr. Freedman was working with hematologist and scientist Dr. Harold Atkins, a professor of medicine at University of Ottawa, to see if a groundbreaking treatment would halt an aggressive form of MS.

When John met with Dr. Freedman, he told him he was interested in participating in this new study. Dr. Freedman agreed he might be a good candidate because he was young, generally healthy, and his symptoms were quickly getting worse.

“If you saw his trajectory, how fast he was becoming disabled going into the transplant.  He should’ve been completely wheelchair bound, or worse, within two to three years,” said Dr Freedman.

John was willing to try an experimental treatment that had the potential to change that trajectory. “MS robbed me of my ability to climb, ski, and walk. I said, ‘I’m going to take a chance.’”

“John was very enthusiastic. That was a very important facet of his recovery,” said Dr. Freedman. “John has never been a quitter. He’s a stubborn guy. His goal was someday to end up on the ski hill again.”

Preparing for treatment

For almost a year, John underwent the exhaustive testing by Dr. Atkins and Marjorie Bowman, the bone marrow transplant nurse, to see if he was physically and mentally suitable for the clinical trial. They wanted to ensure he was prepared to go through the intensive trial treatment and accept the risks, which included death.

“This is fundamentally different than every other treatment,” said Dr. Atkins. “What we’re doing is getting rid of the old immune system and creating a new one that behaves more appropriately.”

“MS robbed me of my ability to climb, ski, and walk. I said ‘I’m going to take a chance.’”
— John Chafe

Replacing his immune system was a rigorous procedure.  John would undergo intensive chemotherapy to help eliminate his immune system.  In November 2001, he was given a dose of chemotherapy to stimulate and move his stem cells into his blood stream.  These stem cells were then collected and cleansed of any traces of MS.

A month later, John was given huge doses of chemo in an attempt to destroy his immune system and started getting weaker and weaker.  On December 13, 2001, after the chemo had wiped out his immune system, John had the cleansed stem cells re-infused by an intravenous  drip.

“I didn’t feel better immediately,” said John, who was only the second patient in the world to undergo a stem-cell transplant of this kind for multiple sclerosis.  “But I started getting stronger in the days following, so much so that Dr.  Atkins released me on Christmas Eve.” He spent three months living with his parents while he recuperated. By spring, he was ready to move back into his own home again.

*John Chafe rock climbing outside Thunder Bay in 1994 after his MS diagnosis.*

Groundbreaking research in Ottawa

Dr. Freedman said that he and Dr. Atkins had anticipated that by rebooting MS patients’ immune systems, they fully expected the disease was going to restart.

“At that time, genetic researchers said, ‘If people are genetically prone to develop MS, there’s nothing you can do to stop it. They’re going to keep redeveloping MS,’” said Dr. Freedman. “If that was true, it would be a matter of time before people started having active disease again.”

Dr. Freedman explained that nobody knows what causes MS. He and Dr. Harold Atkins hoped that through the trial they could reboot a patient’s immune system and monitor it with all the latest immune system  monitoring  and imaging technology, and then watch as the disease restarted and discover the  secret of what triggers MS. However, none of the 24 patients in the trial developed new symptoms of MS again.

“In that respect, the trial was a failure. It halted their disease and in some cases their disabilities went away too,” said Dr. Freedman. “We’ve followed these patients for 18 years, and nobody’s developed anything.”

“Those patients at the beginning, like John, are probably the bravest because there were more unknowns about the treatment,” said Dr. Atkins. “Each patient we’ve treated over the years has taught us something, but we learned more from the early patients at that time.”

A second chance at life

Prior to his stem cell transplant, John had a final exacerbation, which crippled him. After the transplant, his MS did not return. John remained healthy, but the damage caused by the disease wasn’t reversed and he still walks using a cane and walker.

“You almost wonder what would’ve happened to John if he’d had the transplant five years earlier,” said Dr. Freedman. “Today, when we see a patient that has the same profile as John’s, we offer them the stem cell treatment. We’re not waiting years. We’ve become more savvy, able to pick out individuals who warrant this aggressive approach.”

About 77,000 Canadians live with MS. However, only five percent of patients with MS warrant a stem cell transplant. They are generally young and have the most aggressive and debilitating forms of the disease.

After his transplant, nothing was going to hold John back. Three years later, he met Patricia, and they married in 2005. Five years later, his beautiful daughter Mary was born.

*John Chafe with his daughter Mary and wife Patricia in 2013.*

“I recall that as Mary started moving more, she motivated me to get more active again. She became my personal trainer,” said John. “I joined the Canadian Association of Disabled Skiing. I was terrible at first because I didn’t have the strength. But I’m stubborn and refused to give up, and today I can ski independently for hours – albeit with outriggers for balance.”

“I saw John a few years ago. The problem with this business is patients get better and so I don’t see them much afterwards,” said Dr. Atkins. “I do remember him showing me pictures of his young baby, and pictures of him on the ski slope. It is exciting to hear that people can have these treatments and go skiing again.”

John Chafe skiing with his family — *John Chafe, Mary and Patricia skiing at Edelweiss in 2016.*

“I’m not a bank president, but my life is better than incredible. I ski, I dance with my wife, and have an nine-year-old daughter. Because Dr. Freedman and Dr. Atkins were persistent about finding the answers to stop a disease like MS, they saved my life.”
— John Chafe

The following video focuses on Jennifer Molson who was also one of the early patients on the MS clinical trial, and includes interviews with Drs. Atkins and Freedman.

Listen to Pulse podcast and hear Drs. Harry Atkins and Mark Freedman talk about this transformational treatment for MS patients.

The Ottawa Hospital is a leading academic health, research, and learning hospital proudly affiliated with the University of Ottawa.

Categories:

DISCOVERING TODAY. CREATING TOMORROW.

From bench to bedside–bringing stem cell discoveries to patients faster than ever.

From cell and molecular biologists to bioinformaticists, to clinical scientists, and methodologists, specialists are coming to Ottawa to be part of something truly special. Collectively, their expertise is resulting in unprecedented breakthroughs that can move from lab bench to bedside faster than ever. This means in new and exciting treatments, improved care, and ultimately better outcomes for our patients.

“We’re entering a new era. More and more we are going to see regenerative medicine use cellular and molecular tools to treat devastating diseases with no current therapy.”
— Dr. Michael Rudnicki, Director, Regenerative Medicine Program and Sprott Centre for Stem Cell Research

What are stem cells?

Learn More

Regenerative Medicine and Stem Cells Research Update

Innovations thanks to donor support

Stem cells may heal lungs of premature babies

A stem cell treatment soon to be tested in clinical trials at The Ottawa Hospital may help heal the lungs of premature babies.

Biotherapeutics, Creating Tomorrow, World-Leading Research, NICU, Stem Cell

Stem cell treatment banishes disease for patient with multiple sclerosis

In 2001, John Chafe became just the second person in a world-first clinical trial  of its kind  that rebooted his immune system, virtually halted an aggressive form of MS, and stabilized his disease. 

Clinical Trials, World-Leading Research, Stem Cell

Bone research gets a break at The Ottawa Hospital

Dr. Daniel Coutu, the new Research Chair in Regenerative Orthopaedic Surgery will lead research to help understand how bone regenerates, repairs and heals.

World-Leading Research, Stem Cell

Stems cells make dream come true

When Jennifer Molson was 21 years old, she dreamed of becoming a police officer, marrying her boyfriend and dancing at her wedding. Those dreams were shattered when she was diagnosed with multiple sclerosis. Over a period of six years she had multiple relapses. She was in a wheelchair, unable to work, and looking for a miracle. That’s when Drs. Mark Freedman and Harry Atkins from the Ottawa Hospital Research Institute told her about an experimental treatment using stem cells. Jennifer became the sixth patient in a groundbreaking clinical trial during which stem cells were extracted from her bone marrow and transplanted back into her body. Jennifer found her miracle. Today she no longer needs a wheelchair. She is off medication, works full time, and leads an independent life. And, yes, she married her boyfriend and danced at her wedding.

“If you are in the computer business, you go to the Silicon Valley; if you are in oil and gas, you have to be in Alberta; if you are in stem cells, you need to be in Ontario, particularly Ottawa, because that is where the greatest advances are being made.”
— Dr. Bernard Thébaud, neonatologist at The Ottawa Hospital is developing a stem cell treatment to heal the lungs of premature babies.

Two decades in the making — world-first trial aims to heal the lungs of preemies

Health challenges lie ahead

The devastating impact of BPD

What is bronchopulmonary dysplasia?

Saying ‘yes’ to a world-first clinical trial

The next step for this stem cell trial

World-Leading Research

What is Sepsis?

The global impact of sepsis

Putting a face to the impact of the infection

Moving the needle for sepsis treatment

The “little bud” that will grow into future sepsis research

News of MS diagnosis hits hard

Effects of MS on the body

Groundbreaking MS treatment pioneered in Ottawa

MS symptoms were progressing quickly

Time for the MS stem cell transplant

How did a stem cell transplant halt MS?

Was it all worth it?

Listen Now:

A discovery with game-changing potential

The time is right for gene and cell therapy

Collaboration between lab researchers and clinicians key to success

Leveraging our biomanufacturing expertise at The Ottawa Hospital

How to plan a world-class clinical trial

Research program holds enormous promise

What are stem cells?

Two types of stem cells are most commonly used in research:

Pluripotent stem cells

Somatic or adult stem cells

How are stem cells used?

How can I access stem cell therapy?

An unexpected early birth

BPD in Canada

Lack of treatment options

Clinical trial offers hope

First trial of its kind in Canada

A new beginning

A different life after MS diagnosis

Leading-edge clinical trial in Ottawa

Preparing for treatment

Groundbreaking research in Ottawa

A second chance at life

DISCOVERING TODAY. CREATING TOMORROW.

What are stem cells?

Innovations thanks to donor support

Stems cells make dream come true

Be Inspired