Dr. Harry Atkins’ success in treating a rare disease that can turn active, healthy people into living statues is getting the national attention it deserves this week, thanks to the Journal of the American Medical Association’s publication of his paper, Autologous Stem Cell Transplantation for Stiff Person Syndrome.…
Dr. Harry Atkins’ success in treating a rare disease that can turn active, healthy people into living statues is getting the national attention it deserves this week, thanks to the Journal of the American Medical Association’s publication of his paper, Autologous Stem Cell Transplantation for Stiff Person Syndrome.
The JAMA Neurology paper describes two Canadian women who have had all symptoms of their Stiff Person Syndrome (SPF) disappear thanks to a stem cell treatment that Dr. Atkins and his team at the Ottawa Hospital have developed. Readers of this blog will be familiar with one of the women’s stories — we told you all about Tina Ceroni back in December After our post, Canada AM featured Tina and Dr. Atkins.
This week’s reports, including Elizabeth Payne’s excellent news feature (the “living statue” description is hers) that appeared in the Ottawa Citizen and the National Post, explain how SPS, which strikes about one in a million people, triggers episodes in which muscles seize up uncontrollably, leaving a person rigid. Sheryl Ubelacker of the Canadian Press describes it this way in the Toronto Star:
“SPS is characterized by episodes of stiffness in the muscles and painful muscle spasms, which can be brought on by stress, loud noises or emotional distress. Some people with the disorder are so disabled they are unable to walk or move and may isolate themselves at home to avoid triggering an attack.”
Ms Payne’s story also tells of a third woman, a 53-year-old mother of six named Ingrid Steppan who was told she would likely die from her SPS. A recent transplant patient, she has now “put her wheelchair and walker away.”
Success with MS, Crohn’s and other autoimmune conditions
Dr. Atkins has used this technique with other autoimmune diseases, including Multiple Sclerosis (where he led a multi-year clinical trial involving about 30 patients) Crohn’s disease and neuromyelitis optica. The process involves extracting a patient’s bone marrow stem cells, then purifying and fortifying them. After the patient undergoes extreme chemotherapy, in which their immune system is effectively destroyed, the purified and fortified stem cells are put back to build a new disease-free immune system.
A modest and soft-spoken man, Dr. Atkins does not used the word “cured” when he talks about the patients who have had success with this treatment. He refers to his patients as “in remission.” For Jennifer Molson, an MS patient, that remission has lasted more than a decade. And in the case of Jelissa Morgan, the treatment has allowed her to overcome her crippling neuromyelitis optica and will be resuming her nursing career in September.
The success that Dr. Atkins is having is encouraging and offers great hope for the future. But it also harkens back to the past: the procedure he is refining can be traced back to the groundbreaking work the Canadians Jim Till and Ernest McCulloch did in proving the existence of stem cells in the early 1960s. It’s an area of medical science where Canada researchers excel. And people like Tina Ceroni, Jennfer Molson, Jelissa Morgan … and now Ingrid Steppan are living proof.
We recently featured the story of Tina Ceroni, a Burlington Ontario athlete whose life was sidelined by a rare, insidious disorder called Stiff Person Syndrome.…
We recently featured the story of Tina Ceroni, a Burlington Ontario athlete whose life was sidelined by a rare, insidious disorder called Stiff Person Syndrome. Thanks to a stem cell transplant, Tina’s fitness career — and her life — is back on track. To show her gratitude, Tina raised almost $37,000 for the Ottawa Hospital’s Stem Cell and Bone Marrow Transplant Program. When she was presented the cheque to Dr. Harry Atkins on Dec. 4th, Tina met with two other young women whose lives have been rescued by stem cell treatments: Jelissa Morgan and Jennifer Molson. Today we share Jelissa’s story.
From nurse to patient and (soon) back again
When Jelissa Morgan’s neuromyelitis optica flared up again in early 2012, she was devastated.
The rare immunological disorder, similar to Multiple Sclerosis, inflames the optic nerve and the spinal cord. Jelissa was losing her vision — she couldn’t read or watch movies and had trouble identifying who was talking to her. Walking was difficult. She had serious bladder and bowel problems and her skin itched. ‘My body was in pain,” she says, “but didn’t know how to express it.”
That flare-up, the worst of a series the young woman had experienced since finishing high school, transformed Jelissa from hospital nurse to hospital patient.
Until then, her doctors had kept the condition under control with Prednisone, a synthetic corticosteroid that comes with a slew of potential side-effects such as weight gain, sleep difficulties and mood swings. Some patients develop a humped back and a moon face. Jelissa had all of those symptoms and more.
A switch to a different drug, called Imuran, proved futile: she was on it when the worst flare-up struck.
“They were running out of medication options. My neurologist said, ‘What do you think about a stem cell bone marrow transplant?’ She knew about Dr. Harry Atkins and the work he was doing with MS patients.”
For the past several years, the Ottawa Hospital’s Dr. Atkins, working with neurologist Dr. Mark Freedman, has been treating MS patients with stem cell bone marrow transplants. In essence, he takes stem cells from patients with the most severe cases of the immunological disease and purifies and fortifies them. The patient then undergoes an extreme course of chemotherapy treatments that virtually annihilates their diseased immune system. The robust stem cells are then returned to them to rebuild a new — hopefully disease-free — immune system.
The process has worked well with about 30 MS patients whose disease progression has been stopped. In some cases, symptoms of the disease have diminished significantly and — in at least one case — disappeared entirely.
Dr. Atkins is also using the procedure for people who have other autoimmune disorders such as Crohn’s disease, Stiff Person Syndrome — and neuromyelitis optica.
Jelissa decided to have the experimental stem cell treatment.
“It’s kind of one of those decisions where you don’t want to wait,” she say. “You don’t know when the next flare-up will be. That last flare-up was so bad. Luckily, I was able to regain a lot of the functions I’d lost. But it’s like rolling the dice. You don’t know what could happen with the next flare-up.”
Jelissa received her stem cell transplant in the fall of 2012. She has been free of neuromyelitis optica symptoms ever since.
“I feel better than I did even before the disease began. I have much more energy. I’m off all my pills for the immune disorder and the transplant meds. I’m looking forward to getting my life back on track. That includes buying a car, moving out of my parent’s house and traveling.”
She is excited about returning to nursing in the next few months, after receiving a series of vaccinations to replace the ones lost during the chemotherapy treatment.
“From a patient perspective, a transplant is a hard thing to go through. But I survived. I feel like it’s given me a second chance at life. ”
She strongly supports stem cell research to help more people with diseases like hers.
“Stem cell research opens up so many possibilities. It’s like opening a door and entering a room and you don’t even know what’s in the room yet. It could mean treatments for so many things. There is so much hope there.”
The Canadian MS Bone Marrow Transplant Research Study is a MS Society-funded project to re-grow the immune systems of patients with MS using stem cells.…
The Canadian MS Bone Marrow Transplant Research Study is a MS Society-funded project to re-grow the immune systems of patients with MS using stem cells. Led by Drs. Mark Freedman and Harold Atkins at the Ottawa Hospital Research Institute and University of Ottawa, the study began in August of 2000. Ten years later, more than half of the 26 patients enrolled have seen their symptoms stabilize and, in many cases, actually improve, unheard of results in the treatment of MS. Yet few people know about this research. As part of our month-long salute to MS and stem cells, we will speak with people involved in every aspect of this study: the doctors and researchers, patients and their families and the people behind the scenes. Today, an interview with Marjorie Bowman, RN, the Research Coordinator for the study.
A: The study is headed by Dr. Harry Atkins and Dr. Mark Freedman, though there are many people involved. Dr. Atkins is the bone marrow transplant physician, he oversees the stem cell transplants; Dr. Freedman is a neurologist who specializes in MS; and I’m the Research Coordinator. We’ve all been involved in the study from the very beginning, and my role has evolved quite a bit over the past 10 years.
At the very beginning, my job was to get the study up and running — submitting and getting approval from the ethics board — which is a huge process; developing all the data collection tools and forms, and getting everything set up within the hospital. It was big learning experience for everyone involved because the clinics at the hospital hadn’t done a lot of research transplants, so it was very different.
Another big part of my job early in the study was screening and speaking to potential patients. When we received the grant from the MS Society of Canada, there was a big announcement on their website and word spread quickly among the MS community. When I started my position, the MS Clinic had been keeping a list of all the people who had called to inquire about the study. So I had this list of patients that I had to contact and screen. There were 20 patients on this list. With a lot of the inquiries, you know right away that they’re not going to meet the criteria, but you still have to speak with them and explain the trial.
And that was the first year. We developed the protocol, and got everything in place. The first patient received a transplant in October of 2001.
Q: What was it like once you started seeing patients?
A: With the first patient, it was a matter of working through the protocol we had developed step by step, and it was a big learning experience. The protocol has evolved with the study, and we’ve kept up with changes in treatment and technology.
Pre-transplant, we did all the screening. We had to make sure the patient met all the inclusion criteria, and then go through the consent form with them and have them sign it, which is a process. The patients had two counseling sessions prior to signing the consent form, one with Dr. Atkins and one with Dr. Freedman.
Then, the patient goes for a first baseline visit, where they have all the neurological testing and blood work done, and then safety testing to see if they are a good candidate for the study. If they had any illnesses besides MS, they were excluded from the study. So we were testing the heart, lungs, things like that. I coordinated all that, all the testing and visits. I worked very closely with the patients during this process and got to know them very well.
Once they got to the transplant phase of the study, they were turned over to the bone marrow transplant team at The Ottawa Hospital. During that time, I was the first point of contact if they had any problems.
Once they had been through the transplant, I coordinated post-transplant testing. All the patients have to have lumbar punctures and MRI and MRS scans once a year, plus lots of neurological testing. I was kind of overseeing all that testing.
In the meantime, I was still screening patients and talking to potential patients from all over Canada. And that’s something we didn’t expect. The people from out of town need a little extra support to get here and go through the trial. And there were some very brave, dedicated patients for sure.
Q: How many patients have gone through the trial and how much information are you collecting?
A: Of the 26 patients that have gone through the trial, 24 have actually received stem cell transplants. We have 2 control patients — they had the same inclusion criteria but refused the transplant, and we’ve been following them closely to see how their symptoms compare with those who have received a transplant.
In 2008, we received another grant from the MS Society to follow these patients even further. Originally we followed patients for three years after their transplants. But the investigators weren’t satisfied with only three years. They wanted to follow them longer because we need to see that their MS is stable for a sustained period of time. So, we’re now going to be following the patients until 2012. It’s been such a range of time, because the first patient received a transplant in 2001 and the most recent patient received one in December of 2009. So the last patient will have a three year follow up, but the first will have 11 years. It’s great to be able to follow them for so long and to see the changes.
It’s interesting, my role has evolved with the study, now that the transplants are done, compiling and analyzing the data is a bigger part of my job. We’ve collected literally rooms worth of data on the patients. I have four two-inch binders on each patient, so it’s a lot.
We’re working on compiling all of it right now and preparing to present our findings. We’re going to be publishing for the next 10 years by the looks of it, and we’re hoping to publish our first paper in the fall.
Q: What kind of results have you seen?
A: So, this is kind of a summary: we’ve seen stabilization in many of the patients, which was our goal. It has been exiciting to see improvments in some patients. Some have been able to walk without mobility aids, many have experienced decreased fatigue. Some have returned to work and are now focusing on new goals. After getting to know these patients so well, seeing the improvement in their quality of life has been surprising and rewarding for me.
Q: What has been the most rewarding aspect of working on this study?
A: The most rewarding part of the study has been the patients. Just getting to know them and helping them through this trial has been fantastic. Whenever I was having a bad day, I would think about what these patients were doing and what they were going through.
Q: What are your hopes for the future of the study?
A: I hope that this will become standard care for MS, though of course it’s not for everyone. Dr. Atkins has said that only 5-10 % of MS patients would benefit from this kind of treatment.
But there have been a number of interesting findings, not all strictly related to the transplant. One thing I’m hoping the data from the study will show is that we don’t need to give MS patients so many immunosuppressants, so maybe it’ll lead to less toxic therapies for all MS patients.
Marjorie Bowman is the mother of two young children. She enjoys reading and playing shinny.
Rob McConnell’s Crohn’s disease struck about 13 years ago, when he was 20. The Elrose, Saskatchewan farm manager believes the stress of his father’s death had a lot to do with the onset of the debilitating disease — and how hard it hit him.…
Rob McConnell’s Crohn’s disease struck about 13 years ago, when he was 20. The Elrose, Saskatchewan farm manager believes the stress of his father’s death had a lot to do with the onset of the debilitating disease — and how hard it hit him.
The six-footer’s weight dropped to 95 pounds, the result of his decreased appetite, abdominal pain and chronic diarrhea that sent him to the toilet at least a dozen times a day. He underwent more operations than he can remember to remove diseased pieces of his intestines, and when he wasn’t in hospital he “was on enough steroids and pain killers to kill a small horse.”
Crohn’s disease and a related condition called ulcerative colitis occur when the body’s immune system reacts to genetic and/or environmental triggers by attacking the digestive tract. The two conditions are commonly referred to as Inflammatory Bowel Disease or IBD. Canada has one of the highest incidences of IBD in the world, with one in about 150 — about 230,000 Canadians — affected. (For a lively and informative overview of IBD, check out this video at the Crohn’s and Colitis Foundation of Canada site.)
Rob tried every drug and treatment available to combat his Crohn’s. They would work for a while. Some, especially the steroids, came with severe side-effects (moon-shaped face, hair loss, sore joints and brittle bones). But the Crohn’s kept coming back.
“I was going downhill quickly,” says Rob. “I was at the hospital all the time and my girlfriend Teneille would go home and go online looking for other options, especially information about stem cell transplants. She found a blog by Billy Tytaneck.”
In 2008, Billy Tytaneck was able to avoid radical surgery to remove much of his bowel when Dr. Harry Atkins of the Ottawa General Hospital performed a stem cell bone marrow transplant to rebuild his immune system. Dr. Atkins has been featured in this space for his success in treating patients with Multiple Sclerosis, as well as Stiff Person Syndrome and neuromyelitis optica.
Teneille wrote to Dr. Atkins, who asked her to send along Rob’s medical records. “About a week later he responded and told me: ‘You know what? I think you might be a candidate.’ It was late February 2012 when I went to Ottawa for my consultation and right away I had a great connection with Dr. Atkins, who sat me down and went through the whole procedure.”
Three months later, Rob was back in Ottawa for his “Autologous Peripheral Stem Cell Transplant” using stem cells that were extracted from his blood, then purified and fortified. After undergoing extreme chemotherapy to annihilate his diseased immune system, Rob was given back the robust stem cells to rebuild a new immune system.
He sailed through the treatment that others have found excruciating. “I took the chemo relatively well. There was some nausea and I had other things that bothered me, but I didn’t get the whole super illness.”
After staying in Ottawa for follow-up treatments and infection monitoring, Rob went back to Saskatchewan in the fall where, a year-and-a-half later, the Crohn’s is in remission and he feels fine. No more frequent trips to the bathroom. No more cramps. No more weight loss: he’s up to 161 pounds now, his heaviest ever. He no longer takes any medication.
While it is still too early to say whether Rob’s Crohn’s is cured — the condition is known to wax and wane — so far so good. “I eat very well,” says Rob. “Things that used to bother me don’t bother me anymore. There have been no attacks. I used to have a pain twice an hour or more. It has been a long while since I had one.”
And his quality of life has vastly improved. “It is just amazing. I started another business. Teneille and I got married at the end of June. I’m doing so much more and feeling so much better. I really don’t think I would be on this side of the grass if I didn’t get that treatment.”
(Editor’s Note: CNN reports on Dr. Atkins and his work with Stiff Person Syndrome patients.)
A team led by two doctors from The Ottawa Hospital has proved that stem cells, used in combination with chemotherapy can halt the progression of aggressive multiple sclerosis (MS) and — in some cases — help patients recover from the autoimmune disease.…
A team led by two doctors from The Ottawa Hospital has proved that stem cells, used in combination with chemotherapy can halt the progression of aggressive multiple sclerosis (MS) and — in some cases — help patients recover from the autoimmune disease.
In a paper published today in the prestigious medical journal The Lancet, Drs. Harry Atkins and Mark Freedman show that bone marrow stem cell transplants can eliminate all signs of damaging brain inflammation by rebuilding a patient’s immune system.
MS occurs when a person’s own immune system attacks the myelin protective sheath that covers nerves, causing inflammation and damage that inhibits communication between cells in the nervous system — the brain and spinal column.
The researchers took bone marrow stem cells from patients with aggressive MS and purified them in a laboratory. After the patients underwent extreme chemotherapy, their robust stem cells were returned to rebuild new immune systems.
The clinical trial involved 24 patients and tracked their progression over several years. According to The Ottawa Hospital’s news release, after the treatment:
- Not a single participant experienced a clinical relapse (zero relapses in 179 patient-years), whereas before treatment, the participants experienced an average of 1.2 relapses per year.
- Not a single new active inflammatory lesion could be detected in the brains of any of the participants.
- Not a single participant required MS-specific drugs to control their disease.
- 70% of participants experienced a complete stop in disease progression.
- The average rate of brain shrinkage, typically a measure that correlates with MS progression, returned to levels associated with normal aging.
- 40% of participants experienced some lasting reversal of such symptoms as vision loss, muscle weakness and balance problems.
- Some participants were able to return to work or school, regain the ability to drive, get married and have children.
In Jennifer Molson’s case, the treatment eradicated all traces of the MS that had taken over her life. Prior to taking part in the study, she was receiving 24-hour care at the Rehab Centre at the Ottawa Hospital, “learning to how to live with my disability.” She had quit her job and could only walk with the help of forearm crutches or a walker. Life in a wheelchair was imminent.
Now free of MS symptoms for more than a dozen years, Ms. Molson has resumed a demanding career and a busy schedule. As she described in the book Dreams & Due Diligence: Till and McCulloch’s Stem Cell Discovery and Legacy, “I downhill ski, I drive a standard. I can skate. I can dance … Am I cured? I like to use that word. They (Drs. Atkins and Freedman) don’t like to use that word. They’re calling it a lasting remission. I’m very lucky. I got a second chance at life.”
While his recovery was less dramatic, Vancouver’s Aaron Prentice said his quality of life is much improved: “I am now five years post-transplant,” he wrote in the Foundation’s Cellections newsletter last year. “I have not had a relapse and no longer require a cane. My gait has improved significantly and continues to do so. My arms are symptom free.”
The Ottawa Hospital, the MS Society of Canada and the University of Ottawa have produced a video about the clinical trial that can be seen here: https://www.youtube.com/watch?v=vW86owclZes&feature=youtu.be
Marjorie Bowman, trial coordinator and advanced practice nurse at The Ottawa Hospital, expressed her admiration for the remarkable “courage and dedication” demonstrated by the trial’s participants. “We thank the patients from across Canada who participated in this clinical trial, as well as their family members,” she said.
The treatment, Dr. Atkins explained in Dreams & Due Diligence, is a variation on the bone marrow ablation that leukemia patients undergo.
“MS is an autoimmune disease where the immune system is attacking a patient’s brain. The simple concept behind our treatment is, ‘Let’s just get rid of the old immune system and put back the seeds, let a new one grow and hope that it won’t learn the same lesson.’
“Because stem cells don’t carry over immunologic memory. That’s really what we have tried to do. We had a track record for doing transplants for leukemia and knew how we could damage the immune system to remove it. We just applied the lessons we learned in care of patients with leukemia and applied them to this new setting.”
Not for everyone
However, the therapy is not for all MS patients.
“It is only used in very severe cases because participants face a significant risk of infection and other side-effects, including death,” Dr. Atkins said in the media release. “The risks are similar to those faced by leukemia patients undergoing this kind of treatment.”
Indeed, one participant in this study died of liver failure due to the treatment and another required intensive care for liver complications. The treatment regimen was modified to reduce toxicity, but all participants still developed fevers, which were frequently associated with infections.
People who are interested in this therapy should speak with their own neurologist, who can request a referral to The Ottawa Hospital MS Clinic or another major hospital with experience in this area. The Ottawa Hospital cannot treat people without valid Canadian health coverage.
While The Lancet paper is focused strictly on the MS patients, Dr. Atkins has also seen some success treating patients with other immunological disorders such as Stiff Person’s Syndrome, neuromyelitis optica and Crohn’s disease.
And Dr. Freedman has gone on to co-lead a new chemo-free MS clinical trial using mesenchymal stem cells. “These cells have been shown, at least in early studies in humans, to repair — period,” he explained recently. “But they happen, at the same time, to have an anti-inflammatory effect. So they may be able to accomplish both things together. Without the need of any chemo, there is very little risk to the people taking it.”
A turning point for MS
Yves Savoie, President and CEO, MS Society of Canada, called publication of the paper “a turning point” in MS care. “What started as a bold idea has translated into a treatment option for people living with highly active, relapsing MS.”
The $6.47 million trial was funded by the MS Society of Canada and its affiliated Multiple Sclerosis Scientific Research Foundation. The research was also supported by The Ottawa Hospital Foundation, The Ottawa Hospital Department of Medicine and Canadian Blood Services.
This is the third in a series of blog posts about the success the Ottawa Hospital’s Dr. Harry Atkins is having in treating autoimmune disorders with stem cell bone marrow transplantation. …
This is the third in a series of blog posts about the success the Ottawa Hospital’s Dr. Harry Atkins is having in treating autoimmune disorders with stem cell bone marrow transplantation. We previously featured Tina Ceroni, a Burlington Ontario athlete whose life was sidelined by a rare disease called Stiff Person Syndrome, and Jelissa Morgan, a patient with a crippling condition called neuromyelitis optica who is about to resume her nursing career. Today we share the story of Jennifer Molson, who has been free of all traces of Multiple Sclerosis for 11 years.
MS symptoms eradicated by stem cell treatment
Jennifer Molson was working full time and going to school at night in the hope of becoming a police officer. It was 1996 and she was turning 21. When her left arm started going numb for no apparent reason, it was put down to carpal tunnel syndrome.
When thingsbegan to get worse, doctors considered other possible causes. An MRI confirmed it was Multiple Sclerosis.
Jennifer’s disease came on slowly and tended — as MS does — to wax and wane. Within five years, however, it had taken control of her life. Training for the police was out. Full-time employment became part-time work. Eventually she was unable to work at all. Or drive a car. The once unstoppable young woman needed help doing the simplest tasks, such as cutting her food and getting in and out of the shower. “I couldn’t do anything,” Jennifer says now.
She was getting 24-hour care at the Rehab Centre at the Ottawa Hospital, “learning to how to live with my disability.” She could walk only with the help of forearm crutches or a walker. Life in a wheelchair was imminent. Her neurologist, Dr. Mark Freedman, feared that without some kind of an intervention, “she would become very disabled very quickly.”
Intervention came in the form of a stem cell bone marrow transplant to rebuild Jennifer’s immune system where the MS lurked.
For more than a dozen years, Dr. Freedman has partnered with Dr. Harry Atkins, a clinician/researcher, in treating MS patients with stem cell bone marrow transplants. In essence, they take stem cells from an MS patient and purify and fortify them. The patient undergoes extreme chemotherapy to all but annihilate their diseased immune systems. The robust stem cells are then returned to the patient to rebuild a new — hopefully disease-free — immune system.
Tried about 30 times so far, the treatment has shown strong success in stopping the progression of MS. It has also been successfully used in other autoimmune disorders such as Crohn’s disease, neuromyelitis optic and Stiff Person Syndrome.
In Jennifer’s case, the stem cell transplant did much more than shut down the MS. It eradicated all traces of it. The crutches and walker are long gone. She’s back working full time. As she described in the book Dreams and Due Diligence:
“I downhill ski, I drive a standard. I can skate. I can dance, but not well … I have no rhythm. That has always been the case. Am I cured? I like to use that word. They (Drs. Freedman and Atkins) don’t like to use that word. They’re calling it a lasting remission.”
Now free of all traces of MS for more than a decade, Jennifer is an active advocate for stem cell research and development in Canada, the country where stems cells were discovered. She has lent her support to the Campaign for a Canadian Stem Cell Strategy, which is developing a plan for Canada to follow through on its outstanding research legacy to produce more of the kinds of new treatments she has benefitted from.
“I’m very lucky. I got a second chance at life. The Canadian Stem Cell Strategy will allow what happened to me to happen for thousands more Canadians who are dealing with currently incurable diseases.”
A few years ago, when Tina Ceroni first noticed the muscle tightness and cramping in her legs, she put it down to too much exercise. …
A few years ago, when Tina Ceroni first noticed the muscle tightness and cramping in her legs, she put it down to too much exercise. An accomplished athlete who ran a personal training business in Burlington Ontario, she was used to sore muscles. She never suspected that a rare neurological disorder called Stiff Person Syndrome (SPS) was beginning to take a vise-like grip on her life.
But as the attacks increased in frequency and intensity, constricting her whole body so forcefully that she feared her bones might snap, Tina realized something was seriously wrong. After a couple of misdiagnoses, doctors found she had won the one-in-a-million reverse lottery for SPS, a condition that can only be treated with palliative measures such as sedatives to unbind the ever-tightening muscles.
“I was a ticking time bomb,” says Tina, pictured at left. “Everywhere I went, there was a chance that I could have a life-threatening episode. I tried to keep active, but if I was out on the road cycling and had an attack I didn’t have the ability to use my phone and call someone. I wasn’t able to breathe. So I had to have someone with me all the time.”
Ordinary life stressors, such as loud noises or crowded, noisy rooms, could trigger attacks. So could emotional stresses: her muscles seized up at a friend’s funeral and she had to be taken to hospital.
At her worst, in 2010, she was having five to seven attacks a month, each time requiring paramedics to rush her to the emergency ward where doctors, now familiar with her condition, treated her with propofol, the strong sedative implicated in the death of pop star Michael Jackson.
Tina had to stop working, give up her driver’s licence and stay close to her supportive family. The vivacious, outgoing young woman’s future looked bleak.
But in remarkable ‘small world’ circumstances, she found a fellow SPS patient who had undergone a successful stem cell transplant treatment at Ottawa General Hospital under the care of Dr. Harry Atkins, stem cell researcher and bone marrow transplant expert.
For more than a decade, Dr. Atkins, pictured at left, has been treating Multiple Sclerosis with stem cell bone marrow transplants. More recently, he has been using the procedure for patients with other autoimmune conditions such as SPS, Crohn’s disease and neuromyelitis optica.
In essence, he collects stem cells from a patient and then purifies and fortifies them. The patient then undergoes an extreme course of chemotherapy treatments to all but annihilate their diseased immune system. The robust stem cells are then returned to them to rebuild a new — hopefully disease-free — immune system.
The treatment has shown remarkable results, stopping the progress of the patients’ autoimmune diseases. In some cases, patients enter into complete remission.
After consulting with Dr. Atkins, Tina underwent the grueling procedure — the chemotherapy leaves patients extremely weak and near death — in April of 2011. Now, approaching three years later, she is symptom-free. She has resumed her work as a fitness trainer and volunteers at Toronto’s Princess Margaret Cancer Centre, where one of her roles is counseling patients who are about to undergo transplant therapy.
“There are no limits; I’m doing everything I love. I can be in big groups and be around all my nieces and nephews, where before it was difficult because the fear of having an attack was a big concern. I can truly say I’m in remission right now. I feel so thankful.”
With her fear of crowds gone, Tina held an event she called Share a Cell in late October to raise awareness and funds for the Stem Cell and Bone Marrow Transplant (Program at the Ottawa Hospital. More than 200 family, friends and supporters contributed almost $37,000. Today Tina presented Dr. Atkins and his team with a cheque for the amount.
“Before this, I didn’t know much about stem cell transplants,” says Tina. “Now I know how stem cell transplants can change the future. I want to do what I can to raise awareness about stem cell research and what it’s doing for people and how it’s saving lives by affecting all different diseases and conditions.”
In the summer of 2014, Dan Muscat’s fingertips started to tingle. It felt like tiny shards of glass were piercing the skin underneath his fingernails.…
In the summer of 2014, Dan Muscat’s fingertips started to tingle. It felt like tiny shards of glass were piercing the skin underneath his fingernails. Over the next weeks and months the pain crept into his hands.
His family doctor thought it might be arthritis and prescribed a steroid to get things under control. “Within days, the disease sped up,” says Muscat. “It went from the top of my head to my toes. My ankles and my feet were so inflamed I couldn’t walk 20 feet.”
Muscat, who owns a jewelry business in St. Thomas, Ontario, was referred to a rheumatologist who immediately recognized what was happening to him: “Dan had rapidly progressive early onset diffuse scleroderma,” says Dr. Janet Pope of St. Joseph’s Health Care in London.
Scleroderma — from the Greek words for “hard” (sclera) and “skin” (derma) — is the term used for a group of rare autoimmune diseases that, according to the Mayo Clinic, involve hardening of the skin and connective tissues due to an overproduction of collagen. Diffuse scleroderma is a systemic type of the disease, meaning it affects not only the skin and muscle but also the digestive tract and internal organs, ultimately leading to death.
The future did not look good for Muscat: “He had not responded to usual immune suppressive treatment,” says Dr. Pope.
By late 2015, Muscat was growing desperate. He had heard that Dr. Richard Burt at Northwestern University in Chicago was offering a treatment using a patient’s own bone marrow stem cells to rebuild their immune system and shut down scleroderma. Muscat reached out to the Canadian Stem Cell Foundation for guidance.
“I investigated costs of Northwestern University treatment,” says Muscat, “and it was somewhere between $300,000 and $350,000. I thought about selling my business or my home. If I had to, I would have done that. Financially, it would have been a huge burden.”
Because the American treatment is similar to the combination chemotherapy/ bone marrow stem cell transplant treatment that The Ottawa Hospital’s Dr. Harry Atkins has used successfully against extreme forms of multiple sclerosis (MS, which is also an autoimmune disorder), the Foundation suggested Muscat reach out to him. Earlier this year Dr. Atkins agreed to take Muscat on as his first scleroderma patient.
“He fit the profile of the kind of patient best treated with these kinds of stem cell transplants” says Dr. Atkins, who is also an associate professor at the University of Ottawa. “There was a randomized trial carried out in Europe that was very well done and had very good results. I know the doctors involved and we’d talked a little bit about it, but their methods had also been published and are available for everybody. That’s why we publish — to spread the knowledge.”
By the time Muscat arrived in Ottawa in May, the disease had taken its toll. Once an imposing figure who worked out regularly and thought nothing of going for five- or six- miles runs, Muscat had watched 45 pounds fade from his six-foot frame. His brisk walk had turned into a painful shuffle. His boundless energy (he built a log home by himself, is an avid fisherman and collects classic sports cars) disappeared, making it difficult to get through a day. His fingers became like stone claws, curving in at the knuckles. His skin was so impenetrable he worried the Ottawa medical team might not be able to run an intravenous line. And the pain was excruciating.
“On a scale of 1 to 10, about 15,” Muscat says.
Muscat began the therapy in June and spent much of last summer undergoing the treatment designed to destroy the diseased immune system and rebuild it with his own purified stem cells, ideally to produce a new, scleroderma-free one. Though not as harsh as the chemotherapy MS patients undergo, it is not a treatment to be taken lightly. The risk did not dissuade Muscat: “If the treatment didn’t kill me, the disease would.”
He made it through chemo with few noticeable side-effects beyond losing his hair and tolerating a couple of infections. Released from hospital in August, he embraced physiotherapy, doing three to five hours daily to get his flexibility back and rid his body of the collagen buildup.
He is now back in St. Thomas, occasionally working at the store — he has to limit his time in public places because the chemo has left him vulnerable to infections. “My pain is one or two percent compared to what it was before.” His range of motion with his arms and legs is much improved and he can get around on foot again.
He realizes it could take a year before the chemo drugs fade from his system and the full benefits of the treatment kick in. Muscat, grateful for the excellent care provided by The Ottawa Hospital’s bone marrow transplant team, is unswerving in his belief that he can beat this disease. “I feel physically and mentally better. I just don’t feel that I’m being attacked.”
In an email message, rheumatologist Dr. Pope said that while Muscat “has a long road ahead, we expect more recovery with softening of his skin and improved function. His pain is less and he feels that shortly after the transplant a ‘switch’ was turned back to how it should be and he developed a better sense of well-being. I will follow him carefully and I hope for more improvement over the next year or two. He is in physiotherapy and is getting ongoing intravenous drugs for safety as his immune system continues to reconstitute. I am very hopeful that Dan will be even better over time. This is not a cure, but has given Dan a better chance of recovery.”
In Ottawa, Dr. Atkins also urges caution. “It’s going to take months and months to know if this is a real improvement. The changes with scleroderma don’t happen overnight. This is not a curative treatment; it doesn’t make scleroderma go away and never come back. It’s something that helps a population of scleroderma patients: some patients improve, some not. There are patients that progress or relapse again.”
Muscat is positive about the future: “I still have the remnants of the disease, but I can jump in and out of my truck again and I can walk a couple of kilometres with no problems. I feel very optimistic, like I’ve been given a second chance.”
As we reported yesterday, Tina Ceroni, with the help of Dr. Harry Atkins, shared her story with Beverly Thomson today on Canada AM.…
As we reported yesterday, Tina Ceroni, with the help of Dr. Harry Atkins, shared her story with Beverly Thomson today on Canada AM.
Tina is an accomplished athlete and fitness trainer who was diagnosed with Stiff Person Syndrome (SPS), a rare autoimmune disease that affects one in a million people.
In her mid-20’s Tina started experiencing mild spasms and rigidity in her legs. She always assumed they were due to her training regime. She was eventually diagnosed SPS. The contractions and spasms became so severe that she required frequent trips to the emergency ward to save her life.
In April 2011, Tina received a stem cell transplant at Ottawa General Hospital.
The procedure performed by Dr. Atkins is very intense. Strong doses of chemotherapy “kill” the immune system in order to create a new disease-free immune system seeded by stem cells.
“There are risks of severe organ failure or even death,” Dr. Atkins explained on the program.
“It is a challenging process and it takes time to recover from something like that,” added Tina.
Today Tina is completely symptom free. “There are no words to describe how amazing I feel now. I have a whole new lease on life and no limitations on what I can do.”
Click here to watch the interview.
We can add another disease to list of conditions that can now be treated with bone marrow stem cell transplants: myasthenia gravis (MG).…
We can add another disease to list of conditions that can now be treated with bone marrow stem cell transplants: myasthenia gravis (MG).
This week, CTV News in Ottawa told the story of Anne Scott, a 58-year-old Kemptville, Ontario woman whose MG refused to respond to conventional therapies.
MG is an autoimmune disorder like multiple sclerosis (MS). It interrupts communication between the muscles and the nerves, which can make swallowing and breathing difficult.
While most patients can be treated with conventional therapies, some, like Scott, cannot. She had to be placed on life support a dozen times.
Under the supervision of The Ottawa Hospital’s Dr. Harry Atkins, Scott underwent a chemotherapy/stem cell procedure. Essentially, it involves one using a patient’s own blood stem cells to rebuild their immune system after it has been wiped out by chemo therapy. If all goes well, the rebuilt immune system is free of the disease.
Dr. Atkins has had success using this approach to treat patients with MS, Stiff Person’s Syndrome, neuromyelitis optica and Crohn’s disease. The procedure has been used to treat leukemia and other forms of blood-based cancer for decades.
Scott, who was able to attend her daughter’s wedding shortly after undergoing the procedure, was one of seven patients in a study led by Dr. Atkins, the results of which have been reported in in JAMA Neurology All seven patients no longer need treatment for MG and are symptom-free.
You can find out more about the study here.