21
Nov 2016
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Transplant recipient Dan Muscat

Transplant frees scleroderma patient from excruciating pain, gives him hope for the future

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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.”

10 thoughts on “Transplant frees scleroderma patient from excruciating pain, gives him hope for the future”

  1. I am so honoured to know Dan and to see the remarkable change that this treatment has caused in him. I saw him last week and I was so overwhelmed with emotion to see the light back in his eyes. My father died of this horrible disease two years ago and while I wish that he had been given this opportunity I am just glad to know that maybe, through Dan’s experience, we can do more to beat scleroderma.
    My father had a very similar experience in regards to the sudden, crippling onset of this disease but ended up with more severe damage to his internal organs. From what I’ve seen most people with scleroderma do not get it as severely as these men have. In fact, it is generally more of a disease that affects women. The possibilities of how this treatment can help people with less severe forms of scleroderma is very exciting.
    My dad wrote a blog about his journey with scleroderma. If you’d like to read it it can be found herehttp://lvngwthsclrdrm.blogspot.ca/
    Way to go Dan!! I’m so happy for you!

  2. Marie Ormerod says:

    So very happy for you Dan! You have been in our thoughts and prayers. Looking forward to your continued progress!

  3. Mark Wilson says:

    I think you still owe me a fishing trip! Glad to share a laugh with you again. You are far more courageous than I and stand as an inspiration and example of determination.
    Proud to be your friend.

  4. Kim says:

    Mr Muscat, I watched your story tonight on the CTV news and want to tell you that I admire you for your tenacity in finding a treatment and also for the hope that your story gives to others who have this horrible disease. It is also encouraging to know that there are doctors willing to listen and researchers working on finding treatments for autoimmune diseases. I had never heard the word scleroderma until a week ago when I was diagnosed with what seems to be systemic sclerosis sine scleroderma. I thus far have no skin involvement though I do have other symptoms of the disease. I am still waiting to have tests to determine whether or not there is organ damage. I know how frightening it is to have the disease with no skin involvement so I cannot imagine what it must feel like to lose your mobility and independence. May you continue to do well and thank you for allowing everyone a glimpse into your life with this disease and how not giving up in the face of adversity can bring hope.

  5. Heather Perry says:

    My husband was diagnosed 12 years ago. He was never able to bathe a baby, has difficulty getting dressed ( he cannot bend to put his socks on,do buttons, struggles with the mobility we have to even put a jacket on. He has been fired from jobs for asking for help ( he has a red seal millwrighting) and now welds as he is still able to hold the welding rod in his claw like hands. He cannot walk- we will never, it seems, be able to travel and see the world. He is 38. If there is anyone who can help us and give him the chance to play baseball an hockey again, and throw his kids gleefully in the air, or get down to play on the floor our lives would change. This disease is a nightmare for everyone. Please help us see some kind of light at the end of a dark tunnel.

  6. Tam says:

    Hello Dan,
    This is truly inspirational, I have been diagnosed with systemic scleroderma with lung involvement and seem to be reacting well to immunosuppressant therapy. I always wonder what happens when this disease decides to rev up again and the drugs stop working, but now maybe this will be an option. When I hear you say you can feel the disease has stopped attacking, I can actually relate, you can feel the difference. I hope you continue to improve, we will all be waiting and watching. Thank you for your willingness to share your story.
    Sincerely,
    Tam

  7. Diane Curno says:

    Hello Dan I am so happy for you and any one in the future with the advancements in treatment. I was diagnosed with scleroderma at 48 within 6 months I was unable to walk. What I found was you have to keep trying and most of all always have a Positive attitude it’s 20 years for me and I still try and keep Positive. All the best for you now and in the future. Take Care Diane

  8. Yolanda Bencsik says:

    I was overjoyed to see you . I could not believe my eyes on the improvement I saw. We were thinking of you during your treatment. It has given you new hope. All the best for you and your family. God bless you.

  9. Julie Gould says:

    Hello my stem cell transplant brother. I had transplant in Chicago. I look forward to watching you grow and recover on your journey. I’m so excited to spread the word with you about this treatment!

  10. Lynne Lawrence says:

    My daughter went thru the exact same symptoms as Dan and it sounds like she went thru the same rounds of treatments. They also did not work for her. She was a young active mother and wife who tried everything to keep going and battled thru this horrid disease…I feel if she had been diagnosed earlier she could have possibly been given the stem cell transplant before her organs became damaged from the disease. A beautiful life ended way too soon!

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