Dan Muscat

26
Apr 2017
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Transplant recipients Miaya Killips

Scleroderma update: more lives saved through chemo/transplants

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Last fall we told the story of Dan Muscat, an Ontario jeweler whose combination chemo/stem cell transplant treatment helped him recover from scleroderma, an extremely painful, life-threatening autoimmune disease that hardens the skin and attacks internal organs.…

Last fall we told the story of Dan Muscat, an Ontario jeweler whose combination chemo/stem cell transplant treatment helped him recover from scleroderma, an extremely painful, life-threatening autoimmune disease that hardens the skin and attacks internal organs.

We believed then that his transplant, which took place at The Ottawa Hospital over the spring and summer of 2016, was the first time such a therapy had been tried in Canada. It even made news: CTV’s Avis Favaro profiled Dan early this year.

It turns out that other Canadian hospitals are trying — and have been using — the innovative therapy, which involves first extracting bone marrow stem cells from a patient to purify them, then wiping out their diseased immune system with chemotherapy, and finally returning the robust stem cells to the patient. The goal is to have these fortified stem cells rebuild a disease-free immune system.

Calgary’s Dr. Jan Storek has employed the chemo/stem cell treatment on “four or five” patients since 2011 and has contributed to the international Scleroderma: Cyclophosphamide Or Transplantation (SCOT) study, the results of which have yet to be published. He began working with the treatment in the 1990s at the Fred Hutchinson Cancer Center in Seattle.

“I was part of the development of transplants for autoimmune diseases,” says Dr. Storek. “Then the University of Calgary hired me to start a research program in bone marrow transplantation including transplantation for autoimmune diseases.”

He points to the work of Dr. Sharon LeClercq, a Calgary rheumatologist and expert in systemic sclerosis, without whose involvement there would have been no Canadian participation in a randomized study. “Patients participating in studies, particularly in the three randomized studies leading to this success should also be greatly acknowledged,” says Dr. Storek.

One of his transplant recipients is Miaya Killips, pictured above shortly after her 2016 transplant and as she is today. Miaya, who lives in Spruce Grove, Alberta, was diagnosed with scleroderma three years ago. She suffered lung damage, along with hardening of the skin. “I had limited mobility because my joints were tight and I was not able to swallow well — I would choke on food.”

These days, things are much better. “Scleroderma-wise, I’m feeling great. My mobility is much better and I don’t have the pain I had before. I am not choking when I eat and my lungs have stabilized, which is good because before the transplant my doctor had me on a standby list for a lung transplant.”

An electrician by trade, she wants to go back to school as part of a career change. And she and her husband plan to start a family soon, using embryos harvested through in vitro fertilization before she underwent chemotherapy.

Shane Knihnitski received his stem cell transplant in February

We also heard from Shane Knihnitski, a 28-year-old heavy equipment mechanic who underwent a bone marrow stem cell transplant at Saskatoon’s Royal University Hospital in February to treat his scleroderma, which had caused large calcium deposits to form at his joints, resulting in considerable pain and restricted mobility.

“Things are turning around,” Shane says, “and the skin is starting to loosen up. I wasn’t able to see my elbow for two years — it was a big lump like a soft ball — but I can see the bone again.”

Dr. Keltie Anderson, Shane’s rheumatologist, says that while “it’s still very early” Shane has regained “a fairly significant amount of mobility in his hands already.

“When I first started seeing him, he could sort of cup his hands but couldn’t make a fist. Now he can bring the fingers all the way to the palm — almost make a full fist. To have such change already is very impressive.”

That the stem cell transplant procedure is having good results comes as great news to rheumatologists like Dr. Anderson who, until now, had few weapons with which to fight this painful, life-stealing disease. “Basically, you would do cyclophosphamide (a chemotherapy medication to suppress the immune system) and then cross your fingers.

“In Shane’s case he not only has extensive skin involvement, he also had extensive lung involvement. His outlook was quite bleak. And he’s a young guy and (he and his wife) just had their first child.”

Dr. Mohamed Elemary, who directs the hospital’s stem cell transplant program, says Shane was originally referred to Dr. Storek for inclusion in the Calgary study but it had already been completed. “So we started to review the data and see if it was something feasible for him here in Saskatoon. The stem cell transplant unit is run by the cancer centre and by the health region with the University Hospital. We can barely accommodate our patients with malignancies.”

After receiving permission to try the treatment with Shane, making sure he was a good candidate for the procedure, and consulting with Dr. Storek, Dr. Elemary went ahead with the transplant two months ago. “Our concerns vanished because he did tremendously well and was out of the hospital almost two weeks after his transplant. I just saw him a couple of weeks ago and I was amazed at the way that he is responding.”

The treatment does not come without risks. In Shane’s case, he was told there was a 20% chance the treatment could kill him. “But he came through it with flying colours,” says Dr. Anderson. “The last time I saw him he looked great. He didn’t look like someone who’d just gone through ablative chemotherapy with a stem cell transplant. And he was more upbeat than I’d seen him in a long time.”

Dr. Elemary is hoping to get approval to treat another scleroderma patient with stem cells and “with the success of Shane’s story” is optimistic that will happen.

In May, Calgary’s Dr. Storek, with co-authors Drs. LeClercq and Andrew Daly, will publish a report in the Canadian Medical Association Journal, reviewing the challenges of integrating the treatment in Canadian hospitals, given that stem cell transplantation programs are set up to treat patients with malignant cancers. Dr, Storek also

Dan Muscat, whose recovery continues, is delighted that more scleroderma patients are getting the treatment. “I’m trying to get the word out. This should be an option for people when they are diagnosed.”

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