'Transformative' treatment by Calgary researchers gives patients with rare disease new lease on life
Stem cell treatment helps scleroderma patients move, breathe again
By Sarah Rieger, CBC News Posted: Jan 10, 2018 6:12 PM MT Last Updated: Jan 10, 2018 6:12 PM MT
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When Miaya Killips was diagnosed with scleroderma her first instinct was to Google the rare disease.
"And then I sat in my car and I cried," she told CBC News.
Killips, 31, is one of six patients that received a pioneering treatment at the University of Calgary as part of an international clinical trial that doctors say can slow or even halt the disease's progress, giving patients a new lease on life.
Rare, deadly disease
Scleroderma is a rare and deadly autoimmune disease that hardens tissues and organs — approximately 20 in one million people are diagnosed with it each year.
For Killips, her diagnosis started with Raynaud's disease, which causes blood vessels to spasm and cut off circulation to the extremities.
She had just finished trade school and was working as an industrial electrician outdoors.
"How do you work with your hands when your fingers are curling and they go numb from the cold? You can't," Killips said.
Her career was over and her symptoms progressively got worse, impacting her internal organs like lungs and gastric system.
When tissues harden, it means that joints can't move, the face can't express emotions and lung disease can develop, eventually causing death.
Options for treatment were limited — she could pay $165,000 US for chemotherapy in Chicago, or $55,000 US in Russia.
Then she was selected to take part in an international clinical study at Calgary's Tom Baker Cancer Centre and the Foothills hospital, which was covered by Alberta Health.
"The disease is thought to be caused by an immune cell error which, instead of attacking bugs, are attacking the tissues of the patient," said Dr. Jan Storek, a professor of medicine at the University of Calgary (the only Canadian site for the study), which wrapped in 2016.
Doctors extract stem cells from a patient's bone marrow as part of the new treatment, and then give them high doses of chemotherapy to wipe out their immune cells.
Then they reinsert the patient's own regenerated stem cells — essentially "rebooting their immune system" with healthy cells.
The treatment was a risky one, but Killips knew she could die if she didn't try it.
She's glad she did, because it changed her life.
"I can move again," she said. "I have so many opportunities in front of me that I wouldn't have had if I didn't get the treatment."
Don Remizowski, a 44-year-old high school principal in Saskatoon, was also treated as part of the study.
"My rheumatologist at the time basically said to me, you likely have about two years to live," he said.
He received total body radiation and a full year's worth of chemotherapy in just three days, before his own stem cells were transplanted back into his body.
He said the treatment was difficult, but worth it.
"When you're dealt a card like that it's a whole different thought process, that would have never entered my mind but I'm thankful for everyday that I have now," he said.
Storek said the results of the study have been "transformative."
Patients were split into two groups — half received the conventional treatment of just chemotherapy, and half received the new stem cell treatment.
After six years, 51 per cent of patients that received the conventional treatment and 86 per cent of those that received the new treatment survived.
The findings of the study, which took over a decade to complete, were published in the New England Journal of Medicine on Jan. 4.
The scientists will continue to follow the patients to review their long-term progress.
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