Ottawa doctors' high-risk MS treatment yields 'impressive' results, Lancet finds

"We kill you and then we rescue you."

This is what Jennifer Molson remembers doctors saying to her about the high-stakes procedure she would undergo in 2002 as part of an Ottawa study that has yielded some promising results in multiple sclerosis patients.

The 41-year-old Ottawa woman was in a wheelchair before the treatment. She now walks, runs and works full time.

"I had no feeling from my chest down. I could barely cut my food," Molson remembers.

Molson was diagnosed with MS when she was 21, and within five years she needed full-time care.

"It was scary. [The procedure] was my last shot at living."

High-risk therapy

MS is among the most common chronic inflammatory diseases of the central nervous system, affecting an estimated two million people worldwide.

New Canadian research led by two Ottawa doctors and published in The Lancet medical journal on Thursday suggests the high-risk therapy may stop the disease from progressing.

"This is the first treatment to produce this level of disease control or neurological recovery" from MS, said The Lancet in a news release.

But The Lancet also highlights the high mortality rate associated with the procedure — one patient out of 24 involved in the clinical trial died from liver failure.

"Treatment related risks limit [the therapy's] widespread use," The Lancet concludes.

Results 'impressive'

Nevertheless, in the journal's accompanying editorial a German doctor calls the results "impressive."

"This is the first trial ever that showed complete suppression of any inflammatory disease activity in every single patient for a prolonged period," the doctor notes.

The editorial says that for now, the treatment should be restricted to specialized centres, and warns that "all efforts must be taken to avoid stem cell tourism."

The clinical trial, led by The Ottawa Hospital's Dr. Mark Freedman and Dr. Harold Atkins, involved 24 patients over 13 years.

The patients underwent an intensive combination of chemotherapy and stem cell transplants in a procedure known as immunoablation and autologous hematopoietic stem cell transplantation (IAHSCT).

Stem cells removed, replaced

First, doctors harvested stem cells from the bone marrow of their patients, then purified and froze the cells. Patients were then subjected to high doses of chemotherapy drugs, similar to those used to treat leukemia. The preserved stem cells were then returned to the patients. 

The idea is to eliminate the diseased immune system, "resetting" it so it has no memory of attacking the central nervous system. 

The results were striking. According to the Lancet, the treatment fully halted clinical relapses in all 23 of the surviving patients and stopped the development of any new brain lesions without medication.

"I had holes in my brain. It lit up like a Christmas tree," said Molson. "If you see MRIs of my brain before the transplant and after, I have no enhancing lesions on my brain at all. My images are clear. It's amazing."

Results 'floored' researcher

The best the researchers had hoped for was to halt further damage to the patients' nervous systems. Freedman, a neurologist for The Ottawa Hospital, said he was "floored" when patients began showing improvement.

[Jennifer] was holding her own, and one day she comes in for her visit and she comes in wearing heels ... This is a girl who could barely walk with a cane last time I saw her.- Dr. Mark Freedman

"[Jennifer] was holding her own, and one day she comes in for her visit and she comes in wearing heels," said Freedman. "What is this? This is a girl who could barely walk with a cane last time I saw her."

Freedman, who has researched MS for three decades, began to notice remarkable changes in other patients, including the vanishing of involuntary rapid eye movement, called primary nystagmus, common in MS patients.

"I've never seen it disappear. Something really neat was going on with these people that was not explainable by our expectations. They were healing."

Some participants recovered lost vision, regained mobility and have even returned to work.

'Like a miracle'

"It was like a miracle, right?" said Atkins, a bone marrow specialist and medical director of regenerative medicine at The Ottawa Hospital. He's used stem cell transplantation to treat other auto-immune diseases.

"It didn't happen all at once. It was very slow," said Atkins. "But you kept getting happier and happier as you saw the improvement over time."

Stem cell transplants have shown good short-term results in MS patients, but the symptoms always returned. The difference this time, said the Ottawa doctors, is that while previous trials aimed at suppressing the diseased immune system, they wanted to wipe it out.

But the procedure was toxic. For that reason, researchers modified the treatment regimen over the course of the study to mitigate the effect on patients.

According to The Lancet editorial, these factors may have been the reason for the "tremendous anti-inflammatory effect" shown in the Ottawa trial.

'A nice reward for the patients'

The Lancet editorial notes the trial was not controlled by a placebo group, calling that its "most important limitation".

Still the results are particularly encouraging in light of other failed treatments such as the Zamboni method.

"It's a nice reward for the work," said Atkins. "It's a reward for the patients. They put their trust in us and put their lives in our hands. It's a validation of how robust and how important these findings are for the medical community as we move forward."

Despite their success, the doctors remain cautious. Freedman emphasized the combination of chemotherapy and stem cell transplant remains risky, and isn't for everyone. Both he and The Lancet urge wider clinical trials.

"There is a lot of other stuff we have today for the treatment of MS which is very effective. Why go to the cannon when the little pop gun might take care of it?" Freedman said.

Molson, meanwhile, can hardly believe her own transformation. 

"I got married 11 months after my transplant. I walked down the aisle. I danced at my wedding," said Molson. "I was exhausted. But I did it."

She says she takes nothing for granted, and hasn't yet given away her walker and crutches.

"I don't want to jinx myself. I'm just living my life with this second chance."