TORONTO – A long-awaited Canadian study of a controversial treatment for multiple sclerosis known as “liberation therapy” has debunked the procedure, saying it is ineffective and should be avoided.
Liberation therapy involves opening up narrowed veins in the neck, a procedure that an estimated 3,000 Canadians with MS have undergone in clinics outside Canada at a cost of many thousands of dollars each. The experimental therapy is not approved in Canada.
The procedure was first put forward as a potential treatment in 2009 by Dr. Paulo Zamboni, who asserted that narrowed veins in the neck could cause iron deposits to create lesions in the brain, leading to multiple sclerosis.
The Italian vascular surgeon dubbed the condition chronic cerebrospinal venous insufficiency, or CCSVI, and cited dozens of cases of patients who improved after he performed the treatment, known as a venoplasty.
But Dr. Anthony Traboulsee, an MS specialist at the University of British Columbia who headed the clinical trial involving 104 Canadians with the condition, said researchers found no overall difference in outcomes between 49 patients who received the vein-widening therapy and 55 who got a sham procedure.
“The major finding was looking at the patient-reported outcome of quality of life and the most important finding of that was that there was no difference at either 72 hours (post-procedure) or at the end of study, week 48, in terms of change in the measurement for either group,” said Traboulsee.
“So both groups looked exactly the same at the end of study, and that was whether we looked at the physical score or the mental composite score or the fatigue scale or the pain scale — all the scales showed exactly the same thing.”
Preliminary results of the $5.4-million study, which was conducted in Vancouver, Winnipeg, Montreal and Quebec City, were presented Wednesday at the Society for Interventional Radiology’s annual scientific meeting in Washington.
MS is considered an auto-immune disease: the body’s immune cells attack the protective coating around nerve cells, called myelin, impeding their ability to conduct electrical signals and leading to problems with movement, sensation and cognitive function.
The cause of the disease, which can take several forms, is unknown. Although some treatments can slow its progression, MS has no cure.
Zamboni’s proposed treatment brought hope to MS sufferers worldwide, leading thousands to pursue the scientifically unproven therapy at private clinics in several European countries, Mexico and the United States.
Many patients in Canada — which has one of the highest rates of MS in the world — were quick to embrace the procedure and offered online testimonials of its benefits.
Canadian researchers set out to test the therapy by randomly assigning 104 MS patients with narrowed neck veins to receive either a venoplasty, in which a tiny balloon is inserted into the vein and expanded, or a sham procedure, in which the balloon is threaded into the vein but not blown up.
The study was blinded, meaning neither patients nor the doctors who assessed them knew which “treatment” they had received.
While the Canadian trial found no “overall” difference between the two groups in quality of life measures, standard medical assessments or the development of new brain lesions as seen on MRI scans, Traboulsee said about a quarter of patients in each group reported improvements in symptoms.
“When we look at the individuals, we do see that, just like we do in the real world,” he said. “There were patients who reported to me: ‘I know I got (the real procedure). I feel better.’
“At about week 2, we did see an improvement in the overall population (in both groups). It doesn’t mean everybody is feeling an improvement. It’s about 25 per cent of patients have had an improvement in their quality-of-life scale. But then by week 48 it starts coming back down to their baseline.”
Traboulsee said he hopes the findings will persuade people with MS not to pursue liberation therapy, which carries potential risks and has led to a few deaths, at least two of them among Canadians who had the procedure.
Yves Savoie, president and CEO of the MS Society of Canada, which contributed $500,000 towards funding of the study, said it was important for researchers to take the necessary time to assess Zamboni’s theory and test the procedure to answer the question: does it work?
“We now have a definitive answer,” he said.
“Understandably, some people will be disappointed with these findings. Others, I think, will find satisfaction that this brings a confirmatory finding to the question … which is that the treatment is not efficacious.”
One of those who is disappointed — but not surprised — by the negative conclusion is Linda Hume-Sastre, president of the 800-plus-member advocacy group CCSVI Ontario.
“I call this an excellent example of sham science,” said Hume-Sastre, who had the vein-dilating therapy at an Albany, N.Y., clinic in 2011 and said she had marked and sustained reductions in her MS symptoms, including less fatigue and improved balance.
Despite the study’s findings, she advises other people struggling with MS to research their treatment options, including liberation therapy.
“The reason we are members (of CCSVI Ontario) is because we believe in this. We know it works,” Hume-Sastre said Wednesday from her home in St. Catharines, Ont.
Traboulsee said the researchers expected criticism of their study, but he stressed its design was carefully crafted with MS patients’ input to be scientifically robust and unbiased.
“Our goal was not to disprove the treatment; our goal was to test the treatment,” he said, adding that he understands why many people with MS are frustrated with the slow pace of development of new treatment options.
“We gave this proposed intervention the best chance possible to succeed.”