Canada has the highest rate of multiple sclerosis in the world. In 2009, Italian physician Paolo Zamboni published an explosive paper about a new treatment for MS, which became known as MS "liberation therapy." For the estimated 100,000 Canadians who live with the disease, the news was greeted with hope, and many sought the treatment.
Now, Zamboni has done a follow-up study and found it doesn't work.
MS is a disease of the central nervous system. For many people, it causes progressive and disabling weakness, numbness, vision loss and paralysis. Most experts believe it's caused by the immune system attacking the nerves. Medications reduce the frequency and severity of attacks, but they don't stop the progression of the disease, leading many patients to seek other treatments.
Dr. Zamboni developed what was regarded, when he introduced it, a radical new theory to explain the disease; a hypothesis that he called chronic cerebrospinal venous insufficiency (CCSVI). Zamboni said that the veins in the head and neck that drain blood from the brain back to the heart get narrowed and blocked. According to the theory, the narrowed veins delay the flow of blood, and cause it to back up inside the brain and spinal cord. Suspicions have been raised that the iron found in red blood cells accumulates inside brain tissue, and that the iron is what causes the typical symptoms of MS.
Zamboni proposed to fix the problem by doing an angioplasty procedure to dilate or widen the veins, and in so doing help drain the blood. In 2009, he published a preliminary paper of 65 patients with MS who got the treatment — dubbed MS liberation therapy. Zamboni concluded that it was effective, especially in the 35 patients who have the form of MS that tends to come and go. Media reports on MS liberation therapy followed, and it took off like a rocket from there.
The publicity fuelled widespread interest in the treatment from patients, family and friends. For a brief period of time, some doctors in Canada and the U.S. offered MS liberation therapy, but most patients traveled to places like Costa Rica, Mexico, Bulgaria, Egypt, India, and Poland, at a cost of around $6,000 to $10,000 or more, plus travel and living costs.
Many patients came back claiming partial relief of their symptoms. Others said the impact was dramatic. There were patients who said they boarded the flight from Canada in a wheelchair, and came home walking, if not dancing.
But, as testimonials grew, the doctors who treat people with MS were increasingly sceptical about the theory behind CCSVI. Doctors chalked up the testimonial benefits to the placebo effect. Studies have shown that patients who have a placebo benefit from a procedure typically feel much better for a time, but then they relapse. Within a year, most patients who experience the placebo effect are back to where they were before the treatment.
That's pretty much what happened to MS patients who had the treatment. Some patients went abroad a second and even a third time. Few if any of these patients enjoyed the same degree of benefit following the subsequent rounds of treatment they felt the first time.
A few patients who had MS liberation therapy had serious side effects and some died.
You might be wondering what prompted Dr. Zamboni to do a study with more scientific rigour. As time went by, more and more studies were published that challenged his hypothesis. A study by researchers in B.C. published in The Lancet showed that the abnormal veins Zamboni talked about also showed up in people who don't have MS. Another study by the same researchers showed that Zamboni's treatment of opening up the blockages was no better than a placebo.
Zamboni himself acknowledges that his original paper published in 2009 was not a controlled clinical trial, which means the results were at risk of being biased in favour of the treatment. Facing uncertainty about the treatment, Zamboni himself did a randomized clinical trial published last month in JAMA Neurology. The study pitted MS liberation therapy against a sham version of the procedure that was in every respect identical to MS liberation therapy except that in the sham version, the veins were not dilated.
Zamboni's own study showed that MS liberation therapy offers no objective improvement in patients with relapsing-remitting multiple sclerosis, the subtype of MS that Zamboni believed might benefit the most. The Italian doctor noted that there was a trend toward improvement in patients with the relapsing-remitting type of MS, but said the results did not reach statistical significance.
Zamboni concluded that the therapy he devised is a "largely ineffective technique; [and] the treatment cannot be recommended in patients with MS.
You can't get much more definitive than that.
This is not the first debate about a controversial treatment. Still, several things make the one over CCSVI and liberation therapy one for the history books. To my knowledge, this is one of the first in which patient power played a significant role in setting the research and treatment agenda. The treatment was demanded by patients over the strenuous objections of most recognized experts. Patients organized campaigns on Facebook and other social media in favour of the treatment and against MS specialists who criticized it. Some experts were so intimidated that they were afraid to speak to the media.
The controversy developed during the era when social media was becoming a powerful force in society. I think it's fair to say that social media helped proponents of MS liberation therapy find their voice.
The other thing that makes this story unusual is the extent to which some provinces, most notably the Saskatchewan government led by Premier Brad Wall, paid for and actively encouraged research into CCSVI and liberation therapy. In general, governments take care of politics, and leave scientific study to researchers. This was a notable exception.
If scientific evidence were the arbiter, MS liberation therapy would stop. But clinics abroad haven't stopped offering it, and patients from Canada and elsewhere continue to seek it out. The doctors who treat people with MS and the researchers who do studies say millions of dollars have been wasted on a procedure that turned out to be more fake health news than the real thing.
I'm not sure I agree. Given the fact that Canada has the highest incidence of MS in the world, I think it was necessary to determine with a high degree of certainty the value of the treatment.
Still, there are lessons to be learned. For patient power to work in the future, I think it can use a bit more scientific rigour. I'd like to see patient groups consult with their own expert advisors who are well versed in the scientific method, ones who can evaluate the plausibility of new treatments, and the science behind them.
Scientists too must be careful not to oversell their research. If Dr. Zamboni had been a bit more circumspect about his findings in his original paper published in 2009, perhaps patients with MS might have had a more realistic view of his theory and treatment.
Still, let's give a proper shout out to Dr. Zamboni, who brought MS liberation therapy to the world, and who ultimately did the science that was instrumental in debunking his own theory. Not too many physicians would do what he did.