Multiple Sclerosis occupies a particularly terrifying niche among chronic, disabling diseases. While all serious threats to health are to one degree or another fearsome, those we understand least, and that leave us feeling most vulnerable and defenseless, are certainly the most feared.
Variable in its course and at times indolent for years, MS can, when extreme, progress rapidly and culminate in total disability and premature death. Characterized by most as an autoimmune disease, MS is still poorly understood. We don't really know exactly why some people get it, we don't know how to prevent it, and there is treatment, but no cure.
This is a situation that screams necessity, and thus invites inventiveness, such as that of Dr. Paolo Zamboni, for whom the need became personal. Dr. Zamboni, an Italian vascular surgeon whose wife has MS, was frustrated with the ineffectiveness of conventional therapies, which mainly involve suppressing immune system function.
Seeking, initially, to account for the common occurrence of iron deposits in the brains of MS patients, Zamboni conducted brain scans. He noted apparent blockages in the veins that drain blood from the brain in MS patients, consistently absent from the brains of healthy control subjects. He also noted that the blocked veins corresponded well with the location of iron deposits, and that the more severe the blockages, in general, the more severe the MS.
Zamboni next converted his insight into an angioplasty procedure -- now known as "liberation therapy" -- in which a balloon-tipped catheter is used to open up the partially blocked veins.
In Dr. Zamboni's published work, responses to liberation therapy have been very encouraging, although even at best the procedure is not a panacea. Zamboni suggests that the longer veins are blocked before liberation, the more potentially irreversible damage to the brain becomes. Some veins re-occlude after angioplasty. And some patients simply don't respond well. But the testimonials of those who do, including Dr. Zamboni's wife, are quite compelling -- telling tales of restored function and quality of life.
But there are, as yet, dark clouds of doubt wrapped around the silver lining, if not silver bullet, liberation therapy might represent. From the start, many mainstream neurologists have found the entire theory implausible. I myself am troubled by the fact that MS occurs almost exclusively in those born and raised above the 40th parallel, and see no obvious means by which Dr. Zamboni's theory can account for this.
Now, two studies published in the Annals of Neurology -- one from Sweden, one from Germany -- compound that doubt. Both studies used imaging techniques to examine the neck veins of MS patients and matched controls, and neither detected any consistent difference at all.
So, Dr. Zamboni himself, and the patients who claim significant responses to his treatment, believe his observations constitute an epiphany. Much of the medical and research community is far less sanguine, and considers the approach a potential boondoggle.
The Premier of Saskatchewan, Brad Wall, confronted this dilemma with a greater sense of urgency than most. Canada has one of the highest rates of MS in the world, and the prairie province has the highest rates in Canada. So as of July, Saskatchewan became the first Canadian province to dedicate research dollars to study the liberation procedure, and actively encourage clinical trials. While the other provinces and territories look on with increasing interest -- knowing they, too, have residents traveling abroad to seek the as-yet inadequately studied procedure -- Premier Wall has clearly staked out a leadership position.
I was privileged to speak with the Premier and discuss the basis for his decision. I found him knowledgeable, informed, thoughtful and measured.
The Premier knows that liberation therapy is not certain to work, and is certainly not devoid of risk. But like most residents of Saskatchewan, he knows the toll of MS within his own circle of friends and family. And so he knows how fervent is the hope, and need, for new therapies.
The Premier has not endorsed liberation therapy, even in the face of compelling anecdotes from citizens of his own province. Rather, he has endorsed the hope engendered by the therapy, and the need to cultivate that hope by determining, responsibly but swiftly, whether the promise of the remedy is one it can truly fulfill. He is calling on the research community to make that assessment.
In theory, the prime directive of medicine is "primum non nocere": first do no harm. Even the statistical underpinnings of all biomedical research are designed to protect us from a rush to folly: studies conventionally permit up to a 20 percent risk of a false negative result (i.e., concluding something doesn't work when in fact it does), but only a 5 percent risk of a false positive (i.e., concluding something works when in fact it doesn't). But without boldly accepting some risk of doing harm, we have no real potential to do good. And harm can be done by inaction; there are sins of omission, just as there are sins of commission. (Fellow Star Trek fans will no doubt recall that all of the best star ship captains often found that the only ethical way to honor Star Fleet's prime directive -- noninterference in alien cultures -- was to defy it!)
I do not yet know if liberation therapy works. I agree with Premier Wall that we need to find out. I commend the Premier for exercising bold leadership betwixt the perils of commission, and omission. Saskatchewan's policy -- which I hope others will adopt -- will help us determine sooner than later what happens when the hope kindled by liberation therapy is fed the fuel of science.
Between now and then, I urge patience. Hope is a powerful impulse that readily invites a leap of faith; good science, however, offers much much more reliable footing. There is apparent promise in liberation therapy, but we need well-done research to tell us if it is promise the procedure can keep.
Dr. David L. Katz; www.davidkatzmd.com
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