UPDATE: It seems that just as I was publishing the blog below, this press release was also posted: Clinical Trial Testing New Multiple Sclerosis Treatment to Launch in Buffalo. I am thrilled that this research is being undertaken. I am confident that if these preliminary trials go well that this will go far in allowing other researchers to convince their IRBs to allow similar research at their institutions (and result in additional funding from varied sources).
ORIGINAL POST: I guess that I didn't explain my position on research around chronic cerebrospinal venous insufficiency (CCSVI) well enough, as many people reading my blog on Research Directions Around CCSVI seemed to think that I am against treatment trials for CCSVI. It is only because I think clinical trials worthy of publication in peer-reviewed journals (which is necessary for acceptance by the vast majority of doctors) are so important, that I say that there are still many questions to be answered through research before such a trial CAN be undertaken.
Let me explain something - something real, that all of the yelling, screaming and name-calling cannot change, despite how angry, desperate or anxious any of us are. There is a process to conducting clinical trials, especially at major universities in the US. In order to get approval for conducting any trial that involves humans, there is a very, very stringent approval process that has to be passed. A committee called an institutional review board (IRB), also known as an ethical review committee, must approve any study that is to be undertaken at that institution (even studies that just involve asking people potentially sensitive questions about their health have to pass this process). It is the number one job of these committees to make sure that patients are not put at more risk than necessary at these institutions, not only for the sake of the patient, but also as a CYA for the institution itself (no small factor here).
As someone who has worked to get study protocols through this process as a researcher at a university (Johns Hopkins University, to be exact), I can tell you this - there is NO WAY that a treatment trial on CCSVI would make it through an IRB process at this time. No way. There are far too many questions to be answered about:
- causality of the condition itself and correlation with MS,
- diagnostic methods are far from perfect,
- the intervention methods use equipment that is intended for use in arteries,
- the data that would be collected needs to be agreed upon,
- the right specialists (neurologists, radiologists, interventional radiologists, vascular surgeons) need to collaborate on the proposals (and convince their departments that this is worth pursuing).
That is not all - the list goes on of what would need to happen to get to "yes" on this one.
In order to move forward on this, there needs to be preliminary data that provides a solid foundation for these pioneer researchers to jump off of and use to convince universities and hospitals that this is a valid (and safe) direction to go. Believe me, an "I don't know" answer that doesn't directly relate to the hypothesis of a treatment trial (namely, something along the lines of "treating CCSVI - using the methods in this protocol - will result in clinical improvement of symptoms of multiple sclerosis") will kill any trial before it gets off the ground. You can send links of remarkable before and after YouTube videos and links to forums outlining fabulous results, but that does not count as "data" to the people that have to sign off on these studies.
I have learned a couple things over the years about activism and getting results. If we want to move quickly on CCSVI treatment trials, we have to: 1) choose our targets (those people who can get us to the next step), 2) we have to have a very clear vision of what "success" is (I believe it is trials that give us results that are sound enough that the results are accepted by docs, and ideally, insurance companies), 3) we have to understand the steps of how we get to success.
In the meantime, if anyone wants to - and has the means to - get their CCSVI treated, by all means, do so. More power to you. I am very anxious to see this line of research pursued so that EVERYONE who could benefit from this surgery could have access to it - not just the lucky few who are persistent and wealthy enough to find a doc that will do it "off label" or overseas. Unfortunately, this process takes time and a whole bunch of steps that people with MS would like to see skipped.
Read more: Understanding MS Clinical Trials
Here is the press release for the trial at BNAC– there will be appropriate studies.
http://www.prweb.com/releases/2010/06/prweb4199404.htm
It really doesn’t matter what any of us “think.” If there is venous stenosis, reflux and slowed perfusion, the organ which is supposed to be drained is harmed. Budd-Chiari disease is progressive liver failure due to venous stenosis in the portal vein. There are no brain transplants.
As a 51 year old radiologist recently diagnosed with MS (and no symptoms or MRI changes to indicate hidden disease occurring long ago) I’m pretty suspicious of blaming vascular stenosis on a disease that much more typically occurs in younger people. Referring back to Budd Chiari, although sometimes that occurs even in children, it’s more common in older people (I.e. My age and older) than in 20 to 40 years old. I understand the desire to get a “fix” to MS, but the science needs to be done correctly. I worry that those going out of the US will undergo a treatment which doesn’t fix the disease, is costly, and has significant risks itself.
It was actually Dr. B.B. Lee of Georgetown University at the Bologna international conference for CCSVI who likened CCSVI to Budd-Chiari. His statement was that truncular venous malformations (which are congenital and is how the CCSVI malformations are categorized) are present at birth, and grow as the body grows. Some Budd-Chiari patients are not diagnosed until their 20s-40s. A lifetime of congenital venous reflux damages the organ. Liver, kidneys, and the brain.
Again, you’re a voice of reason. Thank you for your thoughtful commentary. Facts are much more compelling than the blame and yelling I have seen on this issue.
A person with venous insufficiency should have every opportunity to have the procedure to unblock the vein(s) without question. They do it all the time.
Indeed no doubt, IRB approvals are demanding. But with all your experience why don’t you suggest ways to get their.
How do we get the data? How do we get diagnostic methods developed? Plenty of noise but except for Buffalo (NY) very little rigourous research is being performed. As such diagnostic research is not invasive and potential benefits are huge it must be relatively easy to demonstrate a favourable balance between risk and benefit.
Any plan starts with the first step and in this case that must be : How do we get an abondance of research focused on diagnosis and correlation between CCSVI and MS like symptoms.
Such data will silence resistance from the medical and pharmacuetical world and open up research for developping vascular treatment on the veins.
Nice example not to do it is the University of Amsterdam where they performed a study on 20 patients using an MRI only. Statistical irrelevant and obvious incomplete method. Such is only creating adverse noise
With all your knowledge please suggest ways how to get the research people of their backside.
There is also a treatment trial currently ongoing at Georgetown University. One is planned at Stanford University (where I brought the published research in 2008) IRBs are being approved, slowly, but surely.
Today’s front page of the health section in the New York Times:
http://www.nytimes.com/2010/06/29/health/29vein.html
Thanks, Julie. I’ve always like reading your blog, esepcially since it’s from the viewpoint of someone in the medical profession.
It was sad to see the vehement comments directed towards you after your last column. It’s really a case of shooting the messenger. But, your comments in this column were dead-on.
BTW, Budd-Chiari syndrome is caused by occlusion of the hepatic vein or inferior vena cava, not the portal vein.
I am so pleased to see the serious and thoughtful comments in response to Julie’s CCSVI posting. I have been following the literature and although this is an interesting development, I am very very leery of anything that is presented as a single solution for this multifaceted condition. Also–as a former cardiac nurse I can tell you that I won’t be jumping in line for CCSVI until there is some definitive research that shows that this really is a direction in which we should proceed. I have seen first hand the danger of cardiac balloon angioplasty and stenting (restenosis, clot formation, damage to the walls of the blood vessels, bleeding, reaction to the x-ray dye or stent material). These dangers are present in cerebral balloon angioplasty and stenting as well.
More to think about re: CCSVI…one person has died as a result of the procedure and one other has had emergency open heart surgery to remove a stent that ‘broke loose’ and ended up in the person’s heart. Basically, because of this, the procedure has been suspended at Stanford. That’s a lot of critical problems for a very small number of cases.
By the way, Julie, your e-mail link doesn’t work for me (I have an old iMac, is that a problem?).
Also, I am beginning Ampyra tomorrow. Anything about people already using it would be appreciated.
Even Dr. Zamboni has said that CCSVI is not a cure for MS but, it can help relieve or lessen many of the debilitating symptoms of the disease.
It’s a quality of life issue.
As I posted before, the IRB route is all well and good. But it should not be the *only* route available to Americans. We should not have to go off-shore for these procedures if we want them and can pay for them.
This is the primary problem of western healthcare: It’s a highly regulated monopoly and such structures never give the best results.
Sidebar to Meg. I filed an FDA complaint against Acorda Therapeutics today regarding Ampyra. I participated in the clinical trial and now, to receive the now FDA approved drug, I have to sign an agreement turning over all my medical records to Acorda and agreeing that Acorda can redisclose them to anyone at anytime. In fact the language of the agreement says my PHI is no longer covered by Federal privacy laws.
Acorda is using FDA approval as a lever to get at your medical records and essentially make them public records. This is illegal — or at least should be.
Check what the authorization you signed. It’s the same for everyone. They worded it in a way which makes it seem reasonable, but the reality is that they can pull all your records any time they like and distribute them to whomever they like. Convenient for them, but wrong for us.