Compassionate Treatment

A forum to discuss Chronic Cerebrospinal Venous Insufficiency and its relationship to Multiple Sclerosis.

Compassionate Treatment

Postby mrhodes40 » Sat Oct 17, 2009 2:50 pm

I am going to throw this highly speculative post in here about compassionate use of new experimental technology.

I believe it is time to acknowledge the truth we all know on some level but do not want to discuss.

Dr Dake can't possibly treat all the MSers who want help.

AND at some point, he will be required to do a trial with inclusion criteria. As soon as he is required to outline an actual trial, they will have to spend their time on the trial participants.

The trial inclusion criteria are not a cruel attempt to deny care th "the rest of us" but a necessary way to have a group that can be evaluated by some meaningful measure that can show how this worked...

Clearly the announcements from Stony Brook and Jacobs mean that studies are going ahead there even now, but treatment is still a couple years away there, at the MINIMUM.

but that means, once again, those of us with "doesn't fit criteria" disease types will be asked to wait on the sidelines, hoping against dwindling function that in the end it becomes our miracle too...

But there may be a way out of this problem.

There is a landmark case that was decided a few years ago AGAINST the FDA. They had contended that they had a right to stop Americans from getting new drugs until they agreed all the needed trials proved not only that the new drug was safe.........but also that it worked.

Safety is one thing, stage 1 trials are safety trials. Carried out on small numbers of people, they do not attempt to answer the question of effectiveness but rather to answer the question of safety for human use.

Stage 2 and 3 trials are done to see "if it works" for the intended patient.

In the case in question the FDA had denied a drug to a woman whose specific type of cancer cell was targeted by a drug that was then in stage 2 and 3 trials. They already had established it was safe. But she wanted the drug because she had exhausted her possibilities on the regular therapies and was dying anyway.

She actually did die and her family pursued this case against the FDA rule that does not allow treatment with things that are already known to be safe but not yet shown to be effective in cases where the patient has no other option, contending essentially that the patient has a right to undertake risk for unknown gain in the fight for life.

The District court remanded the appeal back to the lower court, so the family won the appeal. I read a commentary at the time by a lawyer in which he said "the court basically said that the FDA is like a person who stops a good Samaritan from giving aid to a person who needs it--in other words liable for the harm that comes to the person who did not get aid".

Essentially this seems to be a legal support for the idea that people with no other options should not be denied a potentially helpful treatment even though it may have risks and be as yet unproven, as long as the drug is known to be safe, and the patient in question understands the consequences and risks, and they are mentally competant to make that determination.

Can this apply to CCSVI treatment?

I offer that Revimmune is unproven treatment that has been made available for compassionate reasons while stage 2 studies are ongoing. Though it has a few small studies they are far too small to consider "proven"--obviously; the stage 2 is still mid trial.

The main reasons for Revimmune being available as I understand it is that JH has been able to argue that it "should" help based on the theory related to rebooting, what all the neuro community thinks it knows about MS cause, and the fact that some small studies on ASCT, a different but similar unproven treatment, seem to suggest help for MS. Therefore Revimmune is offered as compassionate treatment in cases where options are exhausted. I believe even medicare is paying for Revimmune treatment.

So what I am offering here is the idea that as more research is published on CCSVI, importantly the paper on the first liberation treated patients, we may be able to lobby for compassionate treatment under the same premise.

This is where there are parallels.
1. Stents, though not previously used in the locations we use them for, are an approved and understood technology that has been around with a good record for over a decade. They aren't new.
2. The stenosis to be treated is visible and diagnosable on MRV or CT, it is not hypothetical.
3. The stenosis can be quantified in terms of percentage of vessel blocked and in terms of pressure gradients across the stenosis, standard measurements of how significant the stenosis is and whether or not to treat. Treatment can demonstrably show that these problems are corrected with surgery.
4. No one knows if treatment of stenosis can alter the course of MS yet, but it is logical on a physiological standpoint to believe it likely would be helpful and that leaving the stenosis as is would likely be harmful.

The risks associated and unanswered question, in addition to the question of MS, is whether or not with time these stents in the high jugular location in particular will prove to be a problem if replacement is necessary. There is an unknown factor there that a person would need to consider and take medical advice on....but for some people that risk may be a fair trade, and mentally competent persons should have the chance to decide that it seems based on the legal decision (below)

I am wondering if our efforts here might focus on perhaps demanding en masse by writing the NMSS and the governing bodies of the various involved medical boards to allow treatment of CCSVI based on compassionate grounds?

What if your neuro, if you had no options in the regular MS treatments, could refer you to a vascular doctor as a last ditch effort, even before this is proven??

What if when they gave you the talk "I am sorry but there are no drugs for your stage/type/level of disease" you had a right to return with "then allow me to seek assessment by a vascular doc" ?

For your perusal I offer this landmark case:
Story of Abigail with links to resources

Actual government legal finding

First the right at issue is the right of a mentally competent terminally ill adult patient to access potentially life saving post phase -1 investigational new drugs, upon a doctors advice where that drug carries risk to the patient.(emphasis mine)

Second we find upon examining "our Nation's history legal traditions and practices" Glucksberg 521 US at 710, that the government has not blocked access to new drugs for the greater part of our Nation's history. Only in recent years has the government injected itself into consideration of the effectiveness of new drugs....snip....the key is the right of the patient to make decisions about her life free from government interference


I am no lawyer --please know that I have no expertise in this area at all but it seems clear that in the case of Abigail who died wanting a cancer drug her namesake organization got agreement that the FDA has no right to stand in the way of a person's pursuit of life when no other options are available.

So, for those of us who have exhausted the standard therapies and who are literally waiting for MS to finish it's course, this argument may be a way to insist on compassionate treatment ahead of the years long process of complete approval.

I just wanted to throw this out there to see if anyone else sees a way to use this information to push for making CCSVI treatment available to the progressive person, who can't even get into a trial, sooner and before the crucial last bits of function are lost.

If anyone has any legal expertise please weigh in ...I have no idea if these are actuallly equal.......
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Postby CRHInv » Sat Oct 17, 2009 3:29 pm

Wow Marie! My foggy little brain just can't keep up. This is very interesting and seem like a very kind thing to pursue.
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Postby radeck » Sat Oct 17, 2009 3:45 pm

Thanks for all those timely thoughts, Marie! Without wanting to make all this redundant, can I ask the obvious question why physicians can't treat this for what it is, CCSVI, as long as treatment is safe. This avenue would save all the legal hassle (so far most insurances are paying for it and they'd had to have a good reason for stopping tondo it in new patients), and would circumvent the potential pressure we're going to see from the pharma industry.
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Postby mrhodes40 » Sat Oct 17, 2009 3:56 pm

can I ask the obvious question why vascular surgeons can't simply add to Dr. Dake's workforce and continue to treat this for what it is, CCSVI, as opposed to what it maybe
that is a good thought but no one has impressed any other doctors to take up the mantle with that argument and I believe the hangup is simply that in spite of the fact we are treating a co factor the reason for treating is still MS and the hope it has impact on it.

Example, I have a stenosis in my vericose vein where I had a clot. It is not getting treatment because it is not a "problem" Only 10% of leg blood is carried by surface veins so it is not expected to impact my overall circulatory integrity. This argument could be offered by skeptical persons: yes there is stenosis but there are many other veins so it should not matter.

I was looking for a way around that, as the fact that it "matters" is that we have MS lesions, assuming the model here. And that is why I mentioned that as this next paper comes out on treatment impact on MS, there is beginning to be a case to be made that MS lesions are impacted by these stenoses.
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Postby patientx » Sat Oct 17, 2009 7:04 pm

I'm not sure what the concern is here. Stent surgery is already being performed at Stanford on people who show CCSVI. If this procedure wasn't already approved, then none of the 20 odd people would have had it performed. It's true that if Dr. Dake does decide to undertake a clinical trial, then, he will have some inclusion criteria, and he will have a comparator group. So, yes, he will have to deny performing surgery on some who may want it. But if someone wants the surgery he or she can still have it. The trick is finding a vascular surgeon willing to perform the procedure.

I offer that Revimmune is unproven treatment that has been made available for compassionate reasons while stage 2 studies are ongoing. Though it has a few small studies they are far too small to consider "proven"--obviously; the stage 2 is still mid trial.

The main reasons for Revimmune being available as I understand it is that JH has been able to argue that it "should" help based on the theory related to rebooting, what all the neuro community thinks it knows about MS cause, and the fact that some small studies on ASCT, a different but similar unproven treatment, seem to suggest help for MS. Therefore Revimmune is offered as compassionate treatment in cases where options are exhausted. I believe even medicare is paying for Revimmune treatment.


I believe the main reason Hopkins was allowed to use Revimmune was that cyclophosphamide was already an FDA approved drug. From what I understand, it had nothing to do with a compassionate basis, and the treatment was completely off-label, not a phase 2 trial. Likewise, Campath, which is already FDA approved for certain forms of leukemia, and is in trials for approval as an MS treatment, can be gotten off-label. It's just a matter of finding a doctor to prescribe it.
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Postby Daisyduck » Sat Oct 17, 2009 9:26 pm

Marie, this is good thinking. As far as Dr. Dake goes, we are the stage 1 trial people, the forerunners, before he has to start the clinical trials, and the information he got by treating us and our follow-ups will be most important to his work and to others after us. Already he has adjusted his MRV regimen and, if I understood this right, the Coumadin has been discontinued as routine treatment? Dr. Dake and I talked about this a little while I was at Stanford in June for 4 stents. I had been under the impression that we were being treated because of the humanitatian aspects as you mention (and maybe because we had stenosis and never mind the MS).
Just before that I was on a regimen of SF1019 and was treated in Utah under that same theory. Thank you for your research. Daisyduck
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Postby ErikaSlovakia » Sun Oct 18, 2009 12:08 am

Hi Marie, you are right.
I have been thinking about this for a long time.
I do not know any person with MS in person, not even in Slovakia, so I do not know what they think. They do not post anything negative so far.
I am active on one Czech forum and 2 Slovak forums.
I inform them as much as I can, until the "other side" wakes up.

Erika
Aug. 7, 09 Doppler Ultras. in Poland, left Jugul. valve problem, RRMS since 1996, now SPMS,
- Nov.3,09: one stent in the left jug. vein in Katowice, Poland, LDN, never on DMDs
- Jan. 19, 11: control venography in Katowice - negative but I feel worse
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Postby sou » Sun Oct 18, 2009 1:35 am

Hi.

It is a pity that the decisions about a patient's health is not to be taken by the medical science, but the political science.

Anyway, I think that balloon angioplasty can be an alternative until Dr Dake's trial is over. It is a temporary solution, but it is not associated with the pain caused by stenting.

I can't accept the argument that there are more veins for the blood to flow, even if one is blocked. Let me cut your hand!

1. You won't die.
2. You have another hand.
3. You even have a leg!
4. Oh, man! You have yet another leg! Come on! Let's cut the hand!

Seriously. Millions of years of evolution have created this similar venous system to all vertebrates. It is very cursory and irresponsible to think that there might not be any problem, while there is a well documented theory of how collateral circles can damage the nervous system and deposit toxic iron amounts within it.

So, let's forget about stenting for a while, but nobody has the right to prevent us from having angioplasty, especially after January, when the Liberation results will be out.

sou[/b]
Shortest joke: "We may not be able to cure MS but we can manage its symptoms."
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Postby Daisyduck » Sun Oct 18, 2009 7:28 am

The eventual legal hassles appear to be inevidible because of all the vested interest in MS, including but not limited to the various MS foundations and the high-priced drug manufacturers. I've felt for a long time that we should focus on the stenosis part of this disease and treat that as reparable, but not act as if this is the answer to MS so as not to start other interests to want to shut down the CCSVI repair process on us. Someone commented that ballooning is better than inserting stents, and it may be found to be true eventually. Apparently Dr. Zamboni prefers that method over stents, but when I asked Dr. Dake he said that he actually tried ballooning (don't know how often) and found the veins to be collapsing again so he went back to stents. There is still so much to be learned, and I am greatful for every bit of knowledge in this regard that is discovered and for the institutions here and in Europe who are pursuing this. I am also immenselty greatful for having had the opportunity to participate, and whatever risk there may be I was and am willing to take.
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Visionary

Postby mormiles » Sun Oct 18, 2009 7:51 am

Marie, I "met" you 3-1/2 years ago and admired your vision and efforts then. But now, you hold the title of VISIONARY in my mind and heart. Thank God for you and Joan and others here who are propelling forward this worthy cause.

A couple of other positive factors in favor of our cause are these:

1) The stellar reputations of the physicians already involved in CCSVI research and treatment.
2) The growing interest and positive response of so many other reputable physicians, e.g., Dr. Elliott Frohman of UTSW.

If and when all this comes down to legal "nut-cutting," there will be some true "heavy-weights" to weigh-in on our side. Now, on the one hand, any one of them might have pressure brought to bear on what they might say by the big pharma funding of their respective institutions and programs. On the other hand, though, early involvement in a medical breakthrough treatment is a terrific "feather-in-the-cap" that adds to the prestige of these doctors, their reputations, and the reputations of their respective institutions. Those "cap-feathers" also result in future revenue.
Joyce (aka cypriane)~caregiver and advocate in Dallas for Steve (SPMS)----"Enter through the narrowgate..."
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Postby skydog » Sun Oct 18, 2009 10:03 am

What does this mean for those of us that are in the stage 1 trial per se? Our continued monitoring and treatment may be needed to preserve our quality of life. We are the foot in the door that cannot be shut. We have a solid case for keeping treatment going for humanitarian reasons. Suns out, going out to breath in the beauty of it all. Peace and Health, Mark
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Postby Arcee » Sun Oct 18, 2009 10:08 am

Quote:
can I ask the obvious question why vascular surgeons can't simply add to Dr. Dake's workforce and continue to treat this for what it is, CCSVI, as opposed to what it maybe

that is a good thought but no one has impressed any other doctors to take up the mantle with that argument and I believe the hangup is simply that in spite of the fact we are treating a co factor the reason for treating is still MS and the hope it has impact on it.


I agree that upcoming clinical trials will narrow the focus of attention (and ultimately provide important evidence), and I agree that compassionate use definitely is an avenue worth exploring, but I also think that this angle is worth pursuing fully.

I don't know the numbers, but how many doctors in the US are capable of doing the stents or angioplasty? How many of them have seen the evidence? Talked with a real person who has gone through the procedure? With someone who likely would benefit?

And how many of these doctors know someone personally who has MS?? This is a huge factor.

I think it is worth trying to strategically focus on these doctors. I have told the doctors I know and other TIMS people that I would be willing to talk with, undergo testing, and even share my medical records with legitimate doctors in the Northeast who want to really understand what the stent approach is all about. (Obviously talking with Dr. Dake would top their list.)
Anyway, just my opinion on it, but I think identifying and networking to these folks so they get educated and have personal exposure is worth the effort.
diagnosed RR in spring '04
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Postby bestadmom » Sun Oct 18, 2009 10:33 am

To embellish on Arcee's post, a small group of us from the NE created a private group on yahoo. We built a database of about 100 interventional neuroradiologists at university hospitals in MA, CT, NY and NJ and started sending emails to them on 10/7. We provide links to research.

We are taking a grass roots approach to reaching the guys who we think will have interest in either contacting Dr. Zamboni, DR. Dake, Dr. Haacke and/or SUNY Buffalo. We've had a few good replies so far - no one knows about this. It is piquing interest in a few and giving us recommendations of other doctors to email to .

We found that by setting up a private group, we can easily email eachother en masse without clogging up our pm system on TIMS and having to triplicate our efforts, and we can share the files we are all building together and updating with our replies.

I hope that others on TIMS will do the same by region and we can all come together on here to make sure we haven't omitted any states. We hope that some of the people we are contacting know or know of Dr. Dake and will reach out for more info. We have to hope that the egos of these doctors, as well as their need to help us, will get us the much-needed surgery ASAP, not in 2 years.

Please feel free to pm me if you wat details on how to set up a private yahoo group.

Michelle
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Postby mrhodes40 » Sun Oct 18, 2009 1:15 pm

I am lining up extra ammo guys. not saying the "treat CCSVI for its own sake' is dead; I wanted to get that legal case bookmarked in case someone needs it---in case we need it. I am hoping we do not need to press such an issue but it is another avenue for us if people find they are at a dead end.

My comment about the vericose veins was likewise not my point of view but rather how general understanding of this system seems to be in some minds: one TIMSer had a vascular surgeon basically say exactly that: these jugular stenoses are meaningless and not needing repair 8O 8O 8O

px Dr Kerr is on record with the compassionate use comment. Off label is compassionate use. The trick is that compassionate use of anything everyone in the field agrees makes sense (like autoimmune approaches) is not questioned by the powers that be--no one is going to give trouble to JH for doing revimmune, not even Teva because it supports their model.

but imagine this: a pharma says to Stanford's medical director that either they stop this treatment before the "proper" trials are done or the funds for the X project will be pulled. No one would tell us. They''ll simply stop treating off label so to speak and a 'proper' trial will begin.

I believe that grass roots are needed here, we need to be ready to have several strategies. This is just another angle another avenue. I love the group contacting people in a organized way on the NE coast. :D :D 8) I think the best group to ask for compassionate treatment is Jacobs or Stony Brook.

Forgive my brutal honesty here, but the compassionate use argument for the progressed person may be able to be used by individuals who can't get into Stanford if it comes to that because some of us will not make even 3 more years--- I was lucky I did get in but don't think for a moment I feel sanguine about it, this needs to be available for everyone and every day I think about the fact that we need to find ways to make this argument, convincingly.

the idea that progressed persons at the end of the rope might have to wait makes me ill.

this is already about 2 decades late.......
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Postby patientx » Sun Oct 18, 2009 3:22 pm

mrhodes40 wrote:px Dr Kerr is on record with the compassionate use comment. Off label is compassionate use. The trick is that compassionate use of anything everyone in the field agrees makes sense (like autoimmune approaches) is not questioned by the powers that be--no one is going to give trouble to JH for doing revimmune, not even Teva because it supports their model.

but imagine this: a pharma says to Stanford's medical director that either they stop this treatment before the "proper" trials are done or the funds for the X project will be pulled. No one would tell us. They''ll simply stop treating off label so to speak and a 'proper' trial will begin.


You seem to be expressing a different argument, or I don't understand the problem. Originally, you were expressing concern that the FDA might ban CCSVI treatment, without proper trials having been completed. Now, it's about big pharmaceutical companies squashing the treatment.

I don't know what record Dr. Kerr used to mention compassionate treatment, but it doesn't really matter. Revimmune is not a new drug; it was alrady approved as a chemotherapy treatment. There were no phase 2 trials, and if you think Hopkins had an easy time getting it installed as a treatment, since it supports the autoimmune model and wouldn't draw anyone's ire, you should re-read this:

http://www.hopkinsmedicine.org/hmn/W08/feature1.cfm

With all of these proposed future conspiracy theories, this forum has gotten a little too strange.
Last edited by patientx on Mon Oct 19, 2009 6:55 am, edited 1 time in total.
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