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
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.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
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.......
I think the best group to ask for compassionate treatment is Jacobs or Stony Brook.