Northwestern accepting stem cell transplant patients OUTSIDE of clinical trial

[ActiveMSers]

Dr. Burt of Northwestern is apparently accepting patients who are interested in getting a hematopoietic stem cell transplant (similar to my HALT-MS experience) outside of a clinical trial. The cost is apparently in the $150,000 range. To my knowledge, this is the only facility in the US doing this procedure without requiring participation in a trial.

The difference with my therapy (BEAM) and this therapy (MIST) is best described by George Goss who runs the well-done blog http://themscure.blogspot.com/

"The other program, called the MIST trial pioneered by Dr. Richard Burt at Northwestern University recognizes that the fully ablative therapy is risky with approximately a 5% mortality. [Post transplant note: BEAM myeloablative treatment at Heidelberg University Hospital is actually closer to a low 1% mortality rate.] So Dr. Burt developed a regimen that is not fully myeloablative, but is instead lymphoablative which does not kill off 100% the bone marrow. This results in substantially reduced risk with a mortality rate of under 1%."

Dr. Burt is also recruiting for the below Phase III trial, which would be the same treatment (but participants are randomized, so one may be put into the control group). Of course it should be noted that going outside a clinical trial, especially if one qualifies, will not further research and will negate any chance of getting insurance to pay for it.

http://clinicaltrials.gov/ct2/show/N...cr=Open&rank=4

[shucks]

Is there a shot of insurance paying for the things that are part of the clinical trial? I hadn't heard that though I am researching hsct and am thinking of getting it done.

[ActiveMSers]

Hey shucks, good to hear from you. The answer is maybe. But you need both a forward-thinking insurance company (they do, actually, exist) and a disease history that warrants HSCT. That means you've tried one or more of the CRAB drugs and/or Tysabri and they have not worked AND your disease is aggressive. I went from using an occasional cane to a walker in six months. I also went through three FDA-approved meds during that time. But even I was still denied.

Here's a link to a blogger who got transplanted at Northwestern and raised some funds to help.

http://activemsers.wssnoc.net/showthread.php?t=434

But that's an unrealistic task unless you are the world's greatest marketer. Getting 150 of your closest friends to donate $100 would be incredible. $1,000? No way. Incidentally, one person in Seattle got his paid for by an anonymous person who read about his plight in the paper. Crazy things can happen with media exposure. But it's unlikely.

[shucks]

I just got an email asking me to come in to meet with Dr. Burt and the other Dr in charge of the program to see if I qualify. Was your treatment the BEAM or the MIST? I have read all of your updates, but I cant remember. The note asked me to not talk about the study with my insurance, just the test and go from there. the reason being that this is not an acceptance to the study, but an invite to a further screening. Their Dr's probably do a much better job describing the treatment and why it should be approved than I can. Chicago is a long way from myrtle beach, so I'm in panic mode trying to see what is in my best interest.
I have never been on a CRAB, as I went undiagnosed for what looks like 7 years now. I hope that would be enough for some sort of exception, but I don't know. If not, I'll start one tomorrow morning to get time in by October. My GP tells me he'll help any way possible, but the 2 neuros have had wait and see mentalities. My worry is that if my numbness continues just a little more, I'm not going to be able to walk. It takes a fair amount of concentration to get from point A to B now, and I walk a few hundred yards each day for lunch, but it's getting tough.
Does anyone think this is completely crazy?
I have posted on several forums looking for folks to talk me out of trying it, but haven't had much negative response thus far. The folks who are negative generally say "hang on and wait for Campath, or try Fingolimod, which has had similar results without so many side effects." The neuros I have seen dont reccomend these treatments until the patient can't walk, due to the risk, and my goal is to not get there unless I have to. I'm almost to the point of checking overseas options. I am all about criticism, so please send me some.

Thanks for all of your help guys.

[ActiveMSers]

Shucks, my treatment used BEAM, but MIST appears to be effective as well and Dr. Burt has been doing this a long time. Since you've potentially had MS for 7 years and have never taken a DMD, I'd say you've done pretty well if numbness is your biggest issue.

It's real easy to let your brain try and predict the future—we all do it. And attacking this disease with the biggest gun you can find is attractive. So no, you're not crazy. But HSCT is expensive, risky, and hard. Although most who have had the treatment are doing well (including myself), there's no guarantee.

Getting on Tysabri would still be guns blazing and you'd have Campath in your holster for 2012 or 2013 if you wanted to go that direction. Insurance would pay for it, you can get treatment immediately, and you don't have to take several months off of work for the treatment much less run a fund-raising campaign every hour that you are awake. (I couldn't have done it w/o financial help from my parents.)

Good luck with whatever you decide. My only recommendation is that you get on a treatment, any treatment, asap.

[shucks]

update on procedure and questions, as always

Thanks for all of the help and advice Dave.

I have been trying for a couple weeks to get approved for Dr. Burt's Mist stem cell treatment for my newly diagnosed MS.
I had several discussions today with the folks in charge of the study. The bottom line is that they told me to come get checked out, and then told me that they wouldn't take me for 6months. I must be on an interferon or copaxone for at least 6 months and get worse prior to qualifying. As I was wrongly diagnosed for the last almost 7 years, I haven't been on anything. It looks like interferons and copaxone are the only things that count, so I need to pick one and get over my fear of needles pretty quickly I guess. I am leaning towards Avonex, as they all seem to have the same efficacy in the numbers, but this one only needs a once weekly injection. At least it would give me six months to get into some fundraising. Who knows, maybe it will work fine and I'll never get any worse.
I am still really looking at this procedure, and hope it will get fast tracked between then and now.
They did tell me that some of the insurance companies involved do cover a portion, but not all, of the costs. They wouldn't tell me if BCBS (mine) was one of them.
My problem is that I am both afraid of needles and a bit of a drinker. I hear further that avonex is tough on the liver.
Can I be a bit of a drinker and still take something? Which one would fit my criteria, or do I need to give up the sauce? Aside from hunting, fishing, and yelling at a jury, it is my solace. I am interested in any advice I can get.

My workout consists of 60 pushups and a few hundred yards of walking a day. I would like to spice it up, but since I twisted a knee in p90x last year, I haven't done much else. the numbness and clumsiness has taken me out of the game so far. That's for another forum though.
Thanks

shucks

[shucks]

Dave, How long would you say you were out of commission due to the treatment? I am trying to get some planning financially and logistically together, and I'm tying to figure out about how long I would have to be out of work for if I went with this treatment. I work with tons of sick strangers pretty regularly and wouldn't want do anything I didn't fully plan for.

[ActiveMSers]

I was feeling 80-90% within a couple months after the treatment. It was a real fast recovery (except for busting my leg when I got too excited about my walking improvement). Lots of folks have trouble with fatigue, but that was never a problem with me. But I don't know how recovery from MIST differs. Everyone is different, so you probably want to plan for worst case and hope for best.

BTW, here's a link to a patient (young guy, 26) who just got out of the hospital at Northwestern after his SCT with Burt. You'll need to join PLM, though, to view it.

http://www.patientslikeme.com/forum/ms/topics/95802

[shucks]

Dave,
Where did you hear about NW taking folks outside of trials?
I neglected to ask before. Thanks for the link, that will be a neat thing to follow.

[ActiveMSers]

I heard this info from another fellow interested in getting the treatment done. He spoke with Burt, who told him that he didn't qualify for the study at the time (6 months ago), but that he could still potentially get the treatment done. But no $ assistance from NW or insurance....