Dr. Richard Burt of Northwestern University has been doing cutting edge research on the use of stem cells for autoimmune disorders for more than a decade. The core idea behind the work that Burt and other research teams are doing is that stem cells may have revolutionary potential to somehow counteract the autoimmunity that causes serious, life threatening diseases in many patients, including those suffering from particularly aggressive forms of Multiple Sclerosis (MS). Some have recently been characterizing Dr. Burt’s MS experiments as potential cures.
Are Burt’s ongoing stem cell clinical trials providing patients with severe MS and other autoimmune diseases a stem cell cure as some are claiming or is it too early to be making such claims? Are the results more intermediate or potentially only temporary? How risky are these trials for patients health-wise, hope-wise, and financially? The fact that interested patients have to pay (either themselves, through donors, or via insurance) $100,000 or more each to be research subjects definitely complicates things. Some patients have even mentioned on the web needing $200,000 for participating in the Burt MS trial.
What’s going on here?
Today’s post is Part 1 of a three-part series on the Burt stem cell trials at Northwestern that tackles the promise and concerns over these trials. The goal is to catalyze an open, diverse, and rigorous discussion. Because there is so much positive buzz about these trials that in some cases borders on hype, I’ve consciously tried to put on a skeptic’s hat (or lab coat). You can read Parts 2 and 3 here and here.
Burt’s trials are more on people’s radar screens lately because a few weeks back the FDA website indicated that it had sent him a lengthy warning letter a couple of months ago. The warning was related to a host of issues with his stem cell clinical trials including failure to promptly report 2 out of the 4 patient deaths. Such reporting delays took around a year or longer when patients dying should have been reported within 72 hours to the FDA and the IRB. For more on the warning letter see this analysis by a different site, but another FDA concern was that the Burt team may not be properly overseeing investigators in their trial in other countries. 
There’s an urgent need for new options for patients with severe MS and Burt’s work has real promise to be game changing. The standard of care for patients with severe MS today is often so poor in terms of outcomes that we need innovation here and some patients are willing to take on sizable risks. However, in addition caution is required as there are so many vulnerable patients looking for hope and no FDA-approved stem cell treatments for MS. Even Burt’s experimental therapy is not yet fully FDA approved because the work is still ongoing. How a trial such as Burt’s is marketed, funded and run has major impact on and risks for patients.
The possible mechanisms of stem cell function in the studies by Burt and other teams in treating autoimmune diseases such as MS include tamping down autoimmunity through a kind of immunosuppressive function or, as is the focus of Burt’s work, promoting a “resetting” of the immune system via partial chemoablation of the immune system. Once specific portions of the existing dysfunctional immune system are killed, the patient is infused with their own isolated hematopoietic stem cells (HSC) and the hope is that these primitive HSCs will then make new mature immune cells that have little or no autoimmunity. It’s an exciting idea with definite promise, but it remains unproven.
Patients challenge me to learn more on the Burt trial
I’ve been following the stem cell work of Burt for years because it is so innovative and also in part because patients have regularly commented on this blog about his clinical trial practices. Some have asked me how the Burt work is different from what stem cell clinics are doing. For instance, see this comment written by a blog reader who has been upbeat about stem cell clinics, and check out other comments in that same thread.
Some patients have challenged me to learn more about the Burt stem cell clinical trials and some even said that he is “charging” patients to receive experimental treatments both inside and outside of clinical trials. That latter kind of “off study” practice is not necessarily against FDA rules if you get permission in advance for what the FDA calls “expanded access”, which is largely synonymous with the more commonly used term “compassionate use”, but it increases risks for patients. For example, investigational stem cell offerings in such contexts with less data backing them could make patients receiving them relatively more likely to suffer adverse outcomes that might otherwise have been anticipated and prevented based on the knowledge that would have been gained from pre-clinical or earlier clinical studies that were never done or are as yet still ongoing.
The FDA even allows charging trial patients in some rare circumstances, but again that kind of practice especially combined with off-study experiments greatly raises the stakes for patients. If not handled carefully such a practice could become more akin to what is referred to as “pay-to-play”. Patient-funded research is not necessarily a bad thing and in fact especially in tight times of funding it could positively advance important clinical research, but it needs extremely careful planning and oversight. Patients should be given realistic expectations with no hype, the trial should have clear, thoughtful and transparent policies on its receiving of funds from patients and how that links up (or not) with specific patient access. Clinical trial participants should also be instructed to avoid promoting or even accidentally hyping the trial to other prospective participants. Overall, there may be ways to do this kind of thing carefully and with transparency. For example, some institutions have innovative care policies and sets of procedures to review and approve innovative approaches to patient care. Still requiring patients to pay to be trial subjects is risky.
For all these reasons, I’ve been asking myself how to learn more about the unusual, high-stakes situation with the Northwestern MS trial and their other trials using stem cells for additional autoimmune conditions. What I found over the past 12-18 months was a complex, mixed bag of exciting ongoing clinical science and some potentially very concerning clinical trial practices. I’ve summarized my concerns in the flow chart below of how patient experiences may unfold in these kinds of trials. Even though I have major concerns about aspects of this trial, I would note that as I’ve been learning about it I have not come across patients involved who said anything negative about Burt or the trial. They tend to be upbeat and enthusiastic. Unhappy patients could be out there, but so far at least I have not seen that.
Burt trial website with medical claims goes private
I started trying to learn more by asking myself, “what’s out there in the public domain?”
Northwestern’s Division of Immunotherapy has had a publicly-accessible stem cell trial page for years and I found that a good place to start to learn more. I was struck by the implied big medical claims as I was reading that trial home page. Note that it is now a password protected website that the public cannot access, which raises major concerns about transparency. But you can see for yourself what it is like in an archived version of the page here on Wayback Machine without a password requirement.
Click around on some of the pages there. Look especially at the videos and bold statements about patient outcomes. What’s your impression? My sense is that at the very least life changing, potential cures are implied there. The page includes an image of a magazine cover on a story on Dr. Burt’s work that unambiguously says “Curing Multiple Sclerosis” (see a version of the story image in the pic at the top of this post). Of course, the Burt team isn’t responsible for a magazine’s article title, but someone is responsible for including it on their patient website.
Keep in mind again that what is offered by Burt in the clinical trials he is doing for MS, scleroderma, and other autoimmune conditions is not yet conclusively proven to be safe or effective for patients overall. Randomized, properly controlled studies are needed and without those, nobody can really be sure how this will all turn out.
To try to learn more I also reached out to Dr. Burt. In the past few years I have emailed him a couple of times in a polite way asking a few questions, but got no reply either time. Their website on stem cells became password protected soon after I emailed him in 2016. I emailed him again last week and this time he replied, but just to say he would not be commenting and to not ask him again. I had hoped that he would be more open to dialogue and transparency given how unusual his trial is particularly in terms of how it has been publicly marketed to patients (see more on marketing and other issues in Part 2 of this series coming up soon) and how much financial and health-risk burden is placed on patients.
Pushing fundraising that publicly releases private patient health info
The Burt/Northwestern stem cell website also has a handbook for patients wanting access to investigational stem cells that contains some striking language. For instance, this section on patients having to pay up front makes it clear that there’s a major financial focus:
“A financial contract of the estimated cost of the procedure will be prepared. The financial contract costs vary depending on what type of stem cell transplant is needed. Your financial coordinator will tell you the cost of your specific transplant. All contracts must be paid in full before any medical treatment can be given. Many people fundraise to help pay for the treatment. Please see Appendix Fundraising Ideas for additional resources and assistance.”
Clinical trials are very expensive to run so the money side of things needs attention and without enough funding a trial often doesn’t even start so that may have been a consideration in the Burt trial requiring patient payment, but how much burden if any is reasonable to place on patients? How much can the average patient get from insurance and how many patients get no insurance coverage for these experimental stem cell therapies? Who knows? The phrasing in that quoted passage from the Burt team patient handbook is also notable in part because it focuses on “medical treatment” even though what’s being discussed there is not a proven, FDA-approved medical treatment, but rather access to a clinical trial with still investigational offerings. Are some patients getting the wrong impression?
The specific fundraising ideas mentioned in the handbook largely consist of encouraging patients to turn to family, friends, and also strangers online or elsewhere to ask for money for the patients to then pay to Northwestern. The below passages promoting fundraising as the way to go to prospective clinical trial participants raise particular concerns:
“Decide on Soliciting Methods/ Ways to Disperse Information
- Internet: fundraising websites, email, blogs, social media
- Face-to-Face: Schedule Appointments
- Written Requests/ Letters
- Telephone
- Media: News, TV stations, Newspapers, Radios”…
- Call your local media including the news stations, TV stations, newspapers and radio.
- Encourage them to do a special feature or human interest story for the cause.
- If planning a special event, ask for a news release a few days to one week prior to the event.”
Is Northwestern actively telling prospective clinical trial participants who are patients to do these things? One could get that impression. Keep in mind that the patient “information” mentioned as to be “dispersed” in the text of the Northwestern handbook is often going to be the patient’s private information including in certain cases some health data. Patients could try to fundraise online without releasing any of their private info, but that’s generally not going to work. You need to include your name, your condition, location, and some personal information to put together a compelling online fundraising story, right? It seems so.
Is it ethically permissible for a health care organization to encourage (in some sense, to require?) some patients to raise money online, on the radio, TV, newspapers, etc. and actively give up aspects of their medical privacy in doing so or otherwise these patients won’t be given access to a clinical trial? This is an extremely serious question. Could that practice, if it is in fact happening and even if it is indirect, be some kind of violation of the duty to protect patient confidentiality?
The big picture concern here is that for some patients there may be a kind of indirect trial coercion to tell the world about their health issues so as to get the funds needed for trial enrollment or else they could be denied access. In my view, it isn’t ethically permissible for there to be pressure even indirectly from an institution for their prospective clinical trial patients to do that or the consequence for the patient is to potentially be denied access. I wonder how often patients who cannot provide the money that Northwestern requires for these trials are turned away? Is it ethical to ever reject patients for a trial based on lack of money, insurance coverage issues, and such? Overall, since there are so many bioethical questions here I wondered at that point in my research whether Burt’s team had a bioethicist on board. There are several excellent bioethicists throughout the Chicago area.
You can read Parts 2 and 3 here and here.
Probably way too late but what the heck 🙂
The SCOT trial: https://clinicaltrials.gov/ct2/show/NCT00114530
Because it’s sponsored by NIH, for Medicare this a “deemed” trial and so CMS will pay. Many (most?) insurers will follow Medicare so while it’s true both the SCOT trial and Dr Burt’s trials cost $$, subjects in the SCOT trial have a very good chance of coverage.
Please correct me but I don’t believe (http://www.sclerodermatrial.org) has anything to with the NIH sponsored SCOT trial. In fact there is no information about who is running the trial or where it’s located. That’s unacceptable and great reason to stay away.
I’m really disappointed that this blog would try to discredit someone who has saved many lives. Especially when not knowing all the facts. MY LIFE WAS SAVED BY DR BURT AND HSCT. I was dying all my organs were failing, I have Systemic lupus in 2016 my kidneys started failing soon after all my organs had begun to suffer as well HSCT was the far out option, other that a slow and painful death, in early 2017 I began my journey to northwestern and HSCT I successfully underwent a transplant and was saved. Close to 5 years later I’m in remission with minimal medications for all the damage I sustained over 20 year of suffering with lupus but NO more STEROIDS, I’m active full of life, I went on to become an RN.
I went through HSCT at Northwestern in 2010. At the time I had a severe case of CIDP a terrible disease in which the immune system attacks the myelin sheath of the peripheral nervous system. When I saw Dr Burt i had had the disease for 4 months, was in a wheel chair and sinking fast. He explained the treatment, the risks, and the potential outcomes. Because conventional medicine, was palliative, and ineffectual and because the form of my disease was malignant- I decided I needed to pursue it. The hospital told me that I needed to either show insurance pre-approval or put up 125K for the procedure. I did not want to wait to fight for preapproval so I put up the 125K. Btw- this seemed like a bargain considering the program was 10 weeks – half in patient half out patient at a world class hospital not to mention it’s saving your life. So here’s what happened:
I was completely bed bound, paralytic, having breathing difficulties and clearly dying when I began the treatment. After the transplant – immediately even – I began to start having feeling in my shoulder area. Whereas prior to SCT everyday was a nightmare of continual loss of function – post SCT everyday day was like Christmas- with the return of function. At the 4 week point I could walk again and at the 3 mos point I had recovered all of my function. I did have some residuals of pain – which was unusual – that lasted 2 years and I do continue to have some slight neuropathy in the feet – but here I am 7.5 years post SCT – off all meds and living a complete and full life! CIDP put me through a living hell – and Dr Burt and the Northwest team gave me my life back. And – my 125K? (That I had kissed goodbye once I gave it to NW?) – well Dr Burt kept working Blue Cross – even weeks after my transplant – and got me that back too.
I am not a miracle boy – I had a good outcome but was fairly typical of the amazing and transformative work Dr Burt has been doing. I met a lot of sick people going through the program and most – had great outcomes. I simply cannot say enough about Dr Richard Burt – he is saving lives everyday. An underpromise overdeliver type – He is the real deal. It is amazing to me that people continue to doubt this technology when it has saved so many lives!
Dr Burt got in trouble because he wanted to do too much. if you read the warning letter it is mostly because he failed to fulfill his duties as a SPONSOR, not so much as a principal investigator. Typically it is a pharmaceutical company that acts as a Sponsor, and it has hundreds of people following clinical trials and making sure all Adverse Events (AE) and Serious Adverse Events (SAE, sometimes deaths) are reported and all regulations are followed. Dr Burt acted as the “Sponsor” of a multinational trial (not sponsored by any one company). Being just one individual he obviously did not have the resources or the Money to
travel to the Clinical Sites in Canada, Brazil, England and Sweden. As far as the science behind his work I want to tell you a couple of things
1) it has been successfully reproduced elsewhere:
for example in Australia
https://www.youtube.com/watch?v=mllc_P3lt9Y
Dr Burt did the first Stem cell transplants for Chron’s disease in the world.
now the hutchinsons cancer center in Seattle and Ringhospitalet in Oslo Norway have replicated them with good results
https://www.youtube.com/watch?v=_YTuvBEad6E
https://www.fredhutch.org/en/news/center-news/2015/07/bone-marrow-transplant-shows-promise-in-crohns.html
There is a group in Paris doing stem cell transplants for Scleroderma under Dr Farge
also see this:
Dr Burt got in trouble because he wanted to do too much. if you read the warning letter it is mostly because he failed to fulfill his duties as a SPONSOR, not so much as a principal investigator. Typically it is a pharmaceutical company that acts as a Sponsor, and it has hundreds of people following clinical trials and making sure all Adverse Events (AE) and Serious Adverse Events (SAE, sometimes deaths) are reported and all regulations are followed. Dr Burt acted as the “Sponsor” of a multinational trial (not sponsored by any one company). Being just one individual he obviously did not have the resources or the Money to
travel to the Clinical Sites in Canada, Brazil, England and Sweden. As far as the science behind his work I want to tell you a couple of things
1) it has been successfully reproduced elsewhere:
for example in Australia
https://www.youtube.com/watch?v=mllc_P3lt9Y
Dr Burt did the first Stem cell transplants for Chron’s disease in the world.
now the hutchinsons cancer center in Seattle and Ringhospitalet in Oslo Norway have replicated them with good results
https://www.youtube.com/watch?v=_YTuvBEad6E
https://www.fredhutch.org/en/news/center-news/2015/07/bone-marrow-transplant-shows-promise-in-crohns.html
This is a very exciting concept-I think in the very near future we’ll see some amazing results from stem cell trials that are just starting out now. I look forward to following Dr. Burt’s stem cell clinical trials for MS.
Yes please follow us. My blog is elliehsct.com
Even if the treatment is 200K (one time for 70% after 5 years) most MS treatments are over 75K a year every year until you die! I think it’s closer to 120k for treatment at NW with some other costs but compared to DMDs it typically would pay for itself in 2 years. The stem cell therapy speeds up recovery the chemotherapy treatment is the curative portion. Look into most MS therapies and you will find many have their roots as chemotherapy agents. A person in theory could go through an intense chemotherapy regimin and have the same effect. There are reputable clinics around the world that offer similar treatments at considerable less cost. This is not the same clinics pushing stem cell treatments as a cure. Your article is uniformed and extremely biased almost if you were on the pharmacutical payroll. Most MS treatments are also associated with severe adverse events including death. Many with one year efficacy on a flare at under 50%. No stopping the underlying cause. Ocrevus the new treatment was a reformulated Rituxan that big pharma pulled from trial since it was running out of patent. This only proves that therapies available and research being done is being stalled to maximize profits at the expense of the patient and exploitation or collusion with the insurance industry. It would be in this country’s best interest to fund non pharmacutical trials to actually find effective open source therapies. Instead the FDA assures billion dollar barriers that only big pharma can navigate and afford for treatments to reach market.
NWU is just one of several locations doing hematopoietic stem cell trials for autoimmune diseases. In fact HSCT trials for autoimmune diseases are run at several major Universities in America, for example, Duke and Seattle, and all these universities require insurance or personal payment . You can find out about them on the web by looking under STAT or SCOT. In fact, the SCOT trial, which was funded and run by the NIH also mandated insurance or personal funding for enrollment, and these trails charged well over 100,000 dollars. This is, in fact, no different than HSCT for leukemia or cancer. Your own University, i.e. the University of California system, demands insurance or personal money, before it will perform stem cell transplants for leukemia. The problem with insurance payment is a problem with the US health care system both private and public including Obamacare, Medicaid, and Medicare. For example California Medicaid and Obamacare refuse to pay for HSCT trials for autoimmune diseases.
All good University stem cell transplant programs, whether for leukemia or autoimmune diseases trials, offer support or direction in how to self-fund raise. What I don’t see is the link that any facility is responsible or supports the release of any private health information. That decision is made by the patient. Is it unfair that patients need to fund raise and in doing so must expose their disease to the world? Yes. But once again that is a US private and government health care issue. You should look at the insurance companies.
You point out patient experiences on a website. From what I can see all trials seem to use patient experiences on their websites. See the SCOT trial web site (http://www.sclerodermatrial.org).There are other sites as well. In today’s world with blogs, YouTube, Facebook groups and other social media apps everything is posted from the patient’s perspective and their experiences. This is a double edge sword because a trail can benefit or be crippled by what the patient community believes to be true. But once again I don’t see this as a NWU issue but as a global issue.
I think your 3 part series on NWH is off target. It seems to be exposing real issues in our health care system and with insurance companies but making it seem that it is only a NWU issue. None of these trails want to exclude patients because of insurance or financial reasons. None of these trials want patients to have to expose their private disease to the world in order to self-fund these treatments.
I would also like to address several statements that you made in regards to NWU that I find either inaccurate or just incomplete.
1- Dr Burt’s trials are more on people’s radar screens lately because a few weeks back the FDA website indicated that it had sent him a lengthy warning letter a couple of months ago. “What you fail to mention is that Dr. Burt received a close out letter dated less than a month later. Here is that letter. https://www.fda.gov/ICECI/EnforcementActions/WarningLetters/2016/ucm548813.htm. You also allude to the deaths that weren’t reported. These were non treatment related deaths that occurred late after treatment. You statement “Patients dying” implies that these were active patients in treatment but in reality died much later and not due to stem cell transplant treatment.
2- You imply that hematopoietic stem cell transplantation for autoimmune disease is unproven. That is also inaccurate. The Scleroderma world has patients that go back to 1998. Patients that didn’t relapse in the first two years live free from the active disease of Scleroderma. These trials only track for 5 years but the Scleroderma community is small enough that we know this is also true 10 15+ years down the road. The treatment has been proven to work in trials like ASSIST, SCOT, and ASTIS and leading universities in America like NWU are leading the way to make it safer.
3- I was struck by your criticism that NWU website is now a password protected website that the public cannot access, which you say raises major concerns about transparency. No one has access to the site anymore except Dr. Burt. This is not a transparency concern but a correct decision to stay away from the impression of advertising. What is not transparent is your website which has advertisements from multiple companies. What financial reimbursements or talks do you get or give from those companies. What is not transparent is that you run a personal blog referenced to the University of California at Davis (UCD). Since you use the UCD name, who at UCD monitors your blog for accuracy? To whom at UCD can we report errors in your blog? Do you report to the IRB at UCD? Since you are part of a University, should you not be accountable to regulatory oversight?
4- You state that stem cell transplant for autoimmune conditions is not yet conclusively proven to be safe or effective for patients overall. You further state that “randomized, properly controlled studies are needed and without those, nobody can really be sure how this will all turn out.” But in fact clinical trials done by Dr Burt and by other Universities in America or Europe have demonstrated that stem cell transplant is effective for scleroderma. And Dr Burt has completed a randomized HSCT for MS (publication pending).
5- You state that you tried to learn more by reached out to Dr. Burt and that In the past few years you have emailed him a couple of times in a polite way asking a few questions, but got no reply either time. You make this sound like Dr. Burt refuses to talk to you because he doesn’t want to have to answer your questions. The truth is that all researchers doing clinical trials must first get permission to talk to any National news media from their University and their IRB. Blogging is not considered National news media. In fact, the distortion of material that you put on your website is exactly why University researchers do not talk to bloggers.
6- As I mentioned in an earlier statement, the issue on your website are not NWU issues but a global issue for all universities treating patients with stem cell transplants. Dr Burt has worked within the system and has made remarkable success in helping advance this field within the current health care system. You inappropriately singled out and criticize one physician and one university. Patients are denied health care every day to multiple clinical trials in the US because their insurance companies or government health care (Medicare, Medicare, Obamacare) refuse to pay for the trial and they don’t have the financial means to pay themselves. Your statement about not being ethical should be directed at both private and public insurance companies that refuse to allow patients the choice on which clinical trial they want to participate in instead of the hospitals and researchers.
@Bill,
Thanks for your interesting comment. You mentioned “all these universities require insurance or personal payment”; can you provide documentation of that such as weblinks, etc.? I have heard that some places will use insurance if possible, but do not require payment by the patient. I have also heard that some trials require minimal payments (maybe a couple thousand dollars) from the patient for certain fees, but that’s a far cry from $100K or $200K. So, I’d be interested in learning more about charging by the places you mentioned. The reason I focused on NW in terms of charging is it was the one I knew of that requires large payments as a condition of enrollment, but if there are others I’d like to learn more about it concretely.
As to stem cell transplants for leukemia, those are proven and fully FDA-approved so charging for them, while I imagine certainly can be a burden on patients, is entirely different than charging for access to a clinical trial.
I’m glad you included the SCOT trial (not to be confused with the SCOTS trial sometimes discussed on this blog) link. In my opinion on first glance, the language on the SCOT trial page is far more conservative than what is on the NW page and in the YouTube videos, etc. I’m going to learn more about it, but again on first glance the SCOT trial page seems appropriately cautious.
Paul
The NIH required the SCOT trail (Scleroderma) and the HALT trial (MS) to either self-pay or have insurance pay before they could enter the trial. In the current STAT trial (Multiple centers) the same is true. Here is the link to that trial and all of those centers. https://clinicaltrials.gov/ct2/show/NCT01413100. Feel free to call any of the centers and walk through the process.
Once again here is the real problem. If insurance won’t pay your only option is to self-pay. These trials will always accept either one. There are no charities or Government funding that will pay for HSCT. Once again this is not unusual in this HSCT space. In the case of Scleroderma there is no real treatment that works like HSCT. The drugs used are not new and they only partially work. HSCT is not the answer long term but for those with aggressive Scleroderma with lung involvement this is the only treatment that currently stops the progression of the disease.
I have to tell you that I personally know of HSCT patients who were covered by Obamacare. That was last year when I got HSCT. At this point I doubt it as it’s become more challenging.
I don’t have wonderful things to say about big pharmaceutical companies at all, but they are not the basic/underlying problem. Effective stem cell research has been demonized and barred from federal funding since the very start, with only a few extremely short windows of opportunity that always ended up getting closed. It’s going to happen again. The politicians who’ve always fought against effective stem cell research– Mike Pence being the best example– now have the power to trash it again, and they will. If the federal government had funded embryonic stem cell research instead of demonizing it all this time, we would not be in this situation in the first place. Research would be so much further along. I really think we’d already have some cures commercially available (for AMD especially.)
We can’t do anything about the real obstacle that has always been in the way until we face it– and it’s been politics all along. There HAS to be more publicity about this issue so that people understand what is at stake!!
The problem is that big pharma is hampering any substantial charity funding into stem cells research so the only alternative is to make patients pay.
It is so sad to see that HSCT has been used in MS for 20 years with incredible sucess and there is only 1 phase 3 study ongoing i.e. Burt study. So thanks to him maybe one day it will be officially approved but I doubt as pharma pressure on FDA is incredible
A lot of people blame big Pharma without actual examples of how and when. Can you support your assertion that they are hampering charity funding?
Yes pharmaceutical companies are for profit businesses. Without them we wouldn’t have any treatment options. I hope they make more money from MS drugs so that more research is funded. One of the main reasons that drugs cost so much is because of current patent laws that force big pharma to recover the multimillion dollars required to conduct research in a very short time before the patent expires.
I agree with a lot of this. I do think it needs to be emphasized though that we would not be in this situation in the first place if certain types of stem cell research had not been demonized this entire time.
Since I can’t edit the post…here is something that should be pointed out. The ad right under this post says “when is an embryo not an embryo.” Look at how companies desperately try to distance themselves from anything that could be related to embryonic stem cell research in any possible way!!
It’s very apparent reading this post that only half of the research has been done on what actually transpires when going through this stem cell transplant at Northwestern. For starters no point in time are we ever told that it is a cure, you are not helped with fundraising at all if your insurance does not cover the treatment as well. As for risks… Dr Burt warns you at the initial appointment that this treatment can be dangerous and may cause death, flat out right to OUR (patients who have undergone HSCT) faces.
Posting on HSCT without experiencing it for yourself can have the same effect you have portrayed above, is your “hype” negativity for a possible huge breakthrough for RRMS?
This post is grossly misleading unfortunately, had you experienced it first hand (and not just read about it) you would have had accurate information.
I take great offense to many comments in your blog, how can you have such an opinion on something you’ve just simply read about? I’m not what’s written on paper… I live the life of an HSCT patients every single day…
I had HSCT at Northwestern in 2014 for MS, if patients are interested in learning about a real patients experience, the good the bad and all in between please feel free to take a look at my Facebook page which I documented my entire transplant and the past years of recovery if you’d like. http://www.facebook.com/heathernicolehsct
Well said. This is exactly my point. Thanks for putting it so well.
Ohh please. Not “transparent”…as a patient who is about to see him it is NONE of your freakin’ business! Yes I know about the deaths, and this is because there were other things going on with these patients than just MS. For as much research as you’ve said you’ve done- it sounds more like trying to find a way to tear this study down. Stem cell treatments are NOT free. It will continue to be this way until the FDA stops trying to regulate a chance patients have of their own body healing itself.
@Victoria,
Of course, you are right that the doctor-patient relationship is just your business.
Overall (but not disclosing specific patient information) a clinical trial actually has legal reporting requirements and on an ethical level openness about specific trial practices in some respects may be preferable and most beneficial to patients.
Also, I’d ask: iisn’t there a contradiction if a clinical trial says essentially to its prospective patients “yeah, go out there and publicly raise money to pay for getting into our trial by telling the world all about your health, etc. (implying something like ‘by the way you may not be allowed to participate if you can’t pay’) and posts things on the web publicly that imply “we are already changing patients lives” or even nearly or fully curing them, but then on the other hand says essentially, “No, we won’t talk publicly about the trial.”
I’m not trying to tear down this stem cell study, but rather I am trying to get a discussion going about ways in which this trial is unusual and perhaps has unique risks. Maybe there are changes that could make it fairer to patients? On a big picture level, I actually hope the trial succeeds and I love stem cells.
As to your statement “stem cell treatments are NOT free”, actually almost 100% of clinical trials do not charge patients just to be in the trial and in a trial what you are getting is not exactly a “treatment” but rather you are serving as a research subject in an experiment that may or may not work, and has definite risks.
The overall point is that related to this trial there are a lot of questions and important things that need more discussion.
My best wishes to you.
Pay to be a research subject. Sounds like every MS drug out there to me. How many diseases do you know of have the Dr say why don’t you research what treatment you may want to try and let me know? You need to walk a mile in the shoes of a MS patient.
I spent 4 years in the CombiRx trial, paid nothing, and got the best medical treatment I’ve ever had. I walked a literal mile between my work and the Neuro’s office every few months. My insurance would have covered nothing. No tier IV drugs, and later no injectables that weren’t insulin or cancer related.
I’ve walked that mile, back when ins cos could utterly screw you for having a major preexisting condition. Drs helping me enroll in trials weren’t nefarious…they were concerned, caring, and the only option I had.
I’m sickened by the idea of ‘for pay’ clinical trials. I’m sickened by promised cures. Clinical trials aren’t the problem. People are.
@Shademar,
Thanks for sharing your experience.
For reference for other readers, here’s a link to the CombiRx trial: https://clinicaltrials.gov/ct2/show/NCT00211887.
Paul
Thanks for the context, and apologies if I come off as overly upset.
The current health care situation has me rattled. I was only able to finish grad school because of a legitimate clinical trial; if I’d have had to pay for that, I wouldn’t be where I am today. Neither would my family.
Best,
Shannon
I’ve spent 2 years looking into this. I was approved by Dr. Burt for treatment but the Dr at my insurance company review who took large payments in 2013 from pharmacutical companies denied it. In the time since my denial on standard approved 85K a year therapy my left side feels like it’s on fire. In your comprehensive research did you find the increase in suicide with MS patients. So wouldn’t it make good sense to have the government fund such research? We are not talking about a new treatment at all but rather a current treatment for a different purpose.
YES exactly…..nobody says anything about cancer patients getting bone marrow transplant. HSCT is the EXACT same thing!!!! It’s just like using wellbutrin for depression when it was called Zyban to quit smoking. One drug two purposes. One treatment multiple purposes. Ugh.
Hi,
It appears to me that you have taken a one-sided approach in this article, though it claims to be a balanced one.
Typically, in my understanding, most trials are sponsored by Pharmaceuticals with deep pockets for investing in research so that they can develop expensive drugs and make their billions from these. This trial at Northwestern is not a drug trial and hence it requires the patient to pay for it. It is not illegal and more importantly, it has turned around the lives of hundreds – including mine.
I came all the way from India, with an Indian salary and paid my way through the treatment, since the protocol logic and published data indicated that it is worth taking the risk. And right enough, it turned my life around. So highlighting in a box ‘patients must pay $ 200K for a risky…marketed as life-changing by Northwestern?’ is pandering to negative sensationalism. Please note, patients have willingly paid the amount knowing full well that the treatment may not work at all. In fact, Dr Burt makes it a point to clearly communicate (he did to me and I know to many others) that the treatment at best will halt the progress. This, even though data indicates relapse free survival at 4 years for MS patients is around 80% and reversal for a substantial %.
Highlighting only the warning letter without taking about the point by point reply further makes this look like a one-sided article.
I also do not see anything unethical in suggesting various methods for patients to raise funds- I believe this is an additional support. The patient has the choice to follow any of this or not. Please do not assume patients are uneducated or unable to decide for themselves.
Many centers across the world are now offering this treatment to patients and makes me wonder why the FDA is sitting on this?
My sincere request is to speak to some patients (you can enter any of the forums on FB) and then give a more balanced view point to the readers. Otherwise, you might end up painting this as a dark scam, whereas in actuality, it is life changing for the patients.
@RamG,
Thanks for your comment and feedback.
Paul
I concur.
Paul sounds like a jilted lover because Dr Burt can’t share unpublished results and doesn’t return his phone calls. And the (mis) leasing questions!
Is it true that Paul has professional jealousy because ISPCELL can’t sell STEM CELL derivatives to these potential patients? Is it true that Paul research will be worthless if Dr Burt and colleagues are successful with this therapy?
Fact: Patients in the trial dont pay.
Fact: When the trial is complete then anyone can get the treatment. You’ll have to wait your turn like the rest of us to get the results to see if your insurance will pay.
Fact: If your wife is laying in bed with excruciating pain from CIPD then yes, you might travel to Brazil to get treatment. You might spend your lifes savings
If your friend shoots himself in the head because of advanced scleroderma with no possibility of a normal future then you might hope for this treatment.
These are autoimmune diseases that are not part of the study.
Fact: The costs for this treatment are lower than non effective existing treatments. Look up the cost of Rituximab for example.
Fact: I have personally talked with his patients and have been very impressed with the outcomes so far.
So thanks for your skeptical lab coat, but for the rest of us we’re foot for Dr Burt!!!!
I like it. I actualy didn’t think about if NW picks up the cost for the actual trail or not. I was accepted on compassionate case outside of trail and my insurance denied it. I guess they would prefer to pay 85K a year for life instead of the 130K one time cost. I have talked to so many people now from the Dr Burt treatment as well as other qualified centers that follow similar protocols. So many doing so well. Medicare was actualy paying for many and then that dried up. Wonder who got paid off by big pharma on that one. Only big pharma has interest in seeing this fail. The big difference here is that the cost actualy is paying for your care instead of lining big pharma pockets.
@PJ,
I don’t think some of your “facts” hold up.
For instance, if patients don’t have to pay according to your “fact”, then why does NW itself say patients do have to pay and patients themselves have told me they had to me? And what about all those people on Gofundme saying they need loads of money to be in the trial?
You wrote that when the trial is complete “anyone” can get it? That is only going to be true if the trial is proven based on properly controlled data to work and be safe for a large group of people. Anecdotal reports don’t prove things.
I hope Dr. Burt’s trial is proven to work.
Whether it is or not has no impact on IPS cell research of the kind that I do.
I actually think his trial seems promising even if it isn’t done yet based on the bits of info I could gather, but I have concerns about how it is being run, marketed, etc. and questions about whether it is fair and ethical to patients.
Paul
I think you should be questioning if the current process to have any trial is fair and ethical. When drug companies can pull drugs from trial on a profitability concern because a patent may expire. It seems that this country needs a mechanism to test cost effective treatments. Instead this country has created a barrier of entry so large that only the members in the price colluding pharma oligopoly have the means to effectively participate. This ties in with bigger issues as well. People need heath coverage and drugs that are affordable. North Western University puts these people up for 21 days in hospital. It’s not like you take a pill and come back in for follow ups every week.
@Paul Hanten,
You make some good points.
Yes, there are completely legitimate questions about the overall clinical trial process. However, from what I’ve seen, and I don’t claim to be a clinical trial expert, there are fair and ethical trials out there including other stem cell trials.
No trial is perfect, but some teams really go the extra mile to do all that they can to respect patients by not hyping things and treat them fairly such as a avoiding as much as possible charging patients or encouraging patients to do fundraising that risks the release of private health info.
Yes, people need health coverage that includes affordable drugs in such a way that patients can access them and not break the bank. Our current system leaves a lot of people behind and also there are things that are just grossly too expensive.
And, yes, as to your point, what NW and others do in the way of stem cell trials, particularly those involving immuno-ablation, are very different in intensity of clinical care associated with it and costs as compared to a trial where someone simply takes a pill or gets a non-living biologic injection and leaves, but I believe that there is so much room for improvement on how stem cell trials like this are managed, burdens on patients are monitored and limited, etc.
Thanks for weighing in.
Paul
You make it sound like NW is hyping this treatment up and pushing people to crowdfund. That is simply not the case. People may be doing this but not by NW direction. Also they accept people off study and those same people still reference the trial. You question ethics yet provide no factual basis to your own claims. Claims of others are not reliable sources. I met with Dr.Burt and his team on multiple occasions. Very professional and up front. Off study and No hidden agenda.
@Paul Hanten,
Thanks for your input as a patient with direct experience. It is really valuable.
But it seems like maybe you didn’t actually read my posts as there is documentation in there.
For instance their own trial website had an image saying “Curing Multiple Sclerosis” on it.
Their patient handbook did, in my opinion, direct or enable patients to go fundraise. If you go over to gofundme and search for relevant terms related to this trial, are you surprised at the # of fundraising campaigns? Maybe not, but I was.
Also take a look at this video below. What do you think?
https://www.youtube.com/watch?v=n-vo-n5awjk
These things seem to me to be very unusual for a still ongoing clinical trial. For that reason and others, they raise legitimate questions and concerns.
Paul
Dr. Burt does not claim this is a cure. I don’t know what website you saw or who created it. As for their website being down I think you’re reading too much into it. As a patient who had HSCT last year I can tell you with the trial being a Phase 3 Trial (which by the way…..shows it DOES work because it was proven in phase 2….) they are winding down and nurses are leaving and probably whoever was managing the website is gone too. Dr. Burt receives a salary from Northwestern- he does not financially benefit at all from continuing to treat patients. I was treated off study. There are different reasons for that. My insurance covered. As someone said above Medicare used to cover but stopped and I find that extremely suspect and agree someone in Big Pharma got to them….
Let me tell you something. Dr. Burt visits his patients every single day he is in the country. Holidays and weekends. The man is a hero.
I, too, strongly suggest you visit the HSCT sites and see what the patients say. He gave me my life back and I’m not in the minority- I’m in the MAJORITY.
I’m glad you are bringing attention to this issue but would prefer to see a more balanced review. I’m new to your blog so I have more to read and hope to see a change of tone by the end. 🙂
I was just recently approved at an evaluation with Dr Burt for this procedure. My insurance has also agreed to pay for it. The trial is over, and Dr Burt himself told me “it’s not a cure, rather it halts progression of the disease”. I have not been misled in any way and if I wanted any other medical procedure out there I would be required to pay for it, so I don’t see what the debate is about. Everything costs something, nothing is free, Dr Burt appears to be a quiet, compassionate man. He probably doesn’t want to talk and have his words misconstrued due to the recent “bad publicity”. I’ve talked too many who have had HSCT done with Dr Burt, and I haven’t found a bad result yet! Most are actually astounding! However, I am realistic and have been warned by Dr Burt’s team that improving results are not a guarantee of HSCT. No hidden message or false hope there.
Great to get your insight into this. Just learned of it last week so it’s an eye opener as I am a very vulnerable person desperately seeking less pain!
I was a normal person seeking a treatment that may prevent progression. Do your own research this man is not helping anyone.
This is such an exciting field but thus far with little or no substantial results.
However there does seem to be a number of Companies taking advantage of peoples desperation and operate without transparency.
Thank you and I look forward to parts 2 & 3.
This is not true. The overwhelming majority of us treated by Dr. Burt have gone back to living our lives. What you have to understand is that drug companies are not going to sponsor HSCT. It would put them out of business. So who is going to pay? Not the National MS Society. They’re in deep with big pharma even though they hide it. In Canada, their MS Society paid for their clinical trial. Why? Because socialized medicine recognizes that it’s less expensive to cure people than to treat them. We’re greedy here. As Chris Rock says “Ain’t no money in the cure- the money’s in the medicine.” https://www.youtube.com/watch?v=r0ArVKsFe3w
Dr. Burt is extremely transparent. He does NOT take “advanced cases” as the author of this blog stated above. He does not claim to repair any damage- only to halt the disease. However many of us, myself included, have experienced amazing results with renewed function.
Other companies out there? I think there are countries who are very good and others who just take money from desperate medical tourists.
I think that your article is somewhat misleading. There is one clinical trial at Northwestern for MS and HSCT. It is limited to 110 RRMS patients that must meet the inclusion criteria. You can’t simply buy into it. Yes, there are other stem cell trials going as well for other diseases, but again there are strict inclusion/exclusion criteria. Also, most clinical trials bill insurance for services that benefit the trial like MRIs, screenings, etc. Your article seems to try and put this research on par with the for profit stem cell clinics or stem cell treatments that are offered in other countries and have no objective data that proves efficacy. This is a University backed trial that follows good clinical trial methodology, meets rigorous standards, and has gone through IRB reviews. The results will provide good clinical data that will be beneficial to all of us. If I could simply buy my way into this trial, I would.
Thanks for the comment, Ken.
Of course, all legit clinical trials have exclusion and inclusion criteria.
But what’s very different about the NW MS trial and perhaps other trials there at NW are several things that warrant open discussion and maybe even debate: (1) large payment as an inclusion criterion, (2) apparent institutional promoting public patient fundraising that releases their private health info perhaps in many cases, and (3) already marketing the trial as successful while it is still ongoing and the jury is still out on how it’ll end.
As to the stem cell clinics: No, as I’ll discuss in future parts of this series and as I mentioned at the end of this post, I don’t believe the NW MS trial is generally similar to the stem cell clinics.
Cheers, Paul
People are leaving this country every day, finding ways to pay for this life-changing treatment – in other countries where they are in a strange place, strange language, away from family and often the things they need to live well. They do this because the US takes FOREVER to approve treatments that have helped almost 80% of those with RRMS stop the disease progression. In the meantime, insurance companies are paying hundreds of thousands of dollars for these same patients to be on medication or have infusion treatments whose side effects can cause debilitating and life altering issues.
I applaud NW and Dr. Burt for doing what they can to get approval for patients to have hope of a better life, and decreased disease progression, in this country.
YES. Agreed. I believe it’s higher than 80% but there have been so many more of us treated that I’m out of date.