Northwestern abruptly ending Burt HSCT autoimmune trials

Selma Blair HSCT

HSCT Chicago Clinic Closing!” is the startling header in all caps on a patient-run Facebook page about a stem cell therapy clinical trial program at Northwestern University.

What’s going on?

Unfortunately, the promising field of hematopoietic stem cell transplantation (HSCT) for multiple sclerosis (MS) and other autoimmune diseases seems to have suffered an unexpected setback here.

Selma Blair HSCT Burt Instagram
Selma Blair post on HSCT via Burt team on Instagram.

Dr. Richard Burt of Northwestern, one of the leading figures in the area of developing HSCT approaches for MS and other autoimmune disorders, is going on a sabbatical and the university is abruptly stopping enrollment in his pioneering trials.

Everything seemed to be going so well.

The most recent published data were encouraging.

Also, the news just recently broke that actress Selma Blair had apparently gotten a HSCT from the Burt team for MS based on an Instagram post (below). In the caption she included “#hsct #drburt”. Talk about great, positive PR.

But just days later, things seem very different now.

The Facebook group with the url “burthsct” apparently in part administered by his past patients has the concerning news (see screenshot) about Burt’s sabbatical and the trials winding down in its new Description with that “Clinic Closing!” header.

Some patients have also independently contacted me about this puzzling, still developing situation. It seems that something radically changed with the program starting in the last month or two.

What’s the backstory here to explain such a dramatic possible end to what seemed to be a promising and very much still ongoing clinical research program at Northwestern? It really felt like things were going full-steam ahead.

I asked Northwestern for comment and spokesperson Christopher King made this statement:

“Northwestern Medicine is grateful for Dr. Burt’s dedication to providing patients with world-class care and wish him well as he transitions to his planned research sabbatical at the end of the year. While the immunotherapy program will no longer accept new patients, we will ensure that current patients continue to receive high-quality, compassionate care. Northwestern Medicine is grateful for Dr. Burt’s pioneering achievements in this field and the care he has provided to his patients. You may follow Dr Burt’s work though http://www.clinicaltrials.gov and through his publications.”

This doesn’t of course explain the “why” behind this news. Hopefully that will become clearer soon.

Richard Burt HSCT Northwestern FB page patients
Screenshot from Richard Burt HSCT Northwestern FB page run by patients.

I have had some past concerns about this program. As much as the idea of stem cells for MS and other autoimmune diseases is potentially exciting, and Burt’s work on partial ablation combined with HSCT for MS specifically has shown what seems to be real promise, in my opinion some thorny questions have remained unresolved over how the trials have been conducted.

As I was looking into all of this a couple years ago, Dr. Burt received an FDA warning letter regarding some issues including delayed reporting of patient deaths, although it seemed the program resolved the issues moving forward. You can read my past 3-part The Niche series from 2017 for more on my specific concerns here starting with part 1, which links out to parts 2-3.

I don’t know if the new development of the trials winding down and Dr. Burt going on sabbatical have anything to do with these past questions and concerns. Maybe the explanation relates to something else. However, my sense is that something major has likely happened here behind the scenes to lead to such an unexpected turn of events.

I’m worried that many patients could be negatively impacted by whatever is going on, and stressed by the uncertainty. This may also be a temporary blow the HSCT field, although other research groups are pursuing this kind of clinical work as well. It’s an area of real promise and hope for the stem cell field overall, in which Dr. Burt has been a pioneer.

19 Comments

  1. The reason he says it makes no sense is because it is the nuclear option. It’s extremely risky as far as elective medical procedures are concerned. And, most patients relapse eventually. Granted, they tend to get many disease-free years after treatment, which is worth a lot (and maybe priceless to patients).

    MSCs on the other hand have been studied in treating autoimmunity in hundreds of papers at this point, and they have been shown to be very effective in most of them. More importantly, though the mortality risk is hugely decreased versus HSCT. And, there have been SLE patients treated by IV infusions of 1*10^6 MSCs per kg body wt who entered 3+ year remission. That’s a remarkable durability considering the safety profile of MSCs and their ease of harvest from discarded tissues like umbilical cord and placenta.

    • @sean “most patients relapse eventually” and where is the evidence? I’ve never seen it.

      I suspect it drug companies or entity that can pull the strings shut it down. There’s too much money in no cure.

  2. @Sean, you’ll find lots of reports indicating the safety of MSC-containing treatment preparations, but good luck turning up one statistically sound paper showing efficacy.

    • This is very interesting to me. I have an immune-related condition (arachnoiditis from spinal tap) that responded incredibly well to IV MSC (and a year later, HSCT – much cheaper! at only $6K not $17K!). I’m 16 months out from that second one and 28 months from second. I still have med-free pain levels as long as I don’t play too much soccer with my kids, don’t jar my back a lot. When I do, I get the characteristic flare about 48 hours later… but nothing like the 2 years of hellish incessant pain and brain fog on gabapentin.

      I have even been considering intrathecal if I have to have another go. That’s madness, I know. But I have been offered it by several doctors I trust. So, anything I can learn about research for CNS immune conditions is of interest.

  3. So, Brad:

    You present the individual reality that some docs confuse with scientific evidence for demonstrated “efficacy.” Efficacy is a distribution term. Although individuals might experience improved outcomes after a treatment, that experience alone does not allow anyone to predict what is likely to occur with the next patient who gets the same treatment. Establishing efficacy means that you have the body of data from scientifically sound analyses that allows a confident prediction of how many people treated the same way in the future will have a similar experience and outcome. To make such predictive determinations, one needs to conduct “clinical trials,” and often several trials are needed to achieve a high degree of confidence that the treatment is safe and, at a predictable rate, will be beneficial in ameliorating or curing a disease, disorder, ailment, or injury.

    You see although you experienced a positive outcome after your treatment, we don’t really know, despite the long period of no improvement before hand, that the treatment you received is, in fact, responsible for your change in status and, importantly, too, for the reasons given. Mechanism of action matters, too. Because if something has a treatment effect for a different reason than believed, it will be difficult to predict the effect in the future and in different patients and to improve upon it. And in medicine, because of the many complexities of human beings, there are many other reasons that improvements in health status can occur besides the one thought. And I don’t mean just placebo effects either. Also sorts of things related to changes in aging, physiology, behavior, environment, activity, other medications, and even the bugaboo of all clinical effect studies, chance variation.

    So, although your treatment experience may absolutely be due to the reasons believed by you and your physicians, that alone does not allow a basis to begin treating other patients with the expectation, and certainly not the promise, that many similar outcomes will occur. If that were true, this discussion that we are having now would already be moot and in the pages of history as having been unnecessary. Quantifying treatment effects in sound clinical studies is essential, because decades of experience teaches that for most of the serious things that ail us, 100% positive treatment effects rarely occur. Not even aspirin for headaches meets this standard. We have to conduct scientifically sound studies to establish if 1) there is any reproducible treatment effect at all; and 2) thereafter define the size of the effect and the types of patients who are mostly like to respond to it.

    I hope that your treatment experience continues to be a good one, no matter what the actual basis.

    Kind regards,

    James @ Asymmetrex

  4. I applied to be in Dr. Burt’s study twice and was denied, my MRIs showed that my MS was progressing, I felt I had to do something quick.
    I found a 60 Minutes Australia story about Kristy Cruise who went to Moscow Russia to have HSCT, long-story-short, I did go to Russia for HSCT in June/July 2018.
    A year ago I had an MRI in Dr. Federanko’s clinic in Moscow Russia, one year later (just a few weeks ago) I had another MRI here in Utah, proscribed by my Neurologists.
    My Neurologist was very, very suspicious and told me not to go but I had done 2 years of research and when anyway.
    My Neurologist here in Utah compared my Russia MRI and my latest MRIs then I got an e-mail from her, she said there was not new lesions, inflammation or scarring in the last year.
    So it looks like HSCT in Moscow halted the progression of my MS.
    Now I doing physical therapy 3 days a week to get stronger!

    I think people will be going out of the country for HSCT in droves.

  5. Hi [email protected],

    As you state:

    ‘Not even aspirin for headaches meets this standard. We have to conduct scientifically sound studies to establish if 1) there is any reproducible treatment effect at all; and 2) thereafter define the size of the effect and the types of patients who are mostly like to respond to it”

    My comment is then, why is Aspirin still manufactured and sold (worldwide) if this has also yet to be proven.
    Mind you we even give Aspirin to our babies and children.

    • Aspirin has been proven as a painkiller many times over since it was first marketed in the 1890´s. And this followed on from the well known analgesic properties of willow (which contains aspirin-like compounds) going back several hundred years.

      For use as a painkiller it was already sold around the world before the FDA was founded and before the clinical trial system and approval processes were organized. I don´t agree with those who say aspirin would not be approved as a painkiller today – the evidence of efficacy and safety is overwhelming.

      But it must still go through clinical trials for new diseases and it has been approved and also rejected by the FDA for other uses.

  6. Shelly,

    You misunderstand my point. My point is that even for medicines that are approved, we know based on the experience of lots of use that they do not always give benefit to everyone. However, because of the way they have been evaluated, we can be confident that they will often have a benefit for many treated patients. That confidence is not based on the reported experience of one or two individuals. It is based on the scientifically evaluated responses of many, many, patients. Aspirin, as an example, has met both requirements 1 and 2. That’s the measure that we need for new stem cell treatments, whether they are in private doctors’ offices or in FDA-authorized clinical trials.

    James @ Asymmetrex

  7. Health Insurance coved my wife’s Non Myeloblative Stem Cell Transplantation for scleroderma back in 2008. Dr Burt and his team saved my wife’s life and allowed her to be a mother and wife to our family. Pioneers in medicine need to be supported and not harassed. Politics, Ignorant bloggers and jealous professionals have hounded the worlds leading pioneer and medical researcher. His patients and family’s know the true impact of living with autoimmune diseases that symptom management regimes have been failing. A treatment that doesn’t just treat just the symptoms but addresses the cause saved my wife. The research we took part in had stringent protocols and due process. People who are suffering will no longer have this chance we have thankfully received, you and the others will have to answer for their suffering.

  8. Patients like myself do not have time for snobby restrictive researchers to get their act together in the U.S. before I get this treatment. Proven? How about the thousands of people who have gotten this around the world in Mexico, Russia, etc. with great success.

    Meanwhile, our good ole USA medical system drags its feet on HSCT for autoimmune diseases in the name of SAFETY! Give me a break! BS meter off the charts! HSCT is done for cancer in the U.S. I guess cancer is a more important disease than mine, huh? Is it too risky for cancer patients?

    I have cried about this disease more than I can count. But it was not because of the disease itself, but for the insane responses and lack of compassion with how doctors treat it. I have no more tears to cry. We have no one fighting for us! Neurologists make more money if HSCT doesn’t get approved in U.S. My own neuro gets an extra 137,000 a year from drug companies for prescribing DMDs. Just look up your docs on this website https://projects.propublica.org/docdollars/ There is no incentive for neuros to support HSCT. MS Society isn’t in our corner. They are bought out by the drug companies too.

    These dmd drugs harm us. My first on wrecked my liver. 2nd allowed multiple nonstop infections and gave me bladder damage and disfunction. 3rd one allowed me to get new lesions on my brain and spinal cord.

    I’m going to Mexico in December for this. I’m not allowing this disease to win while US researchers would rather I take poison pills costing $70,000 a year for my insurance company when a one time treatment only $50,000 can STOP my disease.
    People who don’t have to face a lifetime of pain with a progressive degenerative disease like MS are getting to make the decision for quality of my life. Absurd!

    Just look at the final days of Annette Funicello to see what we would like to avoid. Put yourself in our shoes. You researchers don’t give a damn!

    • I understand your frustration, I had friends back in the 70´s screaming about the lack of AIDS drugs available and that big pharma was holding out and so on. Of course they weren´t and today, 30 years later, AIDS can be managed but not cured. Nobody has been bought out by pharma, researchers are not snobby and nobody in Mexico and Russia is curing anyone faster than the US. You´re one step away for blaming some conspiracy theory or aliens, its an age old trope. You will waste your $70,000 but that´s your choice. I have an incurable autoimmune disease and stem cell therapies will not work and won´t be optimized in my lifetime so I spend my §70,000 sailing around the world and visiting cultures that don´t even know what stem cells are, but are far richer than the bleating masses that refuse to see it as it is. That only leads to a cycle of disappointment reduced quality of life, which defeats the object.

  9. Dear Tamara:

    To share that the disease experience is certainly varied, one of my family’s dearest friends went to Mexico for a stem cell treatment for MS, received no benefit, and died several weeks later. It is still unclear if the treatment were responsible for her death. I was doubly heart-broken, because they made this decision without conferring with the one friend they had who is a stem cell biologist, me. I learned later that they made this decision not to tell me and my wife, because they were pretty sure I would have advised against it. They were right about this. But I could have also advised them that HSCT for cancer has a VERY DIFFERENT AND PROVEN TREATMENT BASIS that does not apply for its use in treating MS. In the case of cancer treatments, the HSCT is not treating the cancer. It’s treating the side effect of the cancer treatment, which is destruction of the body’s blood stem cells by the high dose chemotherapy used to eradicate the cancer. So, in this case, HSCT is replacing blood stem cells that were lost. For MS therapies that involve toxic treatments that require HSCT replacement, the issue is whether the toxic treatment can fix the the problem causing the MS (just like often the chemotherapy does not get rid of the cancer completely). These therapeutic approaches are certainly all still theoretical at best. But in many cases, as for my friend, HSCT (or some other stem cell source) is being given without any other treatment. What the HSCT might provide in this setting is based on the conjecture that preparations containing stem cells have a panacea of disease-curing properties. I can say, confidently, that this is unlikely; but I can’t say for sure that in every case it will not work without having proper studies designed to show that it can have a beneficial effect sometimes. Many of the folks providing these unapproved studies know this, too. I know it’s frustrating, but try to find yourself an authorized clinical trial offering the stem cell treatment that you seek. The ability to do this is getting better. Companies are springing up now to help with matching patients with trials at no expense to the patient. In this way, although no benefit is guaranteed, you will get a safer treatment and your sacrifice will at least contribute to improving stem cell medicine for others in the future, and not just put money in somebody’s pocket who knew they were not doing the best they could for you, which would be getting their patients into authorized clinical trials, either designed by themselves or others…and my friend might still be with us, yes still with MS, but with a better feeling about the still beautiful person that she was.

    Kindest regards,

    James

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