“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.
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.
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.
I did my HSCT in 2006. I met another person through it. We were warned of dangers and could kill us. I had three years to live. It’s a study and we were well aware of what could happen. I’m 17 years out and my friend is. She got into body building. Edss level 7. We got a second chance at life. The pain we incurred with MS was horrific and I would wish it on my worst enemy. Selma Hayek. She is not a spokes person for it. She seems to be doing better but she was like us. Her speaking on TV was very poor taste. It takes a couple years to see full effect of. That hurt us as Doctors Burts patients. She is not spokes person. He had people out further that had three years to live no medicine would it down. We are the real true proof it works. That upset me. We were never promised it would cure us but a chance we could get better. I rather od died in hospital then go 3 the max years of life living in so much agony. This stopped all of it. I still have cognitive issues. Though I can walk. Do you know how that feels being young. Fyi.. I was a model, dancer and did martial arts before getting sick.
Exercise is key to rewire brain. If, I could tell anyone that sitting waiting for things to improve won’t help. Try to stay away from stress. I ran away from my home after transplant for my safety. I also worked with Carolyn H Krause who retired in 2009. She knows my story. So Selma Hayek is not spoke person. I worked politically and also ran away from my own home. Guess what that’s way too much stress but I’m here and so grateful for Dr. Burt. So they need to talk to the people who didn’t have or was given expectations. Dr. Burt never lied to us. We designed like 30 papers knowing we could die. The fact is if I died in hospital fine but if I could help people with MS in the future. The fact so many of us did but you talk about Salem Hayek. I emailed her people about it way before she did it. Study..I’m here. We knew what we faced. Something they changed Russia and Mexico on study. Most people did get a higher edss level after Dr. Burt Study. People need to stand up. Dr Burt knew all his patients. Even passing them on tge street. That a real human being and not a fake doctor. We were not a number. This was his passion. I talk to burt what made him choose to be a immunologist? His answer was dear to his heart. So BS no one knew the risk. We were not promised anything.
My best advise Exercise rewire brain. Hopefully, people in Russia and Mexico know this. I do believe they didn’t undergo as harsh of treatment.
Though Dr. Burt team was the best ever. Start talking to his real patients that had a harder chance. FDA socks. In Europe they knew about stem cells. America has got a thing up their ass approving stuff. They have stuff to help cure cancer. I know two people who work FDA. People are dying because I guess pharmaceutical companies can give you medicine that doesn’t work. I’ve been in office a called one of Doctors out about medicine. I said, would you take it. I saw guy out front pushing and gifts for front desk. Two said, no and gave me me medicine that helped vibration. This is before transplant.
Also, Doctors write notes that they don’t tell patients. By law you can get copy of what they wrote. What they write is not really what they tell in Doctors appointment.
Maybe Carolyn H. Krause State Legislative Rep told me to write my story to help others. I inspired her.
So honestly talk to real patients not people who have lots of money. Ask, how I got through all that stress and recovered from HSCT. I did
My two daughters had the stem cell procedure, it worked on one and not the other. Five years out. I wonder how to get in touch with Dr Burk in CA. Do you know. My daughter is failing rapidly.
You can try this number.
Richard Burt, MD
10710 N Torrey Pines Rd, La Jolla, CA 92037
Scripps Clinic Torrey Pines
1-858-554-8788
He is still there.
I was also told of the dangers involved with the transplant. Dr. Burt explained everything to me before I signed the papers. I am so glad I went through Dr. Burt’s Clinical Trial. I was diagnosed with Progressive Systemic Scleroderma. Dr. Burt’s Stem Cell Transplant, 2/22_2011, saved my life. The transplant stopped the progression of the disease. 2/22/2024 is 13 years for me since transplant. Dr. Burt’s expertise in the medical field is amazing. I will be forever grateful to Dr. Burt and his Staff at Northwestern at Chicago, IL We need more doctors like Dr. Burt, his knowledge and experience speak for itself. Forever Grateful.
So happy for your positive outcome Rosemary. My son was the first patient approved for the hsct in 2001. My patient was the second. Both had intractable Crohn’s and had failed all conventional therapies. My son finished his school year so my patient went 6 weeks ahead of him but my son continues to be the longest disease free survivor of the hsct for an autoimmune disease. Coming up on 23 years. My patient sadly passed away (non transplant related) a few years into the trial but had enjoyed a complete remission of her symptoms up until her death. I’m sad to learn that the clinical trials have ended. So happy that we learned of Dr. Burt’s important work with these trials. It saved my son’s life. He is almost 40 now and while he wouldn’t want to go through it again, he still says his worst days at transplant were way better than his easiest days with Crohn’s. (5 years on chemo therapy and steroids to suppress his immune system, two central lines,TPN and NG tube feedings, fistula, pain, weight loss, malnutrition etc). It has been life changing. Best wishes to you!
I would love to speak with you about this!!
I firmly believe it was hauled not due to patient deaths or lack of him reporting his findings. It 100% believe he was close to a cure for Autoimmune and that scared the pharmaceutical and FDA because it would stop lining their pockets from our expensive medicine. I go for my infusion for MS every 5 months and the cost is 198,000$ yes you read that correctly!! How said that I can’t receive stem cell treatment that would not only help me and possibly stop my progress but also be cost effective in the long run. Our world is sad and not looking out for us only themselves and their income!! It time we take a stand and fight for us!! It very sad that I am at the point of going to a different country for stem cell treatment! The USA is corrupt beyond measure and who is to say just maybe Selma Heyac isn’t one of them trying to destroy the research and but doubt intò all that read or see you speak about this! Very sad!!
Thanks for the speculation! It caused me to do about 30min of Google research and I came up with this Dr. Burt interview from last month.
https://m.youtube.com/watch?v=Tc6s34YII9o
I went through it in 2017 and I’m still in remission. Hopefully he’ll clear up some confusion.
Let me explain something here, for anyone/everyone who may come here to read this. Dr. Burt CHANGED MEDICINE…SAVED LIVES! When this happens…sometimes, you rub some fur the wrong way. I don’t rightly care about those whom have negative stuff to say about him or what he did these past 30 YEARS PLUS (of dedicated service), I only know…he changed MY LIFE…for the better. I’m not someone who wants to be known..(I’m someone who spends my time hiding out (in many ways) and am not looking for any sort of recognition here. All I can tell you is this…Dr. Burt gave a fuck…so did his Nurses/staff…they were fucking some of the NICEST/smartest/most patient/Kindest/loving people you will EVER MEET. I’m thankful he had the balls to not quit…to see it through all the way through…past the critics….the threats to himself and his work. I only pray he would know just how much his former patients like me really do appreciate him. God Bless you, Dr. Burt…keep kicking ass.
I’m 16 half years out of Dr. Burt study. I had edss level 7 and nothing on the market my body didn’t reject. I had three years to live. He saved my life. He also very passionate about the study. He knows your name and can spot you on the street. Great doctor. We signed knowing what could happen. I wouldn’t be here if it wasn’t for him. So I agree that FDA needs to keep this going. I’m proof of it working. Since I did this, I didn’t have anyone to talk to about expectations but I hope it helps reading what I have wrote. I met a friend there who is free and clear also. We’re grateful to him and his team!!
He knows his patients and you cannot say that for most doctors.
Thank you Dr. Burt.
No. We knew ahead of time that the clinical trails were coming to an end. It was not an abrupt ending. My sister was treated by Dr Burt in 2016 w HSCT for scleroderma. He literally saved her life. Sadly, yes there were some patients that didn’t survive HSCT, or passed afterwards. Dr Burt explained the risk of death during the trial. And also explained the chances of relapse. So, his patients were well informed in advance to treatment. He was very thorough, even after my sister returned back to Kansas, he would return phone calls if needed.
@Jama,
Thanks for your comment. I know many patients feel he saved their lives, but other families didn’t have such good experiences and as you said some people even died. Also, while perhaps you knew the trials were coming to an end, many others did not know anything in advance.
I am physician. I worked with Dr. Burt in 2015. Dr. Burt was a dedicated physician. He was very compassionate. His main problem was that he was running the division alone, which was a very daunting work. Also at that time was suffering from a back pain. Dr. Burt used to work more than 10 hours a day. This explains some of delayed reporting that his clinical trials suffered from. However, the results of the trial that he reported were completely correct. I saw the patients improvement as I did some research work with Dr. Burt. Dr. Burt was very strict in his clinical work and was very dedicated to his patients.
He’s a great doctor and thank you for writing this. We knew risk. Truth is there is risk in everything but he saved so many people. He was always kind. New you name. He really put alot into this. After study he would call. He’s one in a million doctors. I’m grateful to him. I enjoyed what you had to say.
Why are certain people trying to make something sinister out of an abrupt closure of the stem cell treatment, it wasn’t that abrupt. Dr. Burt was doing stem cell transplants up until the closure date . my nephew had his done in April of 2019 and they were still taking patients up through October of 2019 . Dr. Burt is a miracle worker in my opinion.. Prior to the transplant my nephew was in and out of the hospital every two months with some paralysis and numerous balance issues he already had been given a wheelchair and a walker. since the transplant he is able to walk completely unaided and hasn’t been in the hospital since the transplant. obviously I wish doctor Burt we’re still doing the transplants for the sake of the other Ms people out there but whatever his reasons it’s not for anyone to speculate.
@Mary, It was a big, abrupt, and not really explained change in plans for him and the program. Many patients told me it was very unsettling and stressful. I am glad your nephew has done well.
Where was this a change in plans? Just saying it was a change because you didn’t know about it doesn’t make your statement accurate. I had HSCT in Dr Burt’s trial at Northwestern in 2015 and this man literally changed my life and I will be eternally grateful. I’ve followed his progress relatively closely, and was made aware well in advance of his looming sabbatical. I didn’t have any access to information that anyone didn’t have, I just took the time to look, if others didn’t, then that’s on them. I understand that it can be stressful for people hoping to have treatment, but that doesn’t make it his fault or the situation ‘suspicious’.
I’m not saying this is necessarily the case with you Paul, but a lot of what I’ve seen that has been negative about Dr Burt, honestly, seems to come from a place of jealousy or pettiness from other doctors… Just because someone has a PHD next to their name doesn’t mean they’re excluded from taking part in some of the less appealing aspects of being human, sometimes it can mean the contrary.
To the best of my knowledge, there was some major change in plans overall in that the program was shut down entirely, which clearly wasn’t the plan. The sabbatical part could have been planned. I don’t know.
After really thinking about it, I don’t have the slightest jealousy for Dr. Burt. I am not in the same field even if he did work with stem cells too. If he was successfully still pursuing HSCT for MS at NW that’d be great if it was being done right. Maybe he is or will continue that work elsewhere, and if so, I wish him good luck on that.
In my view the program was very promising and I do think many lives were likely helped or even saved in some cases, but major mistakes were made on multiple levels. I’m not convinced the program was transparent about negative outcomes and in my view things were hyped. I don’t believe some of the big decisions were in the best interest of patients.
There’s a lot more to this story, some of it very sad, that hasn’t really come out into the public domain so far as I know. I don’t know if it’s worth posting about it after all these years.
I think it’s human nature to speculate, especially since Dr. Burt had been working on this trial for 10+ years. He treats Selma Blair and then just walks away? Why?
I’m sad that this really and most promising treatment for M.S is not available to millions of people that have M.S. their bodies are failing them every day, the progression can be stopped!
Why isn’t the treatment on the market already?
I tried to get into his trial several times but I was denied, so my next option was go to Moscow Russia, which I did 2 years ago.
I had an MRI when I was in Russia and I had another MRI a year ago, my neurologist (here in the U.S. who was NOT happy about me going to Russia) said I DO NOT have new or active lesions, so it looks like HSCT did what it was supposed to do, halt the progression of M.S.
I think people are hurt and upset that the HSCT treatment hasn’t be approved by the F.D.A.
Hi Ivonna,
Thanks for sharing your story and I’m so glad that you feel chemo+HSCT helped you.
I also wonder if Dr. Burt stopping will greatly delay potential FDA approval.
Best, Paul
Dr Burt already has the right to market HSCT as an effective treatment for MS in the US, and healthcare plans in the US, including gov’t ones, have paid for many of the treatments. I think it isn’t appropriate to imply that FDA approval is a hurdle that could be or needs to be cleared.
My understanding is that under US law, HSCT treatment for MS is already a marketable treatment. A bit like an LDT (a Lab-Developed Test run by a lab accredited under or authorized by CLIA), FDA pre-marketing approval isn’t required to market it. There’s no regulatory path that Dr Burt’s HSCT treatment is required to follow that leads to an FDA ruling on approval and the FDA never denied an application to approve it. I think people are hurt and upset that there is no regulatory path to FDA approval that is designed to or has ever accommodated a treatment for a major disease like MS that doesn’t rely on still-under-patent medication (but rather, consists primarily of treatment with off-patent medications). I’m one of them! I think its important to make clear that there is no regulatory hurdle that the treatment failed to clear; that’s my point (which perhaps I’m using too many words to make).
I could be wrong, of course. I’m not saying I’ve just double-checked that I have reliable sources sufficient to prove every claim above; again, I’m expressing my understanding.
When I spoke to Dr Burt during my treatment he said that his main goal (other than to help people) was to get this treatment to a point of being covered by insurance companies, so regular people could actually afford to have it done.
I’m one Dr. Burt patients. I had ms and nothing on the market worked to slow it down. I was in the hospital every month or month 1/2. I was in so much pain.
I got excepted in trial and I’m 14 years free of MS. So Dr. Burt is great doctor and very easy to talk to. I’m not the only one. So I would like to know why they stopped it. You know the risk. I was in too much pain and it was to much tl take. I went MS Society gathering and the Dr. Dismissed my study. I walked in with high heels. I told people about my study before the guy spoke and literally took a jab at another study that I was in and worked. MS Society wanting me to be an Ambassador. I went and didn’t talk about the study there. The new young guy who took over while I left the meeting said, I hope your not telling them. I said, Montel Williams was right. I told him you make me sick. You get money from promoting. A place In Florida was smaller but sent you books, cooling vest and helped. It wasn’t a corporate greed.
So Dr. Richard Burt and the whole staff was amazing.
Your comments, assumptions and innuendo are irresponsible. We’re you just fishing for the controversy? I am per sally disgusted and think your speculation assigning negative motives is horrific.
Have you ever needed this procedure to breath and live? I have.
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
James…. link?
Sorry James but you need to do more research about HSCT. I don’t believe you have the full picture.
James, have you heard of an ‘appeal to authority’ argument? Congratulations that you’re a stem cell biologist, unfortunately, to any informed party here, you just embarrassed yourself by not knowing what the hell you’re talking about. I would suggest you read up on HSCT for autoimmune disorders, and maybe even talk to some people who’ve had it done, before you continue doing damage by spreading misinformation.
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.
Please educated yourself.
It’s the chemo that stops the MS activity. Stem cells (which are from OUR OWN body) are only used to recover our immune systems.
HSCT for MS is now being covered by insurance companies and trials are running all over the US.
I had it done and am here 6 years later on no meds, no assistive devices to walk, and Medicare covered the cost.
16 1/2 years out with edss level 7 and 3 years to live. I’m one of Dr. Burt patients. I’m grateful for procedure. It was a study and we knew risk. Having bad case of Ms and no medication to slow attacks. FDA needs to think about this more. I didn’t have hope because we didn’t know but I know others Dr. Burt patients. No one understands the horrible pain. Hospital every month for attacks. Thank you doctor Burt for my life back!!
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.
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
Hi James@Asymmetrex,
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.
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.
Hi Ivonna, Thanks for sharing your experience. Do you have any insights as to why the Chicago HSCT clinic is apparently closing and trials ending?
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
James Asymettrex , btw a great name, your assertion that one good respond can not be used as ‘predictive’ unless nul hypothesis including the failure of Aspirin to comply is ‘rather’ selfish from an individual or ‘pure researcher’ I was laughed at when I used a very small success data to try to help my wife from being attacked by an anti Yo on her cerebellum. If we had got the protocol initiated correctly as this ‘small’ data or report we knew it would have got the right result. It is high horse to say that quote of the William Russell play in Educating Rita in regards to ‘ tragedy’ for the Bugger who died because for him it was ‘tragic’ So bugger off nul hypothesis let me get that ‘bloody treatment’ Evidence Base is a tool for ‘convinience to dismiss ‘practical experience. ‘ this is your quote 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.
Regards
@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.
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, do you have a few links to peer-reviewed published papers from blinded, controlled trials using MSCs showing they are likely safe and effective?
@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.
I’m still crushed by this loss. Paul Knoepfler, I appreciate the light you’ve shone on this. Yes, there were some significant but relatively minor problems, compared to far more serious ones that receive 0 attention from the FDA. One GIANT VIRUS-ELEPHANT hybrid the FDA gives no attention: https://fdaaa.trialstracker.net : Fines claimed by US Govt $0 Fines collectible but unclaimed: US Govt could have imposed fines of at least
$8,790,588,960. That’s a minimum. @FDAAATracker #FDAAATracker @ebmdatalab Certainly not serious enough to warrant more attention than 9 billion dollars, and probably > 10^6 X more lives squandered than the total # of patients ever injured by legit stem cell therapy.
Doing hemopoietic stem cells for MS makes no sense in the first place. At least MSC’S make more sense.
No, it makes good sense because you 1st harvest, then chemo ablate the auto-reactive immune system components, and then transplant to reconstitute. Somehow the derivatives of the HSCs don’t seem to have much autoreactivity at least for a while in many patients.
Thank you for correcting Dr. Atluri. Marrow ablation makes perfect sense for autoimmune diseases. My son is Dr. Burt’s longest disease free survivor after treatment for Crohn’s in 2001. I worked in cancer research as a research nurse and our clinic conducted clinical trials. The oncologists I worked with are the very ones who told me to get my son to transplant. They all concurred that it was time to wipe out his immune system and start over. Our chief investigator found Dr. Burt’s phase I trial and within weeks, my son and I were on a plane. He found out shortly thereafter that he met the trial criteria. Absolutely life changing. And people need to understand, when you have an intractable autoimmune disease and have failed all conventional therapies, you have no life. My son said that to Dr. Burt and Dr. Oyama during informed consent. They said, “we think this could help you but our primary focus is learning from you and helping those who come after you. We have a 6% death rate so it might kill you”! My son said, “I have no life now, let’s do it”! He was 16, turned 17 at transplant. He has never regretted it! We will forever be grateful to Drs. Burt, Oyama and Craig. They were all a delight to work with throughout the trial. My son felt validated and truly cared for. I’m sorry to hear the trials were suspended.
@Dr. Atluri, where are you pulling your data from? The proof is in the data. HSCT shows better improvements in stopping the disease.
Thank you Trevor… BTW, did someone close to you just have HSCT?
My girlfriend has had MS for 10 years and failed 6 Frontline drug treatments. With limited more dangerous treatments left she was happy to be accepted into Dr Burt’s trial. Since having HSTC 2 years ago she has had zero flares and zero active lesions. I don’t know what caused NW to discontinuing it, but can tell you from personal experience it does work. Amanda is no longer on MS meds and feels better then she has in many years.
@Aaron,
I’m very glad to hear that. My sense is that the trial at NW has produced some great results, but there have been some serious problems with that HSCT program too. More to come.
There are patients of Dr. Burts on FB who are so grateful. I guess I’m one of longest who was talking to his patients. It made me smile. So hopefully 16 half years out can show proof of study working.
I was Dr. Burt’s patient. I was diagnosed with Progressive Systemic Sclerodermaround 2009-2010; a fatal disease. I had a Stem Cell Transplant February 22, 2011 that stopped the progression of the disease. I was around 62-63 years old then. Dr. Burt saved my life. February 22, 2023 will be my 12 years after Transplant. I will be 75 years young in May of 2023. Dr. Burt is an amazing doctor and an asset to his profession. He is my Hero for saving my life.