Can science now cure MS in some patients along with stem cells or is that near on the horizon? What about originate cells for other serious autoimmune disorders? What are the dangers to patients in terms of health, money, and hope? What if patients must find $100, 000 or one-hundred dollar, 000 just to get into a clinical trial for this and insurance policy only sometimes helps in a big way? Will the test conclude with an approved product that works and is safe sufficient? It’ s always complicated with an ongoing clinical demo to make predictions.
Today’ s i9000 post is Part 2 of a three-part series on the Dr . Richard Burt stem cell trials including for MS at Northwestern. You can read Part 1 here where I discussed the big picture of the test, costs and ethical as well as practical issues. The purpose of this blog series is to start an open, diverse debate and exchange of information about this unusual kind of clinical demo. In Part 2, I focus here on the apparent marketing and advertising of the trial.
This NW group has made headlines with exciting, novel approaches to autoimmune illness using stem cells for more than a decade including MICROSOFT. As a stem cell scientist myself, I believe there is true potential here and hope that the trial is successful eventually. While there currently is no approved stem cell treatment for MS, patients understandably have high hopes which includes for the MS trial and you see these hopes articulated by the patients on the web.
‘ I don’ t use the word remedy, but… ’
General, what exactly do patients expect specifically from the Northwestern originate cell trials for MS and other conditions? During these Northwestern trials are many patients expecting cures or at least life-changing outcomes? My sense is that the answer can be often “ yes” based on all the comments and suggestions I’ ve gotten from patients and what I’ ve read online, while other patients have more moderate anticipations. After Part 1 of this series some sufferers already enrolled in the trial told me they believed these people had their life changed by the clinical trial investigational therapy. It feels as though some patients perceive themselves because cured.
A different, but also very important query follows: are patients actively led to believe they will obtain cures or outcomes similar to cures by the language utilized by Northwestern? I’ m not sure, but let’ s have a look at answering that question.
A number of quick Google searches and you can find an assortment of information about the particular NW trials including videos. For instance, you can see a video over with statements from Dr . Burt, those he works together with, and patient testimonials about experimental stem cell remedies in clinical trials that he is testing for a selection of conditions. It’ s a surprisingly glitzy marketing movie and I believe not sufficiently cautious about avoiding giving individuals too much hope from a clinical trial. In fact , it, its music, the cool flashing lights, and the terms spoken make for a very aspirational experience, but what about technology and medicine? Data?
The flashy video clip also makes specific, perhaps still as yet unproven medical claims. Most dramatically, the video claims “ a completely normal life not using medications” (see screenshot) is possible from the trial, the phrase that Dr . Burt also says in the movie.
This big claim is probably going to end up being equal to a cure in the minds of many patients and such the statement may be hard to resist for those suffering. Since I’ ve communicated with patients with severe MICROSOFT, I’ ve tried to imagine myself in their shoes (some even asked me to do that) and in that theoretical scenario a claim of “ a completely normal lifestyle not using medications” would be like a magnet for me to that will clinical trial, especially if I felt that standard chemical substance medications weren’ t doing the job or had bad side effects. I’ d interpret that phrase as meaning a cure or even cure-like outcome being available if I were an MICROSOFT patient.
But I’ m not an MICROSOFT patient and I don’ t claim to have great understanding there. I’ m a skeptical scientist who has observed stem cells over-sold too many times over the years in general even while I wish for proven new stem cell therapies ahead on the stage. If I had to predict, I would be positive that several stem cell therapies will be approved within the next decade, but where we are at today much more complicated. In part for that reason, in my view a “ not out of the ordinary life” kind of statement is a questionable way of describing the still investigational stem cell therapy that may ultimately not have to get proven to be safe and effective for widespread use or that might function only temporarily or only work in a subset associated with patients.
Also included in that same video clip is an apparent trial patient specifically saying “ We are cured” and in that context the patient is similar to a spokesperson for the institution. Speaking of cures, on the now password-protected Northwestern stem cell immunotherapy web site, which although external to the official Northwestern University internet domain has its logo at the top, “ Curing multiple sclerosis” jumps out on the visitor to the site right there in the middle of the page. It’ s there without even a question mark in the form of an image through an article in The Economist about Burt’ s work. As I mentioned in Part 1 of this series, the journal is responsible for its own title choice for an article, but precisely why include that on a page for patients about your own ongoing clinical trials? At the top left of the page the header says unequivocally, “ stem cell therapies are usually changing lives”, which also suggests big success.
Is all of the sufficiently cautious so as not to give patients who are thinking of enrolling the wrong ideas about where things stand? Whilst I’ m not a clinical trial expert, once i see an ongoing trial making bold claims it increases concerns. I don’ t have direct experience of the team and as I said in Part 1, the particular NW folks seem unwilling to have a dialogue on this. Note that some patients have indicated to me that the Burt team does not mention a cure in person as they talk with sufferers and those patients view the NW team as appropriately careful so that’ s important to keep in mind meant for balance.
The state associated with the clinical science including research publications
An important caveat here is that maybe the particular Burt team has amazing preliminary trial data which has convinced them that they can back up big claims, but if to my knowledge that data is not published. If that will data exists it should be peer reviewed and published before big claims are made openly that patients will see. For reference, view the 2016 ISSCR stem cell guidelines that offer a helpful framework on stem cell clinical research.
What about already published papers that might assistance what Burt’ s team is doing? Through the years Burt has published important, novel papers in the team’ s experimental stem cell transplants for MICROSOFT (and other autoimmune conditions including Type I diabetes) and some gave encouraging signs of short-term improvements. I think it really is fair to say he’ s a leader in his field. Nevertheless , the publications do not contain conclusive results and as greatest as I could see the data in those papers upon MS are not from RCTs. Hopefully new papers along with additional data will be published soon that clarify in which the MS trial stands more recently.
The past Type I diabetes stem cell clinical trial function from Burt’ s team is worth a look for evaluation to what is going on now with the MS trial and how it really is characterized.
In 2007-2010 Burt’ s research made a splash of a possible groundbreaking, stem cell-based approach for Type 1 diabetes through work done with pediatric patients along with a team in Brazilian collaborating with Dr . Julio Voltarelli. He said in those days too that he doesn’ t use the word cure, yet there seemed to be pubic indications of at least dramatic modifications for the better in patients. There seemed to be major technology hype by the media itself about this. For instance watch the particular ABC News video above. Also, take a look at this quotation from a Time Magazine piece :
“ I wouldn’ t use the term cure, ” says Dr . Richard Burt, one of the co-authors from Northwestern University. “ But it appears we transformed the natural history of the disease. It’ s the first treatment for patients that leaves them treatment-free — simply no insulin, no immune suppression for almost five years. ”
Exciting, perhaps history-making work ten years ago using a stem cell transplant with partial immune system ablation for an autoimmune condition in the form Type one diabetes?
Whatever happened to this line of diabetes stem cell diabetes research? I don’ t understand. Their 2007 and 2009 JAMA papers were exciting, but from then on I can’ t find anything other than a withdrawn trial clinicaltrials. gov listing . If anybody is aware of what might have led to the achievable end of or long-term pause on that type of research please let us know in the comments.
Once again, I hope the ongoing MS trial is confirmed in the end to be a success because so many desperately ill individuals would be helped, but again clinical trials are tricky items to predict and often do not turn out as people hope. Technology is more generally that way too and we have to see the actual data teach us and what our peers think of the information and their views of our interpretations of the data.
Overall, there are many potential risks associated with elevated individual expectations for an ongoing clinical trial and associated advertising, and these issues are only magnified by requiring patients to pay for large sums of money for access to a trial. In Part 3 of this series I’ ll talk about additional issues including comparisons to other MS stem cellular trial work, hopefully include some patient perspectives through quotes, and look to the future.