Burt Northwestern Stem Cell MS Trial Part 2: Marketing ‘Cure’-Like Outcomes?

Can science now cure MS in some patients with stem cells or is that near on the horizon? What about stem cells for other serious autoimmune disorders? What are the risks to patients in terms of health, money, and hope? What if patients must find $100,000 or $200,000 just to get into a clinical trial for this and insurance only sometimes helps in a big way? Will the trial conclude with an approved product that works and is safe enough? It’s always complicated with an ongoing clinical trial to make predictions.

Today’s 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 trial, costs and ethical as well as practical issues, and Part 3 here. The point of this blog series is to start an open, diverse discussion and exchange of information about this unusual kind of clinical trial. In Part 2, I focus here on the apparent marketing of the trial.

This NW team has made headlines with exciting, novel approaches to autoimmune disease using stem cells for more than a decade including MS. As a stem cell scientist myself, I believe there is real potential here and hope that the trial is successful in the end. While there currently is no approved stem cell therapy for MS, patients understandably have high hopes including for the MS trial and you see these hopes articulated by the patients on the web.

‘I don’t use the word cure, but…’

Overall, what exactly do patients expect specifically from the Northwestern stem cell trials for MS and other conditions? In these Northwestern trials are many patients expecting cures or at least life-changing outcomes? My sense is that the answer is often “yes” based on all the comments and feedback I’ve gotten from patients and what I’ve read online, while other patients have more moderate expectations. After Part 1 of this series some patients already enrolled in the trial told me they believed they had their life changed by the clinical trial investigational therapy. It feels as though some patients perceive themselves as cured.

A different, but also very important question follows: are patients actively led to believe they will get cures or outcomes similar to cures by the language used by Northwestern? I’m not sure, but let’s take a look at answering that question.

A few quick Google searches and you can find an assortment of information about the NW trials including videos. For instance, you can see a video above with statements from Dr. Burt, those he works with, and patient testimonials about experimental stem cell treatments in clinical trials that he is testing for a variety of conditions. It’s a surprisingly glitzy marketing video and I believe not sufficiently cautious about avoiding giving patients too much hope from a clinical trial. Richard Burt Stem Cell VideoIn fact, the video, its music, the cool flashing lights, and the words spoken make for a very aspirational experience, but what about science and medicine? Data?

The flashy video 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, a phrase that Dr. Burt also says in the video.

This big claim is probably going to be equal to a cure in the minds of many patients and such a statement may be hard to resist for those suffering. As I’ve communicated with patients with severe MS, I’ve tried to imagine myself in their shoes (some even asked me to do that) and in that hypothetical scenario a claim of “a completely normal life not using medications” would be like a magnet for me to that clinical trial, especially if I felt that standard chemical medications weren’t doing the job or had bad side effects. I’d interpret that phrase as meaning a cure or cure-like outcome being available if I were an MS patient.

But I’m not an MS patient and I don’t claim to have great insight there. I’m a skeptical scientist who has seen stem cells over-sold too many times over the years in general even as I wish for proven new stem cell therapies to come on the stage. If I had to predict, I would be optimistic that several stem cell therapies will be approved in the next decade, but where we are at today is more complicated. In part for that reason, in my view a “completely normal life” kind of statement is a questionable way of describing a still investigational stem cell therapy that may ultimately not be proven to be safe and effective for widespread use or that might work only temporarily or only work in a subset of patients.

Also included in that same video is an apparent trial patient specifically saying “I am cured” and in that context the patient is kind of like a spokesperson for the institution. Speaking of cures, on the now password-protected Northwestern stem cell immunotherapy website, which although external to the official Northwestern University web domain has its logo at the top, “Curing multiple sclerosis” jumps out at 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 from an article in The Economist about Burt’s work.  As I mentioned in Part 1 of this series, the magazine is responsible for its own title choice for an article, but why include that on a page for patients about your ongoing clinical trials? At the top left of the page a header says unequivocally, “stem cell therapies are changing lives”, which also suggests big success.

stem cells MS Northwestern University

Is all of this sufficiently cautious so as not to give patients who are considering enrolling the wrong ideas about where things stand? While I’m not a clinical trial expert, when I see an ongoing trial making bold claims it raises concerns. I don’t have direct experience with the team and as I said in Part 1, the 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 patients and those patients view the NW team as appropriately cautious so that’s important to keep in mind for balance.

The state of the clinical science including research publications

An important caveat here is that maybe the Burt team has amazing preliminary trial data that has convinced them that they can back up big claims, but if so to my knowledge that data is not published. If that data exists it should be peer reviewed and published before big claims are made publicly that patients will see. For reference, see the 2016 ISSCR stem cell guidelines that provide a helpful framework on stem cell clinical studies.

What about already published papers that might support what Burt’s team is doing? Over the years Burt has published important, novel papers on his team’s experimental stem cell transplants for MS (and other autoimmune conditions including Type I diabetes) and some gave encouraging signs of short-term improvements. I think it is fair to say he’s a leader in his field. However, the publications do not contain conclusive results and as best as I could see the data in those papers on MS are not from RCTs. Hopefully new papers with additional data will be published soon that clarify where the MS trial stands more recently.

The past Type I diabetes stem cell clinical trial work from Burt’s team is worth a look for comparison to what is going on now with the MS trial and how it is characterized.

In 2007-2010 Burt’s research made a splash of a possible revolutionary, stem cell-based approach for Type 1 diabetes from work done with pediatric patients along with a team in Brazil collaborating with Dr. Julio Voltarelli. He said back then too that he doesn’t use the word cure, but there seemed to be pubic indications of at least dramatic changes for the better in patients. There seemed to be major science hype by the media itself about this. For instance watch the ABC News video above. Also, take a look at this quote from a Time Magazine piece:

“I wouldn’t use the word cure,” says Dr. Richard Burt, one of the co-authors from Northwestern University. “But it appears we changed the natural history of the disease. It’s the first therapy for patients that leaves them treatment-free — no insulin, no immune suppression for almost five years.”

Exciting, perhaps history-making work 10 years ago using a stem cell transplant with partial immune ablation for an autoimmune condition in the form Type 1 diabetes?

Whatever happened to this line of diabetes stem cell diabetes research? I don’t know. Their 2007 and 2009 JAMA papers were exciting, but after that I can’t find anything other than a withdrawn trial clinicaltrials.gov listing. If anybody is aware of what might have led to the possible end of or long-term pause on that line of research please let us know in the comments.

Again, I hope the ongoing MS trial is proven in the end to be a success because so many desperately ill people would be helped, but again clinical trials are tricky things to predict and often do not turn out as people hope. Science is more generally that way too and we have to see what the data teach us and what our peers think of the data and their views of our interpretations of the data.

Overall, there are many potential risks associated with elevated patient expectations for an ongoing clinical trial and associated marketing, and these issues are only magnified by requiring patients to pay large sums of money for access to a trial. In Part 3 of this series I discuss additional issues including funding, the future, and patient perspectives.

A nation of stem cell miracles? CNN puff piece on clinic patient & Trump blows it

CelltexWould you believe in a stem cell miracle?

Sometimes the mainstream media stumbles in its coverage of the stem cell world and a recent CNN puff piece on a patient of stem cell clinic Celltex is a prime example of just how extreme this can get.

The CNN piece focuses on a Celltex patient who self-reports perceived big improvement after receiving the non-FDA approved offerings of the Texas stem cell clinic. Celltex is most famous for their number one customer and supporter Rick Perry, former Governor of Texas and now DOE Secretary in the Trump administration. For more background on Celltex including the warning letter it received from the FDA, you can see archived posts here.

CNN stem cells

Jacqueline Howard of CNN wrote about how the patient was invited to and attended Trump’s big speech before Congress recently. Howard mostly blew it on this article as she provided no background on the controversy involving Celltex, its past run in with the FDA, and how its offerings that it sells to patients are not FDA-approved as safe and effective. Minor details, right?

What about the fact that these kinds of treatments cost the average patient thousands of dollars, sometimes tens of thousands of dollars? Not important enough to mention?

And risks? No mention. The reality is that using adipose stem cells grown in a lab has sizable risks. Continue reading

Stem cell wish list for 2017

Last week I posted my list of 2017 predictions for the stem cell field. Today a couple of days into 2017 I’m more focused on hope than realism. What would I wish for in the stem cell and regenerative medicine arena in the coming year?stem-cell-wish-list

More stem cell clinical trial data posted and published. There are few things as exciting as stem cell and regenerative medicine clinical trials across the full spectrum of stem cell types including adult, embryonic, and IPSC. But we need to have actual trial data be peer reviewed and published or at least posted. Clinical trial updates only by press release are not helpful to patients or the field.

Clinicaltrials.gov adapts. This vital resource of trial listings adjusts to new realities. It either filters its listings to screen out for-profit entries that aren’t real trials or it provides more practically useful information such as at a minimum clear indications of whether a listing has an IND (this doesn’t need to violate any confidentiality rules) and whether the listing requires payment as an inclusion criteria. See my interview with the leader of Clinicaltrials.gov.

FDA speaks clearly. Whatever the FDA does or does not do in terms of actual stem cell & regenerative medicine-related actions in 2017, it is clear about it. This year I hope the FDA provides concrete, consistent explanations in the public domain that don’t require an FDA-ese jargon dictionary to try to understand.

FDA and its CBER center are consistent with good & bad citizens of the stem cell arena. The FDA has a tough job overall and its CBER branch focusing on biologics including stem cells has its own specific challenging task set. However, for years CBER has held different players in the stem cell arena to different rules and expectations. Paradoxically, essentially the better a citizen you are, the more the FDA expects from you. On the flip side, if you are for instance a stem cell clinic with no intention of following the rules (no BLA, no IND, no pre-IND, no expertise in stem cells, no data, etc.) CBER has historically generally left you alone.

Continue reading

Huge stem cell hype from Telegraph on new Stanford paper

There is so much stem cell hype out there that sometimes it seems like almost daily up comes a headline that is so full of hype that it could be harmful, but a new piece from The Telegraph really takes the cake.

It is entitled, “Hope of cure for arthritis, MS and diabetes as Stanford makes stem cell transplants safe”.

Just that title alone mentioning cures for a number of major diseases and the blanket statement, “makes stem cell transplants safe” is a big red flag, but the text of The Telegraph article immediately makes things worse:

“Hundreds of thousands of people could be cured of autoimmune diseases like arthritis, multiple sclerosis, diabetes and lupus after scientists discovered how to make stem cell transplants safe.”

Really? Now all stem cell transplants in humans will be safe and kajillions of people cured?

Continue reading

Stem cells on the 4th of July: plenty of fireworks lately

It’s an exciting time to be a stem cell scientist and I feel fortunate to be doing research on stem cells today what with all the developments recently and real promise for clinical impact in coming decades. There’s also unfortunately a ton of hype out there including in academia, biotech, and the stem cell clinic arena. That’s something we all need to be aware of and avoid as much as possible.

stem cell fireworks

Hype and hope are only different by one letter (y versus o), but although they are both out there it is often not easy for patients to distinguish these. The hype is flashy like fireworks of the kind we might see today. But often it is more bang than substance and is harmful to the stem cell field and patients.

There’s so much to celebrate in terms of legitimate research across all the spectrum from the lab to the clinic with stem cells. Making an effort to cut back on hype actually helps the field collectively by keeping expectations realistic (even if optimistic)  and avoids disasters like patients being harmed by hyped stem cells. When powerful people engage in hype of stem cells that is even riskier for patients and the field. I’ve been trying to challenge that.

There’s also been fireworks in the stem cell field over policy debates in the past few weeks and months. The fireworks in the debate over stem cell oversight are not fun, but this kind of disagreement may be a necessary part and sign of healthy debate of different policy ideas. Even if we do not sing a stem cell version of Kumbaya together, that’s OK. Group think and silencing of diverse voices are not helpful to put it mildly.

It’s a big, crazy stem cell world out there. Enjoy all the real fireworks this weekend as well as that in the stem cell world related to science and policy that is likely to continue throughout the rest of 2016.