Today’ s post is the last of a three-part collection on the Burt stem cell trials at Northwestern. This particular piece is focused on funding and the future of the demo, and also includes perspectives from patients.
You can read Part one here and Part 2 here, which focused on issues related to potential support of patient fundraising that unintentionally releases private information and concerns over marketing of the ongoing trial as already known to be something like curative for MS. I’ ve summarized my overall concerns in a flow chart beneath.
Should individuals ever be required to scrape together one or two hundred 1000 dollars to pay to get enrolled in any clinical trial? We need to keep in mind that in such trials patients have no guarantee of any kind of benefits and could be harmed because the patients are analysis subjects. And what happens to the potentially large number of patients who have cannot obtain that kind of money? They are excluded basically on that basis?
There may be certain situations where patients paying for a trial can be a responsible, positive matter. However , this is new territory overall for stem cellular clinical research and there are sizable risks to this various kind of approach as compared to the past and to some other more traditional scientific experiments when sometimes patients were the ones receiving some sort of payment. As I discussed earlier in this series, extremely cautious planning is needed to address bioethical and practical issues that might arise and anticipated risks to patients in such studies where patients have to contribute monetarily.
A few patients have said on the Internet that they are required to pay as much as $150, 000 for participation in the Northwestern MS stem cell trial or in parallel off-study experimental administration of experimental stem cells. Whenever we estimate that about 100 patients have been required to spend (one way or another, such as themselves or via insurance policy or via online fundraising) on average $100, 000 then that is in total $10 million for Northwestern University. Not really a small sum, but clinical trials are expensive. If there were 200 patients paying that much on average the total number can swell to $20 million. The big money involved the following is just one more reason that NW and its IRB need to be extra careful in how it oversees this particular trial.
Do trial participants get any type of discounted price on medical tests and procedures? Does the college cover all the research-specific (versus treatment) costs itself? Just how much does insurance pay for the average participant? How much does philanthropy by NW help the average patient? These same questions might be asked of any other trials that require patient payments too, but there seems to be little if any information that I could find within the public domain on other stem cell IND-based trials that need large patient payments.
One more issue here with the NW work is that as best when i can tell the Burt team has no current NIH funding for this clinical trial work. As many of us understand first-hand, getting NIH funding is really tough these days, yet has his team tried for funding recently? The idea takes a lot of work to get the funding and multiple offer submissions, but it is worth going for it. If the team’ ersus unpublished data is truly groundbreaking then perhaps they have a good chance of getting the funding.
Obtaining NIH funding in this case would shift some of the great economic burden off of the patients and provide another level of peer evaluation. There has been recent NIH-funded stem cell clinical trial study for MS and additional autoimmune diseases by others ( e. g. see here and screenshot above), which to my knowledge did not require large affected person payments, but if I’ m wrong about that last stage on charging please let me know. The point is that in theory a minimum of it is possible to get NIH funding for this kind of clinical test work and that should take some burden off of sufferers.
The right way to view this clinical trial study in the big image?
Quite a few individuals apparently believe Burt saved their lives, but with the particular trial still ongoing, how sure overall can all of us be about broader efficacy and safety? How will this particular experiment end up comparing to standard of care general when the data are objectively analyzed and peer evaluated in the end? What about longer-term potential positive and negative final results? In that regard, will benefits last long term for the typical patient and could they face some longer-term risks?
With all the complicated aspects to this trial including the charging patients big fees, its promotion of what exactly is offered to prospective participants as potentially already known to be healing, its nebulous status in between an experiment and a therapy, unusual large number of off-study patients, crossover of probably nearly all control subjects who know and expect beforehand to be crossed over later, open label status, and much more issues, will the data in the end yield concrete conclusions? Can some of these things be confounding variables? How convinced can reviewers be? Will journal reviewers even be aware of problems? Maybe not. The bigger question is how the medical and scientific neighborhood will view this trial long term.
I’ ve asked readers of this blog including sufferers for comments and here is one from a past recipient of come cells from Burt’ s trial:
“ Imagine you are a patient with an incurable degenerative autoimmune disease. You’ ve taken most of the drugs (Avonex, Betaseron, Aubagio, Tecfidera, and Gilenya) and you’ re nevertheless showing disease progression every time you get an MRI. A brand new radical interventional treatment (not a new drug that will jack shit) becomes available and is already in phase 3. In varying forms it is being administered in several locations all over the world, with extremely promising results. According to expert reviewed literature, it has a higher chance 5 years from preventing relapses than any other treatment. These statistics are usually reasonably consistent across the multiple centers. It might not function, but if it buys you time, then maybe something different will be on the market or an updated treatment will be offered. In the meantime, you may have slowed or even halted progression, for a minimum of a little while.What would you do? ”
As I replied to this patient, honestly I might consider all options if I was in that situation being a patient myself. But as a biomedical scientist I also need to ask myself what I would do hypothetically on the flip side being an investigator considering doing this trial.
I also obtained a comment from another Burt trial patient:
“ Dr Burt completely turned my entire life around. I used to find it impossible to even perform boring tasks such as buttoning up my shirt or strolling without support. Normal medication did not work. The treatment nearly completely reversed this. Dr Burt did not promise a remedy. On the contrary, he said at best this will arrest the condition. This individual also clearly stated the risks. I believe the treatment has assisted hundreds. ”
These patient points of views are helpful and important.
Burt MS stem cell trials as compared to stem cell clinics
Returning to the past challenge given me by some other patients/readers of this blog in the past (see the beginning of Part 1 for more on that challenge), overall what Burt is doing is quite different from the come cell clinic approach. His team seems focused on information and rigorous science. They also work with the FDA and also have regularly published their findings in strong peer-reviewed periodicals in the past. Still, my sense is that there are some concerning issues here even if the trial technically has FOOD AND DRUG ADMINISTRATION permission for much of this and even if this is nearly 100% different than what most stem cell clinics perform.
What would be useful moving forward?
As a start, Northwestern and Dr . Burt should revisit how this demo has been handled. I think they should also be more transparent regarding answering questions with so much at stake in a high-risk, higher reward kind of trial. They should shift more financial problem off of patients, they should consult a bioethicist if they haven’ t already, and they should greatly tone down or even entirely stop what I see as their trial marketing associated with something akin to a cure. As to that last point, taking down all the Internet videos that have possible marketing factors in them might be worth serious consideration. The fact that the NW Immunotherapy stem cell trial website that had a few marketing-like material is no longer publicly accessible (you need a security password now) might be a first step in this kind of direction (contrary in order to how I originally saw its removal from the public domain being a hit to transparency), but we don’ t understand if the team is still allowing prospective trial participants to see the site via a password.
I’ m unsure what NW or its IRB should (or at this time even could) do about the enormous number of their test patients doing public fundraising for the trial on Gofundme and in the process in many cases releasing their private information to the entire world or making medical claims about the trial that they’ ve heard. But that situation is very concerning as well as the fact that many if not most of them were potentially encouraged to get this done fundraising by NW itself raises thorny bioethical queries.
To be clear this type of research is very encouraging. My hope looking to the future at a medical level is the fact that this trial and others like it run by additional groups ultimately prove the new investigational stem cell-based therapies possess a sufficient safety profile and have clear efficacy from extra work including RCTs so that they can help the patients along with severe MS and other autoimmune conditions who are in eager need. The current medications available are just not working and/or have got incredibly bad side effects for a substantial number of patients.
Bigger picture: the new model of trials by which patients pay?
At a wider level we should be asking ourselves, “ how many other groups are usually conducting similarly structured trials? ” Is it possible how the Burt trial is not that unusual and we mostly just don’ t know about the many other trials that also participate in these kinds of practices? One that drew attention this past year is the so-called “ young blood” age reversing trial for which patients must pay $8, 000 to sign up, but what about others?
Is this kind of test model going to become much more common in coming many years? If so, it requires far more discussion in advance about the risks along with bioethical and practical considerations needed for protecting patients upon many levels. The Burt trial is a case study that depending on one’ s perspective either encourages more groups to follow this model or discourages others from subsequent suit. We’ ll see in coming years exactly how it plays out overall.
Last note: It’ s tough for me to do a blog post collection like this. The somewhat concerning information that I found after patients challenged me to learn more, in combination with the recent FOOD AND DRUG ADMINISTRATION warning letter to Burt, are together what motivated me to finally post this series. Some people highly disagree with my perspectives here. In fact , some are already letting me have it on the web since I started posting this course. That’ s no picnic for me. However , I understand that will in many cases they have gone through a lot, they have strong perspectives to talk about, and possess more direct knowledge of what patients go through therefore i don’ t take it personally. In this time of big modify in clinical trial practices, more discussion of how scientific trials and stem cell-based clinical trials specifically are made and managed is needed, and more open, diverse dialogue are unable to help but be beneficial to patients and the field over time. For these reasons, I decided to go ahead with this series.