√ If You’Ve Had A Stem Cell Treatment, How Was Your Experience?

Have you had a stem cell treatment and if so, what was your experience like? (Update, please also take our poll on stem cell therapy cost).

I really value the diversity of readers on this blog from all over the world. I know we have a lot of readers who are patients and have had stem cell treatments. Every week I get emails from people asking about stem cell treatments and clinics.

I encourage you to weigh in here in the comments if you or a loved have had a stem cell treatment. What was it like? If it was positive, why did you feel that way? Same if it was negative.

√ Some Thoughts On Taking Risks In Science Medicine

Are you a risk-taker or do you do everything possible to avoid risks?

Taking risks in science is necessary, but is there a wise way to take the risks you do?

Science and medicine need a certain amount of risk and risk-takers to make transformative leaps forward.

The risks in biomedical science can be conceptual (a eureka! moment of a new hypothesis you are going to put out there) or more practical such as inventing a new device or method. As to the former, embracing a new idea that breaks with dogma can be something viewed positively by peers and can lead to cool new scientific advances, but then again it can and often is unsettling to others. What if you are wrong?

Further, being publicly outspoken about hot-button issues related to science policy is risky. It can not only have positive impact, but also risks people’s wrath as well.

Another level of risk can be devoting precious resources and time to a new, cutting edge idea or project in science or medicine. In terms of resources, for any given scientist, physician-scientist, or lab, science is often a zero-sum game at best. When you focus on a particularly risky area, that means you cannot also do some other thing that perhaps is much lower risk, but perhaps is less interesting.

Risks in science can in addition come with submitting grants. You risk rejection. You risk someone like a competitor knowing about your idea or your preliminary data. You risk submitting a grant on topic A and not on topic B, because there are only so many grants one person can submit…although sometimes it feels like a very large number.

It can also feel like us scientists must work an infinite number of hours each week, but here too there’s a zero-sum game in a sense. If you put too much time into grants, for instance, it means you are doing less of other important things like thinking (imagine having abundant time for reflection on scientific problems), reading, writing papers, mentoring (I think too many people in leadership positions spend too little time mentoring, although I’ve been very lucky with the mentors I’ve had as both a trainee and as a faculty member), etc. and you have less time for other sometimes required aspects of your job such as teaching well. Then again, too much teaching can impair one’s research as well.

In biomedical science there are also possible risks to society and to patients, but again some risk is a good thing. In my main fields of stem cell and cancer biology, over the years I’ve seen some risks now looking like they are going to pay off for patients. But other times not so much. Often patients in clinical trials don’t have any benefit especially in early phases, but even so the patients still take on risks and that’s just largely part of the reality of clinical and translational work.

Clinical trial participants are heroes in my book. We have a responsibility not to hype things, to be compassionate, and to help them be informed. In the bigger picture, whether in academic or industry, risk taking in science and medicine must minimize dangers to patients and help patients understand those risks. Whoever is doing it, biomedical risk taking shouldn’t be primarily driven by a desire for profit and it shouldn’t mean dumping the risks on others like patients.

On the flip side, just playing it safe in every way may be less safe than you think. There are risks to just hiding out in one’s lab as a scientist with your head down metaphorically or not…perhaps plugging away on one safe project. There are risks to not “thinking big” in terms of hypotheses.

There are also risks to not speaking out about problems in the field too such as in our stem cell field.

So where’s the sweet spot for risk in all this?

It’s going to vary for each person and their career stage. And no matter what you do or the focus of your efforts you’re going to have to work your butt off in science to succeed. Still, it’s worth some conscious thought as to the risks you are (or at not) thinking are wise to take…instead of proceeding on autopilot.

What’s the biggest risk(s) you ever took in science? Did it pay off? I’d be curious to hear about it. I’m thinking about my own answer to this question and may post on it. Certainly doing this blog over the year has been a risk.

Sumber aciknadzirah.blogspot.com

√ Patient’S Powerful Story On Multiple Sclerosis And Getting Experimental Stem Cells

Caroline Wyatt, BBC, photo from Twitter

Multiple sclerosis (MS) can be an extremely debilitating disease that ultimately is fatal in some patients and reduces life expectancy and quality overall substantially. An MS patient named Caroline Wyatt, who works at the BBC, had the courage to open up in full detail on the BBC about having MS, her experience getting an experimental treatment of stem cells in Mexico, and how she is doing now. It’s a powerful new piece that brings home the complexities of having MS and of getting this hopeful, but risky stem cell therapy.

While some patients’ forms of MS are manageable, Wyatt’s MS was extreme. It was severely affecting her health as well as her life overall and currently available approaches such as the standard of care drugs did not help. It’s understandable then that she was considering various other options including things that aren’t yet approved such as stem cells. I’ve written extensively about stem cells for MS in the past on this blog.

There’s a lot of hope and hype out there on stem cells for MS. It’s a complicated mixture to navigate to have a clear sense of weighing risks versus benefits. Wyatt did some research and was interested in a new approach to MS with stem cells whereby chemotherapy is used to eliminate the autoreactive immune system and in a sense “reboot” the immune system with putting back in earlier harvested hematopoietic stem cells (HSC).

She wasn’t eligible for participation in formal clinical trials of this new approach in the US or UK, but a place in Mexico is doing what seems to be very much the same kind of clinical experiment, apparently with different inclusion/exclusion criteria. Wyatt was able to get this HSC transplant (HSCT) at a place called Clinica Ruiz in Puebla, Mexico.

(Note, that while Wyatt did not have any bad experiences in Mexico with the HSCT procedure itself, getting an unproven stem cell treatment outside of one’s home country more generally is often likely to be a high-risk thing to do for various conditions. One patient who got a stem cell treatment in Mexico as well as in a couple other international locales ended up with a tumor on his spine. Getting unproven stem cells in the U.S. can also be risky and patients have been hurt including a number who were blinded)

Getting back to the HSCT for MS specifically, it is a very difficult thing to go through for patients with multiple rounds of chemo, stem cell isolation, stem cell infusion, and recovery from the procedure itself. I recommend reading her whole article as it is very interesting and informative about the process. Wyatt is also so open about her experiences with MS as well as what she went through in the process and since.

Wyatt says that she’s not sure if she’s had a net benefit or not from getting the HSCT. It is clear that there are long-lasting side effects and challenges from her HSCT, but there is also hope. For instance, she wrote that her latest brain scan shows no further progression of the disease. Other patients have self-reported a range of experiences after this kind of HSCT for MS. Some patients fared worse (a few have died) and others reported more positive outcomes than Wyatt.

Wyatt described where she’s at post-transplant this way:

“Today, I quite often feel worse than I did before HSCT.

I still need to rest frequently during the day, and when I use my energy for work, I have none left for anything else at all.

But there are sunnier days when I feel a little better than I did immediately before the treatment, and then my hopes soar.”

And looking to the future:

“But I am an optimist, and shall remain so while my immune system finishes reconstituting itself fully by the end of this year, some two years after I started treatment.”

I wish Wyatt all the best. You can follow her on Twitter here:@CarolineWyatt.

At this point for HSCT for MS overall, the jury is still out on whether this kind of new approach will definitely be safe enough and effective beyond the standard of care. A recent report from a meeting brings new hope and we can anticipate a published, peer-reviewed paper on the trial perhaps later this year or in 2019. At that point we’ll have a much better understanding of the big picture.

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√ Update On Stem Cell Treatment Cost For 2018 From Ongoing Poll

I get asked many questions about stem cell therapies, but one of the most common over the years has been about the stem cell treatment cost. For instance, a reporter might ask, “How much does a stem cell treatment for MS cost?” and a patient might ask me, “How much is a fair cost for a stem cell therapy for arthritis?” Or, patients will voluntarily tell me what they paid or mention it in the comments. We hear various numbers thrown around about costs so I decided to do a poll on this. I even did an early update on the results of this poll, voicing my skepticism that the costs paid were worth it.

But the poll has gotten well over 500 responses now so I thought I would revisit it and what it might mean.

You can see a screenshot of the images. It’s fair to say, as much as Internet polls aren’t considered particularly accurate, that this one largely fits with what is reported “out in the field”.

(On a side note, I wish there was such a thing as going out into the field for stem cell scientists as I’ve always been a bit jealous of scientists who really do go out in the field. What do we do, go out in the wild and catch wild or feral stem cells in the bush?)

Patients self-reported most often paying between $2,500 and $7,500 for their stem cell therapy so if we take the average of those we get that $5,000 figure that is what I hear most often from others. Yes, not necessarily very rigorous, but the result makes good sense. Not far behind though were responses in the $7,500-20,000 range.

About 1 in 10 respondents reported paying $20,000 or more, including some beyond $100,000.  That’s a whopping stem cell treatment cost, especially for something most often unproven and unapproved by the FDA.

If we consider these responses, the average cost may be more like $7,500-$10,000.

Notably, about 1/16 respondents indicated their stem cells were free. I’m not sure what that means in terms of how that came to be.

Interestingly, most respondents who also went on to answer a 2nd poll in that post about where they got the treatment indicate it was at a stem cell clinic (scroll down in that Oct. 2017 post and you’ll see the 2nd poll). This 2nd poll has about 200 responses.

So today buying a simple stem cell “treatment”, most often unproven and non-FDA approved, is often not so different in cost than buying a 10-year old used car, while less often it is similar to buy various new cars including at the high end of stem cell therapy cost, some very expensive new cars. This cost and the risks involved are why I have suggested to patients in the past to be assertive when considering a stem cell treatment, ask questions, don’t just accept too good to be true kinds of answers, etc. In short, be at least (or ideally much more) rigorous about unproven stem cell treatments as you are about buying a car.

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√ 15 Still Unanswered Interview Questions For Stem Cells For Vision Scots Trial Docs

There’s a lot going on in Florida with non-FDA approved stem cell offerings even just related to stem cells for vision problems alone, but while US Stem Cell has drawn the most attention related to patient allegations of severe vision loss in Florida after fat stem cell injections in eyes, another group I’ll loosely call the “SCOTS Trial” has also been injecting investigational stem cells (in this case from bone marrow) into patients’ eyes and some patients have made allegations of harm there too in the form of vision loss.

I’ve covered the SCOTS Trial before including those patient allegations and open questions. One particular patient, George Gibson (see image at right below), may have been the first to raise concerns about SCOTS in 2016, and both he and others claim adverse outcomes. Reportedly, eye expert Dr. Thomas Albini examined Gibson and speculated on a link between the eye injections and some retinal scarring:

“When his vision didn’t improve, he returned to South Florida to seek help from Dr. Thomas Albini at the Bascom Palmer Eye Institute at the University of Miami. Albini said he noticed scar tissue on one of Gibson’s retinas.

The extent of the scarring, and how quickly it happened, led Albini to believe it was from the stem cell injections. “When there is a novel treatment, we assume [any problems are] related to the procedure until proven otherwise,” said Albini.”

Overall, SCOTS disputes the patient allegations.

It’s hard for those of us not directly involved to fully understand this situation.

George Gibson, SCOTS trial patient, screenshot of Sun Sentinel video

Early in 2018 I invited the SCOTS Trial physicians, Drs. Jeffrey Weiss and Steven Levy, to do a Q&A interview here on The Niche to try to clarify things. My impression was that they seemed generally receptive to the interview idea by email, but after sending them the 15 questions (which in one email they referred to as “reasonable” questions) in January and emailing a few polite reminders, I still don’t have any answers to the questions from them yet after about 4 months.

Maybe the answers will come, but I thought I’d post the questions now as a way to further the discussion on the SCOTS Trial and the larger issues that relate to non-FDA approved stem cell therapies for which patients must pay. Note that the SCOTS vision trials are run by an organization called MD stem cells.

Importantly, for background keep in mind that not all stem cell injections require full FDA pre-market drug approval. For example, it seems that autologous use of unmodified bone marrow stem cells for many kinds of orthopedic conditions is generally viewed as compliant even without FDA drug-level approval as it is viewed as homologous use most often. I have my doubts as to whether even such compliant offerings are reproducibly effective, but they don’t seem against FDA rules.

Other uses of bone marrow stem cells that might be or clearly are non-homologous in theory could often trigger an IND-requirement, but to me the FDA’s new guidances on this point are not entirely clear. For instance, does the SCOTS trial’s use of bone marrow stem cells injected into the eye for a variety of vision-related conditions in each case meet the homologous use standard? If not, then they would likely need an IND prior to doing this work. In my opinion as a stem cell biologist, it seems that bone marrow stem cells in most cases are not homologous to the specific cells in the eye such as photoreceptors that are lost in some of the most common eye diseases such as macular degeneration. However, SCOTS says it is FDA compliant. It would be helpful if FDA was more concrete with guidance about potential homologous vs. non-homologous use of autologous bone marrow stem cells.

Here are the questions that I sent the SCOTS Trial docs.

• 1. How did you both first get interested in and involved in stem cell research?


• 2. Do either of you have specific pembinaan in stem cells or transplantation biology?


• 3. What is the rationale for using bone marrow stem cells to treat vision loss? If, as is widely believed, hematopoietic cells cannot become cells of the eye, what specifically would the transplanted cells do that is helpful to the eye? In other words, where would the benefit/efficacy come from in terms of mechanisms?


• 4. What are the risks to injecting bone marrow cells into the eye?


• 5. Have you conducted pre-clinical studies in animals to assess safety and efficacy of your approach prior to using it in human patients? If not, why not? If yes, do you have published or unpublished data on that?


• 6. You have at times reported no adverse events in SCOTS, but some patients who participated in SCOTS have chosen to publicly self-report negative outcomes that they believe they experienced and in at least one case reported by the Sun Sentinel an independent physician Dr. Albini examined a SCOTS study subject and Albini is quoted as attributing the scarring likely to the transplanted stem cells. How should those of us in the stem cell field who are not involved in SCOTS reconcile these differences?


• 7. That Sun Sentinel article also raised questions over whether some of your patients were properly consented (http://www.sun-sentinel.com/health/fl-fea-florida-stem-cell-clinics-20171130-story.html). Can you please clarify that?


• 8. Do you have control subjects (e.g. matched, but who do not receive the stem cells) in your trials so you can rigorously assess the outcomes specifically related to the stem cell injections?


• 9. There have been some reports that you do bilateral transplantation of stem cells into both eyes. What is the rationale for that? Doesn’t that raise risk when using an investigational therapy?


• 10. It appears that to participate in SCOTS, patients must pay as much as $20,000 each to enroll. In general, there are bioethical concerns and debate over patients having to pay for enrollment in investigational trials. What’s your rationale for requiring payment? How did you determine the amount that patients pay?


• 11. In a trial that requires payment, what steps do you take to avoid that financial aspect introducing bias into the trial? For instance, do you have third parties who have no financial interest in the outcome of the trial be the ones who determine patient outcomes? Do you plan double-blinded studies in the future?


• 12. Do you have an FDA IND and/or BLA? If not, why not? Do you view your stem cell product in the manner you use it as not being a drug requiring pre-market approval by the FDA? If so, why?


• 13. Have you communicated with the FDA about your trials in the past and if so, how did that go? Are you in regular communication with the agency at this point? Has the FDA visited you and/or SCOTS locations?


• 14. How do you view recent FDA selesai and draft guidances on homologous use and minimal manipulation of stem cells?


• 15. What’s your view of the hundreds of “stem cell clinics” across the U.S. today selling a whole range of stem cell types for dozens of medical conditions? What makes SCOTS/SCOTS2 different in your view?

Sumber aciknadzirah.blogspot.com

√ Arthritis Patient With Pain’S Email On Stem Cell Clinic My Answer

Example of a joint (knee in this unrelated case) with osteoarthritis evident by x-ray.

Stem cells for arthritis and pain are hot topics these days and I’m getting an increasing number of patients reaching out with questions and sharing their situations related to arthritis or pain (or usually both together). I asked one arthritis patient who reached out to me if they would be OK with me sharing their email, with all identifying information removed, along with my answer to them. The point was to use this as example that would be helpful to the community. They agreed. Below you’ll see their slightly edited and redacted email and my answer.

The patient email:

Hi Paul,

I am in my fifties and was scheduled for a Stem Cell Therapy procedure here in —–, CA at —– Therapy with Dr. —–. I cancelled my appointment today because I was afraid of any risks in my future health. I didn’t really research it. I was just desperate to be out of pain due to an injury that has become inflamed in the joint, has loss of cartilage, and now has osteoarthritis degeneration. I was given a book of testimonies but when I was signing the consent forms and release of liability, I became suspicious of it being experimental and not approved by the FDA. Can you shed any light on the liposuction that extracts stem cells from your own fat in the belly and then injected back into the joint? Dr. —– would be in charge of injecting it in the joint after the extraction. He works at —–.

After cancelling my appointment, the receptionist said I should have done my research before making my decision and directed me to Cell Surgical Network. She said Dr. —– is affiliated with that organization. But I could not find how to maneuver through this site. I was supposed to then go to Stem cell resolution(or revelation) Not sure which one. I didn’t find anything. I was truly afraid to go through with it in ignorance. Can you please help me settle my wonderings? I want to know is this safe or not? Will it help or not? Am I foolish or not? Am I passing up an opportunity to be pain free or jeopardizing my health?

Thank you for your time, Paul.

And my answer:

Hi —–,

These are good questions and I understand where you are coming from with the pain.

I personally wouldn’t get a fat stem cell treatment for a variety of reasons. While these cells are unlikely to produce tumors, there are some risks particularly from the systemic injection into the blood. Although the risks probably aren’t high for each individual patient, they aren’t very well understood and aren’t zero. 

Beyond the safety question, a big reason in my opinion to not do it is that there isn’t good evidence that this stuff really works so I think in the big picture that the fat stem cells sold by the various clinics around the U.S. are going to be a waste of hard-earned money. 

I also tell people: At a minimum give it as much thought as you could before buying a car — would you buy a car if you didn’t know it would work? If there was no information on MPG, no warranty, and no crash safety data at all? 

You mentioned the FDA and also a group called Cell Surgical Network. Check out this recent FDA action via the Department of Justice in federal court (scroll down about half way): https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm607257.htm. For me this is another reason to pause.

My best wishes to you,

Paul

Sumber aciknadzirah.blogspot.com