Navigating 4 key kinds of stem cell studies

kinds of stem cell studiesOver the years and in particular after Dr. Oz’s show a couple weeks ago, I’ve heard from many patients who are absolutely convinced that stem cell therapies do in fact work and are safe. They have been focused particularly on hematopoietic stem cell therapy (HSCT) for MS but also other investigational applications using a variety of stem cell types including adipose stem cells.

Almost without exception the kind of stem cell therapy that patients are referring to in these cases are not conclusively proven to work and be safe. In certain instances the type of stem cell therapy some patients mention are not the same kinds of stem cell therapies as what Dr. Oz and his guests were criticizing. We all need to keep in mind that there are different stages to or kinds of stem cell therapy studies.

What are the main different types of stem cell therapeutic studies? How do we tell the differences and what benefits/risks are possible with each type to patients?

First, there are preclinical studies, which can range from work done in a test tube to cells in a dish to transplants in rodents or other research animals. This kind of research can be exciting and lay the foundation for clinical work. On the flip side, some times it can be hyped as being very close to leading to a human therapy. For instance, just because a paper shows that a certain type of stem cell may have benefit in cells in a dish or in rats doesn’t mean it will do the same in people. Often it won’t. But this kind of research is an important first step even it doesn’t mean a human therapy of the same kind is near on the horizon. So for most patients, this kind of stem cell work is not the basis alone for a therapy you will get any time soon or at least it shouldn’t be, but it is worth paying attention to as part of one’s ongoing stem cell “homework”. The risk at this level is giving patients too much unsupported hope. Yes, this category could probably be subdivided into many categories (e.g. in vitro vs. in vivo, etc.), but hopefully you get the point that this is foundational work overall.

Early phase clinical trials are designed to learn more about the drug in question (yes, stem cells can be drugs) such as its pharmacological properties in vivo and especially its safety. The main question at issue here is in fact whether a therapy has adequate safety.  These studies generally will not use placebo control. When most people refer to early “clinical trials” in the US, they mean FDA-approved trials with INDs in place before even one patient gets involved. I believe this is the most appropriate definition of an early clinical trial. Patients who enroll in early trials are taking risks for the benefit of others and to advance knowledge so I think of it as somewhat of a heroic act.

A concern is whether in some instances those who will be administering the investigational therapy will conduct proper consent. Sometimes there could be, perhaps even unconsciously, some indication given to the patient that they derive some medical benefit from an early trial even though these trials aren’t designed to test efficacy and often use sub-clinical doses. Open-label studies can lead to placebo effects or other confounding outcomes. Patients should not have to pay to enroll in these.

Later phase clinical trials further test safety and now start to tell us as a community about efficacy too. The gold standard is the RCT or randomized controlled trial. In these trials, patients can be in control groups (placebo or standard of care) or receive the therapy being tested such as an investigational stem cell drug. Patients are not typically charged for these also, except under what are supposed to be very rare instances pre-approved by the FDA. If conducted rigorously and yielding successful results, in the end these trials can fairly definitively demonstrate that something is safe and effective. Even then, sometimes safety issues can arise later with more widespread marketing and use as we’ve seen with various non-biological drugs, but that’s fairly rare.

Now moving outside the FDA-approved clinical trials process for the fourth kind of experiment….

Stem cell clinic’s “trials” are a different kettle of fish stem cells. In my opinion these are designed in many cases primarily to generate income taken from patients for the benefit of the for-profit businesses running them. Sometimes those running them truly believe the offerings work. Only very rarely (I would estimate ~1% of the time) here in this domain is data ever published.

Patients have to pay to get the experimental offerings from clinics. Also, these experiments usually do not have preclinical data to strongly support them, they lack control groups, they in some cases enroll large numbers of patients for no defined scientific/medical reason, most often they are not FDA-approved, and usually do not have data released publicly or subject to objective peer review by the wider stem cell community.

Now if a stem cell clinic publishes their study data in a legit peer-reviewed journal, even if it is not data from a RCT and even if it isn’t in some fancy top-tier journal, this can be useful for the community and some credit is due. Publishing in a peer-reviewed journal is not only a valuable step scientifically and medically, but also it means that the patients taking risks by being involved in this (and paying for it) are given an additional level of respect in that the information from their participation can help others.

Bottom line

Taking a probing, critical look at the different kinds of stem cell offerings out there such as those falling into one of the four above sections discussed above, is just practicing good science and medicine. Science is all about asking tough questions.

I understand that for some individual patients it may be only human nature to want to defend that “thing” such as an experimental stem cell offering that they believe helped them, but these things have to be rigorously proven and those administering (and in some cases profiting from) as yet unproven stem cell clinical offerings should be open to answering a range of questions. This goes across the board from stem cell clinics to those running FDA-approved clinical trials with INDs — so there’s no double standard!

Kudos to Dr. Oz for stellar stem cell clinic show: time for more action

For me it’s been a wild week of grant and paper writing, grant review, going over data, and more, but finally I had a chance to watch this week’s Dr. Oz show on stem cell clinics in full last night and I give it an A+ grade.

dr-oz-stem-cells

A show producer went undercover with an MS patient to a number of stem cell clinics and let the clinic people’s words do the talking about what’s most important to the clinics: money and not patients’ well-being.

The guests on the show included actor and MS patient Montel Williams and stem cell scientist Dr. Sally Temple, the President of ISSCR. They and Dr. Oz all did great on covering this issue. A special shout-out to Sally Temple for doing the show. Not many leading scientists are willing to put themselves out there to make a difference like that.

The show combined science, medicine, and compelling personal stories together with the undercover videos to expose the stem cell clinic industry for what it actually is: an endeavor almost solely focused on making money taken from vulnerable patients. It’s an industry that collects tens of millions of dollars from patients for experimental offerings that have little-to-no data behind them. No FDA approval.

And there have been bad outcomes ranging from deaths to blindness. Tumors.

If certain stem cells work and are safe for specific medical conditions, you must prove it scientifically and medically, and you have to do that first before you start marketing it. This means putting patients before profits.

Many biotech companies are doing exactly that and there are a host of promising investigational stem cell therapies in various clinical trials. Some will be proven safe and effective, which is so exciting! Others won’t work out. We can’t know the difference in advance of getting the data, but stem cell clinics are pretending they know their stuff works and is safe.

What do I say to patients who believe that the offerings of stem cell clinics do work?

Each of us understandably place great weight on our own individual patient experiences, but the experiences of one, ten, or even many more patients don’t prove things if they aren’t studied carefully with controls and in an unbiased manner. In biomedical science we learn that often, even if we are excited about an idea/hypothesis, once we carefully study our data collected from enough properly controlled experiments and it all gets examined critically by qualified colleagues, we end up being proven wrong. Sometimes we are right. The key thing is you have to let data tell the difference rather than hope or belief.

I appreciated how Dr. Oz issued a call to action at the end for his wide audience to tackle the major problem of stem cell clinics. We all need to work together on this. There’s going to be major positive impact from the show as a starting point to more action that involves the FDA, the FTC, and other governmental agencies such as state attorney generals and medical boards.

Dr. Oz Explosive Exposé on Stem Cell Clinics Airs Tomorrow

American stem cell clinics put thousands of patients at risk each year through hawking expensive, unproven and unapproved medical interventions, and now Dr. Oz is reportedly taking them on in a new show set to air tomorrow. dr-oz

Those running the clinics have affixed the buzz phrase “stem cells” onto a whole range of stuff ranging from A (actual stem cells, but unproven) to Z (zombie cells; aka not really living cells of the stem cell variety.)

While at times in the past the Dr. Oz Show has been criticized for how it discussed unproven health interventions, from what I can tell on the stem cell front now, they are very serious about exposing how risky the stem cell clinic industry has become.

A clip of the Dr. Oz show I was able to see in last week was striking. Another clip above from the show of Montel Williams is quite intense.

I was able to get this quote from Dr. Oz himself about this situation and the show:

“These stem cell clinics are using the potential of legitimate research to take advantage of patients desperate for help. These physicians are violating not only the trust of their patients but also the law and hopefully our show will push the FDA to use its authority to shut them down.”

I’ve set my DVR to record it. If you have any interest at all in stem cells as a patient, scientist, physician, student, grant funder, science writer, FDA person, FTC person, etc., you should check this out.

Dr. Sally Temple, President of ISSCR, is also on the show. I’m told that the stem cell clinic segment will be the second half of the show.

Top 20 Stem Cell Predictions for 2017

stem cell crystal ball

Stem cell crystal ball

Each year I make a list of predictions for the stem cell and regenerative medicine field for the coming new year. Later in this post I list my top 20 stem cell predictions for 2017. In looking at my past predictions I realized this will now be my 7th year doing stem cell/regenerative medicine yearly predictions.

You can see below links to these predictions for past years, which sometimes seems rather far removed from today and in other cases strike me as strangely apropos of our times.

What will 2017 bring? Below are my top 20 predictions in no particular order except starting with a few hopeful visions for the coming year.

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The car shopping analogy for evaluating stem cell clinics

Patients contact me all the time these days asking about American stem cell clinics. The most common question boils down to “should I get a treatment at clinic X and what things should I think about in trying to make this decision?” I recommend checking out my stem cell treatment guide for patients.used-car-sales

In addition, you might consider an analogy to car shopping. I’ve found it is very helpful.

Buying a car is a much less serious decision than getting a stem cell therapy, but it is sometimes not taken as seriously from a practical point of view in terms of what goes into the decision making.

If you are going to buy a car ranging in price from say $10,000 (maybe a used Honda) up to $30,000 or even $50,000 or more for a car, you do your homework, right?

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