I am in the unique position of being a stem cell scientist who has no commercial interest in selling therapies so I can be impartial on giving patients and consumers guidance. In today’s post I give an overview of stem cell therapy and provide an expert, unbiased guide to navigating this area. This post is also based on more than a hundred emails and countless phone calls. The questions come from people looking for authoritative information.
What’s in this post
Stem cell therapy | Stem cells can be a drug | Side effects | Only FDA approved stem cells | Clinics are risky | Not a panacea | Celebrities | Scientists | Profit motive | Clinical trials | Data | References
Stem cell therapy
I know there are thousands of people out there looking for more practical information about stem cell therapy. These folks understandably are using the Internet. They look for some clear, good info on stem cell treatments either for themselves or their loved ones. Too often the info that is out there is either wrong, partially misleading, biased, or overly complex.
In this post my goal is to address this need for trustworthy factual information and no-nonsense perspectives. I am speaking as a scientist, patient advocate and cancer survivor. The post takes the form of 10 key items listed below. They will help you guide your way through the jungle of stuff out there about stem cells.
Keep in mind this is not medical advice and you should absolutely talk to your personal doctor with whom you should make your own decisions.
Stem cells are often a type of drug & possibly stay in your body long term
Yes, stem cells can be extremely unusual drugs, but they are often drugs even if some argue they aren’t. The FDA considers them drugs in many cases. In other cases, such as the use of bone marrow stem cells used for orthopedic conditions, stem cells may not be drugs. Unlike other drugs, once a patient receives a stem cell drug, it will not necessarily simply go away like other drugs because a stem cell drug consists of living cells that often behave in unpredictable ways. What this means is if the stem cells are doing bad things your doctor has no way to stop it. You have no way to stop it. Also because stem cells are alive they can grow inside your body, move around, and change. This can be helpful or harmful, but the big point is that it is not something that is controllable or reversible.
Stem cell therapy side effects
Like any medical product, even aspirin, stem cells treatments will have side effects. Not maybe. Definitely. Our hope is the side effects will be relatively mild and so mild that you don’t even notice. Different kinds of stem cells have variable risk profiles, but if someone tells you that the stem cell treatment they are selling has no risks then that is a big red flag and I would walk away. They are either woefully lacking in knowledge or aren’t being honest with you. Sometimes clinics will either on their websites or in person acknowledge risks almost as sort of a disclaimer, but then they’ll tell more casually that there aren’t really any risks. A common statement is “The only risk is that it won’t work.” That’s false.
FDA-approved stem cell, cell, and gene therapies
If you check out my list of FDA-approved cell and gene therapies, you’ll see that nothing sold at unproven clinics has gotten an okay from the FDA. Most approved cell therapies are related to cord blood, while standard bone marrow transplant for malignancies is not on the list but is okay with the FDA. Also on the list are the combination gene and cell therapies recently approved for sickle cell disease, which was very exciting.
But otherwise there isn’t much there. For this reason be wary of clinics that say they have FDA approval or don’t need it.
Clinics are risky both inside and outside the U.S.
Whether you travel abroad or closer to home, have a knowledgeable physician inside the U.S. guiding you, preferably your primary care doctor who most often is not going to buy into hype. We have to avoid the trap of thinking that only the U.S. can offer advanced medical treatments and these days many American patients get their stem cell transplants here in the U.S. from dubious clinics.
Stem cells are not a cure-all
I am as excited as anybody about the potential of stem cells to treat a whole bunch of diseases and injuries, but they are not some kind of miracle cure for everything. When a doctor offers to inject some kind of stem cells into a patient, we just do not know at this point if it will do any good. The main exception is a bone marrow or hematopoietic stem cell transplant. In some countries stem cells have been approved for other conditions like certain aspects of Crohn’s disease and something called GvHD. You should start getting worried if the clinic tells you that one kind of stem cell such as fat, bone marrow, or amniotic stem cells can treat many different conditions. There’s no science behind that kind of claim.
Stem cells are not a panacea.
Patients often mention to me that the doctors offering stem cell treatments told them that the treatments are proven safe….or that your own stem cells cannot harm you…or that adult stem cells are harmless. I often tell people to think about how much research and how many questions they ask when looking to get a new car. You should bring at least that level of intensity (ideally much more) to getting info about stem cell transplants too as the stakes are even higher. Be skeptical. Ask many questions and if you aren’t convinced, then don’t do it.
Don’t let celebrities be your guide to medical care
The number of famous people getting stem cell treatments is increasing including sports stars and politicians. Don’t let what these folks do influence what you decide to do about your health. Just because they are famous do not believe for one minute that they are any more informed than you or your personal doctor about medical treatments or stem cells. If anything I think sometimes famous people are more reckless with their health than average people like you and me.
Reach out to scientists as a source of info
As a scientist I am always happy to hear from people outside the scientific community with questions about stem cells and other research. I can’t speak for all stem cell scientists but you might be surprised at how likely it is that if you send them a very short, clear email with one or two questions that they will respond and be helpful.
We can’t or shouldn’t offer medical advice, but we can give our perspectives on stem cell research and its clinical potential, etc. Just do not cold call scientists as you are unlikely to find them that way and even if you do, they may be cranky. Email. If they don’t reply try someone else and don’t be offended that we didn’t answer. Sometimes we may not answer because we are super busy. For instance, I often get more than 200 emails a day.
The profit motive in stem cell therapy clinics
As such they will do their best to convince you that their treatment is safe and effective. The more convincing they are then the more money they make. They may offer patient testimonials either from patients who truly believe they were helped or from people who are paid to say the treatment helped them. The bottom line is that the sellers of dubious stem cell treatments are generally in it for the big bucks. Admittedly I do think that some of these providers truly believe stem cells are helpful, but you’ll never see even them offering to give patients the cells at cost. These are instead very much for-profit operations.
Being a “clinical trial” doesn’t necessarily mean its okay
I am contacted weekly or even daily at times by patients or their families. They are rightly focused on getting information that they can trust. Many stem cell clinics call the treatments that they offer by the name “clinical trial”. That’s often misleading.
In the standard meaning of “clinical trial” the experimental therapy being tested has the FDA’s approval to be used in the study. Data support the study. Also, those doing the trial do not charge patients to be in it. You shouldn’t have to pay to be a guinea pig. I think that’s almost always going to be unethical on the part of those giving it. I often suggest that people turn to the federal website clinicaltrials.gov for information and that is still a great resource, but be aware that many pseudo-clinical trials are popping up on there that are really mostly about making money. They do not have FDA approval in many cases and there are other issues of concern. So even on that website use caution.
Ask to see data, preferably published
Before you or a loved one get a stem cell treatment, ask two key questions.
- First, is the treatment FDA approved and if not, why not?
- Second, can you please show me the data that proves your treatment is safe and effective?
See what kind of answer you get. If they demonize the FDA or invoke a plot by “big pharma” to block stem cells then that is a warning flag. If they refuse to show you data, then that is a big red warning flag. They may say it is confidential or that it is not published yet, but as a patient you have a right to see the data, assuming they have any data at all.
These facts will likely change over the coming years, but right now I think they represent reality. I know as patients we need hope, but these unapproved stem cell treatments will at best take your money for nothing, and at worst will endanger your health or that of your loved ones.
The post above is for information only and is not medical advice. All medical decisions should be made by patients in consultation with their personal physicians. The content on The Niche is informed by our Medical Advisory Board.
I note that Frequency Therapeutics are saying clinical trials are/have taken place on small molecule injection to encourage progenitor cell growth for certain conditions (Hearing loss/Multiple Sclerosis) as an alternative to stem cell treatment. Any views?
Thx😥
Well done Dr. Knoepfler, keep writing because authors like you are hard to find. Since I started reading The Niche, I have learned a lot from you.
Thank you for sharing your insight with many readers.
Viola Woodhouse