Patients guide to treatments

Top 10 list of important, easy to understand facts for patients about stem cell treatments

For better or worse, I am in the unique position of being a stem cell scientist and also a patient. Looking on the bright side this gives me a unique perspective on things.

I know there are thousands of people out there looking for more practical information about stem cell therapies and treatments. These folks understandably are using the Internet to 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, misleading, or overly complex.

So in this post I want to address this need speaking as a scientist, patient advocate and cancer survivor in the form of 10 key facts to help you guide your way through the jungle of stuff out there about stem cells.

1) Stem cells are essentially a type of drug or biological and possibly permanent in nature. Yeah, they are extremely unusual drugs, but they are drugs. The FDA considers them 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.

2) 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.

3) The only stem cell treatment explicitly approved by the FDA for use in the U.S. is bone marrow transplantation. What this means is that any other stem cell treatment you see advertised on Facebook or Google or elsewhere that indicates it will be given to you inside the U.S. may in fact be illegal and unsafe. The exception to this is if it is part of an FDA-approved clinical trial.

4) If you venture outside the U.S. for a stem cell treatment, use extra caution and have a knowledgeable physician inside the U.S. guiding you. We have to avoid the trap of thinking that only the U.S. can offer advanced medical treatments, but on the other hand within the U.S. you have the added safety of the FDA, which is trying to protect you. In the vast majority of other countries regulatory agencies are practically non-existent or are far less strict than the FDA.

5) 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 or a stem cell-derived product into a patient either into the bloodstream or into a specific place that is injured such as a shoulder, we just do not know at this point if it will do any good with the exception of bone marrow transplant.

6) 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.

7) 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.

8 ) The people selling you non-FDA approved stem cell treatments want your money. Unlike stem cell researchers, the people out there advertising stem cell treatments that are not FDA approved are only really after one thing: your money.  As such they will do their best to convince you that their treatment is safe and effective. 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 simply want your money.

9) There is no such thing as completely “proven safe” and if something sounds too good to be true, it probably is. I am contacted fairly regularly by patients or their families and they often mention that the doctors offering stem cell treatments told them that the treatments are proven safe…or that umbilical cord blood cannot harm you….or that your own stem cells cannot harm you..or that adult stem cells are harmless. I’ll believe it when the FDA says it is so and you should be skeptical too.

10) The most important thing is data and you have a right to see it before treatment. 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 criticize the FDA 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 hopefully 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 you or 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. 

45 thoughts on “Patients guide to treatments


  1. It truly amazes me how websites such as this one is still not yet shut down because of its completely misinforming data on what stem cells are and what role they play in actually saving people’s lives! FDA is in bed with the Pharmaceutical, they have been for decades, They have approved thousands of ‘legal’ drugs on the market which has resulted in millions of deaths around America, through prescription drugs. Therefore to say stem cells are drugs is a complete and utter nonsense, it is an organ transplant. It has NOTHING to do with FDA or being a drug. In fact the FDA have persistently tried to shut down all stem cell activities as it threatening their playground of manufacturing hard drugs and keeping people sick, as opposed to treating them once and for all. Wake up people!!!


    • Vanessa: I agree with you. The FDA is perceived to be the “protector of our health”. I am presently waiting on the FDA to declare our autologous stem and regenerative cell processing “minimally manipulated” so we can finally offer commercially available autologous stem and regenerative cell laboratory products to patients of US physicians and surgeons across the United States at a cost perhaps 50% or less of the cost for overseas treatments. Our government should encourage an American enterprise offering state-of-the-art technology and therapy to our US citizens that will improve health, create US jobs and help decrease our health care costs. This site is a disgrace. The truth is that researchers extrapolate hundreds of billions of dollars from taxpayers to conduct experiments, create drugs that all have harmful side effects and charge extreme amounts of money for these drugs with harmful side effects. Have you ever heard of FDA APPROVED drugs that are removed from the marketplace? Have you ever listened to the TV ads where the side effects of the “wonder drugs” are worse than the disease? I defy any person to establish that the use of your own unexpanded, “minimally manipulated” stem cells that are transplanted into your own body has proven to cause any negative side effects.
      Dr. L


      • Dr. L,

        You state the reasons why we are all hopeful for regenerative medicine. we want cellular therapies that cure and repair. Trouble is we are not there yet. Throwing some adult stem cells into to body hoping they’ll do something positive and not cause cancer or other problems is irresponsible medicine until we get more data back on safety and efficacy. we all would like the FDA to move faster- hopefully they will. but grassroots medicine leading to experimentation of desperate patients at the doctor’s office is no way to go and is already leading to multiple deaths that could have been avoided.


  2. I am a potential patient doing research. Your remarks about stem cell providers are a far greater disservice to the public than the services of probably 98% of the doctors you deride.

    How dare you presume to know that the motivation of every doctor is purely financial? My life was saved in the 80′s by courageous doctors who were hounded out of practice by the FDA for providing treatments that are now mainstream.
    Doing the work that you do, you are too sophisticated to be ignorant of the actions of the FDA in serving Big Pharma and Big Medicine at the expense of the patient. The only possible explanation for your statements is your collusion and/or your financial dependence.. Your statements betray you.


    • Dear R. Jameson,
      You are very wise as a patient to be doing research on possible stem cell treatments before getting one.
      I disagree with you about my remarks doing a disservice. I also have not derided anyone, particularly not any real doctors.
      It is true that the FDA has a culture of caution that some might argue slows movement of research to the clinic, but it also generally does a good job of keeping the public safe.
      I have no financial interests that you allege.


  3. Doctors abroad are in it for the money, of course. But, doctors in the states are also in it for the money. The FDA is just in the hip pocket of Big Pharma. Too, many drugs and been put out as safe and later people are dying from them. As adults we should have a little more freedom to make our own health decisions.


  4. I am a doctor yes a real doctor stop with the all doctors are in it for the money all people need money to support their families and in most cases mean well train for years and work brutal hours at huge legal risk.are there bad doctors of course there are bad humans in any positions be it church government law etc. It’s all fun and games until you are sick at that point your opinion of doctors will change trust me……I am a doctor….anyway I have a horrible condition called idiopathic polymyoneuropathy hyperreflexia severe muscle pain and fasiculations sounds like fun.Nothing has helped I would sign up for just about anything and hope stem cells can help


    • Doctor, I am recently diagnosed with ALS, looking for something postive in the USA or Canada concerning stem cell trials or treatments. I view the FDA as in the pocket of big Pharma in much the same way that the NTSB is in bed with the airlines or for that matter our Congress is corrupted by various lobbying groups and PACS. Good Luck with your search for a real answer, I certainly know that not all doctors are in it for the money, OMG ! My primary neurologist is great , but so overworked and bogged down in paperwork , I do not envy him at all ! He is my hero !


      • Bill, you have my best wishes on your health. There are a lot of people out there working on stem cells including on hope for ALS, but unfortunately this all takes time…years really. I don’t have ALS so I have never been in your shoes, but I am a cancer survivor of a very serious malignancy. So far I’m doing well, but I know what it is like to be a patient, and us scientists/doctors saying we need more time is frustrating. But you are right there are a lot of heroes out there doing their best sometimes under difficult circumstances to make a difference.
        Thanks for your comment.
        Paul


  5. Paul,
    What are your insights on Autologous Stem Cells.? I have the occasion to work with a physician currently conducting a clinical trial here in southern Nevada. What are your thoughts?


  6. The FDA represents just over 300 million people. The world has about 7 billion people. Without even getting into debates over corruption, money, treatment, etc, surely it is obvious the FDA is only avery small piece f the puzzle.


  7. I am a 52 year old female who, 2 years ago suffered a stroke(right sided cva with left sided hemiplegia) after heart surgery to replace my mitral valve. Despite 12 weeks of intensive therapy in a rehabilitation hospital and on going daily physiotherapy i still have no functionality of my left arm and can only walk slowly with the aid of a hiking pole. Could stem cell treatment help me at this late stage?


    • Gail, I’m not sure I have a good answer for you, but to my knowledge there are no FDA approved stem cell therapies for an injury of this kind. I’d advise a great deal of caution if you are looking into the stem cell clinics out there in the U.S. or abroad that claim to have treatments for stroke. As far as I know there is no good science supporting the safety or efficacy of any of those. Talk to your personal physician and if considering a treatment, ask to see data or publications. My best wishes.


  8. I have advanced COPD. I am searching for an autologous stem cell clinical trial in which to participate. Can you help me?


  9. What is your opinion of stem cell treatment for autism?
    Specifically expanded/non-expanded donor mesenchymal cells and CD34+ stem cells?
    Thanks for your feedback.


    • Celine,
      Thanks for the question. Unfortunately from what I know there is zero convincing evidence of this kind of approach helping autism.


  10. I had an umbilical cord stem cell transplant in China in 2008. Unfortunately I did not see any change in my condition, but fortunately I did not have any side affects. My issue is a stretched cord at T7-T8 with tethered spinal cord syndrome.

    My issue with the industry is that reporting on research progress is inconsistent. I hear about new potential findings then zero after that. As much as we hate big pharma, there is an incentive to produce drugs and get them to market unlike progress with developing treatments using stem cells.


  11. I have herd many opinions most of which was to be optomistic and cautious about stem cell procedures. Does stem cells protect or help revese someone who has very minor hearing loss.


  12. Paul,

    Thanks for the post. As we have discussed, the FDA (and EMA) have both established pathways to bring certain treatment/products to market without official “approval”. I don’t believe the language you’ve chosen to use leaves room for this completely ‘compliant’ category of treatments/products.

    –Lee


  13. Paul, even though I don’t agree with you on most of your points, I very much appreciate how you are always kind in your reply to skeptics and criticism. Having to come to the point of seeking alternative treatment is no fun…What has happened with the MS patient in general is that we’ve totally lost trust in the neurologic approach to MS and FDA approved MS drugs. The drugs don’t work. The drugs are not effective. The drug studies are turning out to be wrong. When you’re newly diagnosed you tow the line and do everything your doctor suggests. Once you’re about 5 yrs. into the diagnosis and your body starts rejecting the maintenance drugs, you go to the internet and start looking for answers. If you’re a part of a support group, you start talking amongst yourselves about how the drugs are failing you as a patients. Most MS patients I know have tried at least 3 different maintenance drugs with huge rates of failure! I’ve NEVER heard even one MS patient brag about how awesome their maintenance drugs is! We think they suck! MS is a unique bugger! No known cause, no known cure, no effective drugs…It kind of sets us apart in the disease community. Ask 10 different Neuro’s their opinions, and you’ll get 10 different answers, as to what MS is….There’s no collective opinion. Lot’s of theories, but no answers. So with all that being said the MS patient has become a professional risk taker. We’ve tested and tried all the black label drugs, the interferon’s with horrible side effects and the new pills that can cause PPML and death. Since we can’t rely on the data and the studies that the FDA put out, we look to personal experience. We look to others who have been successful in an alternative treatment. Many patients as you know, have success stories from becoming medical tourists. These are the ONLY success stories we are hearing. The internet has connected a whole generation of MS patients. Collectively we know the FDA approved drugs don’t work. We don’t mind taking a chance on an unproven treatment. Some people operate their lives from a “show me the proof” kind of mentality. ALL the MS patients that I know operate from a “this is my experience” mentality. I’ve never been a risk taker in my life, but unfortunately “our” FDA has turned me into one….Go figure! I’m new at all this chatting to scientist online, and commenting on articles…one thing I find interesting is that there is absolutely NO consistency in the “science” end of this either…One scientist agrees that adult stem cells are not drugs, and another agree’s! It’s not much different then our doctors. It makes it really rough on the patient! Once again it makes us rely only on our collective experience as patients. I wish the scientists and doctors could come to a meeting of the minds, but I’m sure that’s wishful thinking. I’ve been a fly on the wall in most of these online groups and it’s quite entertaining! It’s like you are all on teams…The adMSC team and the Embryonic team and a few others that I forget the letters to….Well I’m on the patient team and to be quite frank, we’re loosing!


  14. “one scientist agrees that adult stem cells are NOT drugs, and another disagrees” ooops up top! It was late and my comment to you was waaaaay too long! HA! Sorry for my longwindedness!


    • Hi Jennifer. No worries. Thanks for the comments.
      Some thoughts from me….
      While bone marrow transplants, which are stem cell therapies, have been around for decades, other uses of stem cells for medicine are relatively new and there is a lot that we do not know. It is frustrating, but it also makes sense that there is going to be disagreement and even confusion amongst scientists, doctors, and patients. Our lack of knowledge and lack of consensus makes using stem cells as medicines a high risk way to go today. I hope that changes in coming years. I am a fan of the potential of adult stem cells.

      I understand that if one has had no benefit from conventional medicine for a certain disease like MS that taking some risks could be appropriate. I respect where you are coming from on that.

      But I think the potential benefit from the stem cell therapies being offered by clinics is near zero and the risks are very real even if poorly understood. Why? As a scientist who has been thinking about these things for a very long time, the reasoning offered by the clinics just makes no sense. Let’s take the case of MS. Transplanting adMSCs into an MS patient by IV is a common approach, but it makes no sense to me. Most of the cells will be dead or lodged in the lungs within a few hours. It is possible than basically 0% make it to the central nervous system. MSCs also are not known to make cells of the nervous system. Could MSCs “modulate” the immune system in a way that could help MS without even entering the nervous system? I suppose, but I’m not aware of any proof of that. They could also make MS worse by triggering autoimmune reactions.
      I also think that many clinics and doctors offering these “stem cell” therapies are predatory and take advantage of vulnerable patients. Why? Very simple. To make money. These folks are not Robin Hood out to help you simply for the goodness of their hearts.
      Finally, in my opinion the widely held view that there are “teams” or different “camps” in the stem cell world is mostly inaccurate. The stem cell researchers I know who are my colleagues most often work on multiple types of stem cells and like them all. For example, my lab works on adult, embryonic, iPS, and cancer stem cells.


  15. I don’t think that it is scientifically correct to call a stem cell a drug. And there are many types of cell and many types of stem cell and even more contexts for such cells. I just don’t understand why one would try to put them all in the same regulatory bucket, let alone in the same bucket as “real” drugs?

    Regulations should be reasonable. The law makes a fool of itself when it tries to fool nature by using verbal trickery.


  16. I just don’t understand. If your going to die anyway, what difference does it make to try stem cell therapy. Whole different ballgame when you are the one with the disease. In our case, ALS. You try anything.


  17. (admin note: this post was edited XXXX)

    I have FSH Muscular Dystrophy and in the last few weeks I have learned about XXXX center. I have contacted them and I am doing everything I can to be a candidate and qualify for this procedure. I have a response and I’d like to share it, this is what I received back.

    Thank you for inquiring about our services that we believe can be a help to you and your medical condition. Our affiliates at XXXX Center (Dr. xxxxxx) and XXXX Center in XXXX (Dr. xxxxxx) will be more than able to help you with your condition. (We) believe you are a good candidate for our SVF procedure. All our affiliates have the approximately the same pricing structure so there is no need in applying to all the treatment centers. If Dr. xxxxx or Dr. xxxxx do not return your emails please contact me and I will help you in your quest to receive a free phone consultation.

    I left the doctors names out for respect purposes. I am just curious to what people/scientists/ or other doctors may think about this? does anyone know about SVF and if it is legit? has anyone had SVF and came back with promising results? I have seen videos on youtube about many patents receiving stem cells and their outcome they say is positive. One guy even said his Creatine kinase levels are back to average. what that means is before he went in for treatment his Creatine kinase levels were well over a couple of hundred. After his stem cell injections and to this day his levels are at a steady 74, which is equivalent to an average persons Creatine kinase level. for those of you who dont know, Creatine kinase levels are the breakdown of muscle in your body. You can check these levels with a blood test.


  18. I have to agree with Jennifer, I have had MS for 14 years now and I to have exhausted all of the FDA approved therapies which have not stopped but only slowed the progression of my MS. I have been a nurse for the past 15 years but have been out of work due to my illness for the last 5. I consider my knowledge of the healthcare industry greater than average, with seven years of college. I have been researching these stem cell transplants for some time now, and it really cracks me up when people say “we’ll, if they worked, don’t you think Montel would have had it done.” LOL. HOW DO WE KNOW HE HASN’T….. He could have had chemo, who’d of known, he’s already bald. Besides, it would go against all his health food preaching.
    But on a more serious note. I have been communicating back and forth with Mexico, because I have been denied into all U.S. trials ( not enough flair ups in the past 2 years) because of being on Tysarbi. I have asked about their autologous bone marrow transplants. They don’t shut down the entire immune system but do use cyclophosphamide, and mannitol prior to adm. Bone marrow into menengines to enhance crossing the blood brain barrier. So in my opinion, and this is just my opinion, because my neurologist will not collaborate with me because I’m considering going out of U.S., I would think this would be safer than full body irradiation. My opinion is still up for debate

  19. Pingback: Celltex, RNL and phony worst case stem cell scenario comment | Knoepfler Lab Stem Cell Blog

  20. Pingback: Paul Knoepfler: tratamentos com células-tronco – Para Tio Orsini


  21. How about coming out of the school umbrella and see what has been going on in the clinical battlefield for over 7 years I have performed thousands of procedures with autologous BM and adipose stem cells and witnessed lives change for the better. We are there. It is time for the handcuffs of financial restraint to be lifted and allow physicians to do what thousands of research papers have been claiming. It works! And, it works with out any abnormalities for years to come. I would like to see the statements or papers that show it is not ready and the factual basis as to why it doesn’t. Without the laws of HIPPA, I would be so pleased in sharing with you hundreds of patients that have seen their disease halt, reverse, or in most cases diminish all together. And it is across the board in all specialties of medicine.


  22. Stem cells are not drugs when they are not manipulated or expanded. The FDA considers them drugs only when there have been growth factors or other compounds added to them. This is currently illegal. That is like saying blood donating is donating drugs. A blood product is as close to a category I can think of.
    Those with MS And ALS should explore low dose naltrexone. I have personally seen a woman who was totally disabled go back to work. It does not work with everyone but is 20 dollars a month, no side effects, been around 40 years. website is ldnresearchtrust .org.


  23. Hi…Paul,

    Recently few groups have discovered VSELs (Very Small Embryonic like Stem Cells). They are saying that, VSELs are equivalent to Embryonic Stem Cells. VSELs reside in all adult organs, are very small in size. But very few say 1-2 groups report their results reproducibility.

    Can you comment on that regarding VSELs on basis of your experience in Stem Cell Biology field..

    Thank You..!!


  24. Hi Dr. Paul,

    Has anybody heard about a stem cell therapy center for muscular dystrophy, in Bangalore, India? Any data available?


  25. I had a stem cell procedure in march of 2012 m for ms. Helped immediately! Lessions on spine are gone brain lessions still there. Blood brain barrier is very hard to get through. Burning in legs was going same day and have not been back. Until FDA can figure out how to make money on this people will suffer financially and in health!


  26. My wife has been diagnosed with FrontoTemporal Lobe Degeneration. Does anyone know of any clinical stem cell trials that she might be eligible for?

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