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ALS Worldwide
March 15, 2015

Dr. Paul Knoepfler on the subject of Stem Cell Transplantation

Dr. Paul Knoepfler on the subject of Stem Cell Transplantation

Dr. Paul Knoepfler is a biomedical scientist, science writer, advocate, and cancer survivor. His research interests are primarily focused on the epigenomics of cancer and stem cells with a particular interest in pediatric tumors. Knoepfler is an Associate Professor at UC Davis School of Medicine in the Department of Cell Biology and Human Anatomy. At UC Davis he is also a faculty member of the Genome Center, the Comprehensive Cancer Center, the Institute for Regenerative Cures (aka UC Davis Stem Cell Center), and the Institute for Pediatric Regenerative Medicine. He received his BA in English LIterature from Reed College and PhD from UCSD School of Medicine in Molecular Pathology.

He is the author of Stem Cells: An Insider’s Guide, currently the top book on stem cells on Amazon and runs a popular stem cell research and policy blog. He has received a number of awards for science and advocacy.

Stem Cells: An Insider's Guide is an exciting new book that takes readers inside the world of stem cells guided by international stem cell expert, Dr. Paul Knoepfler. Stem cells are catalyzing a revolution in medicine. The book also tackles the exciting and hotly debated area of stem cell treatments that are capturing the public's imagination. In the future they may also transform how we age and reproduce. However, there are serious risks and ethical challenges, too. The author's goal with this insider's guide is to give readers the information needed to distinguish between the ubiquitous hype and legitimate hope found throughout the stem cell world. The book answers the most common questions that people have about stem cells. Can stem cells help my family with a serious medical problem such as Alzheimer's, Multiple Sclerosis, or Autism? Are such treatments safe? Can stem cells make me look younger or even literally stay physically young? These questions and many more are answered here.

A number of ethical issues related to stem cells that spark debates are discussed, including risky treatments, cloning and embryonic stem cells. The author breaks new ground in a number of ways by suggesting reforms to the FDA, providing a new theory of aging based on stem cells, and including a revolutionary Stem Cell Patient Bill of Rights. The book is a guide to near future of the stem cell field and a thoughtful perspective on how stem cell therapies will ultimately change your life and our world.

Reprinted here is Dr. Paul Knoepfler’s article “Patients Guide to Treatments, Top 10 list of important, easy to understand facts for patients about stem cell treatments” from his blog site.

“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.