Stem cell blogger Knoepfler on NPR piece on stem cells
On Saturday, NPR ran a story on the ongoing legal battle between the Food and Drug Administration and the Houston-based Celltex Therapeutics. (Or, I should say, the company formerly based in Texas. Last week, CellTex announced it was moving its patient-treatment center of operations to Mexico.)
The saga between the FDA and Celltex, which is not likely to end any time soon, has been detailed extensively elsewhere. (The coverage by Nature’s David Cyranoski has been excellent.) But I was most interested in today’s take on the NPR story by U.C. Davis stem cell scientist Paul Knoepfler, PhD.
Knoepfler, who writes a well-known stem cell blog, tackles a common refrain expressed by frustrated patients unwilling to wait any longer for what they see as their last chance for treatment: the idea that researchers and FDA administrators are kowtowing to a desire by ‘big pharma’ to keep chronically ill patients from cures that might reduce the pharmaceutical companies’ profits.
I hear this all the time. Desperate patients (and their advocates) have a powerful need for hope. But as I detailed in a Stanford Medicine article in 2011, there are plenty of people ready to take advantage of such hopefulness. Even writing about the topic brings a barrage of angry comments (and I’m sure to see some here). That’s why I’m applauding Knoepfler’s response to the assertion of conflict among researchers and pharmaceutical companies:
Making that kind of accusation, however, is a common tactic of proponents of stem cell deregulation; throw unfounded charges of conflicts of interest against anyone who raises concerns about clinics. Another weapon is to accuse stem cell researchers of being uncaring or disconnected from reality or somehow not as smart or caring as the MDs selling the stem cell treatments. The sad irony is that as the false accusations of conflicts of interest are thrown at stem cell researchers, it is somehow ignored that the MDs involved are raking in millions of dollars of patients’ money.
As I said in my comment on the NPR site, the reality is that these stem cell treatments can be worse than doing nothing. They can literally kill you. They can do unexpected things as was so powerfully illustrated by the bone in the eye facelift case. It is hubris by the individual doctors and clinics to say they know all that is needed to know to put these cells into patients without FDA approval or vetting by other scientists and doctors first.
As I said, this controversy is not going to go away any time soon. But we need to be thoughtful about how we bring stem cells to to the clinic, for many reasons. As Knoepfler concludes:
Rushing unproven stem cell treatments into patients can also do worse than harm one individual too because negative outcomes might set back the entire field many years, affecting hundreds or thousands of patients by delaying future stem cell treatments. So we also owe it to patients of the future to be responsible in our conduct now and do this right.
I’ve excerpted a lot of Knoepfler’s post today here because I think it’s so important. And I encourage you to read other entries in his blog about unproven stem cell treatments. He’s a researcher, a blogger and great communicator.
Previously: FDA audit of stem cell clinic revealed by Houston Chronicle, U.S. District Court rules that stem cells are drugs, and Stem cell researchers challenge clinics’ questionable practices
February 7th, 2013 at 10:30 pm
Krista – Do you have hard evidence of patients being killed after receiving autologous stem cell therapy? Tens of thousands have had treatment and yet we hear of only isolated incidents that most likely were medical malpractice rather than the result of the stem cells themselves. I think it is obvious that everyone but patients has a conflict of interest. That is hardly a reason to deny patients the right to have treatment with a competent physician who uses safe guidelines for therapy. To deny a patient his or her civil rights is uncaring and disconnected and in far too many instances, a death sentence.
February 8th, 2013 at 10:54 am
Your blog reveals the conflict of interest you insist does not exist. There has never been a case of cancer ectopic tissue or contamination in a patient treated in a clinical trial using autologous MSC. meta analysis of many studies reveals it is safe. Insisting it does have severe safety problems is unsupportable.
If you want patients to have safe therapy because you believe MDs who want to provide therapy are purposely taking shortcuts and harming people so they can make a buck then support university doing this therapy for anyone who needs compassionate treatment. Then you’ll be certain that patients who want this get the same standards as people in clinical trials…and I’ll believe you that this isn’t about conflicts of interest.
John Gurdon–Nobel laurete for iPS says patients would be OK to use autologous cells.
February 8th, 2013 at 12:40 pm
I’m the CEO of SevOne, Inc., one of the fastest growing software companies in the world. We’ve essentially built the next generation IT nervous system. At the same time, I’ve been struggling to keep my personal nervous system working. I have Multiple Sclerosis and had the resources to travel to Panama for safe and effective treatment because the FDA has blocked our learned American physicians the right to offer this safe treatment in America. Please see the link below, Safety of Intravenous Infusion of Human Adipose Tissue-Derived Mesenchymal Stem Cells in Animals and Humans. I tried to get into the few, expensive approved clinical research trials in the US, but did not qualify. Furthermore, commercial funding for these trails is unavailable because autologous Healing Cells are impossible to patent. This is a business reality, not an accusation. So, I spent thousands of dollars in the evaluation process and thousands traveling for treatment that should be available in the America. Most Americans can’t afford these studies and can’t afford to travel overseas for treatment. This is a crime.
Roger M. Nocera, M.D., in his book Cells That Heal Us From Cradle To Grave: A Quantum Leap in Medical Science, explains how this medical science discovery is on par with Immunology discovered two centuries ago with the smallpox vaccine, and with the discovery of antibiotics a century later. His book is a primer on how this will completely nullify our current medical health care and pharmaceutical research systems. http://www.cellsthatheal.com/about-the-book.php Nocera, Dr. Roger (2011-02-15).
“The American people would be the beneficiaries of the new Healing Cells therapies, which would rapidly become available quite automatically. Autologous Healing Cells are perfectly safe because they are cells taken out of one part of the body and simply put back into another body part of the same patient. Once our FDA stops denying our nation’s capable physicians their civil right to focus upon it, then Healing Cell treatments will rapidly revolutionize healthcare in a manner that will reduce costs to a fraction of the current system and drastically improve the health of our citizens…The FDA must lift its embargo on this completely natural treatment and allow our learned American physicians to begin to offer this safe treatment and database their results so that standard treatment regimens can be established for each disease and condition.” Roger M. Nocera, M.D.
I returned last month from my second trip to the Stem Cell Institute in Panama. I went a second time because after the first treatment I recovered functionality. This is unprecedented. http://www.cellmedicine.com/about-stem-cell-institute-panama/stem-cell-research-publications/
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Safety of Intravenous Infusion of Human Adipose Tissue-Derived Mesenchymal Stem Cells in Animals and Humans |(by Dr JC Ra on the process used at Celltex)
http://www.ncbi.nlm.nih.gov/pubmed?term=21303266
2. Taking Stem Cells Beyond Discovery: A Milestone in the Reporting of Regulatory Requirements for Cell Therapy
Stem Cells and Development. August 2011, 20(8): 1295-1296.
This article is by leaders in stem cell research at Duke, Tulane and LSU, refers to Dr JC Ra’s study ‘Safety of Intravenous Infusion of Human Adipose MSCs’, as a model for the demonstration that MSCs are safe, with praise for offering a model way to build regulatory frameworks.
http://www.ncbi.nlm.nih.gov/pubmed?term=21510815
3. Stem cell treatment for patients with autoimmune disease by systemic infusion of culture-expanded autologous adipose tissue derived mesenchymal stem cells. Journal of Translational Medicine 2011, 9:181
Dr JC Ra, includes 5 case studies at the end.
http://www.translational-medicine.com/content/9/1/181
Recently published in Multiple Sclerosis Journal:
Mesenchymal stem cells as treatment for MS – progress to date
An exhaustive amount of preclinical data has shown that the intravenous administration of mesenchymal stem cells (MSC) effectively ameliorates experimental autoimmune encephalomyelitis (EAE), a model of multiple sclerosis (MS), through the release of anti-inflammatory and neuroprotectivemolecules. Based on these results, several small pilot clinical trials in subjects with advanced MS have demonstrated that MSC administration is safe and provided an early signal of clinical effectiveness.
http://msj.sagepub.com/content/early/2012/11/01/1352458512464686?elq=d5772cd1683c44a69ca3361a5408027e
February 11th, 2013 at 11:39 pm
Krista, thanks for such great coverage of a hot topic.
It’s hard to disagree with the points made by Knoepfler, yet at the same time I don’t feel it’s easy to substantiate them. But of course, these doctors are raking in a lot of money. And if it is the case that these treatments aren’t safe, it’s nothing short of unpractical to let them remain. It’s a fine line.
You might also be interested in a research being done in China. Dr. Rongxiang Xu is actually working on a nutrient-based composition capable of inducing stem cells in situ.
February 13th, 2013 at 9:44 am
Knoepfler sited adverse affects that sound more like malpractice, so there is no proof of autologous stem cell treatments done properly have any adverse affects. At first I heard that autologous stem cell treatments can cause cancerous tumors. There is not one single published finding on this at all. Now I am hearing on clots and adverse auto-immune reactions. The facts are crystal clear – tens of thousands of treatments have already been performed without any adverse affects. Compared to pharmaceutical drugs having 200,000 deaths a year, I think stem cell treatments are doing pretty good. As for conflict of interest, read up on the after effects of the 1980 Bayle Dole act. It gave your university millions in patent royalties and research grants that is a fact.
February 15th, 2013 at 10:42 am
Knoepfler is wrong, no one is raking in money. The costs of this treatment pale in comparison with many conventional treatments. The immunomodularly drugs for MS cost close to $40,000.00 per year for a pitiful 30% response rate. Surgeries for severe heart disease around $250,000.00, etc.
Because of the FDA adult stem cell therapies have to be done overseas, which adds considerable cost, yet it is still less expensive, but seems like allot because insurance will not cover it, so it must be paid out of pocket.
I challenge Knoepfler to site any proof that the owners or doctors of overseas clinics are making more money than clinic owners and doctors make providing conventional care, It’s just not true. Knoepfler is fear mongering, the usual tactic of the opposition. Does this guy even know what it costs to run a clinic? Knoepfer is pedaling lies. No one is raking in the money on adult stem cell medicine, it’s just a lot of hard clinical work.