The recent NPR broadcast on stem cell therapies lit up my email inbox. Everyone wanted to know if I had heard it. I was working on about 10 things at once that day, but I ended up listening to it while walking my dog, Elvis, later that night.
A few things struck me about the story in both positive and negative ways.
First of all, I hate the title: FDA Challenges Stem Cell Companies As Patients Run Out Of Time.
It immediately casts the FDA as a bad guy and patients as victims. It is not a constructive title with which to frame a healthy, balanced dialogue. I’m a cancer patient and I do not see myself as a victim. The best stem cell patient advocates I know do not view themselves as victims, but rather if they don’t like something they work to make change.
I like the fact that the piece brings in the human side and interviews SammyJo. MS is a devastating disease and there are thousands of very real people suffering from it. Doctors today have little to offer patients with specific types of MS. That’s a reality that is important for readers to know. I do however have to point out that her comment on the NPR website is way off base:
My statement is factual. And to my knowledge I have no direct conflict of interest here.
I found the interview with Dr. George Daly to be thoughtful and convincing. No surprise there, huh? He’s right in what he says and contrary to some comments on the NPR website, I can’t imagine any way that Daly’s raising concerns about for-profit stem cell clinics could benefit him. It makes no sense.
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.
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.
Hello Paul,
I totally agree with you. As a doctor I know how peoples could suffer from diseases such as MS. But I also know how devastating could be a wrong treatment. I understand patients saying that they are out of time, but they must know that they could participate in real clinical trials with very accurate follow-up and cell facility that completely comply with high standards (it seems that this not the case for Celltex). In Europe, where I live, doctors and scientists had set-up a clinical trial to evaluate the benefit of injecting patients’ own MSC in MS. The leader of this European effort is A. Ucelli whose work in MS models and cure is largely recognized. If patients want to participate in such clinical trials, with real endpoints (not money), they could check the website clinicaltrials.gov. I just type “Mesenchymal Stem Cells for Multiple Sclerosis” and found several active clinical trials including one sponsored by The Cleveland Clinic. So patients are not alone and doctors want to help them. But the one involved in clinical trials want first t to prove that the treatment they designed is good for them. Finally, they work for the human community and companies like Celltex, whatever they claimed, work to make a lot of money only.
Congratulations on being a cancer survivor. Who stood in the way of your treatment? There are some doctors who make money and do not have the best interest of the patient. Dr Jones is not one of those Doctors. You may think he is and oppose him. You will be proven wrong. Sammy Jo has no choice. She has been following stem cells as I have for 12 years+. Her option other than Stem cells is a nursing home. With great effort she is forced to work around the FDA, you and others.
Dr Daly holds several embryonic stem cells patens.
Thanks for your comment. No one stood in the way of my treatment, but all my treatments were based on years of published rigorous data from no-fee FDA approved clinical trials on prostate cancer patients. I am ethically opposed to charging patients for clinical trial participation and to treating patients without data to demonstrate the safety and efficacy of the treatment. I respect SammyJo and the fact she can make her own decisions, but I just happen to disagree with her about where the field stands in this area today.
“The reality is that the treatments discussed are not scientifically validated as safe or effected.” – Comment from Paul Knoepfler on NPR story.
I welcome input from a stem cell researcher on this. Here are 6 papers I reviewed that made me feel comfortable pursuing this course of treatment, for my untreatable MS. The review papers each cover further studies. Please point our why this science is lacking, for someone in my condition to rely upon:
1. “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 neuro protective molecules. 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.
– Multiple Sclerosis Journal
http://msj.sagepub.com/content/early/2012/11/01/1352458512464686?elq=d5772cd1683c44a69ca3361a5408027e
2. “Safety of Cell Therapy with Mesenchymal Stromal Cells (SafeCell): A Systematic Review and Meta-Analysis of Clinical Trials”
Based on the current clinical trials, MSC therapy appears safe. However, further larger scale controlled clinical trials with rigorous reporting of adverse events are required to further define the safety profile of MSCs. (Manoj et al 2012)
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3485008/ PMCID: MC3485008
3. Cleveland Clinic is several years into a human trial for MS using expanded bone marrow derived stem cells, and they have isolated a growth factor that is part of the anti inflammatory effect that offers such immediate symptom relief:
“Hepatocyte growth factor mediates mesenchymal stem cell–induced recovery in multiple sclerosis models.”
http://www.ncbi.nlm.nih.gov/pubmed/22610068 PMID:22610068
“Recovery From Multiple Sclerosis By Growth Factor In Stem Cells”
http://www.medicalnewstoday.com/articles/245816.php
The last 3 papers address the specific Celltes method:
4. Safety of Intravenous Infusion of Human Adipose Tissue-Derived Mesenchymal Stem Cells in Animals and Humans
Dr JC Ra
http://www.ncbi.nlm.nih.gov/pubmed?term=21303266
5. “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
6. “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
Thanks for the comment, SammyJo. See my detailed response here: http://tinyurl.com/atw9fhr Paul