About Dr Knoepfler
Dr. Paul Knoepfler is an Associate Professor in Cell Biology and Human Anatomy at the UC Davis School of Medicine, where his lab studies stem cells and cancer. He is also a faculty member of the UC Davis Genome Center and leader of the Cancer Stem Cell Initiative at the UCD Comprehensive Cancer Center. He also teaches graduate courses in stem cells and is an instructor for the histology course at UCD School of Medicine. Dr. Knoepfler did his postdoctoral training at the Fred Hutchinson Cancer Research Center in Seattle. He received his Ph.D. from UCSD School of Medicine in Molecular Pathology and his B.A. in English Literature from Reed College in Portland, OR. He is a cancer survivor, patient advocate, writer, and is currently the only faculty level academic blogger on stem cell research in the world.
Two overall statements:
A. I am not a physician. My answers to these questions are for information only and are not medical advice. All medical decisions should be made by patients in consultation with their physicians.
B. My lab works on pluripotent, adult, and cancer stem cells. I’m excited about all stem cells including adult stem cells. Nothing would please me more than to see propagated adult stem cells be successful in the clinic and help as many patients as possible. I would see that as a tremendously good thing and I can see now way in which that would negatively affect me or my research. I want safe and effective adult stem cell treatments to transform medicine, but we have to do this according to the law and do it safely without taking dangerous shortcuts.
Questions and Answers
1) I am an MS patient who has received expanded stem cell infusions derived from my adipose tissue with wonderful results, like major recovery in vision, sensory and elimination system. You make assertions that your research indicates that expanded stem cells could be a cancer risk; have you published this work, or can you provide citations to other research that proves this?
PK Answer: I’m glad to hear that you are feeling better.
Great question, but one without clear answers in terms of the cancer risk. My lab does not directly study expanded adult stem cell tumorigenic potential when transplanted. However, I have been studying cells grown in culture for 22 years including stem and cancer cells. Growing cells in culture even for just a few weeks fundamentally changes them in ways that make them more like cancer cells. See one example paper for proof of how in just a matter of a few weeks in culture, human adult stem cells can acquire significant mutations that are strongly linked to cancer: http://www.sciencedirect.com/science/article/pii/S1934590911003286
A larger issue is that the responsibility is for proponents of for-profit stem cell treatments to prove expanded cells are safe (which they still need to do via appropriate clinical trials), not for concerned members of the community to prove they are unsafe. The first rule of medicine is “do no harm!” We do not set out to prove something causes cancer but rather to determine a safe and effective way for this technology to benefit people. The FDA has considerable information on the risks of expanded cells. Why not contact them and share with them about your experience. They won’t bite!
2) I've been waiting years for stem cell therapy in the U.S. I didn't want to take risks overseas and I wanted to be a data point in a trial so it might help other patients. Now adipose derived expanded stem cell therapy is available in the U.S. from commercial companies, under IRB monitored trials. Why are you trying to get the FDA to shut this down? Wouldn't patients be safer staying here in the U.S.?
PK Answer: I admire your goal to help others via participation in a clinical trial. I think such participants are making important contributions. If you read the FDA warning letter to Celltex, dozens of issues were raised with how Celltex’s lab operated including basic things like sterility. Then there is the issue that the propagated stem cells are, according to the FDA and supported by a recent federal court case, drugs. That is federal law at this time, which cannot be ignored.
I believe generally that treatments in the U.S. are safer than abroad, but that does not mean that U.S. treatments are by definition safe. Safety depends on operator compliance and expertise. Importantly, there are limited legal protections for those who are recipients of unproven stem cell therapies even if the physician commits malpractice because the laws on deciding these cases are based on standards and procedures established by safety and efficacy over time. Some patients report that their insurance companies say they will not cover complications arising from unregulated stem cells. A blood infection can kill or cost many hospital days and teams of specialists to manage. Organ failure can occur with blood infections.
3) Please provide examples from the medical field where FDA has inserted itself into the delivery of a single medical procedure in an individual beyond testing for communicable diseases in transplants.
PK Answer: This is a political question and has nothing to do with my expertise as a scientist. The FDA is involved with many aspects of medical care for instance testing of the device, equipment, and lab cleanliness during an intervention. There is a free CITI course online (https://www.citiprogram.org/irbpage.asp?language=english ) about FDA, ethics and past decisions and why they are made. You as a group may want to take this so you can learn more about how the system works and be more informed and more powerful contributors. This could make you more effective in stem cell activism or to build bridges to stem cell care.
There is the case of Regenerative Sciences, Inc and now Celltex. Do you mean besides stem cell examples? I believe the FDA has inserted itself into many other cases as well over the years unrelated to stem cells, but that is not an area I am deeply familiar with.
Other issues that arise that in my opinion necessitate FDA involvement are poor laboratory practices that put patients at risk not necessarily from the stem cells themselves, but from infection or immune problems.
4) As a patient, I call foul! I want stem cells - been waiting for years as multiple sclerosis destroys me. I actively read everything about stem cells and I know already of your blog. You state, "I'm a stem cell researcher and cancer survivor who has no monetary interest in the success or failure of any stem cell clinic......". A simple Google search shows that you repeatedly slam MD's in your blog who want to treat patients with adult stem cells. Why do you want to dictate what my doctor and I decide to do in the privacy of the consultation room? They transplant stem cells for cancer already. I'm sick of waiting! An MS researcher said in 2000, it would be 5 years--I'm still waiting! And you are all over the internet talking about danger when adult stem cells are given everyday for cancer and have been for years. Why do you care if I get them for MS? The truth is that you are not unbiased. Your research is related to science where people get pluripotent stem cells that have been manipulated by you rather than natural cells. Having patients get their own stem cells right now before your research is done might slow your funding. My own stem cells are not a Pharma product. They are mine. What do you have to say about all this?
PK Answer: Patients have rights and deserve respect. I totally agree with you on that. At the same time society has a responsibility to do its best to keep medicine as safe as possible and to help patients make fact-based decisions. I believe that stem cell scientists have an extra responsibility in that area. As I said in another answer, the first rule of medicine is “do no harm”. It is not at all clear that stem cell transplants of in vitro propagated stem cells are safe (my great hope is that they are) so I’m concerned that unregulated transplants could do a great deal of harm. American society has decided that what happens between a patient and a doctor is in fact not entirely independent of the law of the land and of regulatory agencies. I think this decision is wise and to the best interest of patients. Doctors cannot just do whatever they feel like to patients even if the patients sign off on it.
Why do I care if you or anyone else gets unlicensed stem cells for MS? My concerns are (A) that to my knowledge there is no scientific or rigorous medical evidence that stem cells work for MS and (B) there is no proof that they are safe. You seem suspicious of my motives, but I can think of no personal gain for me in my efforts to help make stem cell-based medicine safer and educate people. Whether patients get (or do not get) stem cell transplants of their own stem cells has no influence on my grant funding whatsoever. That’s the reality. The doctors selling the unlicensed stem cells of course have a very direct, obvious vested/conflicted interest because they want patients’ money.
You say “I want stem cells” and “they are mine”, but once a clinic isolates your stem cells and grows them, they become a biological product that is different than the stem cell in your body. How are they different? They acquire mutations. They change their antigens on the cell surface possibly sparking immune reactions. They grow faster. They may respond to hormones differently. The list goes on and on.
5) Do you feel that the FDA and PhD scientists such as yourself can make better decisions for patients regarding stem cell therapy, as opposed to the decisions I make with my MD?
PK Answer: Different parties bring different strengths and weaknesses to the table in medical decision making. I’ve spent half my life growing cells and studying them including stem cells over more than a decade. I feel confident in saying I know far more about cell biology and stem cell biology specifically than most MDs. That doesn’t mean I know everything, but I know cells really well. Doctors of course know medicine far better than I do, but they may have some major gaps in knowledge about cells. In addition, when stem cells are offered in a for-profit setting, things get complicated for the MDs involved because of mixed motivations.
In my opinion responsible MDs conduct medicine according to the rules of the land in which they are practicing. If they do not agree with the rules, they do not violate them in their for-profit care of patients, but rather continue to follow the rules while at the same time working to change them. I do not see doctors who break the law as some kind of Robin Hood-like heroes helping patients. A patient and their doctor are not equal partners in decision-making because the doctor has taken an oath to protect and care for you, yet s/he is making a profit off of you by giving you an unapproved, potentially dangerous treatment.
6) On the basis of compassionate use, how can you argue that I can't have access to autologous stem cell therapy, when my other option is lingering miserable death in a nursing home from progressive MS which is non-responsive to any of the FDA approved MS drugs? There are no stem cell trials for progressive MS, so I think that I should be free to utilize any commercial stem cell clinic in the U.S. that I determine has a good and effective service, without interference from the FDA.
PK Answer: I am not an expert in compassionate use, but I understand that it may be appropriate for very specific circumstances. I would, however, question physicians who try to make a profit off of giving “compassionate use” treatment to patients. According to this American Cancer Society webpage (http://www.cancer.org/treatment/treatmentsandsideeffects/clinicaltrials/compassionate-drug-use ), compassionate use is legal only in two circumstances: expanded access (the drug in question must be in Phase III trials, while stem cell drugs are not that far along) or Single Patient Access, in which the patient and doctor must ask the FDA for permission to use the drug. Neither case seems to apply to for-profit clinics offering stem cell treatments that are unlicensed. Note that stem cell clinics and doctors are fighting to avoid stem cells even being defined as drugs. If stem cells are not drugs or medical devices, then to my knowledge compassionate use does not legally apply.
7) Do you hold any patents for your work with stem cells? Do you not see it as a conflict of interest that you are a researcher who depends on research work not just for your own job, but for your university? The dollars must keep flowing and if you position yourself as an industry "expert", then this has to benefit you and your university. If you were to advocate for patients to be able to get treatments as a practice of medicine, your research would be diminished somewhat. The problem patients are facing is that your research and that of others in the field, is not available to patients now and many do not have the luxury to wait years. Why not embrace a stance that helps patients now as well as keeps your own research into riskier stem cell types going as well? It seems that a happy medium would be the best approach rather than condemning sick people to no hope which is basically what you and others are doing.
PK Answer: I have no patents nor patent applications. I do not believe I have a conflict of interest on stem cells. To my knowledge, my funding is not related to my work as a patient advocate and educator on issues of stem cell safety. I have no grant funding for that work. I disagree with your statement about my hypothetical advocating for patients to get treatments diminishing my research. When what I have worked on scientifically becomes a viable medical intervention I will be overjoyed. Where there are results that are evidence based, funding is increased not decreased. Patents are normally held by the lab industry, academic institutions, or organization. Very little if any of the patent benefit gets to the inventing scientist.
8) What on earth does a bioethicist know about my illness? Do you feel that Doug Sipp, who claims to be a bioethicist is qualified to be someone who should be cited by the media as an industry spokesperson? I think patients are getting tired of the type of attacks that some in the industry make who seem to not be qualified or who have very obvious conflicts of interest. This diminishes the impact that true scientists might have in the eyes of patients when the scientific community makes little effort to police itself. There seems to be a lot of retractions and researchers in the news lately too that may have faked data. It's truly upsetting to watch the ISSCR and some in the scientific community condemn most doctors and clinics who want to treat patients while looking the other way at those in the scientific community who need to be called on the carpet.
PK Answer: This is simply a personal attack and an attack on the stem cell field. I believe that Doug (who by the way does not refer to himself as a bioethicist) has something valuable to offer to the dialogue. So do others including Dr. Centeno, ICMS, and Celltex. I organized a debate between Doug and Dr. Centeno on my blog that hundreds of readers found valuable. I also have engaged ICMS via an interview with their President, Dr. Rodriguez. I have personally met with Guv Rick Perry of Texas to talk over stem cell issues. I have invited Celltex to post on my blog. I regularly talk/email with folks who do not see eye-to-eye with me on stem cell issues, but we have productive, respectful dialogue. I believe in building bridges, but with accountability.
9) How is it morally responsible for patients to be denied access to treatments that use their own body parts when these patients have little or no quality of life? Why should someone from a government agency, a university, a company, etc. be the one to hand out a death sentence and expect that the patients should be grateful that this has somehow "protected" them? I think many assume that all patients are simply those who aren't really that ill, don't have a progressive disease and maybe just want to try stem cells because they blew their elbow out pitching balls. The dilemma those of us who are seriously ill now face is going offshore or giving up - thanks to Obama's FDA commissioner and those in the industry who are putting their own interests above patients. I just don't see how some people sleep at night knowing this. There were patients scheduled for treatment at Celltex who are now devastated by the FDA's decision. They were notified that their treatments were cancelled, knowing others that had already been treated were seeing a lot of improvements. Do you have any idea what that does to a person?
PK Answer: By law the FDA is required to regulate stem cells that are grown as drugs at this time. This is the law of the land in America.
There is no scientific evidence that stem cells of this kind actually do patients any good and there are very real risks. For-profit companies are first and foremost in the business of making money. If the FDA was the problem these cells would be demonstrably working in other countries and medical journals would be full of this evidence. This is not happening in any country with or without an FDA.
By way of background, I do personally know what it is like to be a patient with a potentially fatal illness. I was diagnosed just about exactly three years ago with a very serious form of prostate cancer unexpectedly at age 42. I had surgery and I am doing well now, but I know that at any time the cancer could come back. Prostate cancer can also become untreatable at some point in a patient’s life.
Over the last few years I have also talked to dozens of patients who are considering stem cell treatments for various very serious health problems for which today’s medicine has little to offer. I respect them and their concerns, but as I tell them, stem cells are not some kind of magical panacea. They are exciting and powerful, but we have to be careful. Just like any other medicine they must be tested in pre-clinical studies for safety and their use must follow federal and state law. Otherwise they can do more harm than good. Physicians and lawyers of for-profit companies who say pre-clinical and clinical trials (non-profit) are unneeded are, in my opinion, mostly out to make profits and do not want to spend the time and money to do this right.
10) How if your position opposing stem cell clinics offering stem cells as medical care before randomized controlled trials for an indication c/w this statement in the most recent version of the Declaration of Helsinki? Do you support clinics that follow the general guidelines discussed in Helsinki?
35. In the treatment of a patient, where proven interventions do not exist or have been ineffective, the physician, after seeking expert advice, with informed consent from the patient or a legally authorized representative, may use an unproven intervention if in the physician's judgement it offers hope of saving life, re-establishing health or alleviating suffering. Where possible, this intervention should be made the object of research, designed to evaluate its safety and efficacy. In all cases, new information should be recorded and, where appropriate, made publicly available.
PK Answer: Notably, the FDA and NIH have rejected the Declaration of Helsinki for many years for numerous reasons. One concern in particular has been the perceived lack of patient protection. The American Medical Association has had many problems with the Declaration over the years in its different iterations as well.
I do not unconditionally support this one very isolated part (quoted above) of the Declaration of Helsinki taken out of context. I suggest that you read and study the agreement for yourself. Please note that clinics that refuse the FDA pathway are not evaluating safety and efficacy as indicated in that passage. They are making up their own rules.
Dr. Paul Knoepfler is an Associate Professor in Cell Biology and Human Anatomy at the UC Davis School of Medicine, where his lab studies stem cells and cancer. He is also a faculty member of the UC Davis Genome Center and leader of the Cancer Stem Cell Initiative at the UCD Comprehensive Cancer Center. He also teaches graduate courses in stem cells and is an instructor for the histology course at UCD School of Medicine. Dr. Knoepfler did his postdoctoral training at the Fred Hutchinson Cancer Research Center in Seattle. He received his Ph.D. from UCSD School of Medicine in Molecular Pathology and his B.A. in English Literature from Reed College in Portland, OR. He is a cancer survivor, patient advocate, writer, and is currently the only faculty level academic blogger on stem cell research in the world.
Two overall statements:
A. I am not a physician. My answers to these questions are for information only and are not medical advice. All medical decisions should be made by patients in consultation with their physicians.
B. My lab works on pluripotent, adult, and cancer stem cells. I’m excited about all stem cells including adult stem cells. Nothing would please me more than to see propagated adult stem cells be successful in the clinic and help as many patients as possible. I would see that as a tremendously good thing and I can see now way in which that would negatively affect me or my research. I want safe and effective adult stem cell treatments to transform medicine, but we have to do this according to the law and do it safely without taking dangerous shortcuts.
Questions and Answers
1) I am an MS patient who has received expanded stem cell infusions derived from my adipose tissue with wonderful results, like major recovery in vision, sensory and elimination system. You make assertions that your research indicates that expanded stem cells could be a cancer risk; have you published this work, or can you provide citations to other research that proves this?
PK Answer: I’m glad to hear that you are feeling better.
Great question, but one without clear answers in terms of the cancer risk. My lab does not directly study expanded adult stem cell tumorigenic potential when transplanted. However, I have been studying cells grown in culture for 22 years including stem and cancer cells. Growing cells in culture even for just a few weeks fundamentally changes them in ways that make them more like cancer cells. See one example paper for proof of how in just a matter of a few weeks in culture, human adult stem cells can acquire significant mutations that are strongly linked to cancer: http://www.sciencedirect.com/science/article/pii/S1934590911003286
A larger issue is that the responsibility is for proponents of for-profit stem cell treatments to prove expanded cells are safe (which they still need to do via appropriate clinical trials), not for concerned members of the community to prove they are unsafe. The first rule of medicine is “do no harm!” We do not set out to prove something causes cancer but rather to determine a safe and effective way for this technology to benefit people. The FDA has considerable information on the risks of expanded cells. Why not contact them and share with them about your experience. They won’t bite!
2) I've been waiting years for stem cell therapy in the U.S. I didn't want to take risks overseas and I wanted to be a data point in a trial so it might help other patients. Now adipose derived expanded stem cell therapy is available in the U.S. from commercial companies, under IRB monitored trials. Why are you trying to get the FDA to shut this down? Wouldn't patients be safer staying here in the U.S.?
PK Answer: I admire your goal to help others via participation in a clinical trial. I think such participants are making important contributions. If you read the FDA warning letter to Celltex, dozens of issues were raised with how Celltex’s lab operated including basic things like sterility. Then there is the issue that the propagated stem cells are, according to the FDA and supported by a recent federal court case, drugs. That is federal law at this time, which cannot be ignored.
I believe generally that treatments in the U.S. are safer than abroad, but that does not mean that U.S. treatments are by definition safe. Safety depends on operator compliance and expertise. Importantly, there are limited legal protections for those who are recipients of unproven stem cell therapies even if the physician commits malpractice because the laws on deciding these cases are based on standards and procedures established by safety and efficacy over time. Some patients report that their insurance companies say they will not cover complications arising from unregulated stem cells. A blood infection can kill or cost many hospital days and teams of specialists to manage. Organ failure can occur with blood infections.
3) Please provide examples from the medical field where FDA has inserted itself into the delivery of a single medical procedure in an individual beyond testing for communicable diseases in transplants.
PK Answer: This is a political question and has nothing to do with my expertise as a scientist. The FDA is involved with many aspects of medical care for instance testing of the device, equipment, and lab cleanliness during an intervention. There is a free CITI course online (https://www.citiprogram.org/irbpage.asp?language=english ) about FDA, ethics and past decisions and why they are made. You as a group may want to take this so you can learn more about how the system works and be more informed and more powerful contributors. This could make you more effective in stem cell activism or to build bridges to stem cell care.
There is the case of Regenerative Sciences, Inc and now Celltex. Do you mean besides stem cell examples? I believe the FDA has inserted itself into many other cases as well over the years unrelated to stem cells, but that is not an area I am deeply familiar with.
Other issues that arise that in my opinion necessitate FDA involvement are poor laboratory practices that put patients at risk not necessarily from the stem cells themselves, but from infection or immune problems.
4) As a patient, I call foul! I want stem cells - been waiting for years as multiple sclerosis destroys me. I actively read everything about stem cells and I know already of your blog. You state, "I'm a stem cell researcher and cancer survivor who has no monetary interest in the success or failure of any stem cell clinic......". A simple Google search shows that you repeatedly slam MD's in your blog who want to treat patients with adult stem cells. Why do you want to dictate what my doctor and I decide to do in the privacy of the consultation room? They transplant stem cells for cancer already. I'm sick of waiting! An MS researcher said in 2000, it would be 5 years--I'm still waiting! And you are all over the internet talking about danger when adult stem cells are given everyday for cancer and have been for years. Why do you care if I get them for MS? The truth is that you are not unbiased. Your research is related to science where people get pluripotent stem cells that have been manipulated by you rather than natural cells. Having patients get their own stem cells right now before your research is done might slow your funding. My own stem cells are not a Pharma product. They are mine. What do you have to say about all this?
PK Answer: Patients have rights and deserve respect. I totally agree with you on that. At the same time society has a responsibility to do its best to keep medicine as safe as possible and to help patients make fact-based decisions. I believe that stem cell scientists have an extra responsibility in that area. As I said in another answer, the first rule of medicine is “do no harm”. It is not at all clear that stem cell transplants of in vitro propagated stem cells are safe (my great hope is that they are) so I’m concerned that unregulated transplants could do a great deal of harm. American society has decided that what happens between a patient and a doctor is in fact not entirely independent of the law of the land and of regulatory agencies. I think this decision is wise and to the best interest of patients. Doctors cannot just do whatever they feel like to patients even if the patients sign off on it.
Why do I care if you or anyone else gets unlicensed stem cells for MS? My concerns are (A) that to my knowledge there is no scientific or rigorous medical evidence that stem cells work for MS and (B) there is no proof that they are safe. You seem suspicious of my motives, but I can think of no personal gain for me in my efforts to help make stem cell-based medicine safer and educate people. Whether patients get (or do not get) stem cell transplants of their own stem cells has no influence on my grant funding whatsoever. That’s the reality. The doctors selling the unlicensed stem cells of course have a very direct, obvious vested/conflicted interest because they want patients’ money.
You say “I want stem cells” and “they are mine”, but once a clinic isolates your stem cells and grows them, they become a biological product that is different than the stem cell in your body. How are they different? They acquire mutations. They change their antigens on the cell surface possibly sparking immune reactions. They grow faster. They may respond to hormones differently. The list goes on and on.
5) Do you feel that the FDA and PhD scientists such as yourself can make better decisions for patients regarding stem cell therapy, as opposed to the decisions I make with my MD?
PK Answer: Different parties bring different strengths and weaknesses to the table in medical decision making. I’ve spent half my life growing cells and studying them including stem cells over more than a decade. I feel confident in saying I know far more about cell biology and stem cell biology specifically than most MDs. That doesn’t mean I know everything, but I know cells really well. Doctors of course know medicine far better than I do, but they may have some major gaps in knowledge about cells. In addition, when stem cells are offered in a for-profit setting, things get complicated for the MDs involved because of mixed motivations.
In my opinion responsible MDs conduct medicine according to the rules of the land in which they are practicing. If they do not agree with the rules, they do not violate them in their for-profit care of patients, but rather continue to follow the rules while at the same time working to change them. I do not see doctors who break the law as some kind of Robin Hood-like heroes helping patients. A patient and their doctor are not equal partners in decision-making because the doctor has taken an oath to protect and care for you, yet s/he is making a profit off of you by giving you an unapproved, potentially dangerous treatment.
6) On the basis of compassionate use, how can you argue that I can't have access to autologous stem cell therapy, when my other option is lingering miserable death in a nursing home from progressive MS which is non-responsive to any of the FDA approved MS drugs? There are no stem cell trials for progressive MS, so I think that I should be free to utilize any commercial stem cell clinic in the U.S. that I determine has a good and effective service, without interference from the FDA.
PK Answer: I am not an expert in compassionate use, but I understand that it may be appropriate for very specific circumstances. I would, however, question physicians who try to make a profit off of giving “compassionate use” treatment to patients. According to this American Cancer Society webpage (http://www.cancer.org/treatment/treatmentsandsideeffects/clinicaltrials/compassionate-drug-use ), compassionate use is legal only in two circumstances: expanded access (the drug in question must be in Phase III trials, while stem cell drugs are not that far along) or Single Patient Access, in which the patient and doctor must ask the FDA for permission to use the drug. Neither case seems to apply to for-profit clinics offering stem cell treatments that are unlicensed. Note that stem cell clinics and doctors are fighting to avoid stem cells even being defined as drugs. If stem cells are not drugs or medical devices, then to my knowledge compassionate use does not legally apply.
7) Do you hold any patents for your work with stem cells? Do you not see it as a conflict of interest that you are a researcher who depends on research work not just for your own job, but for your university? The dollars must keep flowing and if you position yourself as an industry "expert", then this has to benefit you and your university. If you were to advocate for patients to be able to get treatments as a practice of medicine, your research would be diminished somewhat. The problem patients are facing is that your research and that of others in the field, is not available to patients now and many do not have the luxury to wait years. Why not embrace a stance that helps patients now as well as keeps your own research into riskier stem cell types going as well? It seems that a happy medium would be the best approach rather than condemning sick people to no hope which is basically what you and others are doing.
PK Answer: I have no patents nor patent applications. I do not believe I have a conflict of interest on stem cells. To my knowledge, my funding is not related to my work as a patient advocate and educator on issues of stem cell safety. I have no grant funding for that work. I disagree with your statement about my hypothetical advocating for patients to get treatments diminishing my research. When what I have worked on scientifically becomes a viable medical intervention I will be overjoyed. Where there are results that are evidence based, funding is increased not decreased. Patents are normally held by the lab industry, academic institutions, or organization. Very little if any of the patent benefit gets to the inventing scientist.
8) What on earth does a bioethicist know about my illness? Do you feel that Doug Sipp, who claims to be a bioethicist is qualified to be someone who should be cited by the media as an industry spokesperson? I think patients are getting tired of the type of attacks that some in the industry make who seem to not be qualified or who have very obvious conflicts of interest. This diminishes the impact that true scientists might have in the eyes of patients when the scientific community makes little effort to police itself. There seems to be a lot of retractions and researchers in the news lately too that may have faked data. It's truly upsetting to watch the ISSCR and some in the scientific community condemn most doctors and clinics who want to treat patients while looking the other way at those in the scientific community who need to be called on the carpet.
PK Answer: This is simply a personal attack and an attack on the stem cell field. I believe that Doug (who by the way does not refer to himself as a bioethicist) has something valuable to offer to the dialogue. So do others including Dr. Centeno, ICMS, and Celltex. I organized a debate between Doug and Dr. Centeno on my blog that hundreds of readers found valuable. I also have engaged ICMS via an interview with their President, Dr. Rodriguez. I have personally met with Guv Rick Perry of Texas to talk over stem cell issues. I have invited Celltex to post on my blog. I regularly talk/email with folks who do not see eye-to-eye with me on stem cell issues, but we have productive, respectful dialogue. I believe in building bridges, but with accountability.
9) How is it morally responsible for patients to be denied access to treatments that use their own body parts when these patients have little or no quality of life? Why should someone from a government agency, a university, a company, etc. be the one to hand out a death sentence and expect that the patients should be grateful that this has somehow "protected" them? I think many assume that all patients are simply those who aren't really that ill, don't have a progressive disease and maybe just want to try stem cells because they blew their elbow out pitching balls. The dilemma those of us who are seriously ill now face is going offshore or giving up - thanks to Obama's FDA commissioner and those in the industry who are putting their own interests above patients. I just don't see how some people sleep at night knowing this. There were patients scheduled for treatment at Celltex who are now devastated by the FDA's decision. They were notified that their treatments were cancelled, knowing others that had already been treated were seeing a lot of improvements. Do you have any idea what that does to a person?
PK Answer: By law the FDA is required to regulate stem cells that are grown as drugs at this time. This is the law of the land in America.
There is no scientific evidence that stem cells of this kind actually do patients any good and there are very real risks. For-profit companies are first and foremost in the business of making money. If the FDA was the problem these cells would be demonstrably working in other countries and medical journals would be full of this evidence. This is not happening in any country with or without an FDA.
By way of background, I do personally know what it is like to be a patient with a potentially fatal illness. I was diagnosed just about exactly three years ago with a very serious form of prostate cancer unexpectedly at age 42. I had surgery and I am doing well now, but I know that at any time the cancer could come back. Prostate cancer can also become untreatable at some point in a patient’s life.
Over the last few years I have also talked to dozens of patients who are considering stem cell treatments for various very serious health problems for which today’s medicine has little to offer. I respect them and their concerns, but as I tell them, stem cells are not some kind of magical panacea. They are exciting and powerful, but we have to be careful. Just like any other medicine they must be tested in pre-clinical studies for safety and their use must follow federal and state law. Otherwise they can do more harm than good. Physicians and lawyers of for-profit companies who say pre-clinical and clinical trials (non-profit) are unneeded are, in my opinion, mostly out to make profits and do not want to spend the time and money to do this right.
10) How if your position opposing stem cell clinics offering stem cells as medical care before randomized controlled trials for an indication c/w this statement in the most recent version of the Declaration of Helsinki? Do you support clinics that follow the general guidelines discussed in Helsinki?
35. In the treatment of a patient, where proven interventions do not exist or have been ineffective, the physician, after seeking expert advice, with informed consent from the patient or a legally authorized representative, may use an unproven intervention if in the physician's judgement it offers hope of saving life, re-establishing health or alleviating suffering. Where possible, this intervention should be made the object of research, designed to evaluate its safety and efficacy. In all cases, new information should be recorded and, where appropriate, made publicly available.
PK Answer: Notably, the FDA and NIH have rejected the Declaration of Helsinki for many years for numerous reasons. One concern in particular has been the perceived lack of patient protection. The American Medical Association has had many problems with the Declaration over the years in its different iterations as well.
I do not unconditionally support this one very isolated part (quoted above) of the Declaration of Helsinki taken out of context. I suggest that you read and study the agreement for yourself. Please note that clinics that refuse the FDA pathway are not evaluating safety and efficacy as indicated in that passage. They are making up their own rules.