Jun 04 2008
Dr. Geeta Shroff is an Indian physician who is running a New Delhi clinic offering embryonic stem cell therapies for a large number of various medical conditions. The only thing these medical conditions have in common is that they are incurable. Indian law allows for the use of unproven treatments for terminal or incurable diseases. I cannot know Dr. Shroff’s intentions, but she has rejected the ethics and standards of science-based medicine and in so doing has transformed herself into a dangerous charlatan.
Embryonic Stem Cell Therapy
Embryonic Stem Cells (ESC) are controversial because of the ethical and moral consideration regarding harvesting ESC and the rights of an embryo. But that is not what makes Dr. Shroff’s treatments controversial, and not what I am going to write about here. The question, rather, is the state of the science of ESC therapy.
ESC’s are scientifically interesting because they have the potential to turn into any type of cell in the body. The hope for ESC therapy is that they can be used to replace dead or abnormal tissue in the body, something which is not now possible for many conditions. (Organ and bone marrow transplants are among the current treatments to replace failing tissue.) For example, an injured spinal cord might be repaired by using ESC’s to replace the damaged motor neurons and reestablish a connection between the brain and muscles. Atrophied muscles themselves can be repaired by having ESC’s turn into working muscle cells.
The potential for ESC therapy is amazing, and it is therefore no wonder that it is capturing the imagination, and therefore the hopes, of many who suffer from a disease or injury that ESC therapy might cure. However, for now the hope is only that. ESC therapy requires the development of a tricky new technology, and we haven’t done it yet.
In order for ESC therapy to work we have to figure out how to properly harvest and prepare the cells, how and where to inject or transplant them, and then how to control them once they are inside the host. Controlling ESC’s may be the most difficult challenge. We need the cells to turn into the right kind of cells, and in some cases to also make the right kinds of connections to other cells and tissues. Also, we have to make sure the host doesn’t reject the transplanted cells – just like any transplant. And then there is the potentially biggest problem with ESC therapy – how to keep the transplanted cells from growing out of control and becoming a tumor.
Until all of these issues are worked out, simply injecting ESC’s into a patient may be worthless, or it may even be harmful. Tumors and rejection are harmful things.
The Ethics of Experimental Treatments
Dr. Shroff acknowledges that her treatments are experimental, but she insists that they work. This, of course, prompts the question – how do we know that the treatments are effective, or safe, for that matter?
There is general agreement that it is ethically reasonable to give low probability experimental treatments to patients who are terminally ill and/or incurable (so-called compassionate use). But this does not mean that anything goes. There are strict ethical guidelines for experimental therapies that have evolved over decades and are very thoughtful, compassionate, and practical.
Ethical use of experimental treatments includes the notion of informed consent – which means that the patients need to know that what they are getting is unproven, in some cases they may be getting a placebo, and they need to know everything currently known about the potential risks and benefits, and how they are going to be monitored.
Further, there needs to be the potential that the experimental subjects will be helped by the treatment, and there should be a reasonable expectation that the subjects are more likely to benefit than to be harmed.
It is unethical to lure patients with the hype of a false promise in order to induce them to spend thousands of dollars on an experimental treatment. Dr. Shroff says, “I do not give any guarantees,” but this is not sufficient. Patients are not looking for guarantees – they are looking for hope. In a separate interview Dr. Shroff indicates that she understands this, saying, “My patients often have no other choice. I am their last chance.” Giving them false hope with a lame disclaimer is not ethical.
The Need for Transparency
Science cannot function without transparency. Since effective regulation of health care requires reliable scientific information -regulation cannot function without transparency. The most severe problem with Dr. Shroff’s claims is the complete lack of the usual clinical and scientific documentation – the lack of transparency. We have no idea what she is injecting into her patients, under what protocol, and how they are responding.
Dr. Shroff does not have any legitimate excuse for why she is not following proper scientific protocols. I can only think of one reason – to hide her own fraud. She only says, “Everything I have done has been notified to the Indian Council of Medical Research and there has been no comeback.” But this response is a dodge – she is saying that she is in compliance, but the rules in India are particularly lax in this situation, and the money she is bringing in is a conflict of interest. She does not have an answer for the deeper scientific and ethical questions.
Dr. Shroff claims that she has treated over 300 patients, almost all of them have improved without any side effects. If this sounds too good to be true, it’s because it probably is. But she is asking the world to rely upon anecdotal evidence, which is completely unacceptable for such a claim. Unless side effects are systematically checked and recorded, we really cannot make any statements about the safety of her treatment.
Several cases have been presented through the media as apparent success stories, but they are all completely unconvincing. Their alleged improvements are all mild – quadraplegics who now have some tingling sensation in their legs, or patients who can walk a little better. One patient could breath but not adequately to be independent from a ventilator, and now claims he can breath without the ventilator. All of the cases sound superficially impressive, but they all represent minor changes in actual neurological function.
Legitimate researchers who study neurological disorders are keenly aware of what is called the “cheerleader effect.” This is related to the difference between neurological recovery and functional recovery. In order to say that a treatment is working we need evidence to conclude that there is some neurological recovery – more neurons are connecting more effectively to more muscle tissue, etc. But if all one is observing is net function – the ability to breath and walk – pure functional recovery must also be taken into consideration. What this means is that, even without greater neurological function, patients may have a functional improvement because of the non-specific effects of trying harder (the cheerleader effect) and physical rehabilitation. Even someone who has been paralyzed for many years, if you get them out of their chair onto the parallel bars, or you have them go through a series of exercises and try really hard to walk better – they will. This will happen to a certain degree even if the treatment has zero effect.
The cheerleader effect causing purely functional improvements is a factor that must be considered in all clinical research into neurological diseases and injuries that impair function, from strokes to spinal cord injuries. In order to control for such effects treatments have to be assessed in a blinded fashion, and multiple quantitative measures of function should be used, including measurements of pure physiology where applicable, to show neurological improvement beyond the nonspecific effects of exercises and effort.
What this means is that anecdotal observations of such treatments are highly misleading and are almost guaranteed to produce false positive results – the false appearance of an effect. The stories coming out of Dr. Shroff’s clinic appear to be such cases.
There are also serious concerns about the safety of her treatments. If she is indeed injecting stem cells into her patients there is no indication that she is taking the steps necessary to prevent rejection, or to prevent the transplanted stem cells from becoming tumors. Her anecdotal reassurances are not sufficient.
What if her treatment actually works? While this is doubtful, if this were the case then her failure to provide proper documentation and perform standard scientific testing is depriving the world of an effective treatment. If she has somehow discovered the secret to get stem cell therapy to work today for the diseases she claims she can treat, then she should share it with the world.
Sharing her methods would not only make them available to more people but it would allow medical scientists throughout the world to evaluate her treatments and perhaps figure out a way to make them even better.
There really is no legitimate excuse for the lack of transparency with such an experimental treatment. There is nothing Dr. Shroff can say in her defense – her behavior is unacceptable.
Unfortunately Dr. Shroff is not the only doctor who has set up a dubious clinic charging thousands of dollars to desperate patients for the premature hope of stem cell therapy. Dr. Huang in China has a similar clinic with equally dubious ethics and results. An independent evaluation of some of his cases revealed that his patients do not benefit from his treatments at all (despite their belief that they did benefit) and that many of them were harmed by the treatment due to complications such as infection.
Patients should be very cautious before traveling half-way around the world and spending thousands of dollars on any controversial stem cell therapy. Clinicians need to be aware of these clinics so that they can properly inform their patients about them. The media (which has been very mixed) needs to be careful before reporting about controversial medical claims. All media reporting, even when negative, tends to drive the desperate to such clinics. Wishy washy reporting built around glowing anecdotes from patients is the charlatan’s best friend. It is not enough to include “token skepticism.”
In the end it is likely that the only effective way to protect patients from such clinics is through proper regulation. But this is extremely difficult as such clinics are likely to find safe haven in some part of the world, and patients who are willing to spend thousands of dollars for the hope to walk again are likely willing to travel as well.
All we can do is fight on all fronts and try to minimize the damage by the Dr. Shroff’s of the world.
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