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- Anthony N. DeMaria, MD, Editor-in-Chief, Journal of the American College of Cardiology⁎ ()
- ↵⁎Address correspondence to:
Dr. Anthony N. DeMaria, Editor-in-Chief, Journal of the American College of Cardiology, 3655 Nobel Drive, Suite 630, San Diego, California 92112
That investigators should be extremely enthusiastic about their research and the importance of the findings that it yields is to be fully expected. How could they devote themselves so completely to the uncertain outcome of the pursuit of new knowledge if they were not passionate? It is also completely predictable that the lay press would be highly motivated to inform the public about any medical discoveries. The attention that new health care innovations can attract is obviously very desirable. It goes without saying that the lay public, especially those who might be affected by any new breakthroughs, would be highly receptive to that information. New developments in medicine are a source of security to the well and a beacon of hope for the ill. The result is that the media frequently presents new research findings to the public in the best possible light and in terms of the greatest potential benefit.
The line that separates appropriate presentation of discoveries that generate reasonable hope from exuberant depiction of research findings that induce unrealistic expectations (or hype) is often blurred. This blurring may be influenced not only by the presentation but also by the reception of individuals with serious disease who are particularly susceptible to grasp for remedies. The failure to distinguish rational hope from unreasonable hype can have significant consequences; patients can deny themselves effective therapy, expose themselves to serious side effects, and be encumbered by substantial expenses.
The area of medicine that perhaps best currently typifies the problems in differentiating hope from hype involves stem cell therapy. A variety of debilitating disorders exist for which no cure is possible and even palliation is difficult. In terms of cardiovascular disease, the inability to correct or enhance the function of destroyed myocardium remains one of the last major hurdles to overcome. We have artificial valves, coronary stents and bypass, implantable cardioverter-defibrillators, and so on, but we are powerless in the face of myocardial scar. So, it is not surprising that the notion of regenerating myocardium with stem cells stimulates enormous excitement in both physicians and patients. When preliminary experimental and clinical results suggest that stem cell therapy is feasible and may be beneficial, the findings are optimistically broadcast to the public and stoke the fires of enthusiasm. The fact that it is a daunting challenge to achieve the delivery, engraftment, survival, and effective functioning of cells safely is sometimes lost in the transmission.
The results of exuberant optimism and unrealistic hope can be seen in the numerous stem cell clinics that are operative around the world. A quick search under stem cell therapy on Google reveals over 16 million entries and over 20 pages of listings. Included are clinics offering umbilical, adipose, and bone marrow stem cells in Asia, Europe, and other locations for cardiac and other conditions. One site advertises that they have treated over 500 cardiac patients. This is despite the fact that the consensus of the scientific and medical communities is that the efficacy and safety of stem cells is unproven at this time. In nearly every case, the fee for the services is substantial, and since stem cell therapy is not approved in the U.S., these patients must pay for travel and lodging. The long-term potential of serious adverse effects remains undefined.
The initial reaction to these many stem cell clinics is to suspect financial motivation. In fact, history is replete with examples of medical ruses for unproven therapy foisted upon desperate patients. So, it would not be surprising if some clinics primarily represent an attempt to acquire large sums of money. Such clinics would, of course, be contemptible. However, it seems clear that there are a number of hospitals, clinics, and physicians who feel that sufficient data exist to justify the attempt to treat incurable diseases with stem cells. In some cases, the physicians may have convinced themselves that the benefits of stem cell therapy are established, while in others they may be uncertain but feel that they must act while definite evidence of efficacy is obtained. Nevertheless, even here judgment is questionable. Attempts at regenerative therapy carry significant potential adverse effects such as teratomas. Moreover, since the treatment given is uncontrolled and nonrandomized, almost no useful data or guidance regarding efficacy or safety will be obtained. Thus, patients are being administered unproven therapy at some risk and expense without significant benefit even to society.
While I disapprove of the commercial provision of stem cells, as I suspect most physicians involved with this field do, I suppose I cannot absolutely condemn it. I recently had an interaction with an ischemic cardiomyopathy patient who described Class IV symptoms despite maximal medical therapy. While searching the Internet she came across a clinic in Asia that offered stem cell therapy for heart failure. She was provided with the contact information of a number of prior patients, all of whom reportedly spoke intelligently of the process and the benefits of treatment. Although her husband (a lawyer) thought she was “crazy,” their physician son opined that she had little to lose, and so they traveled to obtain the stem cells. The treatment was supervised by an American-trained physician, was administered in a “spotless” hospital, required 2 weeks, and cost $40,000 to $50,000. Although there was no immediate effect, the patient stated that her exertional capacity gradually improved over 6 months and has remained so for 4 years, such that her husband and son have become believers. The woman is ever so grateful for her progress, has never spoken to a patient who did not improve after receiving stem cells at this hospital, and would go back for additional treatment if she were able. While I have no idea of the nature or the mechanism of such improvement, and obviously cannot rule out a placebo effect, I cannot question that the patient is happier and less symptomatic than before treatment. In this regard, the nature and mechanism of benefit are probably not of great relevance.
Given the circumstances of investigators and patients, it is perhaps not surprising that there is often a thin line between hope and hype. In my opinion, as researchers we have the responsibility to temper overly optimistic interpretation of our findings; as clinicians we have the responsibility to only administer therapies of established value except under investigational protocols. Stem cell therapy represents an issue where the enthusiasm is very high and the medical need very great. The result has been a plethora of worldwide medical facilities that administer stem cell preparations of unproven value to patients with debilitating disorders. Not only is such therapy based upon unfounded expectations, it is expensive and does not move us closer to an understanding of the true efficacy of the treatment. Were stem cells free of any significant side effects, I would agree that desperate patients should be entitled to try any remedy possible; clearly, there are patients that have benefited subjectively after such treatments. However, the safety cannot be assured at this time. Therefore, I think it is our responsibility to make sure that our patients have all the facts available, and that we do everything possible to ensure that the claims made by these clinics reflect these facts. We have an important role in preventing hype and helping our patients distinguish hype from hope.
- American College of Cardiology Foundation