Q: You are taking care of a 61-year-old man you diagnosed with amyotrophic lateral sclerosis (ALS) nine months ago. His daughter has been searching the Internet for new treatments and found a clinic in a Central American country offering stem cell therapy for ALS and other diseases. They have both heard many popular media accounts of stem cell therapy, are impressed with its wonders, and have concluded that he should have it. Patient testimonials on the clinic's website are enthusiastic, reporting improvement in ALS symptoms or remission of disease. The clinic requires wiring $40,000 in advance to cover the costs of therapy. Because the treatment is not covered by health insurance, the patient plans to sell his house to raise the money, and move into an apartment. How would you advise him?
JAMES L. BERNAT, MD, RESPONDS: Most neurologists have cared for at least one patient with a terminal or chronic disease who decided to undergo an unproved, unorthodox, and expensive treatment advertised by a non-establishment clinic, often in another country, and usually requiring cash payment in advance. Who could blame the patient and family members for pursuing a promise of cure or remission?
In the face of a terminal illness, some patients and their families conclude that they must blaze their own path to a cure because the medical establishment cannot provide it. They search the Internet for Web sites and communicate with other patients and family members about possible treatments. They are motivated by the desperation of a fatal disease, the limited efficacy of accepted treatments, the slow pace of therapeutic research developments, and their own dedicated desire to find a cure. They are therefore vulnerable to promises or claims of successful treatment made by unorthodox clinics. They may figure, what have I got to lose? As in this case, they often borrow money or sell assets to secure the payment for treatment.
Several prevailing cultural beliefs promote their search for an unproved cure. First, is the American maxim that action is better than inaction, so doing something is better than doing nothing, even if that something is ineffective at best and dangerous at worst. Further, people tend to believe that natural or “organic” treatments are healthier than artificial treatments. Thus, treatment with plant products, vitamins, or human cells is better prima facie than treatment with a synthesized chemical. Some therapists offer a plausible but unproved scientific rationale justifying particular treatments, such as bee venom therapy for immune-mediated diseases, that patients may intuitively accept as logical because they know that it induces an immune response.
For their own marketing advantage, some therapists exploit the hype generated by the media over promising but potential therapies, such as human embryonic stem cells, as in this case. Some therapists challenge the diagnosis of ALS and claim that, instead, the patient has chronic Lyme disease that can be cured by months of intravenous antibiotic therapy. Finally, some clinics exploit the paranoid fears prevailing in some circles that to prevent cure, organized medicine and science have colluded to suppress important research results showing the effectiveness of certain therapies that these clinics offer.
Despite the intuitive claim “what have I got to lose?” patients may become victimized by several harms resulting from pursuing unproved and unorthodox therapies. First is the cruel hoax of false hope that will become obvious only after the money is spent and the treatment is completed that fails to alter the disease course.
In counseling chronically or terminally ill patients, physicians have the daunting task to provide medical advice that is realistic but does not extinguish hope. Second, unproved treatments may displace more effective established treatments or render the patient unavailable or ineligible to participate in legitimate clinical trials of experimental therapeutic agents. Third, harm to the patient may result from drug toxicity or acquired infections transmitted by therapies they receive in an unapproved and unregulated clinic. Cases of hepatitis B, HIV, and other infections have been reported following intravenous therapy, as have instances of hepatotoxicity and nephrotoxicity from various injected or ingested substances. Finally, the sharing of experiences of unorthodox therapies through patient Web sites and social networks may encourage other desperate patients to seek these treatments.
How, then, should a neurologist respond to the patient's decision? The optimal approach requires compassion, understanding, tolerance, and patience. It is time consuming and may be frustrating. The neurologist should calmly acknowledge that the patient's motivation stems from a sense of desperation and no good treatment choices.
Acknowledgement validates the patient's reasons for considering the unproved therapy. The neurologist should offer to read the clinic Web site and other literature and discuss the merits with the patient and family. The willingness to read the material shows them that you take their plan seriously and are willing to spend the time to review it. It diminishes the development of an adversarial patient-physician relationship that might occur if the neurologist were to summarily dismiss the treatment as unproved, ineffective, or ridiculous. By maintaining thoughtful and respectful dialogue, it keeps the patient and physician on the same side of the battle against the enemy – which is the disease.
The neurologist can objectively explain what is known scientifically about the claims. This opinion can be buttressed by expert reviews on the subject by the medical advisory committee of the ALS Association (or MS Society or other relevant professional society). In nearly all such cases, there has been inadequate, if any, scientifically valid evidence of therapeutic efficacy. In the same calm, impartial, and objective manner, the neurologist can itemize the potential risks to the patient as noted above, including drug toxicity and infection, given the questionable quality control and general absence of regulation by agencies that protect patients' safety when treatment is administered in the United States.
An important element of the conversation is for the neurologist to offer an alternative treatment to the unorthodox one. In addition to disease-altering medical treatments such as riluzole, and symptomatic treatments, neurologists can educate their patients and help them enroll in a valid clinical trial of a new therapeutic agent.
One virtue of enrolling in a clinical trial is the opportunity to receive an innovative and possibly effective pharmaceutical agent before it is available to the public. An often equally valuable benefit of enrolling in a clinical trial is the patient's ennobling experience of contributing to scientific advances that could help other patients. The opportunity to volunteer to further science and help future patients mitigates the patient's suffering by creating meaning from an otherwise devastating and meaningless illness.
Most important is for the neurologist to maintain willingness to continue to treat the patient, even if the patient chooses to undergo the unorthodox therapy. The devotion to stick with the patient throughout the illness cements the patient-physician therapeutic bond and adds credibility to the neurologist's advice discouraging the patient from undergoing the questionable therapy. A strong patient-physician relationship requires physicians to respect patients' autonomy, even to make bad decisions, and is as powerful as any therapy.
Dr. Bernat is professor of neurology and medicine at Dartmouth Medical School. He is the former chair of the AAN Ethics Law & Humanities Committee and is the author of Ethical Issues in Neurology, 3rd ed. (Lippincott Williams & Wilkins, 2008).
van den Noort S. Ethical aspects of unproved therapies in multiple sclerosis, amyotrophic lateral sclerosis and other neurologic diseases. Semin Neurol
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Bernat JL. Ethical Issues in Neurology, 3rd ed. Lippincott Williams & Wilkins, 2008, pp. 342-343, 374-375, 449-450.