December 25, 2005
By Stephen L. DeFelice, M.D.
During 1965 I conducted my first clinical study and made my first medical discovery. I found that carnitine, a naturally occurring substance, was very effective in the treatment of hyperthyroidism. This specific discovery piqued my interest in the general subject of medical discovery. I asked myself, “How are new cures and helpful therapies discovered, and what can we do to speed them up and get them to patients?”
A few years later, while at the Walter Reed Army Institute of Research, WRAIR, I, along with my colleague and friend, Major James Vick, made other medical discoveries, which impact, in large part, led me to launch a career to search for ways to accelerate medical discovery.
I learned then that one of the great obstacles to finding new cures and remedies is the extreme difficulty both in initiating and conducting clinical studies. ShouldnÃt it be obvious that, in order to discover the medical value of insulin, penicillin or a new drug that will cure prostate or breast cancer, it is necessary to test the these medical therapies in patients with diabetes, specific infections and those particular cancers? And, shouldnÃt it be obvious that the more barriers to testing new therapies in patients, the fewer medical discoveries will be made?
Right?
Wrong!
When I first began to express my concern for patients (we are all patients; those of you that believe you are ìhealthyî are misguided; your body is constantly being assaulted by disease causing factors that have not yet manifested themselves because the bodyÃs defenses keeps beating them down), I spoke to a number of professionals in the medical, scientific, journalistic, and political sectors. I ran into a thick, intellectual and emotional stone wall. Not one grasped the message nor felt it important to even substantially delve into the matter. The concept was foreign to their mentality, and there was also a feeling that clinical research was somehow a necessary evil.
Undaunted and with the optimism of youth, I decided to write the book, Drug Discovery: The Pending Crisis, in an effort to educate our country about the importance of the clinical research ñ medical discovery connection. It was published in 1972. I studied very hard in preparation for my first media tour. And guess what happened?
History repeated itself! The media not only did not grasp the message but, instead, almost totally dwelled on the risk involved in clinical research. They, sometimes subtly and sometimes not, treated me as if I were one of Hitler’s or StalinÃs doctors of torture. I donÃt recall anyone who supported or pursued the benefits of clinical research.
Unfortunately, the barriers to clinical research continued – and continue – to rise unabatedly.
During 1976, I came up with what I thought was a brilliant idea. I decided to establish the nonprofit foundation, FIM, the Foundation for Innovation in Medicine, whose primary purpose is to educate the mass media about the importance of clinical research. After all, the mass media has an extremely high level of interest in health matters and would surely take to this message. Then, I believed, after the media saw the light, they would create a ìmighty wave of public opinionî necessary to convince Congress to enact new laws to effectively diminish the barriers to clinical research and spur the discovery of new medical therapies.
But I ran into the same 1972 stone wall affirming my initial experience that the concept of the importance of clinical research is nowhere near the national radar screen, let alone the media’s.
Attempts to obtain contributions for FIMÃs mission reflected the absence on the radar screen. Since FIM was formed to the present (2006), I would estimate that a grand total of about $25,000 in contributions has been donated ñ less than $1,000 a year. Early on, aware that this would be the case, I decided I had no choice but to personally fund FIMÃs effort until Congress got the job done. This included the employment of multiple public relations firms, conferences, books, position papers and other media vehicles. Yet, despite this vigorous effort, the media was stubbornly and inexcusably resistant. As a result, millions of patients have paid a large price by dying prematurely and suffering unnecessarily because of the lack of new therapies that could have been, if the media did their job. I was ready to surrender the effort but then I came up with, what I thought, was another brilliant idea that would undoubtedly arouse the interest of the media and move Congress to act. It was the NREA or Nutraceutical Research and Education Act (visit the NREA section on the FIM website).
Close to one hundred per cent of the health-medical claims of the products sold in health food stores and the supermarket have not been clinically proven to work, and I often wonder why the media has never mentioned this critical fact. Unlike pharmaceuticals, where drugs are proven to be effective and acceptably safe in a number of clinical studies and are approved by the FDA based on the results of the studies, only a very, very limited number of studies have tested whether these health-medical claims are true.
Well over one hundred million Americans swallow dietary supplements daily convinced that they will either prevent or treat disease such as cancer or heart ailments. Over fifty per cent of folks who visit food supermarket read the labels as if they expect to discover that the product bought offers a proven health medical benefit. And the mass media frequently and powerfully supports these claims and dupes the consumer despite the cold truth of the absence of clinical studies to support these claims.
LetÃs return to the NREA: In 1989, after a wonderful meal topped off with a smooth grappa, I took a leisurely stroll in Piazza Navona in Rome, where I then coined the term, ìnutraceuticalî which is now in WebsterÃs Collegiate Dictionary (visit Library News Clippings on the FIM website and read William SafireÃs New York Times article On Language). A nutraceutical is a dietary supplement or food, including special diets, that has been proven in clinical studies to have some type of health – medical benefit as well as being acceptably safe. The next morning I came up with the concept of the NREA which is designed to encourage companies to sponsor clinical research on their products and, very importantly, to have the right to make the health-medical claims based on the results of the study. Poll after poll, including FIMÃs, found that the vast majority of responders were apprehensive about the effectiveness and safety of the products consumed and welcomed clinical research as a solution to their concerns.
FIM then, over the course of almost a decade, held a series of high level nutraceutical conferences mainly in Manhattan, the media center of the world, where we expected heavy media coverage. FIM made a substantial investment in its public relations effort to attract mass media to attend the conferences. Congressman Frank Pallone (D-NJ), convinced of the importance of the NREA, introduced the Act in Congress which, if passed, would have immensely benefited consumers.
The result? With perhaps two exceptions, no mass media folks attended and, as a result, there was no media coverage necessary to support the passage of the NREA! What puzzled and disturbed me the most was that virtually all of the mass media folks were only a cab ride away and didnÃt have sufficient interest to pay the fare. This was enough to convince me that, unless something unusual happened, any effort to convince the media of the enormous importance of clinical research was destined for failure.
After the NREA effort, I must confess, that I was about to throw in the towel until I had dinner with Senator Bill Frist, himself a physician as well as an experienced clinical researcher, and spoke to him about doctornauts (another word that I coined) and the Doctornaut Act.
A doctornaut is a physician who has the right � they currently do not have this right � to volunteer for clinical research studies with fewer restrictions and take greater risks than non-physician volunteers. If we had doctornauts, we would study many more new therapies within a short period of time. In my 1972 book I extensively dealt with the critical importance and rationale for physician volunteers.
Dr. FristÃs eyes literally sparkled, and he asked me to, “Work it up.” FIM then published The Doctornaut Act Proposal calling upon Congress to enact this law. I decided, based on my past experience, that any major publication relations effort would not succeed. On the other hand, I had made, over the years, many connections with the media, and I contacted a number of them. History repeated itself. They didnÃt catch the concept and blank faces prevailed.
I was then asked to find supporters of the Act. I contacted members of the upper echelons of major medical and scientific organizations. I met with dedicated children advocate groups, some of whom I know very well, whose mission is to find new medical therapies for children with diseases and disabilities. As an example, I explained to them that a drug that cured leukemia in an eighty year old doctornaut will also cure leukemia in a child. The reaction? They didnÃt catch the concept and blank faces prevailed.
When there is a wonderful opportunity to reduce premature death and unnecessary mass suffering of patients that is being ignored, it is an unequivocal moral duty and humanistic responsibility of media to alert our country about it as they did, for example, with the Orphan Drug Act.
The first step is for the media to harken to the words of wise old Socrates, gnothi seuton. The following section of the Doctornaut Act Proposal may help the media know something about themselves.
Over and above our national fear and suspicion of clinical research, we have an inexplicable national complacency about medical discovery itself. For example, it is almost the rule to read or hear such messages in the media as, “An effective new gene therapy has been found but it will take years of testing”, or “New drugs fight aging in animals but such developments for humans are years away,” or “New drugs for ParkinsonÃs disease are expected to take years of testing” or “A new protein to treat skin cancer has been tested and may be ready for humans someday.”
Why does it take so long for promising scientific research to be translated into effective medical treatments? Moreover, why doesnÃt anyone in the media ask the question, “Why?”
By far, the most common question asked by my media colleagues is, “WhereÃs the story?” Well, “Here’s the story”. Modern technology, as never before, offers us definite opportunities to increase our medical weapons against disease and disabilities if we can more easily test them clinically. And doctornauts are the most effective way to get it done. With tens of millions of patients needlessly suffering and prematurely dying ½ and a solution with a high probability to significantly help these patients ½ if thatÃs not a story, what is?
It is difficult to count the number of times that I’ve been asked why our country, particularly the mass media, has a blind spot regarding clinical research. My answer has been that I donÃt know, but I do have an idea. During the last century we had highly visible tragic events such as Auschwitz and thalidomide. The media then began to commonly use the term “human experimentation,” which oozes with all types of evil implications, instead of “clinical research.” Then the powerful consumerism movement burst forth which is heavily characterized by a societal concern with safety. No effective counter educational effort regarding the importance of clinical research was made by any segment of the health sector. The media got lazy, didnÃt do its homework and actually, though incorrectly, self-educated itself. And, by so doing, has become the unwitting enemy of the patient.