Stephen L. DeFelice, M.D.
(Over the years I’ve tried by intensive educational efforts, without success, to bring about general change that would speed up the discovery of new medical therapies for the treatment of disease, disabilities and other conditions that ail us by establishing a “health cure” policy. In this article I thought that by presenting FIM’s arguments in a make-believe Congressional hearing on “cure care” would be readily understandable. Regarding health issues, I’ve been following the White House and Congress since the early sixties. The overwhelming majority of hearings and policies have dealt with “health care” such as Medicare, Medicaid, availability of pharmaceuticals at reduced or no costs and the recent Obamacare. None, unbelievable as it may seem, has dealt with “health cure”).
A Senate Health Committee decided to hold a hearing on the current status of research on Alzheimer’s disease. A number of guests were invited including John Salute, M.D., the President of the Alzheimer Association of Concerned Citizens. The veteran Chairman of the Committee, Senator Bruce Byron, commenced the hearing by first calling on Dr. Salute.
“Thank you, Mr. Chairman, for inviting me to this important gathering. As you know our association is very much concerned about the slow pace regarding new effective therapies for Alzheimer’s disease. We’ve spoken to other organizations that deal with a number of diseases and disabilities and they all share the same concern regarding their constituents. I, therefore, feel an obligation to talk not only about Alzheimer’s but also about other diseases that afflict us.
“We believe there’s something radically wrong with our general medical discovery system. For example, with all of our current exquisite technology that can take us to the moon as well as create a living cell why can’t we cure the common cold? Just before this hearing began I spoke to my one of my colleagues from a lung cancer foundation, and he wondered why little progress has been made against this disease over the past 30 years. It’s about time that Congress looks into this matter and finds out the answers.
“Now I mean no disrespect but the federal government, the Congress and the current and past administrations, has almost exclusively dealt with health care and ignored what the people really want. If you were to ask a patient or a healthy person who will inevitably become a patient the question, ‘What do you want?’ “Well, the patient would logically answer, ‘Get rid of my cursed disease’ and the healthy person likewise would say, ‘Prevent me from getting my cursed disease’. Both would emphatically add, ‘We don’t want health care and free hospitalization. We never want to see the inside of a hospital! We want a health cure system along with a health care one.’
“Mr. Chairman, the only way to do this is by speeding up the discovery of new medical therapies. To repeat, Congress has never had a hearing on how to accelerate medical discovery, and we believe that one is urgently needed.”
Senator Byron, clearly surprised and somewhat puzzled by Dr. Salute’s unexpected opening remarks points out, “But Dr. Salute, I’m sure you’re aware that Congress has passed tons of legislation to sponsor medical discovery research projects. Just look at the National Institutes of Health where the annual budget is over $30 billion. Do you and your colleagues believe we’re not spending enough? Is that your point?”
“Unfortunately not, Mr. Chairman. I wish it were so for then the solution would be a simple one by just spending more money. It, instead, has to do with our entire medical system. It’s profoundly counter productive, and what I’m about to say is more than a bit controversial but, in my opinion, the truth. We indeed do generously spend money to fund scientific and medical research to conquer disease but at the same time we paradoxically create barriers to bring these potential new therapies to the people. It’s, in real sense, a medical perversion.”
Senator Byron, not knowing whether to be insulted or curious about these comments chose the latter. He began, “Dr. Salute I mean no disrespect but what you have just said, if true, is one of the greatest national scandals in the history of our country. We always enact health legislation with the best interests of the American people. Though the vast majority of us in Congress are lawyers and not scientists or physicians we do heavily rely on the advice of medical experts when dealing with medical scientific legislation. Not one has ever mentioned what you have just said which, frankly speaking, makes me extremely curious how you have come to this conclusion.
“You are well aware that my mother has Alzheimer’s disease and is losing her battle to hold on to her mind. And let me add that your organization has been the leader in the quest to conquer this awful disease. And you are also aware that the government has heavily funded it to find new effective therapies. Yet what you are saying is that we do it in the wrong way, and, I, as well as my colleagues on this senatorial committee, would surely like to know what you know that we, and everybody else, don’t know.”
There was an uncomfortable pause in the hearing room because of this unexpected and dramatic turn of events. All came prepared to address the issue of Alzheimer’s disease and not the so-called perverse medical discovery system. Dr. Salute, a veteran of Washington politics, was well aware of this and thought about how to get the senator off the hook and yet make his point. He concluded that the best way was to tell the truth.
He began, “Mr. Chairman, I fully appreciate what you have said and it would be impossible to thoroughly cover the issue at this very important Alzheimer’s hearing with the limited time available for my comments. First of all, our great country which, though blessed by untold scores of well meaning charitable people and organizations for reasons only known to God, has a cultural blind spot regarding general nuts and bolts ways to generally increase the discovery of new medical therapies for all diseases. We concentrate, instead, on funding research for specific diseases such as AIDS, breast cancer and, yes, Alzheimer’s. By the very fact that Congress or past administrations have not shown interest or had a hearing on this issue confirms the existence of this pervasive cultural blind spot.
“Let me give you one specific example of a proposal to rapidly speed up medical discovery that’s very clear and difficult to dispute but, unfortunately, only so for a handful of folks. There’s a nonprofit foundation, FIM, the Foundation for Innovation in Medicine that was founded in 1976 by the physician and Chairman, Stephen L. DeFelice.
“His position is simple and easy to implement and, paradoxically, that may be the reason it has been not been understood and ignored. Also, it’s because he’s an individual and not a prestigious blue ribbon expert panel whose recommendations are generally made known to the public and discussed by the experts. He states what should be obvious but is not. The way new therapies are discovered is by testing them in people in a clinical research study not in test tubes or animals. The overwhelming majority of drugs that are effective in laboratory studies do not work in humans. We would have never known whether penicillin cures bacterial pneumonia unless it was given to patients with this disease. And, yes, we don’t know whether a promising new drug in animal studies for Alzheimer’s will be effective until it is tested in these patients. And, to repeat, the probability of success is very low for practically all diseases.
“Now before I go on, Mr. Chairman, I would like to hear from you and the members of this panel whether this message is clear and whether you agree.”
“That’s a very fair and appropriate question, Dr. Salute. It makes sense to me. How about you, gentlemen?” All senators, without delay, nodded in agreement. “Please continue, Dr. Salute.”
“Dr. DeFelice then argues that, if there are powerful obstacles to clinical research or testing new therapies in patients, and then this, by necessity, will slow down the discovery of new therapies. Once more, Mr. Chairman, I’d like to know whether this point is clear to you and your colleagues and whether you agree.”
There was a brief pause, and then the Chairman answered, “You know I have to say it makes a hell of a lot of sense to me.” He turned to the other senators and asked, “How about you?” One senator answered, “It’s tough to argue against what seems obvious.” The others on the panel once more nodded in agreement.
Dr. Salute was both surprised and happy with the agreement among the senators but he knew that things would now change. He continued, “Now here’s the disturbing news. Dr. DeFelice argues that years ago powerful barriers to clinical testing began and are getting stronger every day. He logically concludes that as a result the discovery and availability of new medical therapies have actually been dramatically blocked and the American people have clearly suffered needlessly and died before they should have. He has other things to say but I’m sure you and the distinguished members of this panel have some questions and comments at this point.”
Senator Byron’s facial body language spoke of puzzlement coupled with a tinge of impatience. “Dr. Salute, I think your doctor friend has now gone too far. If this were the case we here in Congress would have known about it and taken action to correct it. I â€¦”
Dr. Salute decided it was appropriate to interrupt the senator before he continued and said, “Mr. Chairman, what I’m about to say you and your distinguished colleagues would not like to hear but it’s important that you do before we go on or else I wouldn’t bring it up. Dr. DeFelice believes that, bottom line, it is the Congress that is the institution most responsible for powerful barriers to clinical testing by tremendously raising the costs and risks to conduct clinical studies. He believes, as I said before, such actions are perverted. Congress funds scientific studies in order to discover new therapies and then it, paradoxically, creates enormous barriers to test them in patients. There’s one more piece to his thinking that I’d like to talk about but I’m sure you and other members of the panel would like to make some comments at this point.”
Bordering on the verge of anger, Senator Byron, his vocal decibel level now elevated responds, “Who does he think he is by accusing us of perverted actions against the sick? Besides, no one has informed us that there are these barriers to clinical research that are robbing patients of new cures or at least helpful ones. Dr. Salute, if it wasn’t for my respect for you, I’d immediately shut down this discussion about this rogue doctor.”
An elderly, well respected white-haired committee senator who recently lost his wife to brain cancer interjected, “Mr. Chairman, suppose this analysis by this DeFelice guy is correct- and, in my mind, there is this possibility because I believe Dr. Salute may think so because he brought it up, and that’s good enough for me- it’s our duty to carefully hear what more he has to say.”
Senator Byron, respected the wisdom of his longtime colleague and replied, “I agree. Dr. Salute, please hold no punches and go ahead with the DeFelice Theory.” “Mr. Chairman, no one may have informed you personally but since FIM was formed Dr. DeFelice has futilely spent substantial amounts of money including the use of multiple public relations firms trying to educate just about everyone that’s important from the media to the Congress not only about the problem but also his proposed specific solutions which I’ll talk about in a minute.
“There are solid unequivocal data that clearly prove that the barriers to clinical research studies have and continue to dangerously increase. I can assure you that this reality would become very obvious during a Senate hearing.
“He had high hopes and counted on the health oriented media to spearhead a national educational effort but they had and continue to have zero interest. Mr. Chairman, we’re talking about a personal intense and expensive effort of over thirty-five years without arousing any significant level of interest in any sector. Regarding Congress, let me tell you he has exerted an enormous effort to arouse interest both in the House and the Senate without success.”
Senator Byron, caught somewhat off guard, said, “If he did all of that why the hell haven’t I heard about it?”
“Mr. Chairman you hit the basic point right on the nose. This is why Dr. DeFelice says we have a cultural blind spot to understanding the critical importance of clinical testing. He opened the door with a few Congressmen and their staffers but then, because of a lack of understanding and interest, the doors quickly closed. There was one hopeful exception which I’ll mention in a moment.
Senator Byron, took a deep breath and then in a voice that expressed a bit of bona fide bewilderment said, “Dr. Salute, that’s hard to believe, very hard to believe. Anyway, please go on.”
“Mr. Chairman, making the assumption that what he proposes is true, he’s come up with a couple of relatively simple solutions. The first deals with a simple specific one regarding the FDA and the second, much more important general one, deals with the Congress.
“To repeat, his main objective is to remove the unnecessary barriers to clinical testing and speed up the creative testing of potential new, promising therapies in patients. Regarding FDA, for example, though it’s recently been a little bit more flexible in certain limited cases, a long time ago it established an anti-patient policy that virtually eliminated the testing of combination drugs in clinical studies. If for example, the combination of three drugs holds promise to be effective for the treatment of Alzheimer’s, it would not be sufficient just to study the three drugs together or, let’s say, against only a placebo but to compare it to each drug individually and every possible combination of them. It’s like evaluating the role of every ingredient in a meal to prove that eating keeps you alive. The cost and risks would be out of sight and this is why combinations are not studied and patients, of course, pay the price. Dr. DeFelice claims that all that FDA has to do is to rescind that policy which would take about five minutes to do which will allow more rapid discovery of new therapies. It is a simple administrative step.
“Before I forget he writes in more detail about this on FIM’s website, www.fimdefelice.org, under the DeFelice Commentary in the article, “Medical Versus Scientific Clinical Research – Time for an Immediate Change!“
Senator Byron, feeling somewhat left out and uncomfortable because of his ignorance of the subject matter, now had something of substance to contribute the discussion. “Dr. Salute, perhaps Dr. DeFelice is not up to date with current FDA policies. It now permits clinical studies in certain diseases with cocktails or combinations.”
“This is certainly encouraging but only applies to a few disease states such as AIDS. Dr. DeFelice believes this policy is also a perversion and should apply to all disease and disability conditions and that there should be no ‘privileged’ patient groups when dealing with suffering and premature death.”
Senator Byron commented, “These are pretty strong words but I wasn’t aware of these privileged groups, and I certainly will look into it. Now Dr. Salute, I am and I can assure so are my colleagues, extremely anxious to hear what Dr. DeFelice thinks Congress can do to speed up the discovery of medical breakthroughs. Please proceed.”
“Well, Mr. Chairman, like that of the combination recommendation, it’s relatively simple. As a young physician, Dr. DeFelice saw that the barriers to conducting clinical testing were not only blocking medical discovery then but would increase dramatically over time. And history has certainly proven his vision correct.
Based on his experience with the natural substance, carnitine, he wrote his first book, Drug Discovery the Pending Crisis published in 1972, a long time ago before, may I say, many of your senate staffers were born, where he came up with the solution. He proposed that physician patients or even healthy ones should be permitted to volunteer for clinical studies to physician clinical researchers much more easily than non-physician volunteers- that means much less FDA control- and also waive the right to sue. They would be able to take greater risks because they, as physicians, understand such risks and weigh them against the potential benefits.
“Later on he called these physicians Doctornauts and proposed the Doctornaut Act. One high level senator met with Dr. DeFelice and was convinced of the merit of the theory. He had a draft of the Doctornaut Act written and circulated. There was little support but, to be fair, it was a confusing draft and before corrections could be made the overwhelming health care issues took over the senator’s agenda. For your information one can find out more about Doctornauts on the FIM website.
I’m not sure but there are about 700,000 doctors in our country which is a huge potential reservoir for volunteers. Many years ago FIM conducted a survey on whether doctors would volunteer for clinical research on natural substances without FDA supervision. Over 50% agreed. Even if a small percentage of the new breed of modern day physicians agree to volunteer for clinical studies there would be thousands of them to tests hundreds and many more innovative new therapies. He believes that, because of our cultural blind spot and fear of pharmaceuticals, there is no other acceptable way to do this. After all, doctors know what’s involved more than others and if they want to volunteer for a clinical study they are, in a real sense, helping to deliver what patients really want and need. It certainly would beautifully strengthen the patient-doctor relationship that our health system is endangering.
“Senator Byron, before we close this discussion we haven’t made it clear enough what Dr. DeFelice means by our perverted general medical discovery system. For the record he has written extensively on this.
“He claims that our country has been and continues to be too heavily indoctrinated against the safety of pharmaceuticals and, largely because of this, a pervasive national fear exist. He, of course, has no unique vision about this for it has been self evident to most of us for a long, long time. He principally blames the media for both creating and perpetuating this national fear. He claims that the media including medical outlets who should know better have virtually completely ignored the issue of the acceleration of medical discovery which is infinitely more important than safety. He says if we cured diabetes we wouldn’t have to worry about drug toxicity because patients would be drug free.
“Now because of our country’s media generated fear of drug safety, The FDA, under constant political pressure to keep us all safe from the bad effects of drugs, has created a policy almost exclusively based on increasing safety of clinical studies by creating all kinds of barriers from permitting them to happen.
“Let me give you one of his examples that describe his concept of perversion. Not too long ago there was a study where gene therapy was given to patients at one of America’s most prestigious medical institutions. One patient died. The media, based on this single death, created a national feeding frenzy of fear of participating in clinical studies which placed the FDA in a bad light and under relentless pressure. And what did the Congress do? It put additional pressure on the FDA for permitting such a “dangerous” study. And what did the FDA understandably do but increase the rules or barriers to conduct clinical research which further slows down the discovery of new therapies while the media, Congress and just about everyone else ignored other cold facts such as there were thousands of people murdered in our major cities last year that went virtually unnoticed.”
There then followed a pause which seemed to signal the end of the discussion of the Doctornaut Act but Senator Byron, being a veteran of the Washington scene and a proud man, wanted to close the discussion with some semblance of being in charge of at least some of the facts. Smilingly, he asked, “Dr. Salute, we in Congress, in both chambers, have been repeatedly told that there is no doubt that new therapies will be increasingly costly as now evident in the bioengineering field. It appears to me that Dr. DeFelice’s proposal will create more of such expensive therapies significantly increasing health care costs when, in fact, we are trying to reduce them. For completion’s sake, he should have factored this in the Doctornaut Act.”
Dr. Salute also smiling answered, “Senator you hit it right on the nose and may I apologize for not addressing it. I asked Dr. DeFelice precisely this question and believe it or not, I have in my briefcase his response. It’s a brief letter. May I read it?”
Senator Byron, without hesitation, nodded his head in affirmation and Dr. Salute read:
“Dear Dr. Salute,
There is no doubt that the Doctornaut Act will lead to more expensive medical therapies but before jumping to conclusions, it will, in time, lead to a substantial reduction in health care costs. Let me address two points:
To my knowledge, future health care costs are projected on the growth of patient populations using the approximate incidence and prevalence figures of disease that we have today not factoring in substantial cure projections. For example, the cost of end stage renal disease, ESRD, is about $40 billion the major part involving renal dialysis treatment. There are currently in the year 2010 over 500,000 ESRD patients. With the aging population this number will steadily increase. But, if we could cure by a prevention or treatment therapy this condition, then the cost reduction would be enormous. In conclusion and generally speaking,, in the short term the costs would be elevated but in the long term particularly in patients with chronic disease such as cancer, rheumatoid arthritis, heart disease, Alzheimer’s and many others, they would be dramatically reduced.
The Doctornaut Act will permit, at a very low cost, the clinical testing of both low and high cost therapies and they will compete with each other in the medical marketplace. Though it is tough to predict there is little doubt that there will be an abundance of low cost breakthrough medical therapies. We must not forget that natural substances are the best potential therapies that we have but because of the difficulty of obtaining patents, very few are tested. The Doctornaut Act will permit such testing .In our great country, patent or no patent, if a major medical discovery is made it will be developed and made available to those in need.
In conclusion Dr. Salute, if the Doctornaut Act is enacted both current and future patients will have much to gain and health care costs would be significantly reduced and millions of Americans would lead happier lives.
Stephen L. DeFelice, M.D.
Senator Byron turned to his colleagues on the panel and remarked, “Dr. Salute has effectively presented a point of view that deserves further consideration for a future date but let us know turn to the subject of Alzheimer’s.”
At the end of the day Senator Byron met with his staffers in his office and reviewed the day’s discussions on the status of Alzheimer research. Before they left for the day, he, in a wondering mode, asked his staffers, “What do you guys think about Dr. Salute’s presentation? It was a novel one but on the surface it kind of made some sense. Maybe we ought to hear from this DeFelice guy in person.” His veteran and politically wise chief of staff replied, “Senator, I don’t see any gain in pursuing this because there’s no support out there and perhaps you’ll take a big negative hit because our country views clinical research studies, as Dr. Salute said, as a necessary evil, and you will be labeled as an Auschwitz perverted nut case. I would strongly recommend that we stay away from this guy and the issue.”
That night while dining with his wife of many years the senator told her about Doctornauts. She surprisingly said that she thought it was a wonderful idea. Maybe it would bring a cure to her crippling arthritis during her lifetime. She asked him what he planned to do. He paused, looked into outer space and replied, “I don’t know, my love, I just don’t know.”