Prominent Physician Proposes A Can-Do Way To Reduce Health Care Costs – By Finding Cures



In Dr. DeFelice’s journey with carnitine, he faced every barrier in our medical discovery system. He believes the Doctornaut Act is the only practical remedy and route to achievable solutions.

“My experience with carnitine and our Barrier System would require a thick book that no one would read,” he said. “A single tragic story concerning cancer clearly demonstrates this. At WRAIR, the Walter Reed Army Institute of Research, Major James Vick, an energetic cardiovascular pharmacologist and good friend, and I showed in animal studies that carnitine blocked the heart damage caused by doxorubicin. This highly effective, broad spectrum anticancer drug is limited in use because of its cardiotoxicity. Our findings, which have been confirmed by other researchers, raise the possibility that we could increase its dose, kill more cancer cells, and save or prolong lives.

“We, much to our surprise, then discovered that carnitine increases the kill capacity of doxorubicin ten-fold against rodent ovarian cells in culture. Later, a distinguished scientist colleague, as a personal favor to me, showed that carnitine, by itself, dramatically killed human ovarian cancer cells in culture and also added to doxorubicin’s kill capacity. Carnitine alone also kills human colon cancer cells in culture as well as some animal types which add to its promise.

“Boy, was I excited! Both carnitine and doxorubicin can destroy ovarian tumor cancer cells. It’s also possible to raise the dose of doxorubicin by protecting the heart and kill even more of them. Carnitine, already in hospital pharmacies immediately available to patients, made it possible to administer this combination on the same day it’s ordered by the oncologist.

“My friend, Dr. Cavazza, agreed to fund a clinical study that I proposed in late stage ovarian cancer patients with a certain rendezvous with death. But I needed some type proprietary or exclusivity protection which the Orphan Drug Act provides. I was successful in obtaining such status with carnitine in the past and was sure it would be a  slam-dunk. But the head of this division, all by himself with no objections, changed the rules, making it more difficult and costly to obtain Orphan Drug status and rejected my application. I’m sure other medical innovators, knowing this, did not even apply. Dr. Cavazza had no choice and reluctantly withdrew his support.

“So I approached a large pharmaceutical company that would have unquestionably benefitted if this low-cost study were positive. Incredible as it may seem, they refused.

”Next, I contacted my colleague and renowned oncologist, Emil Frei, the distinguished Director of the Dana Farber Institute. He was sufficiently impressed with the carnitine-doxorubicin data to propose conducting a clinical study in patients with soft tissue sarcoma. But, for personal reasons, it never happened. He did, however, recommend two famous oncologists to contact regarding the ovarian cancer study, which I did.

“They, and other oncologists I met, all wanted more costly preclinical studies performed before making a decision. I had some good luck and arranged to have the study approved at a local hospital. But patients were tough to come by. I asked a prestigious national foundation that deals with ovarian cancer to help us locate patient volunteers, but they were not interested.

“When I tell this story to people they are incredulous! They simply don’t understand how this could happen. My long experience with carnitine and ovarian cancer is difficult to accept, let alone understand.

“After this experience, the gods on Mount Olympus sent me a message that it was time to give up the ship. And so I did. I am not saying that carnitine is a miracle cure because it’s not. It’s a long shot. But it was the only shot!

“And there’s a reasonable theory as to why it might work. Many tumors prefer sugar to feed on. What carnitine does is to make cells eat fat and this effect may actually cause tumors to starve to death or become more sensitive to anti-cancer drugs and the human immune system.

“This is nothing new. In 1931, Otto Warburg won the Nobel Prize for his work on the anaerobic metabolism of cancer cells and their need for sugar. There appears to be a ‘Warburg Revival’ underway now and this might hopefully be helpful to patients.

“To repeat, we’re dealing with an antipatient cultural mindset. The ovarian cancer patients were at the end of the therapeutic line and doomed to die. And, as I said before, there’s carnitine and doxorubicin sitting on hospital pharmacy shelves immediately ready to be administered. What most disturbs me is that patients were not told about the option. It’s all part of our invisible Barrier System.”

Dr. DeFelice summed up this situation. “What’s the general message of this specific experience? The FDA bureaucrats, the corporate physicians, the medical foundations, and the oncologists form an intertwined, complex system that creates obstacles to promising clinical trials. Money reigns supreme. Lots of it would have overcome the barriers to the ovarian cancer study.”

Dr. DeFelice paused, looked me straight in the eye, which meant something big was coming. “Joe, my experience with carnitine and cancer exemplifies the general nature of our Barrier System. The barriers are the same for all promising therapies. I have had similar experiences with nerve growth factor in multiple sclerosis and a cervical cancer vaccine, to name just two.

“The ovarian cancer story; the example of Rock Hudson on Good Morning America; and others examples send an unequivocal message. We have a huge cultural blind spot to even thinking about of having a Cure Care policy and an absolute blind spot regarding the essential role of clinical research in medical discovery.

“To repeat, the good news is that the simple, uncomplicated Doctornaut Act is the solution. If, for example, female doctornauts with ovarian cancer had existed in the late 70’s, then many patients would still be with us. And this discovery would have led to clinical studies with the combination in other types of cancer.

After Dr. DeFelice described parts of our labyrinthine system, I told him I couldn’t see how The Doctornaut Act could change it—and he surprisingly agreed!

“The system cannot be changed,” he asserted. “It is embedded in our culture, so you have to go add to it. The Doctornaut Act is simply an uncomplicated addition. But here’s the other good news. If the carnitine-doxorubicin combination destroyed ovarian cancer tumors then, by public demand, the pressure would be so great that the  administrative system would have to make it available to doctors and patients as soon as feasible. And don’t forget, that doctors are not bound by the FDA to treat patients for non-approved uses. Public pressure will play a huge role in all major medical breakthroughs.

I then challenged him, “You have tried unsuccessfully for over 40 years, what makes you believe that now is the time to seize the moment?”

Dr. DeFelice replied, “Our culture is rapidly changing its habits and values. People, particularly baby boomers, are paying more attention and are better informed. Although there is much misinformation from the media regarding health and medical issues, the public does hear about promising medical advances. This may help create a sense of urgency which we sorely need to bring about change. Also, there’s also the cost of medical care which combined with the sense of urgency can change our cultural mindset.”

DeFelice switched gears again and said, “Speaking of the media, notice that, after reporting on a potential new therapy, they routinely report that it will take a long time before it reaches the patient. They never—and I mean never—explain why! They themselves haven’t the slightest understanding of the Barrier System and it is tough to find experts to ask why this is so.”

I asked Dr DeFelice the bottom line question. “Would physicians be willing to be Doctornauts?” It’s interesting to note that in Michael Mannion’s book, A Maverick’s Odyssey, about Dr. DeFelice’s quest to conquer disease, a few of his physician friends who are sympathetic to his mission were not convinced doctors would volunteer. Dr. DeFelice dismisses their beliefs for a variety of reasons. Specifically, he learned in his work with prisoner volunteers for clinical trials how strongly people are altruistic and want to help others.

In his research unit in a state prison, and at WRAIR, where he collaborated with two other prison facilities, he serendipitously discovered carnitine’s role in cardiac disease in one of his prisoner volunteers. This opened the doors to its development for Carnitine Deficiency in children.

Dr. DeFelice suddenly smiled. This time it was a cynical one. “Would you believe that later on, the FDA virtually closed down prison research facilities? This created another significant barrier to discovery. And it robbed prisoners of the right to be noble and courageous. The barriers never stop. Once more, who pays the price? The patient!”

In 1983, because of his personal interest in the promise of natural substances, the Foundation for Innovation in Medicine conducted a physician survey asking, “Would you, as a physician-patient, want the privilege to volunteer for clinical research of natural substances under the supervision of a physician-clinical researcher without any FDA, institutional or other restraints?” Over 50 percent said they would. Women physicians were as bullish as the men.

Today, there are over 900,000 U.S. physicians in the U.S. If only 10 percent volunteered, there would be 90,000 Doctornauts, a substantial number for early discovery phase studies where generally only small numbers of patients are evaluated. Dr. DeFelice suggested that foreign physicians might also be permitted to be doctornauts in the United States. Why not?

“Dr. DeFelice, I understand your general concept but how, specifically, would Doctornauts speed up medical  discovery?”

“Joe, generally speaking, Doctornauts would participate in small, short-term clinical studies with potential therapies that offer more than ordinary promise,” he answered. “Doctornauts are not suited for long term clinical studies, such as whether a cholesterol-lowering agent prevents heart attacks. Large numbers of non-patented, logical  combinations of promising therapies, as well as natural substance therapies, will be tested. This will not happen without the Doctornaut Act. Doctornauts are major door openers which will, without doubt, expand our base of medical innovators.

“Here is another great example,” he continued. “Genetic therapy, particularly the newly discovered CRISPR  gene-editing technology, is controversial. People understandably fear it will alter human nature in ways unknown. Costly and time consuming barriers will certainly be erected before the first dose is given in any clinical study, let alone subsequent ones. This is bad news for orphan or rare diseases and disabilities. There are about 7000 of them; 80 percent are due to genetic abnormalities.

“It’s estimated there are 30 million orphan disease patients in the United States, many of them who are children. But with Doctornauts, the barriers would be reduced and discoveries made that could lead to new treatments for  children. If, for example, a drug is effective in doctornauts with leukemia, it could also be given to children. It’s a best kept secret that the vast majority of drugs cannot get to the brain because of the blood-brain barrier. A recent really exciting study in mice reported that, using viruses as the carrier, not only drugs, but also genes can enter the brain. If studies in doctornauts prove this to be true, then this method can be employed in children with multiple types of neurological disabilities and disease and would lead to dramatic medical breakthroughs.

I asked “Why do you still believe that our next president or even Congress would become advocates of the Doctornaut Act?” Without hesitation, he shot back, “The national debt and the impact of health care costs.”

I asked him to elaborate. “Over the years,” he began, “I‘ve come to know conservatives and liberals both in the House and the Senate, as well as influential elites who impact public opinion and public policy. About 25 years ago, I met with one of the most liberal members in the House of Representatives, a thoughtful and sincere man who is still there. I explained the rationale behind the Doctornaut Act, seeking his advice on how to move the Congress to enact it.

“After a long moment of silent reflection, he confidently answered, ‘Make it clear how your doctornauts will reduce health care costs. That will get our attention because no one knows how to substantially reduce costs except by political suicide.’ He was, of course, referring to making big cuts in Medicare and Medicaid services which even President Reagan, in his cost reduction initiative, left untouched.

“I told him that the cost reduction argument may not convince opponents who would raise a legitimate argument: breakthrough therapies would be expensive and increase costs. He agreed that this could be a problem and asked if I saw a solution.

“I smiled and answered, ‘Capitalism.’ He also smiled for he’s not a great fan of it. I explained that, in our dynamic market system, both expensive and inexpensive therapies would soon be discovered and compete with each other in the medical marketplace.

“For example, the estimated cost for Alzheimer’s by the year 2050 is $20 trillion— greater than our current national debt. Also, the money saved by curing Alzheimer’s could be used for research on diabetes, autism and other diseases. It’s a win-win situation.

“Did you know that future health care cost projections do not include the discovery of cures? This is mind-boggling and confirms our cultural blind spot that they won’t happen.

“As I said before, this presidential debate has aroused the interest of the public and media as never before. The people are now listening. Before and during the presidential health care debate, we will present the Cure Care versus Health Care initiative. Through our educational and public relations efforts, we will reach influential leaders who will encourage others to join us. Senator Bill Frist’s previous support of The Doctornaut Act will be very helpful to us.

“What will also help is the Act’s simplicity. Unlike the 2000-page, labyrinthine Affordable Care Act, ours could be about 12 pages long and can be read and understood within an hour!”

Finally, I was curious to learn about his marketing strategy. “I’m depending on what I call a ‘Pascal moment.’ The brilliant French thinker observed that small things can have big impacts. For example, if Cleopatra had a really big nose, Julius Caesar would not have fallen for her. Roman history—and the history of Western civilization—would have been different. Our Pascal moment will be a small, but focused, public education effort that would hopefully have a large impact. Much depends on timing, luck and prayers. And there’s no doubt that I’ll be asking God for any help he can give me. It’s now or never for the Doctornaut Act. Let’s give it our best. We need dedicated leaders to join us. I can’t do it alone.”

Well, I told him that Exceptional Parent certainly will join forces. We plan to form a group of dedicated moms with children with disabilities and diseases, Mothers for Doctornauts, who are committed to spreading the message.

In the final analysis, Dr. DeFelice is the one person who can coordinate and implement the entire approach. Let’s hope that he convinces our next president to seize the moment and successfully push for the enactment of The Doctornaut Act. •

Part I of this article appeared in EP’s July 2016 issue, as well as on

10Stephen L. DeFelice, M.D., is the founder and Chairman of FIM, the Foundation for Innovation in Medicine whose mission is to speed up the discovery of breakthrough medical therapies, including cures. He has proposed the Doctornaut Act as the way to discover such cures as well as substantially reduce health care costs. Visit He brought carnitine into the United States and guided it through our entire system to obtain FDA approval which now saves the lives of children with the previously fatal disease, Primary Carnitine Deficiency.


DOCTOR’S UNITE: Dr. DeFelice with Doctor and former Senator Bill Frist. “Before and during the presidential health care debate, we will present the ‘Cure Care versus Health Care’ initiative. Through our educational and public relations efforts, we will reach influential leaders who will encourage others to join us. Senator Bill Frist’s previous support of The Doctornaut Act will be very helpful to us.”


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