40 July 2016 • EP MAGAZINE | eparent.com

PROMINENT PHYSICIAN PROPOSES A

CAN-DO WAYTO REDUCE HEALTH CARE COSTS –

BY FINDING CURES

There is general agreement that the US health care system isin crisis with no doable solution in sight. The ever-increas-ing cost of health care is the biggest concern of policymak-

ers, politicians and the American people. Access to health care isalso of great concern to millions of Americans who have healthinsurance that, ironically, they cannot afford to use. The controver-sial Affordable Care Act has already substantially raised health carecosts with no end in sight.

Into this chaotic situation steps “A Man with a Plan,” Stephen L.DeFelice, the founder of the Foundation for Innovation in Medicine(FIM), a physician with a long, creative career in medicine. Heapproaches the crisis in health care in a radically different manner.He is not introducing new policies or bureaucratic programs, butinstead, a practical program of action, tobe carried out by brave women and menhe calls “Doctornauts.”

What is a Doctornaut? Simply put, it isa physician-patient who will volunteer forclinical research of pharmaceuticals, nat-ural substances or new medical devicesunder the supervision of a physician-clin-ical researcher with minimal FDA, insti-tutional or other restraints.

Dr. DeFelice has outlined thisapproach in “The Doctornaut Act,” a dis-cussion draft of which was circulated bySenator Bill Frist and available on theFIM website (www.fimdefelice.org).What will most effectively bring down health care costs? The

answer, according to Dr. DeFelice, is finding cures. Who can argue?Cure diabetes and there will be no costs. What is needed to find cures? Clinical trials of promising new

treatments, which now face extraordinary obstacles: New thera-pies, however, can only be discovered in clinical trials. What is his definition of cures? A cure is any therapy that either

prevents or eliminates disease or disabilities by treatment.

What then can overcome the obstacles in the way to the vitallynecessary clinical trials? The Doctornaut Act, which will allow theclinical testing of promising new drugs, natural remedies and med-ical devices quickly and bring about the new cures that are need-ed.

The ancient Greeks had their Argonauts who sailed unknownseas on dangerous journeys. The Russians had Cosmonauts andthe Americans had Astronauts who sailed space craft on danger-ous journeys into the cosmic ocean. All of them took great risks toadvance knowledge and improve the life of mankind. Some ofthem suffered – even died – in this effort. They are consideredheroes for their bravery. Dr. DeFelice suggests we need a new breedof heroes, this time in medicine – Doctornauts who will bravely

and altruistically head into unchartedmedical waters in search of cures in theshort rather than the long term.

While the term Doctornaut may benew, the concept is part of a long tradi-tion in medicine in which physicianshave practiced self-experimentation,trying out new and risky treatments onthemselves first. A century ago, WernerForssmann, a German physician, insert-ed a catheter in his vein and guided it tohis heart. This risky act revolutionizedthe field of cardiology and he wasawarded a Nobel Prize. More recently,

Australian physician Barry Marshall swallowed a concentratedsolution of H. pylori to prove his theory that this bacterium causesgastrointestinal ulcers and gastritis. His brave act was a major med-ical breakthrough for which he also was awarded a Nobel Prize.History is replete with self-experimenting courageous doctors inthe search for cures. In this process, error and harm are unavoid-able, even the possibility of death.

Dr. DeFelice himself is in this tradition. As a young doctor, work-ing with physicians and nurses in Yugoslavia, he acted as a true

• PHOTOS PROVIDED BY FAYZAL MAHAMED40 July 2016 • EP MAGAZINE | eparent.com

INTERVIEW OF STEPHEN L. DEFELICE, M.D., BY JOSEPH M. VALENZANO JR.

PA R T   I   o f a S E R I E S

PHOTO PROVIDED BY STEPHEN L. DEFELICE, MD FROM THE NEW YORK TIMES

eparent.com | EP MAGAZINE • July 2016 41

Doctornaut to move one of his studies forward. He injected him-self with two different carnitine solutions in separate arm veins totest for safety. The results permitted him to proceed with other for-eign and U.S. clinical studies which were instrumental in obtainingFDA approval for carnitine that saves the lives of thousands of chil-dren, both in our country and abroad.

Today, our risk-averse culture is not will-ing to take such chances. That is why Dr.DeFelice is working so hard to make theDoctornaut Act a reality. He is convincedthat history and current trends indicatethat physicians will step up to the plate andtake the risks others fear to take or whichour cultural rules prohibit. In order to avoidthe misconception that we are dealing withdoctors gone wild, Dr. DeFelice emphasizesthat the vast majority of such clinical stud-ies will not be life-threatening becausephysicians understand, better than others,what the benefits / risks are.

Dr. DeFelice is doing his best to see thatthis actually occurs through Doctornautsand the approach he calls “Cure Care vs. Health Care” and howthey are related.

If President Obama can undertake an ambitious $1 billion“Cancer Moonshot” to eliminate cancer in his last year in office,perhaps the next President can start out by supporting the

Doctornaut Act which will deal with all diseases. This will increaseinnovation in medicine and accelerate the discovery of cures forthe costly major diseases that plague humanity, including diseasesand disabilities in children. “Cure Care” will deliver those treat-ments to the American people – soon.

I first met Dr. DeFelice in the 1980s at aFIM conference. I was intrigued by hisstraightforward, no–nonsense mes-

sage: the best way to reduce health carecosts is by curing disabilities and disease byprevention and treatment.

But what intrigued me even more washow he proposed to discover these cures.Years ago, he proposed that Congress passthe Doctornaut Act. He had support fromSenator Bill Frist, a physician and thenSenate Majority Leader. It’s based on thepremise that the only way to discover newtherapies is to test them in clinical studiesin patients. For example, penicillin couldnot be discovered until tested in patients

with bacterial infections, and insulin in diabetic ones. There isindisputable, published evidence of the enormous obstacles toclinical testing of new therapies. Dr. DeFelice calls this the BarrierSystem in which large numbers of promising therapies have notbeen and never will be tested. For this reason, the discovery of

The Doctornaut Act will

increase innovation in

medicine and accelerate

the discovery of cures

for major diseases that

plague humanity,

including diseases and

disabilities in children. 

TO BOLDLY GO: An early photo shows Dr. Stephen L. DeFelice (far right) working with with a team of nurses during an intravenous

procedure. He suggests that we need Doctornauts, a new breed of heroes, who will bravely and altruistically head into uncharted

medical waters in search of cures in the short rather than the long term.

42 July 2016 • EP MAGAZINE | eparent.com

cures is a rarity despite our exploding tech-nology of which our culture doesn’t get theconnection.

Few appreciate the enormous sums ofmoney – billions upon billions of dollars –spent on research on diseases, such as can-cer, cardiovascular, mental, neurological,arthritic and pulmonary and many others,without the discovery of cures. The NIHannual budget for medical research isapproximately 32 billion dollars. Over thepast decade the NIH, apart from the phar-maceutical industry, has funded close to 50billion dollars on cancer research withoutthe discovery of major cures. There areclose to three million patients with the pri-mary diagnosis of epilepsy, with 32 drugsavailable as treatment, none of which is acure. Patients over the age of 65 take a dailyaverage of five drugs, none of which is acure.

Oftentimes, less prevalent conditions,including disabilities such as Down’s syn-drome, escape sufficient national attentionand how our aging population is changingthe status quo of the disabled and theirfamilies. In the past, these children left usin their twenties, but due to modern thera-pies they can now live up to the age of 60,when their parents, however, are mucholder and afflicted with the costly chronicdiseases of aging. So we are dealing withthe long-term suffering of two very costlyand suffering patient populations in a sin-gle household without the availability ofcures – an unacceptable outcome, if thereever was one.

The no-cure list is long. Dr. DeFelice hasunsuccessfully attempted to have ourcountry ask the challenging question,“Why are there so few cures?”

The Doctornaut Act will rapidly over-come the barriers which block the discov-ery of cures as well as more effective ther-apies. It will permit physician volunteers tofreely volunteer for early clinical trials,some risky and, importantly, waive theirright to sue. If enacted, the base of medicalinnovators would immediately broaden;more promising therapies would be tested;more medical discoveries would reachpatients, curing many. Because of his experi-ence, he also believes doctornauts wouldimmensely benefit children. And these bene-fits would occur in the short-term.

Despite decades trying to convinceCongress to pass the Doctornaut Act, he

has, with the exception of Senator Frist,repeatedly run into a stone wall. But he’sbetting that the current presidential racewill produce an opening for his innovativeideas.

Dr. DeFelice believes the next presidentcould seize the moment and help acceler-ate the discovery not only of cures but alsoof low cost medical breakthroughs throughthe Doctornaut Act. He plans to deliver his

message of Cure Care versus Health Care tothe candidates during the presidential race.

When I asked what sparked his passionto pursue the passage of the DoctornautAct, DeFelice attributed it to three personalexperiences: his grandmother’s diabeticcoma; a child with leukemia; and his dis-covery and pursuit of the natural sub-stance, carnitine – an interesting triad, tosay the least.

When he was 12, his grandmother, or“nonna,” was in diabetic coma lying on abed in the dining room without hope ofrecovery. There was a 24-hour vigil by fam-ily and friends. He couldn’t accept the factthat she would die and he talked to her, try-ing to elicit some type of response, whichfailed. He then went to the local CatholicChurch and made a deal with God promis-ing to do good things if He saved her life.He was convinced he had made a deal. Butshe died that night.

He unexpectedly felt two powerful emo-tions: an intense hatred of disease and astrong conviction that disease must andcan be conquered. He met only one person,‘Doc’ Druckenmiller, a country doctor whohe made rounds with when he was a med-ical student – $3 an office visit and $5 ahouse call – who proclaimed hatred for dis-ease. About 15 years later, as a third-year

medical student covering the pediatricward, Dr. DeFelice cared for a nine year-oldchild with terminal leukemia. The motherand father were kneeling by her bed silent-ly praying. He said, “The scene of Christand the manger came to mind. The firstscene dealt with life; the one before mewith death. About an hour later, when I wasalone with her, she expired. It hit me hard.One moment she was alive, the next goneforever. Incomprehensible!”

Only a handful of people know that itwas Dr. DeFelice who brought carnitine toAmerica in 1965. He conducted the firstsuccessful clinical studies on it. Afterrepeated failures, he found funding fordevelopment through his friend, the lateClaudio Cavazza, proprietor of Sigma-TauPharmaceuticals.

Together, they guided its way to FDAapproval for the treatment of the fatal dis-ease in children, Carnitine Deficiency, andalso for patients on renal dialysis. It’s alsogiven to premature babies who fail to thriveand other conditions. His unparalleledexperience in all sectors of clinicalresearch qualifies him to be consideredone of the world’s top experts.

As we discussed his third experience,with carnitine, his adrenalin productionskyrocketed. He began, “Carnitine taughtme about the entire Barrier System whichbegins with the identification of the drugitself to FDA approval and beyond. If youunderstood the entire Barrier System, youwould conclude that it was devised by asadist who finds happiness by creatingobstacles to keep promising medical thera-py from being clinically tested and reach-ing physicians and patients.”

I n his first book, Drug Discovery, thePending Crisis, published in 1972, Dr.DeFelice predicted, “Our present sys-

tem of drug discovery is almost designednot to cure the great diseases that confrontus. There is no doubt that many will becured in the distant future, but it is unfortu-nate that many must wait.” In this book, hefirst proposed physician volunteers or doc-tornauts for clinical studies as the solution.

According to Dr. DeFelice, the complicat-ed Barrier System includes the nature ofthe drug, patents, funding, patient avail-ability, doctors, universities, hospitalInstitutional Review Boards (IRBs), the FDA,the National Institutes of Health (NIH), the

The Doctornaut Act will

overcome barriers that

block the discovery of

cures, permitting

physicians to volunteer

for early clinical trials

and, importantly, waive

their right to sue.

eparent.com | EP MAGAZINE • July 2016 43

pharmaceutical and medical device indus-tries and many other factors. But the cul-tural mindset is the governor of the othercomponents of the aforementioned.Interestingly enough, he knows of no onewho has traveled through the entire sys-tem.

“How would you describe this culturalmindset?” I asked.

“It’s a syndrome characterized principal-ly by fear combined with ignorance, apathyand the absence of knowledgeable leaderswho represent the patient. It’s simply toodifficult and costly to conduct clinical stud-ies. Since the thalidomide tragedy and therise of safety–obsessed consumerism, weview clinical research as a necessary eviland something to fear. An over-emphasison safety permeates all aspects of theBarrier System.”

Dr. DeFelice continued, “Often, themedia labels clinical research as ‘humanexperimentation.’ This connotes an evil act.If an astronaut dies, he’s considered a hero.If, however, a patient in a gene study dies,all hell breaks loose. The doctor and hospi-tal are somehow considered as baddies.The FDA and IRBs, responding to pressure,create further regulations and rules thatprofoundly inhibit clinical research andmedical discovery which, ironically, arewelcomed in the name of safety. What isignored is the primary concern ofpatients – to be cured!”

I asked Dr. DeFelice to give us a simpleexample what best demonstrates our cul-tural blind spot to the critical importance ofclinical research. Without hesitation, hereplied, “Rock Hudson,” the famous moviestar who died of AIDS in the early phases ofthe epidemic. “He was a man who waswell–liked and well–known to mostAmericans. Inaccurate media coverage hadproduced a pervasive national fear of anAIDS epidemic. There were no effectivetherapies back then.

“An anti–viral drug was in the researchphase in France which might have helpedRock Hudson. But the FDA ruled that it did-n’t meet their requirements and could notbe given to Mr. Hudson in the United States.He had to fly to France to be treated! Heshould have been able to be treated withthis drug in the United States.”

The popular TV show, Good MorningAmerica, learned about Dr. DeFelice’s posi-tion and invited him and the head of the

FDA to a debate. “I sincerely believed thatthis was the golden opportunity to finallypierce our cultural blind spot about clinicalresearch,” Dr. DeFelice said. “I stressed thatMr. Hudson should, for example, be able toreceive the therapy at Memorial Sloan-Kettering where the experts are. The FDApolicy on clinical research is a huge barrierand should have no role in this early med-ical discovery phase.”

The FDA official was evasive, notaddressing Dr. DeFelice’s point. “I was con-fident I made the point clearly,” he said,“and fully expected that I had started anational discussion on the urgent need toreduce the barriers to early clinicalresearch. Good Morning America has mil-lions of viewers and the AIDS phenomenonwas of great national concern bordering onnear hysteria as if it were another bubonicplague. It seemed to me to be a perfectmedia storm.

“I alerted Patricia Park, my indispensablesidekick for over 40 years, to ‘man’ thefoundation telephone. The response? Zero!And I mean zero! Not one call from themedia, the foundations, the medical com-munity or individuals. If that’s not a cultur-al blind spot, what is? And who pays theprice? The defenseless patient!”

Rock Hudson’s diagnosis with AIDS wasa huge story. The thousands of others whowere ill and dying was a big story. What,then, could account for the lack of atten-tion to the need for clinical trials?

“Joe, I wish I knew, but I have a theory.Our society is simply not interested in thegeneral issue of why we don’t have cures.Over the years, I’ve asked hundreds of menand women in different walks of life, manywith serious and fatal diseases, ‘When was

the last cure?’ The overwhelming responsehas been silence, coupled with blank faces.The few who did respond mostly men-tioned the polio vaccine which happenedin the fifties!

“When I informed them that, despite ourbooming technology, there are few cures,the almost unanimous lack of curiosity andconcern regarding the reasons why wasand remains striking. When I explain therole of clinical research in medical discov-ery, blank faces and lack of curiosity stillprevailed. Many, influenced by persistentmedia coverage, mentioned concernsabout the dangers of clinical studies. Manymore inquired whether there are new ther-apies on the horizon for what specificallyails them or their family and friends. Theseexperiences bespeak of a blind culturalmindset which is unbudgeable.

“Even Christopher Reeve, the thenextremely popular actor who played therole of Superman, couldn’t make a dentregarding the importance of clinicalresearch. In the mid-nineties he fell off hishorse, partially severed his spinal cord inhis neck and became a quadriplegic – par-alyzed from the neck down. He laterformed the Christopher and Dana ReeveFoundation which, to this day, is dedicatedto funding research for cures for spinalcord injury.

“He observed that, although there wasmuch promising research in laboratorystudies, particularly with rodents, few werebeing tested in clinical studies. His emo-tionally moving declaration, ‘If I were onlya rat’, which basic on-target message is thedifficulty of conducting clinical researchwent virtually unnoticed and unheeded.” •

Part II of this article will appear in EP’sAugust 2016 issue, as well as onwww.eparent.com

ABOUT THE AUTHOR:

Stephen L. DeFelice, M.D., is the founder and

Chairman of FIM, the Foundation for Innovation in

Medicine whose mission is to speed up the discov-

ery 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 www.fimdefelice.org.

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.

“I’ve asked hundreds of

men and women in

different walks of life,

‘When was the last cure?’

The overwhelming

response has been

silence, coupled with

blank faces” 

24 June 2016 • EP MAGAZINE | eparent.com

PROMINENT PHYSICIAN PROPOSES A

CAN-DO WAYTO REDUCE HEALTH CARE COSTS –

BY FINDING CURES

In Dr. DeFelice’s journey with carnitine, he faced every barri-er in our medical discovery system. He believes theDoctornaut 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 singletragic story concerning cancer clearly demonstrates this. AtWRAIR, the Walter Reed Army Institute of Research, Major JamesVick, an energetic cardiovascular pharmacologist and goodfriend, and I showed in animal studiesthat carnitine blocked the heart dam-age caused by doxorubicin. This highlyeffective, broad spectrum anticancerdrug is limited in use because of its car-diotoxicity. Our findings, which havebeen confirmed by other researchers,raise the possibility that we couldincrease its dose, kill more cancer cells,and save or prolong lives. “We, much to our surprise, then dis-

covered that carnitine increases the killcapacity of doxorubicin ten-foldagainst rodent ovarian cells in culture.Later, a distinguished scientist col-league, as a personal favor to me, showed that carnitine, by itself,dramatically killed human ovarian cancer cells in culture andalso added to doxorubicin’s kill capacity. Carnitine alone alsokills human colon cancer cells in culture as well as some animaltypes 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 ofthem. Carnitine, already in hospital pharmacies immediatelyavailable to patients, made it possible to administer this combi-nation 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 ren-dezvous with death. But I needed some type proprietary or exclu-sivity protection which the Orphan Drug Act provides. I was suc-cessful in obtaining such status with carnitine in the past and

was sure it would be a slam-dunk. Butthe head of this division, all by himselfwith no objections, changed the rules,making it more difficult and costly toobtain Orphan Drug status and rejectedmy application. I’m sure other medicalinnovators, knowing this, did not evenapply. Dr. Cavazza had no choice andreluctantly withdrew his support. “So I approached a large pharmaceu-

tical company that would have unques-tionably benefitted if this low-coststudy were positive. Incredible as itmay seem, they refused.”Next, I contacted my colleague and

renowned oncologist, Emil Frei, the distinguished Director of theDana Farber Institute. He was sufficiently impressed with the car-nitine-doxorubicin data to propose conducting a clinical study inpatients with soft tissue sarcoma. But, for personal reasons, itnever happened. He did, however, recommend two famousoncologists to contact regarding the ovarian cancer study, whichI did.

• PHOTOS PROVIDED BY FAYZAL MAHAMED24 August 2016 • EP MAGAZINE | eparent.com

INTERVIEW OF STEPHEN L. DEFELICE, M.D., BY JOSEPH M. VALENZANO JR.

PA R T   I I   o f a S E R I E S

PHOTO PROVIDED BY STEPHEN L. DEFELICE, MD FROM THE NEW YORK TIMES

EP Health Care Issue

eparent.com | EP MAGAZINE • August 2016 25

“They, and other oncologists I met, all wanted more costly pre-clinical studies performed before making a decision. I had somegood luck and arranged to have the study approved at a localhospital. But patients were tough to comeby. I asked a prestigious national founda-tion that deals with ovarian cancer to helpus locate patient volunteers, but they werenot interested.“When I tell this story to people they are

incredulous! They simply don’t under-stand how this could happen. My longexperience with carnitine and ovariancancer is difficult to accept, let aloneunderstand. “After this experience, the gods on

Mount Olympus sent me a message that itwas time to give up the ship. And so I did.I am not saying that carnitine is a miraclecure because it’s not. It’s a long shot. But itwas the only shot! “And there’s a reasonable theory as to

why it might work. Many tumors prefer sugar to feed on. Whatcarnitine does is to make cells eat fat and this effect may actual-ly cause tumors to starve to death or become more sensitive toanti-cancer drugs and the human immune system.

“This is nothing new. In 1931, Otto Warburg won the NobelPrize for his work on the anaerobic metabolism of cancer cellsand their need for sugar. There appears to be a ‘Warburg Revival’

underway now and this might hopefullybe helpful to patients.“To repeat, we’re dealing with an anti-

patient cultural mindset. The ovarian can-cer patients were at the end of the thera-peutic line and doomed to die. And, as Isaid before, there’s carnitine and doxoru-bicin sitting on hospital pharmacy shelvesimmediately ready to be administered.What most disturbs me is that patientswere not told about the option. It’s all partof our invisible Barrier System.” Dr. DeFelice summed up this situation.

“What’s the general message of this specif-ic experience? The FDA bureaucrats, thecorporate physicians, the medical founda-tions, and the oncologists form an inter-twined, complex system that creates

obstacles to promising clinical trials. Money reigns supreme. Lotsof it would have overcome the barriers to the ovarian cancerstudy.”Dr. DeFelice paused, looked me straight in the eye, which

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.”

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.”

PHOTO PROVIDED BY STEPHEN L. DEFELICE, MD

26 August 2016 • EP MAGAZINE | eparent.com

meant something big was coming. “Joe,my experience with carnitine and cancerexemplifies the general nature of ourBarrier System. The barriers are the samefor all promising therapies. I have hadsimilar experiences with nerve growthfactor in multiple sclerosis and a cervicalcancer vaccine, to name just two. “The ovarian cancer story; the example

of Rock Hudson on Good MorningAmerica; and others examples send anunequivocal message. We have a hugecultural blind spot to even thinking aboutof having a Cure Care policy and anabsolute blind spot regarding the essen-tial role of clinical research in medicaldiscovery.“To repeat, the good news is that the

simple, uncomplicated Doctornaut Act isthe solution. If, for example, female doc-tornauts with ovarian cancer had existedin the late 70’s, then many patients wouldstill be with us. And this discovery wouldhave led to clinical studies with the com-bination in other types of cancer.

After Dr. DeFelice described parts ofour labyrinthine system, I told himI couldn’t see how The Doctornaut

Act could change it—and he surprisinglyagreed! “The system cannot be changed,” he

asserted. “It is embedded in our culture,so you have to go add to it. TheDoctornaut Act is simply an uncomplicat-ed addition. But here’s the other goodnews. If the carnitine-doxorubicin combi-nation destroyed ovarian cancer tumorsthen, by public demand, the pressurewould be so great that the administrativesystem would have to make it available todoctors and patients as soon as feasible.And don’t forget, that doctors are notbound by the FDA to treat patients fornon-approved uses. Public pressure willplay a huge role in all major medicalbreakthroughs.I then challenged him, “You have tried

unsuccessfully for over 40 years, whatmakes you believe that now is the time toseize the moment?”Dr. DeFelice replied, “Our culture is rap-

idly changing its habits and values.People, particularly baby boomers, arepaying more attention and are betterinformed. Although there is much misin-formation from the media regarding

health and medical issues, the public doeshear about promising medical advances.This may help create a sense of urgencywhich we sorely need to bring aboutchange. Also, there’s also the cost of med-ical care which combined with the senseof urgency can change our cultural mind-set.” DeFelice switched gears again and said,

“Speaking of the media, notice that, after

reporting on a potential new therapy, theyroutinely report that it will take a longtime before it reaches the patient. Theynever—and I mean never—explain why!They themselves haven’t the slightestunderstanding of the Barrier System and itis tough to find experts to ask why this isso.” I asked Dr DeFelice the bottom line

question. “Would physicians be willing tobe Doctornauts?” It’s interesting to notethat in Michael Mannion’s book, AMaverick’s Odyssey, about Dr. DeFelice’squest to conquer disease, a few of hisphysician friends who are sympathetic tohis mission were not convinced doctorswould volunteer. Dr. DeFelice dismissestheir beliefs for a variety of reasons.Specifically, he learned in his work withprisoner volunteers for clinical trials howstrongly people are altruistic and want tohelp others. In his research unit in a state prison,

and at WRAIR, where he collaborated withtwo other prison facilities, he serendipi-tously discovered carnitine’s role in car-

diac disease in one of his prisoner volun-teers. This opened the doors to its develop-ment for Carnitine Deficiency in children.Dr. DeFelice suddenly smiled. This time

it was a cynical one. “Would you believethat later on, the FDA virtually closeddown prison research facilities? This cre-ated another significant barrier to discov-ery. And it robbed prisoners of the right tobe noble and courageous. The barriersnever stop. Once more, who pays theprice? The patient!” In 1983, because of his personal inter-

est in the promise of natural substances,the Foundation for Innovation in Medicineconducted a physician survey asking,“Would you, as a physician-patient, wantthe privilege to volunteer for clinicalresearch of natural substances under thesupervision of a physician-clinicalresearcher without any FDA, institutionalor other restraints?” Over 50 percent saidthey would. Women physicians were asbullish as the men.Today, there are over 900,000 U.S.

physicians in the U.S. If only 10 percentvolunteered, there would be 90,000Doctornauts, a substantial number forearly discovery phase studies where gen-erally only small numbers of patients areevaluated. Dr. DeFelice suggested that for-eign physicians might also be permitted tobe doctornauts in the United States. Whynot?“Dr. DeFelice, I understand your general

concept but how, specifically, wouldDoctornauts speed up medical discovery?”“Joe, generally speaking, Doctornauts

would participate in small, short-termclinical studies with potential therapiesthat offer more than ordinary promise,”he answered. “Doctornauts are not suitedfor long term clinical studies, such aswhether a cholesterol-lowering agent pre-vents heart attacks. Large numbers ofnon-patented, logical combinations ofpromising therapies, as well as naturalsubstance therapies, will be tested. Thiswill not happen without the DoctornautAct. Doctornauts are major door openerswhich will, without doubt, expand ourbase of medical innovators. “Here is another great example,” he

continued. “Genetic therapy, particularlythe newly discovered CRISPR gene-editingtechnology, is controversial. Peopleunderstandably fear it will alter human

“Did you know that

future health care cost

projections do not

include the

discovery of cures?

This presidential debate

has aroused the

interest of the public

and media as never

before. The people are

now listening.”

eparent.com | EP MAGAZINE • August 2016 27

nature in ways unknown. Costly and time-consuming barriers will certainly be erect-ed before the first dose is given in anyclinical study, let alone subsequent ones.This is bad news for orphan or rare dis-eases and disabilities. There are about7000 of them; 80 percent are due to genet-ic abnormalities. “It’s estimated there are 30 million

orphan disease patients in the UnitedStates, many of them who are children.But with Doctornauts, the barriers wouldbe reduced and discoveries made thatcould lead to new treatments for children.If, for example, a drug is effective in doc-tornauts with leukemia, it could also begiven to children. It’s a best kept secretthat the vast majority of drugs cannot getto the brain because of the blood-brainbarrier. A recent really exciting study inmice reported that, using viruses as thecarrier, not only drugs, but also genes canenter the brain. If studies in doctornautsprove this to be true, then this method canbe employed in children with multipletypes of neurological disabilities and dis-ease and would lead to dramatic medicalbreakthroughs. I asked “Why do you still believe that

our next president or even Congresswould become advocates of theDoctornaut Act?” Without hesitation, heshot back, “The national debt and theimpact of health care costs.” I asked him to elaborate. “Over the

years,” he began, “I‘ve come to know con-servatives and liberals both in the Houseand the Senate, as well as influential eliteswho impact public opinion and publicpolicy. About 25 years ago, I met with oneof the most liberal members in the Houseof Representatives, a thoughtful and sin-cere man who is still there. I explained therationale behind the Doctornaut Act, seek-ing his advice on how to move theCongress to enact it. “After a long moment of silent reflec-

tion, he confidently answered, ‘Make itclear how your doctornauts will reducehealth care costs. That will get our atten-tion because no one knows how to sub-stantially reduce costs except by politicalsuicide.’ He was, of course, referring tomaking big cuts in Medicare and Medicaidservices which even President Reagan, inhis cost reduction initiative, leftuntouched.

“I told him that the cost reduction argu-ment may not convince opponents whowould raise a legitimate argument: break-through therapies would be expensive andincrease costs. He agreed that this couldbe 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. Iexplained that, in our dynamic marketsystem, both expensive and inexpensive

therapies would soon be discovered andcompete with each other in the medicalmarketplace. “For example, the estimated cost for

Alzheimer’s by the year 2050 is $20 tril-lion—greater than our current nationaldebt. Also, the money saved by curingAlzheimer’s could be used for research ondiabetes, autism and other diseases. It’s awin-win situation. “Did you know that future health care

cost projections do not include the discov-ery of cures? This is mind-boggling andconfirms our cultural blind spot that theywon’t happen.“As I said before, this presidential

debate has aroused the interest of thepublic and media as never before. Thepeople are now listening. Before and dur-ing the presidential health care debate, wewill present the Cure Care versus HealthCare initiative. Through our educationaland public relations efforts, we will reachinfluential leaders who will encourageothers to join us. Senator Bill Frist’s previ-ous support of The Doctornaut Act will be

very helpful to us. “What will also help is the Act’s simplic-

ity. Unlike the 2000-page, labyrinthineAffordable Care Act, ours could be about12 pages long and can be read and under-stood within an hour!”

Finally, I was curious to learn abouthis marketing strategy. “I’mdepending on what I call a ‘Pascal

moment.’ The brilliant French thinkerobserved that small things can have bigimpacts. For example, if Cleopatra had areally big nose, Julius Caesar would nothave fallen for her. Roman history—andthe history of Western civilization—would have been different. Our Pascalmoment will be a small, but focused, pub-lic education effort that would hopefullyhave a large impact. Much depends ontiming, luck and prayers. And there’s nodoubt that I’ll be asking God for any helphe can give me. It’s now or never for theDoctornaut Act. Let’s give it our best. Weneed dedicated leaders to join us. I can’tdo it alone.” Well, I told him that Exceptional Parent

certainly will join forces. We plan to forma group of dedicated moms with childrenwith disabilities and diseases, Mothers forDoctornauts, who are committed tospreading the message. In the final analysis, Dr. DeFelice is the

one person who can coordinate andimplement the entire approach. Let’shope that he convinces our next presidentto seize the moment and successfullypush for the enactment of The DoctornautAct. •

Part I of this article appeared in EP’s July2016 issue, as well as on www.eparent.com

ABOUT THE AUTHOR:

Stephen L. DeFelice, M.D., is the founder and

Chairman of FIM, the Foundation for Innovation in

Medicine whose mission is to speed up the discov-

ery 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 www.fimdefelice.org.

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.

Exceptional Parent will

join forces with

Dr. DeFelice. 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.