WESTFIELD, N.J., May 23, 2013 /PRNewswire-USNewswire/ — Stephen L. DeFelice, M.D., Founder and Chairman of FIM, The Foundation for Innovation in Medicine, released a new video criticizing mainstream health care cost projections of Medicare, Medicaid and Obamacare, among others, because they omit the discovery of cures. He cites a boom in technology, including gene modification and new organ growth as inevitable in curing many of our major costly diseases. View the video here:
“The Alzheimer’s Association projects that the cost of managing this life stealing disease over the next 40 years is 20 trillion dollars, more than the national debt. Current annual costs for diabetic care, in another example, are about $250 billion, which will rise over time approaching the Alzheimer’s level over the next 40 years,” the Doctor points out.
He cites formidable problems blocking discovery of cures under our current system.
“When was the last cure?” he questions, and finds it puzzling that few ever ask this question. In his book, Drug Discovery: The Pending Crisis (1972) he warned of a huge, unrecognized barrier effectively preventing the discovery of cures. “Our present system of drug discovery is almost designed not to cure the great diseases that confront us. I have no doubt they’ll be cured in the future, but many must wait.
“Discovery of new therapies can only happen if they are tested in patients in clinical studies, but we have made testing and research so costly – and risky – that promising new therapies remain in the shadows.”
But Dr. DeFelice says we have a cultural blind spot to the critical role of clinical research and, instead, view it with suspicion. He proposes that doctor-physician patients have the right to be much freer to volunteer for clinical studies than non-doctors. He later coined the term “Doctornauts,” to describe their role and subsequently proposed the Doctornaut Act to Congress, which grants the doctors this right.
Dr. DeFelice sees encouraging news as Congress recognizes the importance of clinical research’s role in medical discovery. He collaborated with then Senate Majority Leader, and physician, Bill Frist, who circulated a discussion draft of the Act which is posted on the FIM website (see below). Recently, Congress, largely supported by Senator Tom Harkin, agreed to fund the Translational Science initiative which is based on the need to help doctor researchers conduct clinical studies. This initiative was sparked by two directors of the NIH, Drs. Elias Zerhouni, the former director and Francis Collins, the current one. “Any effective way to diminish the barriers to clinical research will lead to cures in the short term and to dramatically reduced health costs,” the Doctor points out. “And the Doctornaut Act can make that happen much sooner, he says.
Dr. DeFelice is the physician who brought the orphan drug carnitine into the United States and conducted the first clinical trials on it.
Dear Parents of Children with Diseases and Disabilities: Pay Attention for They Desperately Need Doctornauts. It’s Time to Get Angry- and Active!
In previous posts I emphasized how physicians or doctornauts who volunteer for clinical studies can rapidly speed up the discovery of medical breakthroughs and cures . In the past, I also emphasized that doctornauts, though adults, can do the same for children. Unfortunately, I wasn’t successful in getting the message across to you, their parents.
Last week I had dinner with my old and kind friend, Joseph Valenzano, the dedicated founder and President of Exceptional Parent (EP), a long –standing, highly respected educational organization which deals with children with diseases and disabilities. During dinner he urged me- to tell the truth, he really gave me hell- not to give up on educating you, the parents, on how doctornauts can dramatically help your children. Joe’s a very persuasive guy, so here I go again!
There are two categories of childhood maladies, one that are relatively common and the others rare. The latter are oftentimes called orphan diseases. In the former category are conditions such as cerebral palsy, asthma, diabetes, leukemia, brain tumors, autism, and ADHD. In the latter category it is estimated that there are plus/minus 6000 conditions most of which are of genetic origin. Because of their small populations and , therefore, for economic reasons, relatively few clinical studies are conducted on them. One source reports that 30% of children with rare diseases will die before their fifth birthday. The encouraging news is that genetic discoveries in more common children’s diseases will be effectively applied to many of them.
Let’s take an example on how adult doctornauts with Parkinson’s disease can help children with Cerebral Palsy. Parkinson’s is caused by the destruction of certain brain cells. There are various ways to treat this disease. One is with pharmaceuticals. But there is often a problem with promising drugs in animal studies because of what is called the blood brain barrier- which prevents them from getting to the brain. Successful research on ways to eliminate the blood brain barrier in doctornauts with Parkinson’s can also be applied to cerebral palsy children. Discoveries made in doctornaut Parkinson disease patients with stem cell implants, insertion of genes into cells and even the use of electric devices to control movement among others are all applicable not only to Cerebral Palsy children but also other central nervous system diseases of children.
Here’s another example: let’s say a brilliant researcher has an out-of-the –box idea on a new therapy for leukemia which FDA and hospital committees would not approve for clinical studies because they believe it’s too risky. But because of the Doctornaut Act a physician with leukemia can and does volunteer, and the therapy proves to be very effective. This same therapy would then be available to children with this disease as well as adults.
( FYI, there are many bright researchers with creative therapeutic ideas which are never tested let alone made public because of the barriers to clinical research. And they remain unknown because there is no place for them to go to have their ideas evaluated in a clinical study. In addition to the establishment, these folks are essential to speed up medical discovery).
Awhile back- before some of you were born!- I brought carnitine into the United States and conducted the first clinical study in adults in patients with hyperthyroidism. Because of its availability it was tested clinically in children with the fatal disease, Primary Carnitine Deficiency, and eventually approved by the FDA saving a number of children’s lives.
You may wonder why there has been little interest in the Doctornaut Act. Despite my personal efforts, with the exception of physician and former Senate Majority Leader, Bill Frist, who had a discussion draft of the Doctornaut Act circulated but failed to attract support, I don’t have a clear answer except the well accepted general rule that there is an establishment system which very rigidly regulated where bold ideas are not well received. There’s an old saying that progress is disagreeing with the established experts. Also, patients are not aggressive and understandably surrender their lives to the rules of the system. But there has been one dramatic exception to this rule that tells us that mountains can be moved. It’s AIDS patients. In the early days of AIDS there was little support to conduct research on this fatal disease. The angry AIDs patients then got together and launched an extremely effective public relations campaign , strongly supported by mass media, resulting in enormous sums of money poured into AIDS research. And this effort handsomely paid off with the discovery of very effective, life-saving breakthrough therapies.
Physician Doctornaut Survey on Natural Substances
Because of my experience with the natural substance, carnitine, I, being trained mainly to study artificial, patented pharmaceutical drugs, turned my attention to the non-patented, largely ignored world of natural substances be they legally considered a food or a drug. And what a discovery it was! The promise is enormous, but because of the difficulty of obtaining strong enough patents to economically justify the investment required to at least conduct a single solid clinical trial on them, their promise continues to be largely undiscovered.
To overcome the lack of economic incentives to conduct clinical studies on natural substances, FIM conducted the first and, I believe, only physician doctornaut survey at that time- and perhaps since- by mailing the following postcard to 3,100 doctors, medical doctors and osteopaths, asking them to respond to the question, over 50% responded in the affirmative.
I then conducted a personal phone call survey to many of them asking whether they would do the same with pharmaceuticals and medical devices and the response was also similar. Today there are approximately 900, 000 physicians in the United States. If 50% are willing to be doctornauts, then there would be over 400,000 physicians willing to volunteer for potential new therapies at low cost dramatically increasing the speed and breadth of medical discovery!
Even if, today, it were only 20%, it would leave us with an enormous pool of courageous doctors. Certainly, in our current culture there would have to be some type of regulation which the Doctornaut Act would provide but, if structured properly, the impact would be the same.
Professor Stephen L. DeFelice, M.D.
Plenary Lecture September 9, 2014
XXV National Congress of the Italian Chemical Society – SCI – The University of Calabria.
This year marks the 25th anniversary of the coining of the term nutraceutical by Dr. DeFelice.
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