Reprinted from eparent.com, September 2004
Dr. Stephen L. DeFelice, M.D.
In 1976 Stephen L. DeFelice, MD founded the Foundation for Innovation in Medicine (FIM). The mission of FIM is to rapidly accelerate medical discovery so that new, promising treatments can be made available for adults and children with disabilities and disease much sooner than our present system permits.
FIM has recently proposed “The Doctornaut Act” to Congress. A doctornaut is a physician who volunteers for early clinical research under the supervision of his colleagues. Doctornauts would have fewer restraints than non-physician volunteers and would be permitted to take greater risks. Dr. DeFelice firmly believes that doctornauts can accomplish the goals of FIM. If enacted, the doors that effectively block the availability of new treatments would immediately be unlocked.
Dr. DeFelice states, “There is a huge reservoir of modern technology that can attack disease now. But we need a system to make these new therapies happen.”
Senator Bill Frist, MD
Good news for the proponents of “The Doctornaut Act”is that Senator Bill Frist, MD, the only physician in the Senate, is highly attracted to the idea. One of his major goals in Congress is to “speed up medical discovery from the laboratory bench of the scientists to the physician at the bedside of the patient.”That is exactly what doctornauts will do!
The idea for doctornauts came about in 1965. As a young physician, heavily involved in clinical research, Dr. DeFelice concluded that there are many potential medical uses for carnitine, a natural substance found in every cell of the body. But he soon learned that the barriers to clinical testing were, more often than not, insurmountable. This was true not only for carnitine, but for many other new medical therapies. If a new treatment could not be tested in people, then it can’t be discovered. For example, penicillin was successfully tested in a test tube but remained undiscovered until it was tested in a patient with an infection. It is a simple but largely unrecognized and unappreciated fact.
One of the principle events that led Dr. DeFelice to create FIM happened during the Vietnam War, where he was Chief of Clinical Pharmacology at the Walter Reed Army Institute of Research. He was a member of the team that developed drugs for the Department of Defense, including anti-malarial and anti-radiation compounds that could protect our troops against drug resistant malaria and nuclear fallout. His interest in carnitine motivated him to conduct a number of laboratory studies on carnitine in his free time. He worked with Major James Vick, an expert scientist in cardiovascular pharmacology. One study they worked on included adriamycin, a powerful anti-cancer drug. Adriamycin was effective at killing cancer cells but could only be given in limited doses. Too high of a dose causes cardiac toxicity and heart failure. The doctors’ tests demonstrated that carnitine blocks the cardiac toxicity of adriamycin but not its anti-tumor activity.
His tests showed that in an ovarian cancer tissue culture, carnitine dramatically increased adriamycin’s ability to kill these malignant cells.
Additional testing by other researchers have confirmed their findings. They were joined by Dr. Sam Barranco, a colleague and cancer research scientist, who made the most surprising discovery. His tests showed that in an ovarian cancer tissue culture, carnitine dramatically increased adriamycin’s ability to kill these malignant cells.
Excited by the reasonable possibility that adriamycin could now be given with carnitine to various types of cancer patients at higher, much more effective, doses with the possibility of curing or at least prolonging the lives of many cancer patients, Dr. DeFelice began his long journey to find someone who would sponsor and conduct the clinical definitive trials of these two substances in certain cancer patients. Unfortunately, almost forty years later, he is still searching.
After a persistent effort over the course of many years, carnitine was finally approved by the FDA as an orphan drug for certain rare fatal diseases in children with carnitine deficiencies as well as for carnitine deficient renal dialysis patients.
Assuming that carnitine is effective, Dr. DeFelice wonders how many people who have gone through the torment of cancer could have been saved or at least could have had a prolonged and better quality life.
Unlike vast oceans or high mountain peaks, the barriers to clinical research are not ones of physical dimensions. Instead, they are man-made and hard to see, easily escaping national notice. The barriers of clinical research come in the forms of bureaucratic regulations and red tape, threats of litigation, faulty bioethical judgments, lack of proper consideration of the needs of patients, lack of consideration of the importance of clinical research, and weak or nonexistent patents. These barriers are just some reasons why carnitine, along with many other promising new medical therapies, has never been adequately tested or tested at all in human volunteers.
What Can You Do to Help?
Your support is extremely important to encourage members of Congress to support the Doctornaut Act. Remember, stem cell or anti-cancer treatments discovered in doctornauts can rapidly be applied to children. You can help by contacting Dr. Bill Frist at http://frist.senate.gov, along with your local senators and representatives. You can access your local information from the homepage of our FIM website http://www.fimdefelice.org or go directly to both http://www.congress.org or http://www.senate.gov.
FIM strongly holds to the belief that our window of opportunity is unique and very narrow, but must not be lost! FIM also believes that it is you, the parents of children with disabilities and disease, who can be the most important force to help convince Congress to pass the Doctornaut Act. We must work together and tell others to join the effort!
As a final note, after a persistent effort over the course of many years, carnitine was finally approved by the FDA as an orphan drug for certain rare fatal diseases in children with carnitine deficiencies, as well as for carnitine deficient renal dialysis patients. It is important to note that carnitine was first tested in adults before being given to children. Unfortunately, its other major medical promises, such as the carnitine-adriamycin combination, will remain in medical limbo because of the bolted doors to medical research. Doctornauts can change this.
For more information contact:
The Foundation for Innovation in Medicine
411 North Avenue East, Cranford, New Jersey 07016
(908) 272-2967 Fax: (908) 272-4583
Reprinted from eparent http://www.eparent.com