Promising Opportunities for Doctornauts: Exploring Gangliosides and the Carnitine-Doxorubicin Combination in Ovarian and Other Cancers
By Stephen L. DeFelice, M.D., Founder and Chairman | March 24, 2025
On the FIM home page a Doctornaut is described as a physician who is substantially freer to volunteer for clinical studies. This would include the vast world of substances without patents such as natural substances. As a result, the cornucopia of promising new therapies would be tested for their clinical effectiveness, a significant percentage of which will be available much sooner than usual. Also, the discovery of cures and low-cost therapies will result in the reduction of healthcare costs beginning in the short term.
Take the GM1 ganglioside as a doctornaut opportunity. Gangliosides are natural substances found in neurons that create nerve growth or sprouting. Now, there are many neurological diseases and injuries in which nerves are damaged and die (apoptosis). But normally, because of the high costs and risks to obtain FDA approval for a single disease or condition, a company would choose to develop GM1 for a single disease or condition, provided, of course, it has sufficient product ownership such as a strong patent. But if doctornauts were available, many health organizations and small groups covering a broad range of neurological conditions from spinal cord injury to stroke to degenerative diseases, among others, would—patent or no patent—sponsor small probing clinical trials designed to detect clinical activity in a relatively brief period of time instead of years—if, of course, doctornauts were available.
Carnitine and doxorubicin offer another opportunity for doctornauts. Doxorubicin is an extremely broadly effective anti-cancer drug. The dose, however, is limited because of its cardiotoxicity. During the Vietnam War, while at WRAIR, the Walter Reed Army Institute of Research, cardiovascular pharmacologist James Vick and I teamed up and demonstrated that carnitine blocks doxorubicin’s cardiotoxicity, which has been substantiated by others. This offered the clinical promise that the dose of this chemotherapeutic agent could be raised, thereby increasing its efficacy. Working with others, it was shown in laboratory studies that the combination of carnitine and doxorubicin was effective in various neoplastic models, but carnitine itself had an apoptotic or killing effect on ovarian cancer cells.
I ran into a stone wall in an attempt to demonstrate this effect in a clinical study until I succeeded in obtaining approval at St. Joseph’s Hospital in New Jersey in 2007. But the physicians refused to subject their ovarian cancer patients to enter the study. The following is the rationale that was presented to the hospital committee, but more general studies, including clinical, have been published since:
Summary of the Rationale of the Carnitine-Doxorubicin Combination for the Treatment of Ovarian Cancer presented to St. Joseph’s Hospital Committee for Study Approval (IRB) (Ovarian Cancer Summary)
Now, just imagine if the St. Joseph’s study was conducted in 2007 and confirmed that the combination was extremely effective. How many ovarian cancer patients would have benefitted, and suffering avoided? Also, imagine if women doctornauts existed, and the aforementioned happened much sooner?
Physician Survey of Male and Female Physicians Confirming Their Willingness to be Doctornauts
Because of my particular interest in carnitine and the urgent need to conduct more clinical research on natural substances, in 1983, FIM conducted a survey on 2,100 male MDs, 500 female MDs, and 500 DOs potential doctornauts asking the following question: “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 FDA, institutional or other restraints?”
Over 50% in each category responded in the affirmative! But here’s another encouraging finding. We called about 100 doctors asking the same question but adding, “if you had a disease such as Alzheimer’s or another disease or disability,” would you volunteer? Almost 100% said they would! This entire experience is described in my book, From Oysters to Insulin: Nature and Medicine at Odds.”
In my next post on the Nutraceutical Research and Education Act or NREA and the Doctornaut Act, I will describe my collaboration with then Senate Majority Leader Bill Frist on the Doctornaut Act and how, with our booming technology including AI, doctornauts can jump-start the discovering of medical breakthroughs, including cures—and how those breakthroughs can reshape healthcare.