Month: March 2025

Promising Opportunities for Doctornauts

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.

A Promising Recent Neurological Clinical Study

Breakthrough Study on GM1 Ganglioside Offers New Hope for Neurological Disorders

By Stephen L. DeFelice, M.D., Founder and Chairman | March 24, 2025

A recent clinical study on GM1 ganglioside has shown significant promise for treating a variety of neurological diseases and disabilities. (See the study here).

Previously, the Italian pharmaceutical company Fidia developed a method to extract gangliosides from bovine brains, marketed under the brand name Cronassial. Gangliosides are vital substances found in the brain and neuron membranes, playing a critical role in neuronal growth and development.

The product was approved in several countries but was later removed from the market due to safety concerns, specifically regarding Guillain-Barré syndrome and the purity of the extract.

At the request of Fidia, my organization assembled a peer group of medical experts to review the available data. They concluded that there was sufficient evidence to support the initiation of clinical trials. As a result, I took the responsibility to plan and manage U.S. clinical studies on the bovine extract, administered in daily intramuscular doses of 40mg.

A double-blind study conducted on ALS patients under the leadership of Walter Bradley, Chairman of Neurology at the University of Vermont, showed no significant activity. Similarly, another study on diabetic neuropathy, conducted by renowned neurologist Mark Hallet at Peter Bent Brigham Hospital, also yielded no noticeable effects.

Dr. Hallet, who later became Chief of the Medical Branch of Neurology at the NIH, shared with me the findings of a Chinese clinical study that demonstrated the impressive efficacy of GM1, a purified ganglioside extract, in reducing ataxic dysfunction in patients with Spinocerebellar Ataxia Type 3, a neurodegenerative disorder.

Our negative findings in the U.S. may have been due to several factors:

  • The dose used in the study may have been too low.
  • Pure GM1 could be more effective than the mixture used in our trials.
  • In contrast to the U.S. trials, the purified GM1 was administered intravenously, ensuring optimal availability in the body.

The Chinese study indicated that the high dose of GM1 was effective—a 400mg loading dose followed by 200mg daily. Notably, the 40mg dose in our study was ineffective, suggesting that a higher dose of Cronassial may also yield positive results.

The positive clinical results from the Chinese study were evident after just 12 weeks of GM1 administration. This opens up opportunities to conduct cost-effective, short-term efficacy studies, including the use of biomarkers, with an acceptable risk-benefit profile. This approach could replace the need for lengthy, expensive studies.

Furthermore, GM1 can be synthesized, providing a potential commercial opportunity. While I am unaware of GM1 being marketed in any country, it’s important to note that I have no financial stake in this pursuit.