Carnitine and Doxorubicin: Potential Promise for the Treatment of Ovarian Cancer

Carnitine is an extremely safe naturally occurring substance present in most body tissues with a particularly high concentration in the heart, body muscle and sperm tail. It is FDA approved as a drug or pharmaceutical and also available as a dietary supplement or true nutraceutical in health food stores and other outlets.  It has a number of actions two of which are the transfer of fatty acids into mitochondria, the furnaces of the cell, to produce energy and also as both a preventive and treatment antidote for many toxins. Because of its many modes of action it has abroad range of activities both in medicine and health.

During the Vietnam War I was stationed at WRAIR, the Walter Reed Army Institute of Research, where I met the late Major James Vick, an expert cardiovascular pharmacologists and a lover of life. He held rank over me for I was only a Captain. One sunny day, I remember it well, we lunched at the Officer’s Club where I told him about the medical promise of carnitine and the urgent need to conduct more research on its therapeutic possibilities. Well, the Major needed no more convincing, and we planned a series of laboratory experiments, one of them on the commonly used anti-cancer drug, doxorubicin, better known by its commercial name, Adriamycin. (Doxil, is another form of doxorubicin).

For certain reasons, the Department of Defense had an interest in finding treatments for toxins, a number of which can be used in biological warfare. Doxorubicin was on the Major’s list. It’s an effective anti-cancer drug for many tumors types such as breast cancer, but its dose is limited because it is also causes heart or cardiac toxicity. This toxic effect limits what dose can be administered reducing its potential anti-tumor effectiveness. If we reduced the cardiotoxicity with carnitine, we reasoned, then higher doses of doxorubicin could be given which would destroy more cancer drugs and save more lives or at least permit cancer patients to live a longer, toxicity- free life.

Our first experiment was a spectacular success. Carnitine completely blocked doxorubicin’s cardiotoxicity. While we were celebrating this success over martinis at the Officer’s Club it suddenly occurred to us that carnitine may also block the anti-cancer activity of this drug. It otherwords, it blocked all of doxorubicin’s actions. Let me tell you that this possibility took the wind out of our celebratory sails! Fortunately our colleague, Sam Barranco, was an expert on anti-cancer agents and volunteered to conduct a study on Chinese hamster ovarian culture cells to test this possibility. Wow! Were we surprised by the results and once more entertained ideas that we were on our way to a Nobel or some type of prestigious award. Carnitine not only did not block doxorubicin’s cell kill capacity but increased it tenfold! The message was twofold: Carnitine could dramatically increase doxorubicin’s ovarian cancer cell kill capacity while, at the same time,  protect the patient against its cardiotoxicity.

But then came the disappointing reality of our medical discovery system: I approached a number of pharmaceutical companies to further pursue this exciting lead, but without success. So I gave up the ship.

Then years later while having dinner with my late wife, Patrice, I was told by one of the waiters that the 25 year old attractive and soft spoken hostess was recently diagnosed with ovarian cancer. I called her over to the table, and we talked about it. My late kind wife, remembering the WRAIR study with the Major, who, by the way, became a Colonel before his tragic death, urged me to do something about it. So I did. As fate would have it, at that time I was discussing another carnitine study with a very distinguished physician, Professor Gordon Bernard at Vanderbilt University Medical Center. He introduced me to Dr. SK Dey, a very highly respected expert in fertility who happened to have available in his laboratory human ovarian cancer cells in culture, or what is equivalent to a test tube. This distinguished, innovative man volunteered to conduct a study at no costs.

What he discovered brought my energy levels back to pursue a clinical study. This opens the door to three potential way to destroy ovarian cancer cells:1) carnitne, by protecting the heart, makes it possible to increase the dose of doxorubicin  to more effective levels, 2) carnitine itself kills a high percentage of the cells and 3) carnitine adds to doxorubicin’s – or vice versa- tumor cell kill capacity. In my opinion, that’s an exciting and hopeful profile. Not only did carnitine increased doxorubicin’s kill capacity on these ovarian tumor cells but carnitine itself had a dramatic killing capacity!

I decided to test the combination in women with late stage ovarian cancer who have a certain rendezvous with death. There is no truly effective therapy and nothing much in the immediate research pipeline. I approached certain pharmaceutical companies to sponsor a clinical study, no names mentioned. I thought it was a no-brainer, but I was wrong. They expressed no interest in sponsoring the study. So I decided to sponsor the study myself and was fortunate enough to find an investigator at a local hospital to conduct the study. But there was one formidable problem. It was extremely difficult to find patients because of the nature of our clinical research system. I then went to ovarian cancer meetings and set up a booth trying to recruit patients without success. Ironically, our booth was next to the booth of a top ovarian cancer foundation but not one member of this group was curious enough to ask us about our study. I did the same at another of their meetings and, once more, no one asked. Tough to believe? Not so, for, to repeat, that is the nature of our clinical research system. Promising new therapies are only tested if they arise from the establishment such as the NIH, pharmaceutical industry and foundations. This is one of the best kept secrets from patients. There is little room for independent, creative clinical researchers particularly with non-patented drugs. They are not welcomed as part of the system. In addition, there are the tremendous costs and risks involved in testing potential new therapies in patients.

But I gave it one last try. At that time I still had a few powerful physician friends in academic circles who introduced me to a few oncologist leaders. I pointed out to them that the human ovarian cancer cell study was no fluke. Other scientific studies showed that carnitine kills human colon cancer and mouse liver cancer cells. They were good hearted guys but decided not to help because the study fell outside our current system. Also, before I forget, I didn’t have substantial personal funds to sponsor the study, a “killer” indeed.

In a previous post I warned that this anti-patient system was a growing monster .   In 1972 my first book, Drug Discovery: The Pending Crisis, I sadly predicted that there would be relatively few medical breakthrough therapies and fewer cures for a long time to come. And history has, unfortunately for most disease and disabilities, proven the prediction correct.

That’s why I proposed the Doctornaut Act. It would permit physician patients to volunteer much more easily to physician researchers  for clinical studies effectively reducing the costs and risks and waive the right to sue. As a result many more potential therapies would be tested and more discovered. For example, if there were late stage female physicians with ovarian cancer, they would have the right, which they don’t have now, to volunteer for carnitine- doxorubicin study without the current barriers.  If the Act were passed then my study would have been completed and, if positive, the results known by now and the treatment immediately available to all women because both carnitine and doxorubicin are FDA approved and already available by prescription.

Conclusion: So here we are in the year 2012 with tens of thousands of women stricken with ovarian cancer many of whom have a certain rendezvous with death because of a lack of effective therapies. In addition, the near term research pipeline of potential new therapies is not exciting and a breakthrough may be a long way off. Sitting on the shelves of hospital pharmacies and immediately available for treatment of patients are two drugs, carnitine and doxorubicin, which offer hope. And nobody is interested let alone angry about it. Something is wrong!

In order to recruit patient volunteers for the clinical ovarian cancer study, we formed the website Though the attempt to find patients was not successful, we decided to maintain the website as a source of information for both patients and doctors.

6 Responses to “Carnitine and Doxorubicin: Potential Promise for the Treatment of Ovarian Cancer”

  1. As an Ovarian Cancer survivor and trained scientist I am hoping you can provide more information about your research. Can you provide information regarding your OC cell study? Has your research been published? Have you done mouse studies prior to trying to test it on women? Are their adverse side effects for taking L-carnitine? If it can affect the your heart there have to be other significant organ affects. I can understand how difficult it is translating bench research into treatments in terms of cost and the difficulty recruiting women for clinical trials (or for any cancer clinical trial study). I believe most Late stage women will be looking to the larger NCI comprehensive cancer centers and the NIH for clinical trials.

    • Stephen L. DeFelice, M.D. Reply April 10, 2012 at 8:11 pm

      Dear Dee,
      The carnitne-doxrubicin journey has not been a main part of my career but because of its potential I had to go forward. All the information is on the carnitine website. You are correct in that women and doctors will stay in the system for that’s the general rule for all clinical research. But, historically, many discoveries are made by researchers outside the system. H. pylori for the treatment of gastric ulcers is a recent example.Doctornauts or physician volunteers would permit creative physicians to evaluate potential breakthrough therapies outside the system on their colleagues.And there are many such therapies out there..

      Carnitine is as safe as a substance can be with no significant adverse effects..I’ve been taking it for 40 years.

      And finally, I’m very happy to hear that you are a cancer survivor. God bless.

      Dr. DeFelice

  2. Surely it comes down to intellectual property? $10 million is a lot to invest unless there’s some prospect of commercial protection.

    • Stephen L. DeFelice, M.D. Reply April 10, 2012 at 8:21 pm

      Dear Flossie,

      You hit it on the nose. It’s just too costly for an individual creative physician to clinically test his ideas. Doctornauts or physician volunteers patients would dramatically lower the costs and permit many more therapies to be tested and discovered. But for some reason we have a blind spot to this obvious rapid road to discovery. Just try to find out whether your friends understand the critical importance of clinical research. I’ll bet the first thing they’ll say is how dangerous it is. Let me know.

      Dr. DeFelice

  3. Articles like this really grease the shafts of knowgedle.


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