The Promise of the Combination of Adriamycin and Carnitine
For the Treatment of Cancer
By Stephen L. DeFelice M.D.
During the Vietnam war, Major James Vick, an expert cardiovascular
pharmacologist, and I were stationed at the Walter Reed Army Institute
of Research (WRAIR) where we conducted animal studies which showed that
carnitine dramatically reversed myocardial ischemia lack of oxygen to
Carnitine is a naturally occurring substance that transports fatty
acids into mitochondria, the furnace of the cell, where they are
metabolize to produce ATP or energy. It is present in high
concentrations in high energy-requiring cells such as those found in the
heart as well as sperm cells. We postulated that carnitine works by
maintaining the function of the mitochondria preventing cellular death,
or apoptosis, which commonly follows ischemic attacks.
At that time, we were also interested in the toxicity of
adriamycin, an anti-cancer drug, that is highly effective in various
types of malignancies.
Though it is extremely effective in killing cancer cells, its dose,
unfortunately, is significantly limited because of cardiotoxicity that
can lead to fatal heart failure.
Major Vick and I wondered whether carnitine could eliminate or at
least reduce adriamycin's cardiac toxicity. If so, then higher doses
could be given to cancer patients increasing its tumor-kill capacity
resulting in the saving or prolonging of many more lives.
We initiated a series of acute and chronic animal studies ranging
from isolated dog hearts to intact monkeys. In every laboratory model
carnitine dramatically reduced adriamycin's cardiac toxicity!
We also studied the effect of carnitine on daunomycin, a derivative
of adriamycin, which has similar anti-tumor activity and
cardiotoxicity. Carnitine worked equally as well.
It is important to note that other investigators have subsequently confirmed our findings for both substances.
During our initial excitement a sobering- and discouraging, may I
add- thought occurred to us. What if carnitine also blocked adriamycin's
We then collaborated with a cancer research specialist and
colleague, Dr. Steve Barranco, to plan a laboratory study that would
test this possibility.
The study, using exponentially growing mono layered cultures of
Chinese hamster ovary cells, showed that carnitine did not block
adriamycin's cell kill capacity but actually increased it by a factor of
ten! (1) Other investigators also have confirmed that carnitine does
not block the anti-tumor activity of adriamycin. (2)
After our discovery, we popped open a bottle of champagne and a can
of peanuts to celebrate this potential medical breakthrough for cancer
But the celebration was short-lived. For over thirty years I have,
as an individual, have tried to find a company or organization to
sponsor a clinical trial to determine whether this promise would hold in
certain cancer patients.
I've failed. Despite an intensive personal effort there was, and remains, little interest.
I then made available $50,000 of my own money as an grant for the
medical institution that would conduct a preliminary clinical study to
evaluate the promise of the combination in a small number of certain
It was not enough! The medical institutions all requested that I
indemnify them because of the fear that carnitine might not block
cardiac toxicity and the probability of being sued would be high. The
cost for the amount of indemnification left little funding for the
clinical trial costs.
If Congress had passed the Physician Volunteer (Doctornaut) Act
that I proposed thirty years ago which would permit physician patients
to volunteer for clinical studies at much reduced costs and risks, our
hypothesis would have been tested a long time ago.(3) If confirmed, it
is likely that hundreds of thousands of cancer patients -- and perhaps
more -- who have succumbed to their neoplastic diseases would be with us
1. Vick: J.A., DeFelice: S. and Barranco: S. Potentiation of the
Cytotoxicity of Adriamycin by the Cardioprotective Drug Carnitine.
Cancer Research 8: 312, 1978
2. Alberts: D.S., Peng: Y.M., Moon:
T.E., and Bressler: R. Carnitine Prevention of Adriamycin Toxicity in
Mice. Biomedicine Nov; 29(8): 265-8
3. Fimdefelice.org: DeFelice
Commentary: Doctornauts, Physician Volunteers for Clinical Research: The
Time Has Come for Congress to Act.