Home > Uncategorized > Off-label Drugs and its potential use against cancer – An Intro by Raymond Chang MD

Off-label Drugs and its potential use against cancer – An Intro by Raymond Chang MD

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The off-label use of a drug is the prescription or taking of a medicine for other than what it was originally intended for as described or approved by the US FDA or similar regulatory body. Simply put, medication usage for what is not on the official label of the drug is “Off-Label”.

Although hardly uncommon, there are distinct reasons why off-label prescribing is not as common as it ought to be if based on scientific evidence of efficacy alone. Firstly, it is unlawful to market, advertise or promote the off-label use of drugs (See an example of the intricacies and complexities as well as the conundrum of off-label regulation by FDA, US Congress and the Courts as evidenced by the recent saga of the Neurontin case in this 2004 paper by Robert Kaufman of the Harvard Law School).

Secondly, the insurance industry frequently invoke the off-label nature of a prescription to decline payment (ie they will not pay if they can find a reason not to pay, why would they? See illustrative story here), although Medicare in recently expanded its coverage of off-label treatments for cancer (See related news here), although it mainly applies to the use of an agent already approved for the coverage of some form of cancer to be covered when used for a different cancer, which is quite different from the drugs presented here ( approved for some other condition than cancer, to be applied for use as an anti-cancer), ie it is off-label use of a cancer drug rather than off-label use of a drug for cancer.

Finally, although the FDA does not regulate the individual physician’s prescription of a drug as long as it has been approved,  the legal liability for the physician is deemed higher especially if harm arises out of the course of its use and if it seems to deviate from “standards of care” which is how physicians are legally judged. Furthermore, physicians may be deemed to be engaged in human experimentation when prescribing drugs off-label (See a balanced discussion by Maxwell Mehlman JD on the legality and bioethics issues of off-label prescribing here)

It is because of potential legal risk on the part of the prescribing physician, limitations of insurance reimbursement, as well as the lack of knowledge about the potential off-label usefulness that limits the broader use of such drugs.  The unlawfulness of pharma related marketing or promotion and their lack of interest in investing in new clinical trials to demonstrate new indications when a drug has already gained FDA approval is often a factor limiting the broader use off-label treatments.

Back to the cancer patient: my purpose here is just to broaden the awareness of the science behind the usefulness of some very common and some not so common drugs that could jointly or otherwise enhance a patient chances of overcoming cancer, and to disentangle the healing process from insurance red-tapes and legal suffocation (See Disclaimer).

More useful info on Off-label Drugs for cancer can be found at the NCI site here.

TOPICS in this blog include:

Introduction to Off-label drugs for cancer

Posts (Publisjed, Most Recent or Recently Updated First)

  1. Statins, pleiotropic anti-cancer (6.10)
  2. Gamma-delta immunotherapy (2.09, updated 6.10)
  3. Urso as chemopreventative (5.09)
  4. Noscapine  near perfect (5.09)
  5. Naltrexone (2.09)
  6. Bisphosphonate (2.09)
  7. Clodronate for breast cancer (2.09)
  8. Gossypol (11.08)
  9. Metformin (10.08)
  10. Disulfirim (11.08)
  11. Dipyridamole (11.08)
  12. Cimetidine (11.08)

Draft Topics (To Be Published)

  • Cox-2 inhibitors (e.g. Celecoxib i.e. Celebrex)
  • PPAR agonists
  • Heparin
  • Anti-coagulants
  • Coumadin
  • Tetracyclines as Anti-angiogenics
  • Clarithromycin against lung cancer
  • Artesunate and Antimalarials for cancer
  • Lithium as immunomodulator
  • Anti-depressants against cancer
  • Cannabinoids
  • Benzodiazepines
  • Theophylline for B cell leukemia (CLL) / lymphoma (NHL)
Categories: Uncategorized Tags: , ,
  1. Phenix
    July 14, 2011 at 8:54 pm

    I am working on statins anti-cancer therapy, this is the best review of this field. I can’t believe Dr. Chang didn’t published it in professional journals like nature reviews.
    Many thanks for your free share these information.

    • July 14, 2011 at 11:02 pm

      Very kind of you, I am glad you enjoyed the blog. R

  2. ISO 9000
    August 9, 2011 at 1:54 pm

    Hey, very nice site. I came across this on Google, and I am stoked that I did. I will definately be coming back here more often. Wish I could add to the conversation and bring a bit more to the table, but am just taking in as much info as I can at the moment.
    iso 9000

  3. Michele
    December 23, 2011 at 2:24 am

    Dear Dr. Chang:

    I would like to ask if you have any thoughts on Procaine? It looks to good to be true, but the constituants, paba, choline, diethylaminoethanol are controversial w/regard to cancer.

    Procaine, an ester anesthetic, is metabolized in the plasma by the enzyme pseudocholinesterase through hydrolysis into para-amino benzoic acid (PABA), (derivatives of 4-aminobenzoic acid)

    Procaine has also been shown to bind or antagonize the function of N-methyl-D-aspartate (NMDA) receptors as well as nicotinic acetylcholine receptors and the serotonin receptor-ion channel complex.

    Procaine Is a DNA-demethylating Agent with Growth-inhibitory Effects in Human Cancer Cells
    Procaine and procainamide inhibit the Wnt canonical pathway by promoter demethylation of WIF-1 in lung cancer cells.

    MAO inhibitor

    reduces cortisol

    Improved Therapeutic Index of Cisplatin by Procaine Hydrochloride

    Procaine Turns-Off Telomerase And Destroys Cancerous Cells – A Potential New Treatment Of Cancer

    Thank you.

    • January 11, 2012 at 1:16 am

      Yes, I have been busy because my book Beyond Magic Bullets just came out and have been working on that past few months, but hopefully get back to this blog again soon. You have very good suggests and will definitely get to review them. Thankyou Michele !

  4. Johnie
    February 5, 2012 at 5:46 pm

    Doc; chemobrain results from upregulation of IL-1 in brain and subsequent general inflamation and confusion…etc. A very nice way to prevent or remove chemobrain is to place the pt on modafinil(provigil) and or minocycline ,or doxycycline. They work very nicely because each one of them cause a down regulation of IL-1 thru different mechanisms.
    the result is very impressive and allows the pt to maintain their mind inspite of chemo.

    just thought i would share this with you
    keep up the great work Doc! 🙂 jb

  5. Johnie
    February 5, 2012 at 5:52 pm

    Doc –just a thought on theanine and doxorubicin…. Theanine inhinits the efflux of doxorubicin thru inhibition of nucleoside transport systems in cancer cells therby increasing the doxo concentration in the cancer cells. It spares normal cells. the reasons are complex but understandable.
    It is also provides cardioprotection against doxo induced cardiac damage..

    refs are in pubmed.

    you do great work doc!

  6. mena
    May 1, 2012 at 8:35 am

    doc — ethacrynic acid (edecrin) , messes with the binding of lef-1 and beta catenin in B-cll cells and causes lots of apoptosis —-
    just fyi —-ref on pubmed. 🙂

    • May 2, 2012 at 3:58 pm

      Edecrin is an interesting pleiotropic diuretic with potential off-label use in cancer. There is quite a bit of in vitro research on this interesting compound. Thanks for the pointer !

    • mena
      August 9, 2012 at 8:49 pm

      doc– regarding chemoresistance —- verapamil,loperamide,quinine,reserpine,celexicob,egcg,theanine,dipyridamole,trental,methylxanthines, can each inhibit specific efflux pumps,mdrs,nucleoside transporter,topoisomeraes,glutathione based pumps,…etc to help overcome chemoresistance in some of the various commonly used chemo drugs(doxorubicin,platin-based chemo)…etc
      like they say cancers dont become chemoresistant thru magic—-there is usually a mechanism. and where there is a mechanism , there is usually some way to screw with it.
      keep up the great work Doc!! 🙂
      all the references for the above agents are found in pubmed.

  7. mena
    May 1, 2012 at 8:39 am

    Doc —-celebrex and indomethacin each inhibit multidrug resistance efflux pumps in cancer cells(and bacteria) ….verapamil inhibits p-glycoprotein based resistance mechanisms in some cancer cells. 🙂

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