Off Label Drugs for Cancer

  • Aspirin             – Heart Disease
  • Artemisinin    – Malaria
  • Metformin      – Diabetes
  • Mebendazole  – Worming tablet
  • Doxycycline    – Antibiotic
  • Naltrexone      – Used to treat drug addiction in high doses


The medicines above are showing promise in the treatment of cancer. Research is early stage and as yet there are no large scale phase 3 clinical trials demonstrating efficacy and safety. However they are all pretty cheap with good side effect profiles. So why not use them? Well, we don’t really know for sure that these agents work. They may make the situation worse. Doctors like to see a robust phase 3 clinical trial and more before they feel comfortable prescribing a new drug. Things could go wrong and if the drug is unlicensed then the practitioner may be vulnerable to a complaint or lawsuit. The use of these medicines for cancer would have been made much easier if the recent “Off Label Drugs” Bill had not been filibustered  in Parliament last year, but don’t get me started on that…..

So what does one do if articles appear promoting the use of medicines for the treatment of cancer in the absence of big clinical trial evidence? Doctors are busy and “evidence-based” guidelines may be out of date. The huge volume of research being published means some important developments get lost in transit. Some doctors will prescribe – for instance my oncologist has advised me to take low dose aspirin – risky with a bowel problem but the evidence is pointing to good effects of aspirin in patients with gastro-intestinal cancers.

The inspiring survival of Professor Ben Williams who recovered from  one of the most deadly brain tumours – grade 4 glioblastoma multiforme clearly demonstrates the benefit of using appropriate off label drugs under appropriate supervision and monitoring.




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