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Non-standard options, such as off-label drugs, are available for improving treatment outcomes, reducing cancer risk, or managing side effects. These are sometimes referred to as repurposed drugs for cancer.
These therapies are not standard treatment, but off-label use of drugs is very common in cancer treatment. Even so, they may not be offered as options by either conventional or complementary medicine practitioners, even though substantial evidence is available for many of them. These therapies may enhance conventional treatments, improve survival and reduce the risk of cancer or recurrence, while others may help manage side effects and symptoms such as pain or fatigue.
This group includes several types of therapies:
If you choose to explore these therapies, it is strongly recommended to seek guidance from a qualified professional who can help you choose the off-label and other novel therapies that are right for you and that are safe to use with your other therapies. Some of these therapies are readily available at low cost, but others may involve considerable expense and perhaps travel for access.
Useful Resources:
Yes to Life Radio Shows on this therapy:
Further Reading:
Off-label (repurposed) drugs covered here are: Statins, Metformin and Low-dose naltrexone (LDN)
Statins are cholesterol-lowering drugs used to reduce mortality and illness for people at risk of cardiovascular disease. They are sometimes used as an ‘off-label’ cancer treatment, with preliminary evidence suggesting they may be a potential future anti-cancer therapy.
Statins are a class of drugs that are currently widely used to lower cholesterol. A secondary relationship between statins and cancer is being researched, with studies potentially linking certain lipophilic statins (that combine with or dissolve in lipids/fats) to with anticancer effects. This is because statins may have inhibitory effects on tumour growth, promoting cell death and preventing metastasis (1).
However, research is still unclear on the anti-cancer effect of statins, as studies exploring their effects tend to be observational, and thus involving patients with a range of health condition including heart diseases and diabetes. Therefore, understanding the exact effects of statins on cancer is difficult and requires further research and clinical trials (2).
Importantly, certain statins have also been linked to an increased cancer risk in elderly patients. However, alternative studies have countered this, suggesting there is no increased cancer risk from statins. This highlights how further research is still important to understand the potential role of statins as a cancer therapy (3).
Since the 1950s scientists have believed that increased cholesterol levels are a risk factor for heart disease. Thus, finding a drug to reduce cholesterol levels was important. In 1976, Japanese scientist Akira Endo identified the statin ‘compactin’ from a fungus. Compactin was found to lower cholesterol in many animal species, and later clinical trials began finding promising effects in humans. In 1978 another statin was identified by Alfred Alberts, named lovastatin, with clinical trials beginning in 1980. In 1987, lovastatin was the first approved statin in the US (4).
Adoption of statins in medical practice was slow in the 1980s/90s, however in 1994 a study claimed that there was a 30% reduction in mortality in patients with coronary heart disease who had been treated with statins compared to a control. Now several strains of statins are routinely used worldwide to lower cholesterol (5).
– Statins can be prescribed on the NHS to lower the level of low-density lipoprotein (LDL) cholesterol in the blood and come as tablets to be taken daily. – It is important to understand that statins can have serious side-effects, and considering their off-label use for cancer treatment needs to be carefully considered and discussed with a doctor. This is because statins are not recommended for all people to take and can also interact unpredictably with other substances/medications.
(1) Jiang, W. et al. (2021) Statins: A repurposed drug to fight cancer – journal of experimental & clinical cancer research, BioMed Central. Available at: https://jeccr.biomedcentral.com/articles/10.1186/s13046-021-02041-2 (2) Barbalata, C.I. et al. (2020) Statins in risk-reduction and treatment of cancer, World journal of clinical oncology. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7443827/ (3) Statins (2023) CancerChoices. Available at: https://cancerchoices.org/therapy/statins/ (4) Meštrović, Dr.T. (2023) Statin history, News. Available at: https://www.news-medical.net/health/Statin-History.aspx#:~:text=The%20discovery%20of%20statins,(HMG%2DCoA%20reductase) (5) On the history of Statins (2019) Clinical Correlations. Available at: https://www.clinicalcorrelations.org/2019/01/11/on-the-history-of-statins/
Axpinet, Diagemet, Glucient, Glucophage, Metabet
Metformin is a drug predominantly used to treat type 2 diabetes. There has been some research to link metformin to decreased cancer risks, and thus some people suggest it may be used ‘off-label’ to treat cancer. However, it is still unclear whether there are improved cancer outcomes for people without diabetes/metabolic issues who take metformin.
Metformin is a first-line medication to treat type 2 diabetes, which works by lowering blood glucose levels. It is also used by people with polycystic ovarian syndrome (PCOS). More experimentally, it has been suggested to be useful in extending lifespan, treating Alzheimer’s disease, and potentially cancer (1).
Research into metformin and cancer specifically is limited, and thus the relationship between the two is still unclear. It is particularly unclear as to whether metformin impacts cancer outcomes in patients without metabolic issues/diabetes. Evidence suggests that metformin is effective in reducing insulin and glucose levels in people with cancer. Moreover, people with type 2 diabetes have an increased risk of cancer, thus treatment with metformin of diabetes may help reduce cancer risk through reducing the cancer-promoting effects of type two diabetes. There is also some evidence that suggests metformin may improve cancer-specific mortality and tumour proliferation for certain patients. However, the available evidence highlights that any effects of metformin on cancer outcome may be limited to people with diabetes or other metabolic abnormalities (2, 3).
Metformin was first described in scientific literature in 1922. In the 1940s, the value of metformin in lowering blood sugar was highlighted by Filipino physician Eusebio Y. Garcia. Metformin became available in the UK by 1958, however it was not widely used until the 1970s – only approved in Canada in 1972 and the US in 1994. It is now considered the most widely prescribed antidiabetic medication. More recently, there is renewed interested in metformin in the field of ‘healthy-ageing’ to treat a range of age-related conditions (4).
– Side effects of metformin include nausea, vomiting, diarrhoea, stomach aches and a loss of appetite. It can also cause a deficiency in vitamin B12. – Metformin should not be taken unless it is prescribed, and if it is being explored as an option for cancer should be discussed with a qualified and experienced doctor first.
(1) Nasri, H. and Rafieian-Kopaei, M. (2014) Metformin: Current knowledge, Journal of research in medical sciences : the official journal of Isfahan University of Medical Sciences. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4214027/ (2) Metformin (2023) CancerChoices. Available at: https://cancerchoices.org/therapy/metformin/ (3) Lord, S.R., Harris, A.L. Is it still worth pursuing the repurposing of metformin as a cancer therapeutic?. Br J Cancer 128, 958–966 (2023). https://doi.org/10.1038/s41416-023-02204-2 (4) Bailey, C. and Day, C. (2004), Metformin: its botanical background. Pract Diab Int, 21: 115-117
Revia, Vivitrol
Low-dose naltrexone (LDN) is the off-label use of naltrexone. Naltrexone is normally used to treat addiction, and it has been suggested that a much lower dosage may be useful in treating cancer and other diseases.
Naltrexone is an opioid antagonist, meaning it can be used to inhibit the action of opioids. This is through blocking GABA receptor action and thus stopping the release of dopamine. Naltrexone can therefore be useful to treat opioid/alcohol dependence by blocking the effects of alcohol/opioids. Low-dose naltrexone is off-label use of naltrexone at low doses (1/10th the usual dose size) to treat conditions unrelated to addiction. These include Crohn’s disease, HIV/AIDs, Multiple Sclerosis and Parkinson’s disease (1).
LDN therapy has been proposed as a potential treatment for chronic pain, autoimmune disorders and cancer. For cancer specifically, LDN may interfere with tumour growth by affecting cell signalling and the immune system. However, there is need for further research and more robust trials before definitive conclusions on its effectiveness can be reached (2).
Preliminary studies suggest that LDN treatment may be promising for primary cancer patients, and more broadly may reduce inflammation and lessen pain (1).
Naltrexone was synthesised in 1963 by a company in New York, and later characterised in 1965 as its advantage as a strong opioid antagonist was found. It was patented in 1967, and clinical trials for its use to treat opioid dependence began in 1973. After its approval by the FDA in 1984, it has been used to treat both alcohol and opioid dependence (4). The low dose method was later developed by Dr Bihari in New York, a specialist in neurology (5).
While it is generally safe, LDN is linked with side effects that include nausea, vomiting, abdominal pain, decreased appetite, constipation, headache, anxiety (6). Moreover, a lack of research means there is still a lack of clarity on the dose and treatment best suited to treat conditions aside from addiction. Thus, considering LDN for cancer therapy is not widely recommended, and requires careful discussion with a doctor.
(1) Low-dose naltrexone (2023) CancerChoices https://cancerchoices.org/therapy/low-dose-naltrexone/ (2) Couto, R.D. and Fernandes, B.J. (2021) ‘Low doses naltrexone: The potential benefit effects for its use in patients with cancer’, Current Drug Research Reviews, 13(2), pp. 86–89. doi:10.2174/2589977513666210127094222. (3) Liu, W.M. and Dalgleish, A.G. (2022) ‘Naltrexone at low doses (LDN) and its relevance to cancer therapy’, Expert Review of Anticancer Therapy, 22(3), pp. 269–274. doi:10.1080/14737140.2022.2037426. (4) Chapter 4—oral naltrexone – Incorporating Alcohol Pharmacotherapies Into Medical Practice Available at: https://www.ncbi.nlm.nih.gov/books/NBK64042/ (5) Low dose naltrexone (LDN) choices booklet (2023) MS. Available at: https://ms-uk.org/low-dose-naltrexone-multiple-sclerosis-choices-booklet/ (6) Naltrexone (oral route) side effects (2023) Mayo Clinic. Available at: https://www.mayoclinic.org/drugs-supplements/naltrexone-oral-route/side-effects/drg-20068408
For more research and science on off-label, overlooked, or novel cancer approaches, visit CancerChoices.
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