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Drug Repurposing in Oncology

Reconsidering drugs already available for other diseases as options for cancer treatment is a unique opportunity
21 Oct 2015
Bioethics, legal and economic issues;  Anticancer agents & Biologic therapy

International collaboration between researchers and foundations in Belgium, Italy and the USA proposes a new approach to cancer treatments using drugs already in our medicine cabinets, in an eye-opening article published in Nature Reviews Clinical Oncology.

The cost of cancer drugs is going through the roof. Reconsidering the large arsenal of drugs already available for other diseases as options for treating cancer is a unique opportunity for patients and for national healthcare systems, according to a press release issued by the Anticancer Fund.

The continuing reduction in the cost of DNA sequencing is paving the way for ‘precision medicine’, and an increasing number of clinical trials are being designed specifically for patients with a certain genetic profile of their tumours. However, researchers and doctors are having to deal with a new and complex scenario.

Firstly, the few National Healthcare Systems (NHSs) who can afford new “targeted” cancer drugs can no longer bear the cost. The average cost of treatment with a new cancer drug rose from 100 USD per month in the 1990s to 10,000 USD per month in 2011. Unfortunately, there is no sign that this will improve over the short or medium term.

Secondly, the availability of new cancer genomic data has revealed a greater complexity than was anticipated, and this situation makes it even clearer that we need treatments based on a combination of therapies instead of simply using a single agent. However, this does not fit the traditional strategy of the pharmaceutical industry, whose agenda is centred around clinical trials designed to achieve registration and approval for the clinical use of one new drug for one indication at a time.

In light of this situation, in an article in Nature Reviews Clinical Oncology entitled “Drug repurposing in oncology—patient and health systems opportunities”, Francesco Bertolini of the European Institute of Oncology in Milan, Italy, Vikas Sukhatme of Harvard Medical School, Beth Israel Deaconess Medical Center and GlobalCures in Boston, USA, and Gauthier Bouche of the Anticancer Fund in Brussels, Belgium propose a new way forward with the potential to deliver significant benefits to patients and NHSs alike.

They wrote in their article: “To date, successes in oncology drug repurposing have been limited, despite strong evidence supporting the use of many different drugs. A lack of financial incentives for drug developers and limited drug development experience within the non-profit sector are key reasons for this lack of success.”

Oncology drug repurposing

There is a certain amount of drugs already available for clinical use in different diseases and which have the potential to be reused in oncology, a strategy known as "drug repurposing"; this presents unique opportunities for new combination therapies. Preclinical and clinical development will certainly be safer because the side effects are already known, but also faster and cheaper as existing knowledge about the drugs reduces the amount of work and time needed for further development. Clinical development can be supported by partnerships   between governments, NHSs, and not-for-profit foundations, with obvious potential benefits for all partners.

Success stories involving the repurposing strategy in oncology have so far been limited, but have had a big impact, exemplified by thalidomide, a drug made infamous for serious effects on unborn children in the sixties, and now used with great success to treat multiple myeloma. Good old aspirin is another successful case of repurposing, in light of the results of its role in preventing colon cancer.

The European Institute of Oncology, Global Cures and the Anticancer Fund are already actively pursuing repurposing on various fronts. Each of these organisations is running pre-clinical and clinical trials focusing on drugs with low-cost and low-toxicity. A commitment to this research is now required from governments, NHSs and other funding sources, as this would significantly facilitate this type of therapeutic approach with the potential for rapid and significant benefits for cancer patients worldwide.

The full article can be accessed via the ESMO journal access programme (ESMO members only).

Anticancer Fund is a private foundation providing research-based information on cancer therapies and selectively funding the development of promising therapies. Anticancer Fund is ESMO partner, based on non-financial agreement, in creating guides for patients that follow information available in the ESMO Clinical Practice Guidelines.


Bertolini F, Sukhatme VP, and Bouche G. Drug repurposing in oncology—patient and health systems opportunities. Nature Reviews Clinical Oncology 2015; Published online 20 October. doi:10.1038/nrclinonc.2015.169

Last update: 21 Oct 2015

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