Repurposing Drugs for Cholangiocarcinoma

Repurposing Drugs for Cholangiocarcinoma

by Helen Scott

Over three years ago I was diagnosed with an incurable cancer, at the age of just 39 when my 3 children were under the age of 5. Since then I have undertaken countless treatments including 30 chemotherapy cycles spanning 4 types, radiation, cyberknife, SRS, ablation, dendritic cell therapy, targeted drugs and immunotherapy, always with the knowledge that these were unlikely to cure me, but merely delay the inevitable. And like many other patients I complemented these treatments with a smorgasbord of natural supplements such as turmeric and fish oil. However, while most other cancer patients stick to a typical mix of chemo and supplements, I have long been exploring a whole other sphere of cancer medicines in addition, many of which may already be lurking at the back of your bathroom cabinet: repurposed pharmaceutical drugs.

Repurposing a drug refers to the investigation of existing drugs, approved for one medical condition, for new therapeutic purposes. The most famous example of this is perhaps Viagra. Originally designed as a drug for angina and hypertension, when many of the male subjects on the trial experienced erections, Pfizer was quick to redesign the drug for this market, an opportunity with potentially far more upside (no pun intended!).

As for cancer, there are hundreds of other drugs, regulatory approved for other conditions, which have shown potential in oncology, such as metformin for diabetes and statins for lowering cholesterol, but because many of these drugs are off patent, the pharmaceutical company has little incentive to repurpose them as there would be no financial gain. Trials can cost up to half a billion dollars to fund, yet aspirin, another repurposing candidate, is just a few dollars a pack. But imagine if they did… If these cheap generic drugs could play a role in cancer treatment, alongside conventional treatments, cancer patients might live longer lives with an improved quality, while also reducing the huge financial burden on national health services.

Fortunately, there are various organisations and individuals championing the cause of repurposed drugs, one of which is the Anti Cancer Fund (ACF), based in Brussels. Set up by the entrepreneur Luc Verelst in 2013, when his sister was diagnosed with endometrial cancer, his ultimate aim is to find the cure for cancer while sharing reliable, scientifically backed cancer treatments and new options with patients.  It is 100% funded by private donations rather than pharmaceutical company investment, in order to remain totally independent and to always act in the best interest of the patients. The salary of the employees is paid by the founder, Luc Verelst, and the donations go directly to clinical trials.

One of the ACF’s achievements has been to curate a database of 291 non-cancer drugs, mainly off patent, which show evidence of anti-cancer activity, titled the ReDO project (Repurposing Drugs in Oncology):  Data comes from peer-reviewed studies, case reports and clinical trials. The table tells you whether the drug has been involved in oncology studies in vitro or in vivo, or in trials, human or otherwise. Even the non-medically trained can recognise the names of many of these drugs which include paracetamol and Prozac. The table also has links to any research published on the drug from an anti cancer angle including papers written by the ACF team themselves such as Dr Pan Pantziarka’s paper on the commonly prescribed NSAID, Voltaren:

However, the ACF does not simply research repurposed drugs, they have also spearheaded various trials involving them such as CUSP 9, a phase I trial using a basket of 9 repurposed drugs (including Aprepitant and Celecoxib) constructed for the treatment of the most aggressive brain cancer, glioblastoma multiforme:

Funding is another area with which the ACF can help, either by directly financing a project or facilitating introductions. For example, in 2019, it is seeking Requests for Application for funding in clinical trials for cancers with high recurrence rates.

It was my first sighting of the GBM trial in ACF’s trial portfolio on their website, along with the knowledge that the ACF particularly likes to support the more rare cancers, which are subsequently underfunded and under-researched, that led me to contact them in late 2018 to propose that they develop a similar basket geared towards cholangiocarcinoma. I was then delighted to hear that their Board accepted my idea in early 2019, thereby instructing their medical team to begin the preliminary research. This involved trawling Pubmed and other medical databases for studies already performed on repurposed drugs targeting cholangiocarcinoma. For legal reasons, this work can’t be shared publicly, but the ACF will help individual interested patients to put in perspective possible treatment options, including repurposed drugs and clinical trials. Please email:

Otherwise, I and the Cholangiocarcinoma Foundation remain in talks with the ACF about the best way to use this data to benefit patients going forward and will keep you posted. To read more about the work of the Anti Cancer Fund, check out their website:

Donations are gratefully received online at:

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