How crowdfunding could repurpose a drug for cancer patients
Dr Yolanda Augustin, St George’s, University of London, UK
Can you explain what artesunate/artemisinins are normally used for?
Artesunate comes from a family of drugs known as artemisinins that are established anti-malarial agents. These drugs have been used for more than thirty years to treat millions of malaria patients worldwide. They were initially discovered following a research project initiated by the Chinese government in the 1970s and come from a herb, or plant rather, used in traditional Chinese medicine called quinghao or sweet wormwood. They have been found to have an excellent tolerability profile when used in malaria patients but now we’re going to be doing further studies to see whether or not these drugs might also be effective in the treatment of cancer.
How is artesunate being repurposed in cancer and why?
At St George’s we are planning to run a phase II study of artesunate given to patients with colorectal cancer two weeks prior to their surgery. The reason for this is because over the last ten years there have been over 400 different publications on the anti-cancer properties of artesunate or artemisinins that now require further investigation in patients. We conducted a phase I study at St George’s which was reported in January 2015, in January this year, in EBioMedicine and this was a phase I study so it was a small study. It involved 23 patients but we basically randomised half the patients to receive artesunate and half the patients to receive placebo. The drug was well tolerated and in actual fact in the patients who received the artesunate we saw only one cancer recurrence at 42 months whereas in the patients that received placebo there were six recurrences. We therefore feel that this is obviously an area that requires further investigation to confirm if indeed artesunate can reduce the risk of recurrence in operable colorectal cancer.
What made you initially decide to focus on colorectal cancer?
At St George’s I’m a clinical research fellow and I’m working with Professor Krishna who is an infectious and tropical disease specialist and Professor Kumar who is a consultant colorectal surgeon. They came together a few years ago after seeing the emerging evidence and a publication is coming out on the anti-cancer properties of artesunate in cancer cell lines in terms of lab-based research. We therefore put together a phase I study of neoadjuvant artesunate in colorectal patients in order to look at safety and tolerability. Following this phase I study that was published in January this year we now would like to take this work forward with a phase II study in 140 patients aimed at looking at whether or not artesunate can improve survival after colorectal cancer surgery.
How did the preliminary trial turn out?
It was a small study and patients were randomised to either receive artesunate or placebo daily for two weeks prior to their operation. In terms of tolerability the drug was well tolerated and there were no long-term side effects that were noted. Also in terms of molecular studies we found that in patients who received the artesunate drug there was a reduction in Ki-67 levels which was not seen in the placebo group. We did look at overall survival and at 42 months there was one recurrence in the patients who took artesunate versus six recurrences in the patients who were in the placebo group. Obviously, this being a phase I study, we’re not able to draw any conclusive conclusions about this data but this has prompted us to now design a phase II study in 140 patients in order to take this work forward to see if indeed artesunate can have an effect to reduce the rate of recurrence after colorectal surgery.
What made you decide to look at crowdfunding the larger trial?
We decided to turn to a crowdfunding model for our research project because at present it is quite difficult for researchers to get the necessary funding through conventional funding streams and also because our drug is an established off-patent drug that wouldn’t be able to receive the funding from pharmaceutical companies or the backing of pharmaceutical companies. So we therefore decided that we wanted to basically do a public appeal to encourage the general public to basically engage in the idea of our scientific project and therefore back our study through a crowdfunding model. We’ve also seen that crowdfunding has become increasingly popular over the last few years for technology and the arts and so our feeling was that if arts and technology can do this then why not medical research and science.
So we teamed up together with a crowdfunding platform called Futsci which is also run by a scientist and a consultant dermatologist. We were actually pleasantly surprised with the response that we’ve seen so far. Having only opened the project for about 15 days we’ve actually managed to raise 70% of our crowdfunding goal, which is £50,000, in order to run our clinical trial. The response from the general public has truly been amazing and what’s been even better is actually to see how ordinary people and lay persons are actually really engaging in the idea behind our study, asking lots of interesting questions, getting in touch with us and communicating their thoughts and reflections and why they think this research is important. So, to me, that has actually been quite a powerful learning exercise as a researcher and also as a cancer doctor and it’s opened up to me the idea that the general public and scientists and the medical profession should really look towards working more closely together in the future for trying to get the necessary funding to run important trials that may be able to answer some of the questions that are important to all of us.
What are the clinical implications of this research?
If this research shows that artesunate given once a day for two weeks before surgery for colorectal cancer can indeed improve recurrence free survival and overall survival from this disease I think it does have a potential to certainly change practice worldwide. At present overall survival for patients with colorectal at five years is about 60% and many of the chemotherapy regimens and treatments that are being used are expensive and also come with significant side effects. The pricing index also means that a lot of these chemotherapy agents are not available to patients in the developing world. Artesunate, on the other hand, is an off-patent drug that is relatively affordable, one dose is priced at about 70p per day, and also from having been used in millions of patients worldwide for malaria we know that it seems to be very well tolerated. So if we’re able to actually give patients medication that’s well tolerated with minimal side effects and is also relatively affordable in order to improve their survival from colorectal cancer, I believe this could make a big impact on their treatment and the treatment of cancer patients worldwide.
What do you think other doctors can learn from this experience?
I think this experience in terms of setting up a phase II study of repurposing a drug for a new use in cancer, as well as the experience of setting up a crowdfunding campaign for cancer research, has taught me to actually think out of the box in terms of trying to find innovative solutions to some of the problems that we sometimes face in our medical and research practice. In terms of the crowdfunding exercise it has been a very positive exercise for me in terms of engaging with the general public and various disciplines that have come together in order to make this campaign a success. Essentially it’s obviously at the core of what we should be aiming to do as doctors, and what we all hope to do as doctors, which is to actually improve treatment for patients and focus on the issues that really matter to patients all over the world.
