What does Marmite – the well-known spread seen on British breakfast tables – have to do with chemotherapy for cancer?
Marmite is made from yeast extract which has a high folic acid content. In the 1920s, it was discovered by chance that Marmite helps to normalise the production of blood cells (haematopoiesis) in a particular form of anaemia. Dr Sidney Farber, the renowned American pathologist, who started his medical studies in Heidelberg and Freiburg, knew about this. In 1947, hypothesising that folic acid might also be beneficial in other blood disorders, he began giving it to patients with leukaemia. Unfortunately, the results were disastrous; instead of treating the condition, its progression was accelerated. Naturally, Dr Farber discontinued administration of folic acid immediately. In today’s terms, it would be said that his study had been terminated on the basis of real-world evidence.
In the same year, Dr Farber asked himself whether a recently discovered antagonist of folic acid – aminopterin – might help patients with leukaemia, given that the effect of folic acid itself had been so detrimental. In December 1947, he administered aminopterin to three-year-old Robert Sandler, who was on the verge of death from acute leukaemia. Robert recovered within a few weeks and, two months later, seemed as well as his twin brother. He survived another 18 months. This was the first time that a drug or a chemical substance had enabled even a partial response in a patient with a non-solid tumour and it caused a sensation in the oncological community. Without the timely information that Sidney Farber was able to access in the medical world of the past, this success would not have been possible.
Treating cancer today…
In the last 70 years, the world has become much more complicated. Thanks to coordinated global research efforts, the outlook for people with cancer is far brighter. But progress has brought new challenges, which the modern oncology community has to face. In the last five years alone, 89 new cancer drugs have been approved, and – over the last ten years – the number of investigational products in oncology has doubled, reaching a total of 900. These products are mainly innovative compounds that target genetic changes in cancer cells. They work in a very different way to traditional chemotherapeutic agents, which are partly derived from plant-derived toxins or chemical warfare agents.
Some of these new products have proved beneficial in patients who have not responded to past treatments. However, they are usually only effective in small subgroups of patients with specific genetic mutations. The growing need for a precision approach to cancer treatment has meant that research and routine care are both becoming more and more expensive, with a cost rise in the double-digit percentage range year on year. The question of how to ensure financial sustainability while pursuing the fight against cancer and maintaining quality of care now looms large.
Creating the ODN, a new European data network in oncology
While Sidney Farber was able to achieve a landmark breakthrough 70 years ago with just a couple of pieces of information to guide him, much more effort and coordination is needed today to equip oncologists with the data they need to shape and align with best practice. Randomised clinical trials – while still key – cannot keep pace with the increasing need for individualised care or reflect the heterogeneity seen in cancer clinics. It was for this reason that, three years ago, IQVIA initiated the creation of a network of data-sharing cancer centres across Europe: the Oncology Data Network or ODN.
The ODN promotes innovation in the field of drug-based cancer therapy by networking oncology centres at European, national and regional levels. Collection of a carefully defined dataset is completely automated and extracted from existing hospital systems with little requirement for human resources or manual effort. Information is collected, validated and analysed in strict accordance with data protection regulations. Patient data are rendered fully non-identified using a series of encryption procedures. In addition, processing of data from German treatment centres takes place in several independent steps, some of which are done in the pharmacy computer centre in Darmstadt, where the data is stored. Once processed, data analyses are made available to member sites in almost real time. Centres can benchmark their clinical decision-making with other centres in Europe and independently interrogate their data using a state-of-the-art analytics portal. Deep insights into the use of cancer drugs in routine practice may be gained and the initiation of new research studies expedited.
Can data heal?
A few days ago, I gave a lecture in my Rotary Club on the topic “Can data heal?” My response to this is ‘no’; it would be a stretch to say that data alone represents an advance in cancer care. But without good data to generate promising hypotheses, effective research cannot be done. The ODN is an innovative approach to obtaining quality real-world data at speed, for the benefit of patients, physicians and the general public. I hope that many more centres will join the ODN. Currently there are 120 member sites across Europe, 30 of which are in Germany. The network is up and running – and starting to generate outputs.
I would like to thank you for your interest and the board of the CDGW for the opportunity to present this initiative which has become my “heart’s project.” If the ODN is awarded the Future Prize of the cdgw, we will donate the prize money to two charities that put their heart and soul into helping children with cancer, Help for Children with Cancer and the Frankfurt Foundation for Children with Cancer.