30 years ago, the idea of doctors being able to prescribe medicines according to a patient’s individual characteristics was simply a theory or, according to some, a fantasy. Scientists simply didn’t have enough knowledge of the biological causes of disease, let alone how to identify patients who were most likely to respond to certain treatments. Now, with precision medicine research, all that has changed. In 2018, 42% of medicines approved by the US FDA were linked to a diagnostic test or disease sub-group2
Of course doctors have always sought to personalise treatment for their patients. Hippocrates (BC 460-370)3 described how physicians in ancient times moved away from a “one size fits all” approach. In modern times, ambitious projects such as the Human Genome Project were a huge leap forward in helping scientists discover what underlies and drives human health and illness at a genetic level. This technology rapidly expanded to allow us to identify genes responsible for disease, particularly in cancers where we were able to develop single gene tests to identify patients and treat them with targeted therapies.
Since then, the research community has been able to map gene products (such as proteins, enzymes and urinary biomarkers) in addition to the genome, and we are starting to uncover how they are linked to disease. This knowledge is helping guide precision medicine approaches for more complex diseases, so we can ensure that more and more patients are matched to those medicines most likely to benefit them. We are also aiming to treat patients at early stages of disease, when there is the best chance to transform disease. Taking this approach to discovery and development of medicines is a step-change in patient-centric healthcare, enabling targeted treatments that provide more predictable and better outcomes for patients as well as more efficient use of medicines.
Building a precision medicine approach
At AstraZeneca, we first started applying a precision medicine approach to our drug discovery over 10 years ago, identifying and testing specific biomarkers that might guide the development of candidate drug molecules, starting with oncology. At the time, we focused on candidate medicines that were late in development and retrospectively analysed data from our clinical trials to identify patients who had responded to treatment. We soon started to look across the spectrum of cancers, identifying genes responsible for tumour growth and developed single gene tests alongside the process of drug development, so we could identify and enrol patients right at the start of clinical trials who were most likely to benefit from a targeted therapy.
Evolving technology is now allowing us to move from single genetic tests to a wide range of diagnostic approaches using rich scientific data from genetic and molecular biomarkers. In addition, our teams are also developing tests that consider clinical and patient use right from the start of the drug discovery process. We have built global partnerships with emerging and established diagnostic companies, so that we can include the latest technology advances into our testing approaches. This is also beneficial for physicians and patients to select the best treatment path earlier in disease. We work closely with regulatory agencies and other healthcare institutions to determine how we can reduce access barriers across the globe.
Leading precision medicine into the future
Precision medicine is now being applied across 90% of our portfolio, we are taking our learnings and success from oncology and applying them to complex chronic diseases, like asthma or heart failure, using the 5R research framework (right target, right patient, right tissue, right safety, right commercial potential).
With this expansion across our therapeutic areas comes new areas of science and technology. Using artificial intelligence, or AI, we can test up to a million biomarker models in early phase clinical trials which is something that would be impossible by humans alone. In addition, we are building digital apps, similar to those used during the COVID-19 pandemic, to simplify patients’ experience when they enrol for diagnostic testing in our clinical trials.
To support the development of expertise and leaders in precision medicine, our Precision Medicine Academy launched in November 2021. This unique academy is targeted to early career researchers and will enrol cohorts from across varied geographical locations and from diverse backgrounds. Over the three-year course, members will have the opportunity to work with leading mentors from industry and academia, engage in theoretical learning and gain valuable practical experience from the start, working on drug-diagnostic projects.
Not only are we developing talent in precision medicine, we are also pushing the boundaries of technology. Collaborations with leading academic and biotech partners have driven advances in sequencing technologies where we can now sequence an entire genome in a matter of hours with a device small enough to fit in your pocket. Historically this would have taken years to achieve and multiple rooms’ worth of computer technology. This unprecedented progress is aligned to initiatives from industry and academia to bring diagnostic testing nearer to patients worldwide. Our aim is to make testing more convenient for patients, hence helping those eligible gain access to the best treatments for them.
Our ambition in precision medicine is to dramatically accelerate biomarker testing to change patient outcomes. As technology improves and our scientific knowledge becomes more refined, we are better placed to advance research and development, as well as transform future monitoring of health and illness. For example, we are currently testing a model of adaptive treatment depending on diagnostic test results. This therapy switch concept could monitor a patient’s response to treatment using circulating tumour DNA (ctDNA) where, if levels in the blood are not diminishing, this will indicate the need to change to an alternative therapy. To achieve this we are exploring diagnostics that can detect ultra-low levels of ctDNA with enough sensitivity and specificity that a physician can use it to tailor treatment.
Find out more about the history of precision medicine and some of the key milestones from the industry as well as from our research and development over the last few decades.
A brief history of precision medicine
