Precision medicine has seen tremendous success in oncology, but has been more limited in other fields of medicine. However, efforts are underway to accelerate the application and use of precision medicine in areas such as neurology, immunology and women’s health, among others. We explore what has made precision medicine so successful in oncology and discuss what will be needed to advance the field to new areas of application.
Precision Medicine and Its Success in Oncology
Precision medicine is an approach to diagnosing, treating, monitoring – and potentially preventing – disease that considers the variability between individuals in terms of genes and protein expression, environmental influences, and lifestyle. The goal of this approach is to better predict the most effective and safest treatment for each person, rather than relying on the one-size-fits-all approach that has characterized many treatment regimens in the past. Moreover, precision medicine approaches enable the generation of data that can support pricing and reimbursement, by demonstrating clearly to a payer that a particular drug works well for a defined set of patients.
To date, the greatest gains for precision medicine have been in the field of oncology, which has been driven by several key factors:
Understanding of Cancer Biology
The field of cancer biology has seen major advances over the last 20 years, furthering our understanding of what drives tumor development and growth. Large-scale programs such as the Cancer Genome Atlas (TCGA) took advantage of emerging genomic and proteomic technologies, and significant resources have been dedicated to understanding cancer at the molecular level. This has provided researchers and pharma companies with a strong foundation for identifying biomarkers and druggable targets.
Strong Focus on Innovative Tools and Technologies
Adoption of new and innovative tools, both in the R&D and clinical settings, has supported advances in precision medicine. Technologies like next-generation and whole genome sequencing, as well as applications of liquid biopsy and other molecular tests, are being increasingly integrated into routine cancer diagnosis and care, allowing precision medicine to become part of standard practice.
Patient and Medical Advocacy
The demand for improved cancer care and treatment has helped advance precision medicine. Several precision medicine oncology initiatives have been launched by government and private groups, raising billions of dollars dedicated to generating new knowledge, tools and technology. Furthermore, efforts to improve patient access to such advances have been key to driving the field of precision medicine in oncology and ensuring that it does not remain in the hands of a few top hospitals.
Challenges to Extending Precision Medicine to Other Areas
There is clear acknowledgement amongst the industry that the promise of precision medicine can and should be applied to other therapeutic areas. But, as the name implies, there is no one-size-fits all approach, especially given the unique complexities and characteristics of many diseases. As a result, the broad adoption of precision approaches outside of oncology has lagged. We looked at some of the challenges to advancing precision medicine in three broad areas:
The field of neuroscience is highly diverse, encompassing neurodegenerative diseases, motor neuron and other movement disorders, stroke, epilepsy and neuropsychiatric conditions. Many of these conditions have variable manifestations, onset, disease courses and outcomes, as well as pleiotropy and varied penetrance. As a result, identifying commonalities between patients that enable their classification into discrete disease types has been challenging. A further complicating factor to identifying useful biomarkers is the inability to easily access neural tissue prior to patient death. As a result, the molecular approach to precision medicine successfully pursued in oncology doesn’t work as well for many areas within neuroscience. Compared to oncology, few molecular biomarkers have to date found utility as neuroscience research tools, much less in clinical medicine. Thus, the focus of biomarker research has been broadening to imaging studies (especially in Alzheimer’s and Parkinson’s disease), digitally measured behavioral or fine motor changes, and a variety of -omics approaches. Each condition needs to find its own disease classifications and biomarkers, given the complexity of what falls into the neuroscience field.
Developing a precision approach to immunology is challenged by the complexity of the immune system. It is subject to dynamically changing cellular states and interactions, as well as the many factors that can influence an immune response, including environmental and microbiome-related impacts. Molecular and clinical heterogeneity also is common to many of the diseases falling into this category, which includes autoimmune and inflammatory conditions that can affect different organ systems. As a result, advancing precision medicine in this field and identifying useful therapeutic targets and biomarkers will likely require a systems biology approach that goes beyond genomic / proteomic measures alone to consider cell-type specific and disease state specific measures. Longitudinal studies and dynamic analyses will also likely be needed to identify risk and prognostic markers, as well as those useful in determining treatment efficacy.
- Women’s Health
Gynecological and breast cancers have benefitted from the overall advances of precision medicine in oncology, but few other areas of women’s health have been a priority for such approaches. While this situation is beginning to change, the future of women’s health and precision medicine would benefit from greater awareness and advocacy to drive advances in discovery and clinical research, and dismantle the bias against women with male-skewed clinical trials. Companies like Celmatix are leveraging genomics and proteomics to generate a large multi-omic dataset and inform discovery efforts, in the hopes of developing new and more targeted therapeutics for conditions such as ovarian senescence and polycystic ovary syndrome. More research is needed, particularly in the areas of women’s cardiology, and sexual and reproductive health.
There is much that can be learned from the success of precision medicine in oncology and applied to advance patient care in other fields of medicine. We believe that building a better understanding of disease biology is a fundamental success factor, supported by the adoption of the relevant tools and technology, be it -omics or digital tools. Stakeholder engagement and advocacy also play a critical role in driving advances in precision medicine, bringing policy changes and awareness, and fostering collaboration. The time is now for companies to ride the wave of success from oncology and advance the promise of precision medicine to new areas within our healthcare industry.