During his State of the Union address last week, the president announced a new initiative to increase funding into promising new research to deliver the right treatments to patients at the right time. He called this project the "Precision Medicine Initiative", and it could be an historic investment of great importance to cancer patients. What is personalized medicine exactly? As you probably learned in high school biology class, we all inherit genes from our parents that determine specific traits like our physical features, natural talents and sometimes our health. A decade-long federal investment in the "Human Genome Project" made it possible to catalog the over 20,000 "standard" human genes we all have. Each gene serves a purpose in the normal function of our bodies, and when a gene is mutated - or does not function the way it is supposed to - it can result in disease. Some mutations are inherited from our parents and can lead to genetic disorders like cystic fibrosis, hemophilia, or in some cases this type of mutation can also lead to cancer. Most cancer-causing mutations are not inherited, but rather develop during one's lifetime, a process which can be greatly affected by lifestyle factors like smoking or exposure to UV radiation. Personalized medicine seeks to understand specifically which gene or gene mutations cause a disease, which will hopefully allow more targeted and effective therapies to be developed and delivered. After the Human Genome Project gave us the roadmap to our genome (the collection of all the genes we carry), subsequent personalized medicine research, like the federally funded The Cancer Genome Atlas (TCGA), has helped us link cancers to the specific genetic mutations that are at the heart of each person's cancer. As an example, we now know that there are well over a dozen unique different types of lung cancer, each with a different genetic mutation causing the cancer.
We are still making new discoveries about the underlying genetic roots of cancer every day. Understanding the genetic basis of a cancer is a vital first step. The second step in the movement toward personalized medicine is the development of therapies that can directly target the results of different mutations. In the case of lung cancer, we have examples of targeted drugs for a couple of the many mutations (EGFR and ALK) that make up the array of lung cancer types. In other types of cancer, we also have targeted therapies, for example against melanoma with a BRAF genetic mutation and in breast cancer with abnormal genes for a protein known as HER2. While we have seen the development of a number of targeted drugs, we have only begun to scratch the surface of the potential for new targeted therapies. The research needed to continue to develop personalized medicine spans the spectrum from basic research like that performed by The Human Genome Project and TCGA, to more applied drug development that builds upon that basic science. Additional research resources are needed all along the spectrum, which is why the president's announcement is so welcome. One of the paradoxes of personalized medicine is that as we further divide common cancers into genetic subtypes, the number of patients with any given subtype gets smaller and smaller until we are faced with a collection of individual rare cancers that are related, but may each call for a different approach to treatment. Small patient populations pose a unique challenge to conducting research and developing drugs, so collaboration is extremely important in personalized medicine. Lung-MAP is an example of a public-private partnership formed to leverage research resources and accelerate the development of targeted therapies in lung cancer. It is a partnership between the National Cancer Institute, five different drug companies, a molecular diagnostic company and non-profits led by Friends of Cancer Research. The American Cancer Society and ACS CAN are actively supporting the development of personalized medicine. ACS CAN has been advocating for increased federal funding of research initiatives such as the one announced during the State of the Union, and we are also direct supporters of research, like Lung-MAP. As new personalized medicines make their way into the clinic we will see significant changes in the way cancer is diagnosed and treated. Our health care system will have to be ready to adapt to the information demands and increased specificity of therapies. We have also seen an explosion of new diagnostic tests offered to patients, and ACS CAN has also been actively supporting appropriate oversight of these tests to make sure that patients and their doctors can trust the results of these tests. Our understanding of cancer and the opportunities to make progress have never been greater, but it will take the commitment of our nation's policy makers to ensure that the resources are available to realize the promise before us. *Image by Duncan Hall on Flikr.
Mariah Forster Olson, an ACS CAN Ambassador Constituent Team Lead (ACT Lead), traveled to Washington, D.C. this week to share her story as a childhood cancer survivor and to advocate on behalf of other patients and survivors during the 2019 Childhood Cancer Action Days.