Bonnie J. Addario

 the consideration of individual patient characteristics so that doctors, working directly with patients, can develop the best treatment plans for them as early in their care as possible. Treatment strategies should consider the whole person, including their age, medical history, ethnicity, biological characteristics, and other factors. This involves using technologies such as diagnostic tests, molecular profiling platforms, and therapies that directly target disease-causing genetic mutations.

I see the profound impact of precision medicine on patients every day. In 2006, my family and I founded the Bonnie J. Addario Lung Cancer Foundation (ALCF). We started another nonprofit, the Addario Lung Cancer Medical Institute (ALCMI), in 2008. ALCF works with patients to support research and to advocate for innovative lung cancer medicines and treatments. Both the ALCF and the ALCMI facilitate, fund, and drive research. We work with thousands of patients and their families around the globe, providing free education and support programs, connecting patients with doctors and clinical trials, and funding innovative research.

A lung cancer diagnosis is grim—only 18% of patients survive the disease. In the United States alone, 450 patients die from lung cancer every day. Lung cancer is the top cancer killer of men and women, killing almost twice as many women as any other type of cancer in the United States. Responsible for 26% of all cancer deaths, lung cancer is the second leading cause of all deaths in the country.

With the rapid pace of developments in precision medicine, I am excited about the potential to shift the paradigm on how we diagnose and treat lung cancer and save lives. Furthermore, in this era of personalized medicine, we have an opportunity to vastly improve clinical trial design, thereby fostering an environment for innovation that can lead to the development of even more novel treatment strategies.

In 2016, for the third year in a row, targeted medicines and diagnostic tests accounted for more than 20% of the new molecular entities approved by the FDA (

 A recent study sponsored by the Personalized Medicine Coalition and conducted by Tufts University found that 42% of all medicines and 73% of cancer medicines in development are potential personalized medicines (

At the ALCF, we strongly believe that patients are our most important partner in clinical research. There would be no clinical research without patients participating in studies and donating their specimens. The traditional clinical trial design process overlooked patient input, which led to profound inefficiencies. Researchers often needed to scrap or redesign their clinical trials to address patient needs and circumstances. We have an opportunity at hand to vastly improve the process. Including patients in clinical trial design and giving them an opportunity to own their data provide a platform to streamline the process by getting it done right, arriving at the endpoints that matter most, and finding ways to most effectively improve health. We need processes for greater patient participation in clinical research. Now is the time to move from concept to practice, and lung cancer is a prime example of where it can work.

I advocate for the need to shift the way we look at clinical research. The current biomedical innovation paradigm, of discoveries moving from the bench to the patient, needs to shift its focus so that clinical researchers first take into consideration the patient and new discoveries move from the patient to the bench and then back to the patient. This will transform research to ensure it is patient driven and truly personalized.

Consider the example of Corey Wood. While attending the University of California, Berkeley, Corey was diagnosed with stage 4 lung cancer. A triathlete and marathon runner, she immediately began doing research about targeted therapies and genomic tests. Once her oncologists zeroed in on the rare genetic mutation that was responsible for Corey’s tumor, they treated her with targeted therapy. Corey now leads the normal life of a 25-year-old thanks to precision medicine. She is passionate about furthering research in the hopes that researchers can develop more effective targeted therapies. Corey owes her life to medical research.

Still, these new and more effective targeted treatments are not useful if we cannot get them to patients. Research and innovation in personalized medicine are surging, but its adoption into clinical practice is relatively slow. In most cases, doctors do not even discuss personalized medicine at the point of care. A recent public survey showed that only 4 of 10 people are aware of personalized medicine and just 11% of patients say their doctor has discussed or recommended personalized medicine treatment options to them.

Another survey found that most healthcare organizations are unprepared to implement personalized medicine

 and some hospital systems may be placing implementation programs on hold.

 This lag is caused by novel challenges encountered by healthcare delivery systems as they adapt to the new treatments and practices associated with personalized care. What we lack are more patient-driven healthcare delivery approaches and processes that would ensure access to personalized medicine, including comprehensive genomic testing and multidisciplinary care.

Empowering patients to be more engaged in their healthcare through awareness programs and education on the promise of precision medicine would be a good start. The strategy should focus on:

		Patient involvement in learning about the healthcare system

		Improving patients’ engagement with their physicians

		Shifting the control of individual genetic data to patients

		Encouraging patient participation in the development of treatment guidelines and clinical pathways

Support for precision medicine and patient-centered research is strong, as indicated by several recent legislative initiatives and public policies. Patient-centered directives are included in the Affordable Care Act, and the recently passed 21st Century Cures Act focuses on involving patients in research and care design. The Precision Medicine Initiative, an effort to recruit 1 million volunteers to donate their genetic information for research aimed at finding more effective ways to improve health, and the Cancer Moonshot Initiative, an effort to cut in half the time it takes to discover new personalized cancer treatments, were authorized in the 21st Century Cures Act.

These policies underscore the importance of ensuring patients are receiving the appropriate diagnostic tests and personalized treatment. Lung cancer is not one-size-fits-all. Patients who feel they are not receiving the care they need can share the information with regulators by visiting 

So, what can we do to advance personalized medicine? Patients must have a seat at the table to provide valuable data to drive faster cures. This includes ensuring that there is appropriate patient-driven trial design, clinical trial participation, diagnostic testing, endpoint determination, and data aggregation. We can change the clinical medicine research ecosystem and adapt it to be more patient-centered. This will bring new treatments forward on a shorter timeline.

We need to ensure that community hospitals and physicians are appropriately engaging patients and promoting personalized medicine. The ALCF is doing this by designating hospitals as Lung Cancer Community Centers of Excellence, where patients are assured of multidisciplinary care, tumor boards, genomic testing, and early screenings in these facilities. We can conduct clinical trials at community hospitals so that patients do not have to travel hundreds of miles to participate in one.

The ALCF just launched its patient-powered Lung Cancer Registry,

 a place to gather and store detailed information for patients with lung cancer. The registry directly involves patients in the collection of their information, which allows medical professionals to quickly analyze data to improve patient care. By creating a centralized registry, patients, healthcare professionals, researchers, the pharmaceutical industry, and policy makers have open access to information. Programs such as these will ensure that patients are empowered to educate themselves about their disease so they can receive the best, most personalized treatments possible.

Bonnie J. Addario is chair, Bonnie J. Addario Lung Cancer Foundation, Addario Lung Cancer Medical Institute.

Daryl Pritchard, PhD, is vice president, Science Policy, Personalized Medicine Coalition.

E-mail: dpritchard@personalizedmedicinecoalition.org

1. Cancer stat facts: lung and bronchus cancer. National Cancer Institute website. https://seer.cancer.gov/statfacts/html/lungb.html. Accessed July 10, 2017.

2. Pritchard D. Personalized medicine at FDA: 2016 progress report. 

3. The Personalized Medicine Coalition and PhRMA. Biopharmaceutical companies’ personalized medicine research yields innovative treatments for patients. Personalized Medicine Coalition website. http://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/ pmc-phrma-personalized-medicine-investment-21.pdf. Accessed June 22, 2017.

4. Miller AM, Garfield S, Woodman RC. Patient and provider readiness for personalized medicine. 

5. Survey: most healthcare organizations unprepared for precision medicine [press release]. Salt Lake City, Utah: Health Catalyst; January 20, 2016. https://www.healthcatalyst.com/news/survey-healthcare-organizations- unprepared-precision-medicine/.

6. Slabodkin G. Many healthcare organizations not preparing for precision medicine. HealthData Management website. https://www.healthdatamanagement. com/news/many-healthcare-organizations-not-preparing- for-precision-medicine. Published January 27, 2016. Accessed June 22, 2017.

7. Lung Cancer Registry website. https://www.lungcancerregistry.org/. Accessed June 22, 2017.

Articles

The Bonnie Addario Lung Cancer Foundation has developed a registry that can serve as a repository for data on patients with lung cancer-a means to empower patients and assist care providers to deliver personalized medicine in a patient-centered manner.  

Patient Engagement Is Mandatory at Our Table

Medical advancements, over the past year, have been game changing for lung cancer patients. In 1971, President Richard Nixon declared war on cancer, and now, we finally have a war on lung cancer.

In 2015, the FDA approved 7 new treatments for lung cancer patients, and 5 of these approvals came over a 2-month period. FDA programs, such as the Breakthrough Therapy, Accelerated Approval, Priority Review, and Fast Track designations, have allowed the expedited approval of life-saving lung cancer medicines that address unmet medical needs and provide clinical benefit to patients.

As we march into the era of personalized precision medicine and improved clinical trial design, genomic profiling allows us to target and treat specific cancer mutations, by identifying and prescribing the right drug to the right patient, at the right time. Another fantastic addition to our armamentarium is, immuno-oncology (I-O), a therapy that completely upends traditional treatment options using medicines to boost a patient’s own immune system to fight their disease.

Many of us now have a second chance because of the doctors, scientists, and medical researchers working on our behalf, to provide new medicines and treatments for lung cancer patients.

Eleven years ago, I was at the peak of my career as a CEO when my world came crashing down around me. After 14 hours of surgery, a battery of nurses and doctors, an army of radiation and chemotherapy treatments, blood clots, procedures, and tubes, I lost my right lung to cancer. I am a survivor.

Today, we not only have new treatments; we have new surgical approaches; new medicines, and skilled, passionate researchers fighting for people with lung cancer; we have information, patient resources, and collaboration among patients, researchers, clinicians, and physicians. It is finally the century of patient-driven lung cancer research!

Throughout my diagnosis and treatment, I realized the huge unmet need for patient resources and information on lung cancer. Survival gave me a new goal: address a deficiency in lung cancer healthcare resources and research initiatives.

In 2006, my family and I founded the Bonnie J. Addario Lung Cancer Foundation (ALCF), where we work with patients to support research and advocate for innovative lung cancer medicines and treatments. In 2008, we started another nonprofit, Addario Lung Cancer Medical Institute (ALCMI), a global consortium dedicated to facilitating and driving research. We work with thousands of patients and families worldwide, providing free education and support programs, connecting patients with doctors and clinical trials, and funding innovative research where the need is greatest.

Clinical research holds the key to discovering the causes of lung cancer, developing effective treatment options, and delivering those treatments to patients, in a timely manner. What can we do to predict lung cancer risk, detect the disease at early onset, and prevent it? Are there novel drug combinations to pre-empt and overcome cancer cells? How can we personalize and provide targeted treatment for each individual patient?

For the first time in decades, we have momentum and hope in the progress made with immunotherapy and companion drug trials. Timely approval and availability of breakthrough therapies allow more families to think of lung cancer survival in terms of years, not just months, and provide hope to make lung cancer a chronic, manageable disease by 2023. It can’t get more personalized than using your own immune system to fight your cancer!

Immunotherapy is a game-changer; a breakthrough that oncology desperately needs. Now, for the first time, we can begin to talk about long-lasting effects, even a 

Unlike other therapies, immunotherapies afford a sustained, durable response that continues even after treatment is stopped—a first for diseases like lung cancer that have had poor prognoses.

This is a quantum leap forward—a moonshot. Before now, patients had limited options, especially after their disease progressed beyond the current standard of care therapies.

Unfortunately, immunotherapy does not work with every patient, every time. Approximately 70% to 80% of cancer patients do not respond to immunotherapy treatments, which may be an outcome of clinical trial design, including selection of  the appropriate patient population.

Among patients who do respond to immunotherapy, not all have dramatic, long-lasting effects. Several patients present with pseudoprogression, meaning the disease appears to get worse before it gets better. Physicians, patients, and their caregivers need to be prepared for this to avoid discontinuing a potentially effective therapeutic regimen.

The Need to Redesign Clinical Studies for Maximal Benefit

On a broader level, we also need to examine clinical trial design in order to change the current dismal patient accrual rates of 3% to 5%. Clinical trial design needs an overhaul that would allow trials to be more inclusive, patient friendly, and easier to access, so that precious research dollars can be maximized to speed up the development of newer diagnostics, prognostics, and therapeutics. We need clinical trial data that is publicly available, so that it may advance the current state of lung cancer science and research, and inform the design of future clinical trials. 

At ALCMI, we have successfully implemented a remote clinical study participation platform that allows young, lung cancer patients, from countries around the world—Italy, Turkey, Brazil, and New Zealand&mdash;to participate in the Genomics of Young Lung Study (GoYLC) trial

 that is investigating mechanisms of tumor initiation and progression in patients under 40 years old. It is important that the field continues to change paradigms of how trials and studies are run, so that patients can look forward to better outcomes and more hope. 

Not only do we need the clinical medicine ecosystem to change and adapt in order to bring newer treatments forward, we also need the patient to take a seat at the table, join clinical trials, and provide valuable data to drive faster cures.

Take Jeff Julian. Diagnosed in 2015, the 39-year-old former Olympic Trials finalist and Rose Bowl Aquatics head coach was shocked when he found out he had stage IV lung cancer. Because of his participation in a clinical trial for nivolumab, a breakthrough I-O drug, his tumors have significantly regressed and his disease is under control. Jeff knows, firsthand, that innovative treatments save countless lung cancer patients’ lives.

This year alone, more than 200,000 people in the United States received a lung cancer diagnosis.

 Innovative medicines, like immunotherapy drugs, have and will save countless lung cancer patients’ lives. We need to continue to support the researchers and doctors pushing the envelope, and seeking new approaches and treatments to cure lung cancer.

Medical innovation works best when patients, donors, clinicians, pharma, biotech, payers, government, public, and private enterprise all take a seat at the table to collaboratively drive research. The lives of patients, around the world, are possible because of the stream of new research and novel drugs that help us fight lung cancer. It is a promising time for lung cancer patients and we are just getting started.

Personalized Medicine Should Lead the Way

Across the board, we see a heightened focus on precision medicine and personalized medicine, from President Obama’s Precision Medicine Initiative

 to the FDA’s Breakthrough Therapy and Fast Track approval process.

 Precision medicine empowers oncologists to test, profile, and target genomic mutations that cause cancer to grow and spread. Personalized medicine takes a step back and looks at the whole person, his or her age, medical history, ethnicity, and other factors that might influence treatment decisions.

As an advocate for personalized medicine, the ALCF has established 20 Centers of Excellence in community hospitals, nationwide, to ensure all patients receive genomic testing that can guide the treatments they receive. The ALCF also collaborated with 16 other lung cancer organizations on the Don’t Guess.Test. campaign

 to expand awareness and educate patients about the importance of comprehensive genomic testing in lung cancer, so that each patient receives the right treatment at the right time. We have created both, an online patient portal and an app for lung cancer patients; we fund research initiatives, such as the GoYLC study and the Clinical Trials Innovation Prize. We are working hard to move the dial to improve the 16% survival rate for lung cancer patients.

We now have an arsenal of new resources for lung cancer patients who are living well longer than previously imagined; yet, in the United States alone, 450 patients die from lung cancer every day.

 Lung cancer is the top cancer killer of men and women, killing almost twice as many women, as any other cancers. It accounts for 27% of all cancer deaths and is the second leading cause of all deaths in the country.

As a lung cancer patient and a patient advocate, I know the stakes have never been higher for patients waiting for new medicines that could improve treatment, save lives, and offer hope to families. Supporting, funding, and protecting the research and development of new medicines and new treatments is more critical, now than ever, to provide better treatments and better outcomes to all cancer patients. Patients deserve it. 

Bonnie J. Addario is a stage IIIB lung cancer survivor and founder of the

Bonnie J. Addario Lung Cancer Foundation (ALCF) and the Addario Lung Cancer Medical Institute (ALCMI). For more information please visit 

		Genomics of Young Lung Cancer study.  Addario Lung Cancer Medical Institute website.

		American Cancer Society. Cancer facts & figures 2015. 

http://www.cancer.org/acs/groups/content/@editorial/documents/document/acspc-044552.pdf

		Fact sheet: President Obama’s Precision Medicine Initiative [press release]. Washington, DC: The White House; January 30, 2015. 

https://www.whitehouse.gov/the-press-office/2015/01/30/fact-sheet-president-obama-s-precision-medicine-initiative

		Walia G. Breakthrough medicines usher in hopeful era for lung cancer patients. Bonnie J. Addario Lung Cancer Foundation website. 

http://www.lungcancerfoundation.org/patients/news/breakthrough-medicines-usher-in-hopeful-era-for-lung-cancer-patients/

. Published December 18, 2015. Accessed January 7, 2016.

Supporting, funding, and protecting the research and development of new medicines and new treatments is more critical, now than ever, to provide better treatments and better outcomes to all cancer patients. 

Author | Bonnie J. Addario

Articles

Patient Engagement Is Mandatory at Our Table

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