Real World Health Care Blog

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Multiple Myeloma Treatment: Too Many Choices?

Real World Health Care continues our multiple myeloma series with an interview with Edward A. Faber Jr., DO, MS, associate director of the Blood and Marrow Transplant Program managed by Oncology Hematology Care, Inc. in conjunction with the Mercy Health and The Jewish Hospital in Cincinnati. He also serves as principal investigator for OHC’s multiple myeloma clinical trials. Dr. Faber is a member of OHC’s board of directors and the Dayton Clinical Oncology Program and serves on the board of trustees for the Leukemia and Lymphoma Society. We discussed his clinical trial work and his views on promising new treatments for multiple myeloma.

Early Work in Multiple Myeloma

Real World Health Care: What attracted you to the field of multiple myeloma and what keeps you inspired?

Dr. Edward A. Faber, Oncology Hematology Care, Inc.

Edward Faber: I initially sought a career in bone marrow transplantation. During my training and fellowship, I decided to pursue a career in academic medicine, at a time when multiple myeloma was almost in its infancy. There have been so many advances over the past decade, not only with new individual medications but also with new combination therapies.

The inspiration comes from the patients, as quality of life and survival have improved over the past 10-15 years. This is a testament to the collaborative effort between academia, cooperative groups and pharmaceutical companies.

Clinical Trials

RWHC: What is the significance of your recent clinical trials?

EF: Over the past five to six years, we have been involved with clinical trials involving carfilzomib, pomalidomide, panobinostat, and daratumumab. We’re also comparing combination therapies between carfilzomib (KRd) versus bortezomib (RVd). These trials have demonstrated the efficacy of the combination of carfilzomib and panobinostat. For example, daratumumab, a potent choice for our patients with multiple myeloma, may offer benefits towards patients with smoldering myeloma.

Bone marrow transplant is an accepted treatment option, and the large Eastern Cooperative Oncology Group (ECOG) trial comparing KRd and RVd with lenalidomide maintenance may prove to offer a viable strategy outside of transplant. Quite simply, the research that we have been participating in has led to improvements in combination therapies with the newer medications for patients in the current community.

Research Collaboration

RWHC: What are some of the biggest challenges facing multiple myeloma researchers?

EF: The challenges facing multiple myeloma researchers are not unlike those facing all researchers, from the standpoint of availability of clinical trials, and making clinical trials available to large portions of the population.

Funding is always an issue for basic science researchers, bench top researchers, and clinical scientists.

Staying up-to-date with the current large cooperative groups, as well as the national scientific groups such as the American Society of Hematology and American Society of Clinical Oncology, is more important than ever, in order to learn and understand which available therapies offer more promise and move those therapies forward so that they eventually can lead to FDA indications.

Sequencing Therapies

RWHC: How are clinicians overcoming treatment challenges in multiple myeloma?

EF: The availability of a large number of drugs to treat multiple myeloma patients is both positive and a challenge for clinicians because there can be just too many choices.

The most important step begins at initial diagnosis and knowing how to sequence available therapies — especially combination therapies — over time to maximize benefit. These combinations must be interpreted in the context of the patient’s other medical issues. Certain side effects, such as cardiac toxicities, need to be avoided in certain patients. Patients should be encouraged to develop a good relationship with a physician who has focused interest in multiple myeloma. If that physician is aligned with a large academic center, he or she will be in a better position to help translate recent research and trial successes to hospital exam rooms, where the majority of myeloma patients are treated.

 

Multiple Myeloma: A Rare and Complex Cancer

We continue our series on multiple myeloma with an interview with Gareth J. Morgan, MD, FRCP, FRCPath, PhD, professor of hematology and director of the Myeloma Institute at the University of Arkansas for Medical Sciences (UAMS). Dr. Morgan also serves as deputy director of the Winthrop P. Rockefeller Cancer Institute at UAMS.

Dr. Morgan is responsible for all clinical, research, and administrative operations at the Myeloma Institute, which is one of the largest programs in the world focused on the research and treatment of multiple myeloma and related diseases. Clinically, he directly oversees 8 physician specialists, 14 mid-level providers, and 7 hospitalists, all experts in the management of myeloma and related plasma cell diseases. He also manages 8 research teams, which represent an integrated program of the genetics and biology of myeloma.

Mechanisms of Malignant Transformation

Real World Health Care: What got you interested in the field of multiple myeloma and what keeps you inspired?

Dr. Gareth Morgan, University of Arkansas for Medical Sciences

Gareth Morgan: Scientifically, I saw the opportunity to exploit the transition of MGUS (monoclonal gammopathy of undetermined significance) to smoldering myeloma to multiple myeloma as a model to understand the mechanism that drives malignant transformation. I reasoned that if we understood these, we should be able to manipulate them therapeutically.

I continue to be inspired by the ability to translate the biology and genetics of myeloma into clinical practice, where better and less toxic treatments can and are being developed to help patients achieve better outcomes and increased survival. Patients are the true source of inspiration.

Myeloma Genome Project

RWHC: Tell us about your recent research work and its significance to the multiple myeloma patient community.

GM: This is an exciting time for the field of myeloma, and our research investigations have led to some exciting discoveries in the biology of myeloma based on the genetic variations within the human genome. With our colleagues in Europe, have identified eight new genetic variations that could be linked to an increased risk of developing myeloma.

We are also focused on developing a molecular classification of myeloma based on patient subgroups with distinct pathogenesis and clinical behavior. We have partnered with Celgene and the Dana–Farber Cancer Institute in establishing a global collaboration called the Myeloma Genome Project. The goal of this project is to compile and analyze the largest set of genomic and clinical data to design a molecular classification system to improve the diagnosis, prognosis and treatment of myeloma. Up to this point, we have collated the data that we already have, and now we are bringing other national and international investigators in to really form a global consortium. This data will help us identify patients who have distinct clinical outcomes, and allow us to take a stratified-risk approach to designing treatment strategies. This initiative could really lead the way in developing specific clinical trials for targeted treatments for patients in the future.

Personalized Medicine: The Future of Cancer Care

RWHC: What are the most promising new treatments for multiple myeloma? Are there any on the horizon that hold the possibility for a cure?

GM: I believe that stem cell transplantation remains the backbone of treatment for many patients, and we don’t want to move away from a strategy we know to be successful for many. So, we are now incorporating new treatments to increase cure rates and to give more patients higher and better responses overall. This involves moving away from the one-size-fits-all approach to a more directed, personalized one which includes the use of novel agents, immunotherapy, and targeted-based treatments.

Immunotherapy is one of the more exciting developments for patients with relapsed/refractory disease, and we are now looking at incorporating these agents into the newly diagnosed setting. Using different combinations of antibodies that address the different components of the immune system is going to be a really important way forward. In addition, the increased understanding of cancer genomics has given us a wealth of information about the biological processes involved in the initiation and development of myeloma cells, which has really powered the concept of developing targeted therapies directed at specific mutations at the molecular level. This approach, also known as personalized or precision medicine, clearly represents the future of cancer care.

Underlying Causes of Myeloma

RWHC: What are some of the biggest challenges facing multiple myeloma researchers?

GM: Some challenges myeloma researchers face include gaining a better understanding of the underlying causes of myeloma and designing prevention and early intervention strategies, as well as developing strategies to prevent precursor states of myeloma—MGUS (monoclonal gammopathy of undetermined significance) and smoldering myeloma—from developing into active multiple myeloma. Of course, funding remains one of the biggest challenges faced by researchers, and it probably always will be.

Increasing collaborations with university and industry partners provides an opportunity to gain access to much larger datasets and to develop clinical trials to help answer some of these important questions.

Overcoming Resistance to Multiple Myeloma Treatment

RWHC: What are some of the biggest challenges facing clinicians treating multiple myeloma patients?

GM: Over the last decade, we’ve seen an unprecedented improvement in multiple myeloma as novel agents and treatment combinations expand. Despite the vast improvement, there remains a proportion of patients who relapse and are more likely to have aggressive disease that is refractory to therapy. I think one of the challenges in myeloma is how do we treat and design strategies that can overcome treatment resistance for these high-risk patients to improve their long-term outcomes.

This challenge requires a change that focuses on the use of genetic analyses to segment myeloma at the molecular level to enhance risk segmentation to develop biologically-stratified treatment approaches. We are also exploring the use of imaging studies to recognize high-risk and DNA-based features. Collecting the sequencing and imaging data and analyzing it consistently provides a resource for the myeloma community that can continue to grow into the future.

Another important challenge facing clinicians is patient access to a myeloma specialist. Myeloma is a rare and complex cancer, so oncologists can’t use concepts developed for more common cancers, such as breast or colon, in myeloma. The key is having a focused strategy that is directed by a myeloma expert. Unfortunately, some patients don’t have access to a specialist because of location, insurance, or other financial restrictions. At the Myeloma Institute we incorporate a team approach, whereby our team of myeloma experts direct the treatment strategy and for practical purposes, patients can receive much of their treatment locally. So, if myeloma experts can partner with local oncologists who can then deliver some of the treatment, then it’s an ideal setting for patients, because they receive the benefit of a myeloma-specific strategy and risk stratification upfront, and in the long-term, they have the comfort and security of being close to home.

 

Multiple Myeloma & Cardiac Risk

Real World Health Care continues our series on multiple myeloma with a discussion about the cardiac effects of cancer treatment with Dr. Robert Frank Cornell.  Dr. Cornell is the clinical director of the plasma cell disorder program and the director of the plasma cell/lymphoma clinical trial research program at Vanderbilt University Medical Center.

Dr. Cornell’s research focuses on translational and early phase clinical trials for the treatment of multiple myeloma. He also conducts research and clinical trials for relapsed/refractory multiple myeloma and other plasma cell disorders. He also researches cardio-oncology to better understand the effects of chemotherapy on the heart and vascular system.

Effective Treatments Carry Risks

Real World Health Care: What initially intrigued you about the cardiac effects of cancer treatments?

Robert Frank Cornell, MD, Vanderbilt University Medical Center

Robert Frank Cornell: I was initially drawn to study cardio-oncology due to some cardiac toxicities observed with a drug used to treat myeloma called carfilzomib. While this drug was found to be very effective in treating myeloma, it had a small but definitive increased risk of cardiac toxicities including heart failure, arrhythmia, hypertension, coronary syndrome and rarely sudden cardiac death.

I continue to be inspired by researching cardiac toxicities because it is through our research efforts we have determined means to both predict patients at risk for cardiac toxicity and mitigate the severity of the effects in order for patients to continue to receive this effective therapy. As more drugs are developed, such as CAR T-cell therapies, there will be ongoing need to understand any potential cardiac risk associated with treatments with the goal of improved patient outcomes and quality of life.

Many Myeloma Patients Already at Risk

RWHC: Can you describe some of the common cardiac complications involved in multiple myeloma treatment?

RFC: Since the average age of diagnosis for myeloma is around 68 years, many patients already have risk factors for cardiac toxicities such as hypertension, hyperlipidemia, diabetes mellitus, obesity and kidney dysfunction. The addition of chemotherapy in this patient population increases the risk of possible cardiac complications.

One of the most common drugs used to treat myeloma is dexamethasone. This drug is a corticosteroid and can result in fluid retention and high blood pressure. Another treatment commonly used for myeloma is high dose melphalan followed by autologous hematopoietic cell transplantation (stem cell transplant). During the course of the transplantation process, patients are monitored for development of cardiac toxicities. It is not uncommon for patients to develop atrial fibrillation (a fib) or have an exacerbation of preexisting a fib during the course of transplant due to the added stress on the heart.

Carfilzomib, as mentioned above, also increases the risk as cardiac complications. The drug class of immunomodulatory/cereblon-binding agents including lenalidomide and pomalidomide increases the risk for thromboembolic events including deep vein thrombosis (clot in the deep veins of the leg) or pulmonary embolus (clot in the artery of the lung). While this is not a traditional cardiac complication, many cardiologists monitor for this since it is thought that the blood vascular system may play a role in the development of these.

The majority of other FDA approved treatments for myeloma have very low risk for cardiac toxicity such as the drug class of reversible proteasome inhibitors including bortezomib and Ixazomib (though there are case reports of cardiac problems with bortezomib) and monoclonal antibodies including elotuzumab and daratumumab.

Collaborative Care

RWHC: What sort of challenges do clinicians face when treating multiple myeloma patients in terms of minimizing cardiac complications?

RFC: The biggest challenge I see is how best to manage a patient considered to be at very high risk for cardiac complications during the course of treatment. This patient is identified according the medical history as already having a known history of cardiac difficulties. The best strategy to overcome this is with a collaborative cardiac and oncology effort to optimize the patient’s cardiac status prior to and during the course of treatment. This includes optimization of a patient’s blood pressure, cholesterol, diabetes, heart failure, rhythm control and volume status. Should cardiac problems arise during the course of treatment, then prompt evaluation by a cardiologist should be considered, particularly in high-risk patients.

Underlying Causes of Cardiac Complications

RWHC: How can researchers overcome the challenges they face when studying cardiac complications of cancer therapies?

RFC: One of the biggest challenging for the research of cardiac complications is the determination of the underlying cause for the cardiac complication. Since patients with myeloma already often have risk factors for cardiac events, it can be challenging determining if the cardiac event occurred from the treatment itself, from supportive care as part of the treatment such as intravenous fluids, or simply occurred by chance since the patient was at higher risk for cardiac complications regardless of treatment. The cardiac event may have also occurred due to a combination of these factors. To overcome this challenge, ongoing research and collaboration between hematology and cardiology are needed to better understand the cardiac complications, develop strategies to prevent or mitigate the events and optimize patients care and outcomes to both improve survival and quality of life.

Multiple Myeloma: Promising New Therapies, But Challenges Remain

This week, our series on multiple myeloma continues as we talk about immunotherapies and other promising new treatments with Shaji Kumar, MD. Dr. Kumar is a professor of medicine in the division of hematology at Mayo Clinic, where he chairs the myeloma group across all three Mayo sites. Dr. Kumar conducts National Institutes of Health-funded research on resistance mechanisms to common myeloma drugs and epidemiology of disease progression. He also receives funding from the Multiple Myeloma Research Foundation to study the relationship between molecular profiles, treatment regimens for patients with multiple myeloma and outcomes.

Inspirations

Real World Health Care: How did you become interested in the field of multiple myeloma?

Shaji Kumar, MD, Mayo Clinic

Shaji Kumar: I did my fellowship training at Mayo Clinic. Mayo is one of the most renowned institutions in the field of plasma cell disorders. Training at Mayo, and having the fortune of working with people like Professor Robert Kyle, was very inspiring and led to my interest in this group of disorders. The patients I see keep me inspired. While we have made some progress in this area, much work needs to be done and we need to develop a cure for this disease. The progress so far convinces me that we can reach this goal if we keep at it.

High-Risk Multiple Myeloma Patients

RWHC: What is the significance of your research work to the multiple myeloma patient community?

SK: I am involved in several aspects of myeloma research. In the laboratory, we try to understand how the myeloma cell survives, especially how the other cells in the body help them grow, and try to develop new drug combinations that can lead to better treatments. I try to translate these findings to the clinic in the form of early clinical trials, which if successful, can lead to larger phase 3 trials. I am the principal investigator of several phase 1, 2 and 3 clinical trials. Another area of great interest to me is risk stratification and identification of high risk myeloma. What we have seen is that patients with high risk myeloma continue to do badly despite all the new therapies. Moreover, there are still significant issues with the current systems for identifying high risk patients. We are trying to develop new approaches for risk stratification in myeloma and develop new treatment approaches for these patients.

Monoclonal Antibodies and Immunotherapy

RWHC: What promise do monoclonal antibodies and chimeric antigen receptor (CAR T-cell) therapies hold for the treatment of multiple myeloma? Could a cure be around the corner?

SK: Immunotherapy is the next frontier for myeloma. The results so far have been spectacular. The monoclonal antibodies have opened up a new class of drugs, and daratumumab especially has led to high response rates and deep responses in patients with myeloma. The next step is to combine the current classes of drugs to develop the most effective regimens. Other immunotherapy approaches, especially CAR T-cells, while early in the testing phase, has shown significant benefit among patients with very few options left. Other approaches to enhancing the T-cell immunity such as BiTE platforms as well as vaccination approaches are in clinical trials. All in all, this is an exciting time for myeloma and I am convinced that a cure is around the corner. What needs to be determined is if this will come from treating myeloma with these approaches or whether we need to intervene at the smoldering phase.

What Triggers Multiple Myeloma?

RWHC: What are some of the biggest challenges facing researchers studying multiple myeloma?

SJ: There are many challenges facing researchers. These include the lack of understanding the triggers for development of the disease, and the ability to identify patients who progress to myeloma in a specific fashion. Lack of good models for testing the myeloma cells outside of the patient, both for its behavior and responses to treatment, remains a problem. In the clinical trial arena, the ability to do clinical trials fast and also have faster readouts using surrogate endpoints remain a challenge.

The Multiple Myeloma Landscape

Editor’s Note: This article originally appeared in Biotech Primer Weekly. For more of the science behind the headlines, please subscribe.

Emily Burke, BiotechPrimer.com

Multiple myeloma is a cancer formed by a type of white blood cell called a plasma cell. These cells are the antibody-producing cells of our immune system and play a critical role in our defense against infections. If they begin to grow and divide in an uncontrolled manner, however, they form a plasmacytoma – a mass of cells within the bone marrow that no longer function in our defense but instead simply take up space and interfere with the functions of healthy cells. Instead of producing normal disease-fighting antibodies, plasmacytoma cells produce abnormal antibodies called M proteins, which don’t provide any benefit to the body and crowd out normally functioning antibodies.

Easily Confused: Plasma Cells vs Blood Plasma

Plasma cells are specialized white blood cells that produce infection-fighting antibody proteins. Most plasma cells are found in the bone marrow. Blood plasma is the straw-colored liquid component of blood that holds blood cells in suspension, made up of water (95%), proteins, glucose, clotting factors, electrolytes, hormones, carbon dioxide, and oxygen.

Picking Apart Plasmacytoma

Plasmacytoma formation can lead to a host of problems with recognizable clinical symptoms. Because all blood cells are formed in the bone marrow, over-production of plasma cells can essentially “crowd out” normal blood-forming cells. This can lead to anemia, caused by a shortage of oxygen-carrying red blood cells; increased bruising and bleeding due to a reduction in clot-promoting platelets; and an increased risk of infections due to lower levels of healthy infection-fighting white blood cells.

Although multiple myeloma is classified as a blood cancer, it has a significant impact on bone health. As the plasmacytoma grows, bone-forming cells called osteoblasts are suppressed. At the same time, production of a substance that activates bone-reabsorbing cells, osteoclasts, is increased. The resultant damage to the bone structure results in soft spots or lesions which may extend from the inner bone marrow to the outside surface of the bone. Bone lesions result in significant pain and increase the risk of fracture. Bone destruction also releases excessive calcium into the bloodstream, which leads to a range of symptoms including changes in urination, restlessness, confusion, increased thirst, nausea, and loss of appetite. Excess blood calcium, combined with high levels of M protein, also contributes to the impaired kidney function seen in multiple myeloma patients.

Unmasking Multiple Myeloma

There is no one diagnostic test for multiple myeloma. Blood and urine tests to detect some of the symptoms listed above such as low blood cell counts, elevated blood calcium levels, and impaired kidney function may suggest multiple myeloma. These tests can be followed by a bone marrow biopsy for confirmation.

Most cases of multiple myeloma have no known cause, although some research suggests that regular exposure to herbicides, insecticides, petroleum products, heavy metals, and asbestos increases the risk of developing the disease. And although there is not a specific gene yet associated with multiple myeloma, abnormalities in chromosome structure or number are associated with the disease.

Multiple Myeloma Treatments

Once considered incurable, there are now a number of effective treatments for multiple myeloma, and several more are in the pipeline.

Currently, there are two FDA-approved monoclonal antibody therapeutics approved to treat multiple myeloma.  They work by recognizing and binding to proteins on the surface of multiple myeloma cells, activating the patient’s immune system to destroy those cells.

Another type of approved therapy for multiple myeloma is a small molecule proteasome inhibitor therapy. A proteasome is a specialized compartment within the cell that gets rid of damaged proteins by digesting them. If the proteasome is inhibited, damaged proteins build up within the cell. This triggers a process called apoptosis – essentially, cell suicide. In other words, the cancer cell kills itself.

Small molecule histone deacetylase (HDAC) inhibitors have also been shown to be safe and effective in treating multiple myeloma. HDACs are enzymes that modify chromosomes (strands of DNA that contain our genes) and influence how often specific genes are activated. Some cases of multiple myeloma are associated with changes in gene activation. By inhibiting HDACs, this faulty gene expression can be corrected.

In the Pipeline

Two novel drugs in the multiple myeloma pipeline are Mivebresib and Selinexor.

Mivebresib influences the activation of specific genes by inhibiting a group of proteins called Bromodomain and Extra Terminal motif (BET) proteins. In some types of cancer, genes are activated or deactivated inappropriately due to BET activity. By inhibiting BET, normal gene activity may be restored to these cells.

Selinexor helps to increase the number of tumor suppressor proteins present in the nucleus of cancer cells. These proteins help to protect against cancer by detecting DNA damage and promoting apoptosis in those cells that have high levels of DNA damage. In many types of cancer cells, tumor suppressor proteins are transported out of the nucleus, where they can no longer do their job of detecting DNA damage. Selinexor blocks this transport and enables tumor suppressor proteins to do their job of triggering apoptosis in cancer cells.

A number of CAR-T therapies are also in development for multiple myeloma, with several early stage clinical trials ongoing. 

Multiple myeloma is a complex type of cancer. In recent years, a better understanding of the disease has led to the approval of several new therapeutics. In the coming years, we can look forward to additional approvals as novel therapeutics move through the pipeline.

New Real World Health Care Series: Multiple Myeloma Research and Treatment

Krista Zodet, President, HealthWell Foundation

Susan, from Columbia, S.C., is among the millions of Americans struggling to manage a chronic and life-altering disease without the financial means to afford needed medication. Here at HealthWell, we’re honored to be able to help Susan and other patients like her. It always warms our hearts to receive letters like the one she sent:

“I have been a multiple myeloma patient since December 2007 and, unfortunately, there is presently no cure for this cancer. Thankfully, I am responding to treatment, but it is expensive. My oral maintenance medication costs over $11,000 per month and it gets more expensive every year. I am so very thankful for Medicare, but we have a $3,600 deductible that has to be met before insurance pays the claims. That’s a lot of money to come up with each year on a worship pastor’s salary. The charity I was receiving help from can no longer help multiple myeloma patients, but they referred me to HealthWell Foundation and it could not have been easier! The HealthWell representatives I have spoken with have been professional, caring, and efficient. I am thrilled with my experience. Thank you to the wonderful and generous donors who make this possible.” – Susan, Columbia, SC

Susan’s story is not unique. It is however, unfortunate, and one that we hear at the HealthWell Foundation all too often. Cancer is not easy and it’s not inexpensive. That’s why it’s important for us to continue to use this blog to highlight advances in current research, therapies, and what’s on the horizon for treating devastating diseases like Multiple Myeloma.

About Multiple Myeloma

Multiple myeloma is a type of blood cancer that affects the plasma cells found in bone marrow. It is the second most common blood cancer and is considered incurable. It is a treatable disease, however, thanks to recent advances in cancer research which are improving the life expectancy of multiple myeloma patients.

According to the American Cancer Society, more than 30,000 new cases of multiple myeloma are expected to be diagnosed in 2017 and more than 12,000 deaths are expected to occur. The disease is more prevalent among men than among women.

Promising New Therapies

Our new Real World Health Care series will shine a spotlight on the individuals and organizations driving research on new multiple myeloma therapies, including monoclonal antibodies, CAR-T cell therapy, checkpoint inhibitors and other immunotherapies. I share the hopes of these researchers that these new therapies, or others just coming to the drawing board, will allow multiple myeloma to be treated easily and effectively, and with the possibility of a cure.

Supporting Multiple Myeloma Patients

The HealthWell Foundation, sponsor of Real World Health Care, is proud to support the multiple myeloma patient community with copayment and premium assistance. We have helped more than 9,000 multiple myeloma patients afford their treatments since launching our Multiple Myeloma Medicare Access Fund in 2015 — thanks to the generous support of our donors. We invite corporations and individuals to help us by contributing to our Multiple Myeloma Medicare Access Fund, so no one goes without essential medications because they cannot afford them.

Skin Cancer Awareness and Prevention Efforts in Focus at American Academy of Dermatology

This is the last week of Real World Health Care’s brief hiatus, during which we are re-publishing some of our most popular interviews on oncology-related topics.

The editors of Real World Health Care, along with our sponsor, the HealthWell Foundation, understand that cancer takes a huge toll on patients, their families and loved ones. About 1.6 million new cases of cancer were diagnosed in the United States last year, and nearly 600,000 people died from the disease.

Real World Health Care is pleased to shine a spotlight again on the researchers, clinicians and organizations dedicated to the future of cancer care. We also are proud of the work being done by the HealthWell Foundation, which provides a financial lifeline to underinsured Americans through grants for cancer patients across a variety of funds, such as multiple myeloma, bone metastases, and chronic myeloid leukemia for Medicare patients, to help them afford the medical treatments they so desperately need.

In May, Real World Health Care continued our recognition of Melanoma and Skin Cancer Awareness Month by highlighting the work of the American Academy of Dermatology. We spoke with the AAD’s new President, Henry W. Lim, MD, about the organization’s mission and some of the challenges and opportunities associated with preventing and treating melanoma and other skin diseases.

Real World Health Care: Please tell our readers about the overall mission of the American Academy of Dermatology.

Henry W. Lim, MD, American Academy of Dermatology

Henry Lim: The American Academy of Dermatology promotes leadership in dermatology and excellence in patient care through education, research and advocacy.

As the largest, most influential and representative dermatology group in the United States, and the largest such organization in the world, the AAD works to make sure its values reflect this mission. The AAD’s values include putting patients first, encouraging its members to adhere to an uncompromising code of clinical and ethical standards, fostering an interest in our members to pursue lifelong learning, encouraging collaboration and working within our communities and embracing diversity.

Public Education: Sun Safety

RWHC: How does the AAD’s mission address melanoma?

HL: It is estimated that 161,790 new cases of melanoma will be diagnosed in the U.S. in 2017.  That is a staggering number that could be reduced if people incorporated skin cancer detection and prevention behaviors into their lives.

The AAD works to increase public awareness of skin cancer and its risks through its SPOT Skin Cancer campaign, which is designed to create a world without skin cancer through public awareness, community outreach programs and services, and advocacy that promote the prevention, detection and care of skin cancer.

The first step toward a world without skin cancer is educating the public about prevention. The Academy has long communicated sun-safety messages to the public about the importance of skin cancer prevention and detection.

In addition, dermatologists have led the medical community in finding and treating skin cancer. For more than 30 years, dermatologists across the country have hosted 2.5 million free SPOTme® skin cancer screenings that have detected 28,822 suspected melanomas and 256,329 suspected skin cancer lesions.

To assist the public with learning more about skin cancer prevention and detection, the AAD offers a variety of free, online videos, downloadable handouts and skin self-exam resources, including a body mole map, as well directories to find a dermatologist and skin cancer screenings.

Melanoma & Skin Cancer Awareness

RWHC: What is the AAD doing in 2017 to recognize Skin Cancer Awareness Month?

HL: The AAD’s 2017 SPOT Skin Cancer campaign, Check Your Partner. Check Yourself, encourages the public to be aware of changes on their skin that could be signs of skin cancer. Research has shown that women are more likely to detect suspicious spots on others.  Men over the age of 50 have a higher risk of developing melanoma, than the general population, so the campaign encourages women – often the health care decision makers of a household – to check their partner’s skin regularly, check their own skin, and to visit the AAD’s SpotSkinCancer website to find a free SPOTme® screening in their area.

RWHC: Do you have additional initiatives you’d like to highlight?

HL: In addition to the activities for Skin Cancer Awareness Month in May and the SpotSkinCancer™ website, the AAD works with state dermatology societies and state legislatures to introduce and support laws and regulations that protect consumers and promote awareness about skin cancer prevention and the dangers of indoor tanning. As a result, 42 states have enacted tanning bed restrictions to potentially reduce the risk of melanoma and other forms of skin cancer.

The AAD’s Shade Structure Program awards shade structure grants to schools and non-profit organizations across the country in order to protect children and adolescents from the sun’s harmful rays.  Since its launch in 2000, the AAD’s Shade Structure Program has awarded 350 shade structure grants, which provide shade for more than 600,000 individuals each day.

The AAD also has a strategic social media presence on Facebook, Twitter, YouTube and Pinterest, designed to raise awareness about skin cancer detection and prevention.  Social media, including paid, promoted posts, reach our targeted audiences – the public, our members and the media – with links to AAD resources.  We encourage our followers to like, share and re-tweet our skin cancer awareness videos and tips.

Melanoma Research

RWHC: Does the AAD underwrite or otherwise support research into melanoma detection and/or treatment?

HL: While AAD is not a research funding organization, the AAD does provide annual awards for Young Investigators in Dermatology.  These awards recognize outstanding basic and clinical/translational research by young dermatology investigators and some of the projects are related to melanoma.

The purpose of the award is to acknowledge research contributions by individuals at the start of promising research careers that further the improvement of diagnosis and therapeutics in the practice and science of dermatology.

RWHC: What do you see as the biggest challenges facing researchers studying melanoma treatments and clinicians treating melanoma?

HL: The rapidly changing health care environment presents major challenges to researchers and clinicians in all aspects of dermatologic care, not just those studying and treating melanoma. 

A significant challenge is the inadequate funding for research, together with the pressure to increase clinical revenue generated by clinician researchers.  For many years, the American Academy of Dermatology Association (AADA) has been active in advocating increased research funding by NIH to dermatology research, including through our support of the 21st Century Cures Act.

The current health care system also presents barriers that impede patient access to the best possible care from a qualified physician.  To combat this, the AADA is working with all dermatology care providers and other physicians to confront these challenges.

In particular, the AAD recently launched a new specialty positioning campaign, SkinSerious, to raise awareness of the serious impact of skin disease. Our goal is also to improve access to dermatologists’ expertise and increase collaboration with our physician peers to ensure high-quality patient care. We know that when dermatologists work with other physicians as part of the health care team, everyone can benefit from improved patient outcomes and lowered health care costs.

Other concerns within the health care environment that the AADA is closely monitoring include the rise of big data and the growth of teledermatology.  We closely follow developments at the federal and state levels and, when appropriate, the AADA will take action on issues that can be influenced positively for dermatology and pursue opportunities to impact health care policy. 

Promising Melanoma Treatments

RWHC: What do you see as the most promising or breakthrough melanoma treatments on the horizon?

HL: This is an exciting era in melanoma research.  In-depth understanding of the molecular pathways of melanoma development has led to the availability of immune checkpoint inhibitors; combinations of these medications are being looked at in clinical trials.  Metabolic manipulation of the peri-tumoral environment to inhibit the growth of melanoma is being actively investigated.  Understanding of the genes responsible for melanoma resulted in the availability of gene expression profile (GEP) test that can be used to determine biologic behavior of melanoma.

Melanoma Prevention

RWHC: What are the biggest challenges facing the medical community in terms of increasing awareness of and adherence to melanoma prevention efforts among the general public?

HL: The challenges facing the medical community around melanoma prevention are two-fold.

One is the misconception that a tan is a sign of health.  Tanning is a protective physiologic response of our skin to damage caused by ultraviolet radiation.  There is no such thing as a healthy tan, yet people continue to seek the sun or use indoor tanning, thereby increasing their risk of skin cancer.  This is a particularly challenging message to get across to young women and men, who feel peer and societal pressure to be tan.

The AADA was instrumental, along with several other organizations, in having the FDA re-classify tanning lamps from the Class I to Class II medical device category, which requires more supervision and restriction in their purchase and use.  For the past several years, the AAD has released a new public service advertisement that focuses on the dangers of tanning, particularly targeting young women.  We know that melanoma is the second most common cancer in young women, and this may be due in part to their tanning habits.

The 2016/2017 public service advertisement is called “Arms,” and features two young women comparing their tans at various stages in their lives. The emotional ad concludes with the two friends clasping hands in the hospital as one of them reveals she has advanced stage melanoma.  This PSA, and our previous ones, have resonated strongly with young women, especially on social media, where they have liked and shared the video with their friends.

The second challenging misconception is that many people believe that sun exposure is the best source of vitamin D.

While our bodies need vitamin D to build and maintain strong, healthy bodies, the AAD does not recommend getting vitamin D from sun exposure or indoor tanning because of the increased risk of skin cancer.  In fact, it has been demonstrated that sun exposure that results in increased vitamin D levels is directly correlated with DNA damage.

Vitamin D from food and dietary supplements offers the same benefits — without the danger of skin cancer — as vitamin D obtained from UV light.  Vitamin D cannot be used by the body until it is processed by the liver and the kidneys. The usable form of vitamin D created by this process is the same, regardless of how it enters the body.

The AAD recommends dietary sources (foods naturally rich in vitamin D, fortified foods and beverages) and vitamin supplements as sources of vitamin D that are available year-round and can easily be incorporated into a healthy lifestyle. Good sources include fortified milk, cheeses and yogurt, fortified cereal, and oily fish like salmon and tuna. Research shows that vitamin D supplements are well tolerated, safe, and effective when taken as directed by a physician.

The fact is these myths are harmful because the consequences of this misinformation could be potentially fatal.

RWHC: What personally inspires you to build awareness of the importance of preventing melanoma?

HL: Having been in dermatology practice for 40 years, I see on a regular basis the devastating effects that melanoma has on patients and their family.  The risk of developing melanoma can be significantly decreased by sensible photoprotection, and avoidance of tanning beds.  The exciting new developments in the treatment and genetic profiling of melanoma reflect the value of investment in scientists and research projects, and I look forward to additional treatments in the future that will benefit patients.

A MESSAGE FROM OUR SPONSOR:

The HealthWell Foundation, sponsor of Real World Health Care, is proud to have supported the melanoma patient community in recent years with copayment and premium assistance. We have helped more than 2,230 melanoma patients afford their treatments since approving our first Melanoma grant in 2011 — thanks to the generous support of our corporate partners. Due to high patient volume, our melanoma fund is temporarily closed until we receive additional funding. We invite corporations and individuals to help us meet this demand by contributing to our Melanoma-Medicare Access Fund, so nobody goes without essential medications because they cannot afford them.

 

Categories: General, Melanoma

Non-Small Cell Lung Cancer: EGFR Mutations and Targeted Therapies

For the next several weeks, Real World Health Care will take a brief hiatus as we re-publish some of our most popular interviews on oncology-related topics.

The editors of Real World Health Care, along with our sponsor, the HealthWell Foundation, understand that cancer takes a huge toll on patients, their families and loved ones. About 1.6 million new cases of cancer were diagnosed in the United States last year, and nearly 600,000 people died from the disease.

Real World Health Care is pleased to shine a spotlight again on the researchers, clinicians and organizations dedicated to the future of cancer care. We also are proud of the work being done by the HealthWell Foundation, which provides a financial lifeline to underinsured Americans through grants for cancer patients across a variety of funds, such as multiple myeloma, bone metastases, and chronic myeloid leukemia for Medicare patients, to help them afford the medical treatments they so desperately need.

Continuing our series on non-small cell lung cancer, this week Real World Health Care speaks with Lecia V. Sequist, MD, MPH, Associate Professor of Medicine at Harvard Medical School and the Mary B. Soltonstall endowed chair in oncology at Massachusetts General Hospital. Dr. Sequist’s research focuses on studying novel targets and targeted agents for lung cancer treatment, particularly those that target the epidermal growth factor receptor (EGFR) and in detecting and studying the significance of tumor cells circulating in the bloodstream.

Real World Health Care: Tell us about what you do at Massachusetts General Hospital, especially in relation to research and treatment of non-small cell lung cancer.

Lecia V. Sequist, MD, MPH, Harvard Medical School

Lecia Sequist: I’m a medical oncologist with a busy practice, seeing and treating patients with lung cancer. I also conduct clinical and translational research on new drugs, looking at the molecular aspects of tumors and biopsies as patients go through various forms of treatment. My focus is on personalizing treatment for each patient.

RWHC: Can you share some highlights of your recent research in non-small cell lung cancer?

LS: Most of my recent research has revolved around EGFR mutations. One of the biggest advances in lung cancer in recent years is that we’ve come to understand lung cancer is not one disease. It’s many diseases. We can now tell the difference between one cancer and another by looking at the tumor genetics. These are not the genes we inherit from our parents, rather they are genes that reside only in cancer cells. These genes are at the core of what causes cancer. By identifying these genes in a lung cancer patient’s tumor, we can be more successful with treatments that target those genes and the proteins they produce.

EGFR mutations were first discovered here at Mass General, right around the time I started in oncology. It was a very exciting time, and ushered in a new era of personalized treatment for cancer. Since those early days, we’ve done a tremendous amount of research with patients who have the EGFR mutation, and we’ve found treatments that work better than standard chemotherapy.

RWHC: What are some of the biggest challenges you face as a researcher studying non-small cell lung cancer?

LS: I think of the challenges in two categories: scientific and societal. From the scientific point of view, we can’t currently identify mutations in every lung cancer, though we’re constantly working to uncover more of them. The group of lung cancer patients who have no identifiable mutation, or who have a mutation with no matching drug therapies at this time, are effectively left out of the “molecular revolution.” For those groups, the challenge is to find alternative approaches. Luckily some of the newer immunotherapies may work particularly well in such patients. Then down the road, we know that targeted therapies eventually “wear off,” in the sense that cancer cells get smart and find ways to work around the roadblocks we put in their path. For example, we saw this with the first generation of tyrosine kinase inhibitors (TKIs) developed to target EGFR mutations. Most patients initially responded, but subsequently developed a resistance after about a year, because they developed a second mutation that prevents the TKIs from binding to the cancer cells. Last year, a new EGFR drug was FDA approved that is able to effectively target this second mutation. Now we’re racing trying to learn how the cancers may get around the newer drug and also looking at strategies to prevent resistance.

From a societal standpoint, one of our biggest challenges in the lung cancer research community is the stigma that still exists around lung cancer. In the United States, we were fortunate to have had a very successful public health campaign around the dangers of smoking over the last generation. Those dangers are important to understand, but one of the unexpected consequences of this was to popularize the opinion that lung cancer is a self-inflicted disease and therefore patients carry some degree of blame. Not only does this end up negatively affecting individual patients, it also cuts into research funding. The fact is, some smokers get lung cancer while others don’t. And more importantly, many lung cancer patients have never smoked. No one deserves lung cancer and research must push forward to stop this, the deadliest of all cancers.

RWHC: What are some of the biggest challenges you face as a clinician treating patients with non-small cell lung cancer?

LS: There are promising treatments being studied in clinical trials, but many patients don’t have access to those treatments because the trials are concentrated in academic centers. Even if patients have geographic access to research studies, clinical trials have fairly high thresholds for eligibility, so if a patient has other medical conditions — which many lung cancer patients have — or if their cancer has certain characteristics, they won’t be eligible for the trial. We need to keep pressure on the pharmaceutical industry to include broader groups of patients in trials so all patients can get access to promising new treatments.

RWHC: What do you think are some of the biggest opportunities for advancement in how we research non-small cell lung cancer and treat people with the disease?

LS: Immunotherapy has really changed the paradigm for non-small cell lung cancer. Years of failed vaccine studies led us to believe that it wasn’t possible to affect the human immune system in meaningful ways against lung cancer. Now that we’ve hit upon a different way to activate the immune system, new discoveries are tumbling out the door every day. Unlike past treatments, immunotherapy has true promise for long-term disease control. There are already three FDA-approved lung cancer immune therapy treatments over the last year and likely many more to come. I think someday we’ll look back on this time and say that this is when the needle really started to move.

RWHC: Why did you get into this field of research? What continues to inspire you?

LS: I was initially drawn to studying lung cancer when I was in training by the doctors who were mentoring me and the patients I met. At the time, there weren’t many treatments available for non-small cell lung cancer, so there was a lot of room for improvement. This was attractive to me as a clinician and a researcher and it has remained a vibrant and ever-changing field. I enjoy being involved in the exponentially increasing number of treatments available and how these new treatments can bring hope to patients. It has ended up being an intellectually stimulating and extremely fulfilling career and I continue to be inspired by the patients I meet every day.

Pain Management: Opioid Adherence in Cancer Patients

For the next several weeks, Real World Health Care will take a brief hiatus as we re-publish some of our most popular interviews on oncology-related topics.

The editors of Real World Health Care, along with our sponsor, the HealthWell Foundation, understand that cancer takes a huge toll on patients, their families and loved ones. About 1.6 million new cases of cancer were diagnosed in the United States last year, and nearly 600,000 people died from the disease.

Real World Health Care is pleased to shine a spotlight again on the researchers, clinicians and organizations dedicated to the future of cancer care. We also are proud of the work being done by the HealthWell Foundation, which provides a financial lifeline to underinsured Americans through grants for cancer patients across a variety of funds, such as multiple myeloma, bone metastases, and chronic myeloid leukemia for Medicare patients, to help them afford the medical treatments they so desperately need.

This week, Real World Health Care speaks with Salimah H. Meghani, PhD, MBE, RN, FAAN. Dr. Meghani is an associate professor and term chair in Palliative Care at the University of Pennsylvania School of Nursing. She is also associate director, NewCourtland Center for Transitions and Health. Her main research interest involves palliative care, specifically understanding and addressing sources of disparities in symptom management and outcomes among vulnerable patients.

We asked her about her study on analgesic adherence and health care utilization in outpatients with cancer pain, recently published in Patient Preference and Adherence. We also discussed the role of non-pharmacological approaches in treating cancer pain.

Opioid Adherence Patterns

Real World Health Care: Last year, you published an article: Patterns of analgesic adherence predict health care utilization among outpatients with cancer pain. Can you provide a brief summary of the article and talk about the study’s implications for cancer patients with pain management issues?

Salimah H. Meghani, University of Pennsylvania School of Nursing

Salimah Meghani: This is the first study to understand how opioid adherence patterns, over time among cancer patients, relate to health care utilization outcomes. We used objective measures of adherence (Medication Event Monitoring System – MEMS) and novel adaptive methods recently validated by the co-author, Dr. George Knafl from UNC-Chapel Hill. We found that inconsistent adherence patterns of analgesics over time was significantly associated with hospitalization over a 3-month observation period. The interaction of inconsistent adherence and strong opioids (WHO step 3 opioids) was one of the strongest predictors of health care use. It should be noted that this was a serendipitous finding. We did not plan to study adherence patterns and health care utilization. It therefore needs validation in hypothesis-driven study.

RWHC: Are you currently involved in any new research programs studying pain management in cancer patients? If yes, can you briefly describe?

SM: Yes, I am studying outcomes of opioid adherence and adherence patterns among cancer outpatients. This is an important topic as few recent U.S. based studies exist on the topic despite all the recent guideline contentions (e.g., CDC guidelines for managing chronic pain including chronic cancer pain and ASCO response) and national policy debates on opioids.

How Patients Manage Cancer Pain

RWHC: What do you think are the biggest challenges facing researchers studying pain management in cancer patients? How can those challenges be addressed?

SM: One of the biggest challenges is that we know very little about how patients manage their cancer pain. We know that opioids are widely prescribed, but we also know that there is poor adherence to prescribed opioids. Other treatments such as acupuncture are not consistently covered by health insurance or lack data on clinical effectiveness. There is a need to understand how patients are managing their cancer pain and what health care systems can do better to address the great burden on unrelieved cancer pain. Future work should also include improving access to effective non-opioid treatments for cancer patients. My previous research has also documented racial and ethnic disparities in cancer pain treatment for African Americans, which requires continued attention.

Safe Opioid Use

RWHC: What do you think are the biggest challenges facing clinicians treating pain in cancer patients? How can those challenges be addressed?

SM: There is a lot of confusion among clinicians about the role of opioids and the safe and rational use of opioids among cancer patients. Unfortunately, there is little empirical evidence base about the outcomes of opioid treatment among cancer patients. A look at the recent CDC guidelines on managing chronic pain would indicate that cancer patients frequently, if not invariably, have been excluded from the studies of the outcomes of chronic opioid therapy. More empirical evidence is needed to help clinicians develop comfort in opioid prescriptions.

Non-Opioid Treatments

RWHC: What do you think is the role of non-pharmaceutical pain management therapies for cancer patients? How can clinicians integrate both pharmaceutical and non-pharma therapies for cancer patients?

SM: I think access to non-pharmacological treatments is the biggest problem. While the NCCN guidelines for cancer pain identify a number of non-pharmacological modalities, they are not readily accessible to cancer patients. I have argued this in a recent letter to JAMA Oncology about the CDC opioid guideline that recommends that non-opioid treatments should be the first line therapy for chronic pain. This paradigm assumes easy and consistent access to non-opioid treatments. Also, access to effective non-pharmacological treatments are very different among poor, minorities, those with limited literacy.

Global Disparities

RWHC: What initially attracted you to this field? What continues to inspire you about it?

SM: My original research interest was global disparities in opioid availability for cancer pain management and the role of the International Narcotics Control Board. After migrating to the United States, I became familiar with racial and ethnic disparities in pain care and the toll it has for patients and families. This work continues to inspire me.

American Lung Association: Research Focused on Improving Patient Care and Saving Lives

For the next several weeks, Real World Health Care will take a brief hiatus as we re-publish some of our most popular interviews on oncology-related topics.

The editors of Real World Health Care, along with our sponsor, the HealthWell Foundation, understand that cancer takes a huge toll on patients, their families and loved ones. About 1.6 million new cases of cancer were diagnosed in the United States last year, and nearly 600,000 people died from the disease.

Real World Health Care is pleased to shine a spotlight again on the researchers, clinicians and organizations dedicated to the future of cancer care. We also are proud of the work being done by the HealthWell Foundation, which provides a financial lifeline to underinsured Americans through grants for cancer patients across a variety of funds, such as multiple myeloma, bone metastases, and chronic myeloid leukemia for Medicare patients, to help them afford the medical treatments they so desperately need.

As part of our series on non-small cell lung cancer (NSCLC), Real World Health Care spoke with Susan J. Rappaport, MPH, vice president for research and scientific affairs, American Lung Association. Rappaport provides leadership and direction in the development and implementation of the American Lung Association public health education and research programs. She works with leading scientists to develop research and scientific policies relevant to lung disease.

Here, Rappaport shares insights on how the American Lung Association supports research for NSCLC and other types of lung cancer.

Real World Health Care: How does the American Lung Association fund, or acquire funding, for the research it supports?

Susan J. Rappaport, MPH, American Lung Association

Susan Rappaport: The American Lung Association has been funding research for more than 100 years. We receive our support through public donations, including our annual Christmas Seals® campaign, Fight for Air Climbs and LUNG FORCE Walks. We have a long history of connecting with people and communities in support of lung health.

Our organization was founded in response to tuberculosis — the most feared disease at that time. Now, with tuberculosis largely controlled in the United States, we have turned our sights toward defeating lung cancer and working toward a world free of lung disease.

Research is a critical part of our LUNG FORCE initiative, which focuses on lung cancer in women, to raise awareness and more lung cancer research funds. Through LUNG FORCE, we have already invested an additional $1 million in lung cancer research. The Lung Association’s Awards and Grants Program supports a rich array of studies in lung cancer to help improve methods of early detection and develop better treatment options for patients. In the past four years, we have funded more than $4 million in lung cancer research grants and have doubled our investment in lung cancer since 2015. This year, the Lung Association is funding more than $6.5 million in lung disease research.

RWHC: How does the Lung Association determine what research it supports, either through direct funding or through its advocacy work?

SR: It is important to the Lung Association that we fund the best projects available on a host of lung disease issues. We solicit grant applications each year, and successful applicants are identified through a scientific peer review committee system modeled on the one used by the National Institutes of Health (NIH). These peer review committees are comprised of accomplished and diverse researchers with the necessary expertise to review and assess each proposal. Proposals are funded based on the results of this process, ensuring that we only fund those applications considered of the highest quality, and with the best chance to advance our understanding of diseases, improving patient care and ultimately saving lives.

We know our chances of significant improvement in patient lives and of finding a cure increase when we work together. That’s why we collaborate with other organizations and advocate for increased research funding at NIH.

RWHC: What is the Lung Association currently doing to promote and/or fund research into NSLC? What are your priorities in this area?

SR: Our overall priority is to fund the best research that has the greatest chance of a scientific breakthrough and making a difference in patient care and quality of life. With increased funds available to lung cancer researchers, we attract and retain brilliant, motivated investigators to the field.

As NSCLC accounts for 85 percent of all lung cancer cases, many of the proposed projects do focus on this specific issue. However, the nature of scientific discovery has shown us that answers from one area of research can also work more broadly. Areas that drive our lung cancer research — all of which can address NSCLC — include:

  • Development of new and combination therapies
  • Biomarker discovery and validation
  • Targeted therapies and resistance
  • Screening implementation and novel screening for the non-high-risk population
  • Lung cancer initiation and growth

RWHC: What are the biggest challenges in NSCLC research and how is the Lung Association working to overcome them?

SR: Among our biggest challenges is to be able to fund all the qualified research applications we receive. Each year, we turn away qualified researchers and projects due to the limited availability of funds. LUNG FORCE seeks to raise additional funds for lung cancer research and raise awareness about lung cancer as the leading cause of cancer deaths. Again, we strive to overcome gaps in funding by leveraging our resources, collaborating with other organizations and advocating for increased federal funding for lung cancer research.

RWHC: What would you say have been the most important advances in NSCLC treatment over the past 10 years?

SR: Scientists have discovered somatic mutations — called “driver mutations” — that drive the development of lung cancer. These discoveries, made over the past decade, have transformed how to identify and treat the disease. Now, lung and other tumors can be tested for these mutations.

There are now specific therapies that can address those genetic changes that keep the cancer cell growing. These therapies target the mutations in different ways, are more specific and have fewer side effects. These advances in precision medicine have led to lifesaving discoveries and treatments.

Another sea change in lung cancer treatment is immunotherapy. Cancer cells have found ways to keep the immune system from identifying and destroying them, as they do for infectious invaders. Immunotherapy medicines work to activate a person’s own immune system to recognize and kill cancer cells. So far, immunotherapy has only been approved to treat some forms of NSCLC. Currently, only a minority of patients respond to immunotherapy. However, a large proportion of those who do respond have improved survival.

RWHC: What do you think will be the next biggest advance in NSCLC treatment in the near future?

SR: Our scientific advisors believe that the Lung Association should invest in the following areas, which they identified as having the potential for the most important breakthroughs — all of which can apply to NSCLC:

  • Additional precision medicine in treatment of early-stage lung cancer
  • Precision medicine to identify patients who would benefit from newly developed lung cancer screening, irrespective of smoking history
  • Non-invasive biopsy strategies
  • Comparative effectiveness studies to improve clinical outcomes and cost-effectiveness after treatment

RWHC: Does the Lung Association’s LUNG FORCE research innovation project address NSCLC?

SR: This award will address all types of lung cancer. To better understand the impact of lung cancer in women, the American Lung Association has created a new research award to examine gender differences in lung cancer. Sharad Goyal, M.D., is the first-ever recipient of the LUNG FORCE Research Innovation Project: Lung Cancer in Women Award.

Dr. Goyal’s project focuses on ionized radiation exposure during common cardiology procedures and how that affects the risk of developing lung cancer for women. The project leverages two large population-based data sets that include both cancer and cardiac information. Through analyses of these data sets, Dr. Goyal will evaluate the factors influencing the relative risk of developing lung cancer in a diverse group of people after this type of radiation exposure. This has not been previously studied, and it will take two to three years to complete the analysis.