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After seeing promising effects of CAR T cell therapies in patients with relapsed/refractory leukemia and lymphoma, researchers began to explore their potential in relapsed/refractory multiple myeloma. First, researchers needed to find a target for the T cells —one that was detected in multiple myeloma cells. Scientists found a target in B-cell maturation antigen (BCMA), which is also found in healthy plasma cells, and created a new family of CAR T cell therapies. Now the question is whether these investigational medicines will help prolong survival in patients with relapsed/refractory multiple myeloma.

Dr. Nina Shah, associate professor in the Department of Medicine at University of California San Francisco, discusses how CAR T cell therapy may further transform the treatment of relapsed/refractory multiple myeloma and how researchers are looking to optimize these treatments for this still incurable blood cancer.

DR. NINA SHAH

DR. NINA SHAH FROM THE UNIVERSITY OF CALIFORNIA SAN FRANCISCO BELIEVES THAT CAR T CELL THERAPY HAS THE POTENTIAL TO IMPROVE OUTCOMES FOR SOME PATIENTS WITH RELAPSED/REFRACTORY MULTIPLE MYELOMA.

What may CAR T cell therapies offer patients with relapsed/refractory multiple myeloma?

“The hope is that CAR T cell therapy may be a way for some patients with relapsed/refractory multiple myeloma to have a chance for a long-lasting treatment response. Clinical trials are still in the early stages.

Currently, multiple myeloma is treated with several repeated cycles of different therapies. Patients would most likely still go through these treatment regimens before receiving CAR T in a later line of therapy, but it would be one of the few single agent treatments these patients would receive.”

Will CAR T cell therapy be as effective in multiple myeloma treatment as it has been in other blood cancers?

“It remains to be seen. Every tumor has different properties. So it will depend on many factors, including whether targeting BCMA kills the cells that drive the cancer and whether the CAR T cells themselves disappear or stick around.

As we continue to see follow up research, we’ll look at the longevity of these CAR T cells as well as the duration of the response. Will finding these CAR T cells in patients long after treatment correlate with efficacy and survival? If so, we would try to use that information to create T cells that last longer in patients.”

What is the toxicity profile of CAR T in multiple myeloma?

“In clinical trials, patients with multiple myeloma have tolerated CAR T cell therapy as anticipated, but further investigation is needed. We can’t compare across disease states as patients themselves may experience the treatments differently in some ways. While it is uncertain why, what we do know is that as toxicities and disease states are better understood, along with further optimization of dosing, we may be in a better position to manage these responses. We have also learned from our colleagues who have been using CAR T cell therapy for longer in cancers such as in leukemia and lymphoma.”

CAR T cell therapy research offers hope for patients with relapsed multiple myeloma who have already been through several lines of treatment.

How would you characterize the early results in multiple myeloma?

“CAR T cell therapy research offers hope for patients with relapsed multiple myeloma who have already been through several lines of treatment. But we shouldn’t be overzealous.

In general, we have to work harder to understand why not all patients respond and which patients are most likely to receive a benefit from CAR T cell therapy. If we can predict that and learn why others don’t benefit and why relapses occur, then maybe we can improve how we engineer CAR T cells.”

Could CAR T cell therapy be combined with other treatments for multiple myeloma?

“There may be the potential for combinations worthy of investigation as we look toward the future with therapies such as immunomodulatory regimens and checkpoint inhibitors. Moreover, further exploration of CAR T cell therapies given simultaneously with other agents may show the potential to boost their activity. In any case, such combinations would require additional studies and careful attention should always be given toward the potential toxicities that may occur in combination strategies due to T cell overreaction.”

What other ways could researchers optimize CAR T cell therapy?

“We see lots of creativity in how we design these therapies. Some researchers are trying to grow CAR T cells under conditions that would allow them to last longer once they are given back to the patients, and others are trying to add an on/off switch to CAR T cells, so they only activate when they encounter signs of a tumor.

Meanwhile, we’re looking at when we use these therapies. Right now, CAR T cell therapies are being studied in patients who have tried and relapsed on multiple treatments. Using it sooner might be useful for patients with high-risk disease who don’t have very durable responses to traditional treatments, but this will require additional research. So I’m looking forward to seeing the results of clinical trials as we learn more about CAR T cell therapies in the multiple myeloma field.”

To learn more about the ongoing research into CAR T cell therapies, read “CAR T Cell Research Continues to Advance.”

While most people living with follicular lymphoma—the most common subtype of slow-growing indolent non-Hodgkin’s lymphoma—respond to treatment, studies conclude that approximately 20 percent of patients relapse within two years of receiving first-line therapy. One area of research to understand this is the tumor microenvironment, which researchers have recognized as a significant driver of this blood cancer.

At the University of Texas MD Anderson Cancer Center in Houston, Dr. Loretta J. Nastoupil leads the Lymphoma Phase I Drug Development team, which is exploring the role of the tumor microenvironment in lymphoma. In this Q&A, she discusses how targeting the microenvironment may change how we treat follicular lymphoma and why treatment combinations may be helping to eliminate these tumor cells.

What is the tumor microenvironment?

DR. LORETTA J. NASTOUPIL

DR. LORETTA J. NASTOUPIL FROM THE UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER IS EXPLORING THE ROLE OF THE TUMOR MICROENVIRONMENT IN FOLLICULAR LYMPHOMA.

“Recently, researchers have been paying increased attention to the tumor microenvironment—the cells, molecules and pathways that surround and support the growth of tumor cells.

When B-cells, a type of white blood cell, become cancerous in follicular lymphoma, they have migrated from the bone marrow, where they are produced, to the lymph node. The microenvironment within the lymph node is essential in maintaining this disease. Complex interactions between cells in the nodes keep the cancerous B-cells growing.”

Why is targeting the microenvironment in follicular lymphoma worth studying?

“We tend to consider follicular lymphoma as more of a chronic disease than a fatal one. Patients generally do well with treatment, but we can’t cure it. Current therapies can put most people in remission, when there are no signs of cancer, but it usually comes back. If we can understand the tumor biology better, we hope to develop treatments that lead to a more durable remission.

We know that the microenvironment plays a critical role in maintaining this disease, helping it to thrive and to resist treatment. Right now, characterizing the microenvironment is our best predictor of how well someone with follicular lymphoma is going to do. If we can figure out better ways to target whatever structures support the lymphoma, instead of helping to stop DNA replication like some current therapies do, we might progress how we treat patients with follicular lymphoma.”

Why can’t we just target the genetic drivers of follicular lymphoma?

“With solid tumors, immune cells infiltrate in small numbers. If you can target or block that mutation, you can stop or slow that tumor from growing. But in follicular lymphoma, it’s not the same.

About 90 percent of patients with follicular lymphoma have an abnormal rearrangement between parts of two chromosomes, called a translocation. But you can’t target that abnormality for treatment. Acquiring the translocation is just the first in a multi-step process that leads to follicular lymphoma. The other steps lead to more genetic alterations, which provide some survival advantage to that cancer cell, including masking them from the immune system’s search-and-destroy process.”


Do any current therapies target the microenvironment?

“People with follicular lymphoma are often treated with anti-CD20 antibodies that interact with effector cells of the immune microenvironment. Essentially, you’re putting a flag on the follicular cancer cells, which express the CD20 proteins on their surface, and hope the other key immune system players see those flags and eliminate the cancer cells. Significant developments in treatment over the last two decades have led to an improvement in the relative five-year survival rate for patients with follicular lymphoma, which increased from 70 percent in 1990 to 89 percent in 2010.

Therapies that target the microenvironment offer a promising approach that has the potential to improve outcomes in follicular lymphoma.”

How important is combination therapy in helping the immune system to eliminate follicular lymphoma cells?

“With follicular lymphoma, when you block one pathway, usually cancer cells find another way to escape the immune system.

Now, it’s critical to be more rational about the combinations we’re studying. We could do a better job selecting combinations if we understood the key players in the microenvironment and their roles in maintaining the lymphoma.”

We want 100 percent of patients with follicular lymphoma to be looking at an average life expectancy, and we want to achieve that with as little impact on their quality of life as possible.

How important is targeting the microenvironment to the future of treating patients with follicular lymphoma?

“With follicular lymphoma, many patients do well with the current standard of care treatment options. So why do we focus so much on the 20 percent that relapse early, within the first couple years? Why do we talk about it so much at our meetings and in our research? It’s because we want 100 percent of patients with follicular lymphoma to be looking at an average life expectancy, and we want to achieve that with as little impact on their quality of life as possible. Targeting the tumor microenvironment is the next step forward towards accomplishing this goal.”

To learn more about novel combination approaches for some patients with lymphoma, read “The Goal to Treat Lymphoma without Chemotherapy.”

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Over the past decade, researchers have begun to uncover the complex biology behind myelofibrosis. Nearly 5,000 people in the United States are estimated to be diagnosed with this blood cancer each year.

RUBEN MESA, M.D.

RUBEN MESA, M.D., FROM THE UT HEALTH SAN ANTONIO MD ANDERSON CANCER CENTER, BELIEVES ONGOING RESEARCH IN MYELOFIBROSIS OFFERS HOPE THAT THERE WILL BE MORE TREATMENT OPTIONS IN THE FUTURE.

“We do not yet fully understand the biology behind why myelofibrosis advances in patients,” explained Ruben Mesa, M.D., director of UT Health San Antonio MD Anderson Cancer Center. “Improving our understanding can help us advance treatment beyond the current approved and investigational therapies, which are still being examined to determine how they might slow progression of disease.”

What Is Myelofibrosis?

Myelofibrosis is a rare blood cancer that starts in the stem cells of the bone marrow, leading to the production of faulty blood cells that don’t mature or function properly. Eventually, the disorder results in scarring of the bone marrow, further stifling its ability to produce healthy blood cells.

While some patients may live years without symptoms, others see their disease progress rather quickly. Without enough healthy red blood cells, these patients can develop anemia, leading to fatigue, weakness and shortness of breath. Myelofibrosis can also cause an enlarged spleen and the disorder has a higher incidence in people over age 50.

In addition to monitoring patients closely for any signs of disease progression or other conditions, physicians face the challenge of tailoring treatment. “Myelofibrosis is a variable disease,” Mesa said. “A better understanding of the role of genetic mutations that cause the cancer and refining the prognostic scores could help doctors have a much more detailed assessment of prognosis and determine which treatment options are best for each individual patient.”

Understanding Options

While a stem cell transplant is potentially curative in some cases of myelofibrosis, doctors reserve it for patients fit enough to endure the procedure due to the risk of life-threatening complications. Eligibility depends on several factors, including prognosis, age, overall health and the availability of an appropriate donor.

“The average age of diagnosis for myelofibrosis is about 60 years old, which is toward the latter end of when transplantation is considered safe or effective,” Mesa said. “So it’s not an appropriate therapy for all patients.”

For many patients, the goal of treatment is focused on relieving symptoms, reducing an enlarged spleen and improving blood cell counts. Therapy options for those who are ineligible for a transplant include JAK inhibitors, chemotherapy, immunomodulators and corticosteroids. Not all of these agents target the signs and symptoms associated with myelofibrosis, and researchers are working to find additional options.

There are many reasons to be hopeful in myelofibrosis research – a better understanding of the disease, better understanding of its genetic drivers and better therapies.

Hope for the Future

As with many rare diseases, several unmet needs in myelofibrosis are attracting attention, according to Mesa. “Currently, there are very few targeted therapies approved for myelofibrosis, and we do not have effective therapies for patients with the most advanced forms of the disease.”

There is hope. At December’s American Society of Hematology (ASH) Annual Meeting, researchers presented data from more than 20 clinical trials in myelofibrosis. Treatment options being investigated include JAK inhibitors and combinations that incorporate other agents, like immunomodulatory therapies which adjust the body’s immune responses to disease. Researchers are also exploring treatment strategies that target multiple disease-signaling pathways, which are showing promise in early studies, according to Mesa.

These are positive developments for patients with a disease like myelofibrosis. More targeted therapies may one day allow doctors to tailor a patient’s treatment to their specific symptoms and unique genetic makeup of the disease.

“There are many reasons to be hopeful in myelofibrosis – a better understanding of the disease, better understanding of its genetic drivers and better therapies,” Mesa said.

To learn more about Celgene’s commitment to myelofibrosis and other rare diseases, read “Supporting Research to Find Cures for Rare Diseases.”

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What do space, the ocean and the desert have in common with health care? That is the question posed by Healthcare’s Great Expedition, a unique interactive experience created by Celgene and focused on innovation in medicine.

The display made its debut at the 2018 World of Technology and Science (WoTS) congress in Utrecht, Netherlands, and Celgene plans to take this Expedition experience on the road, including to the 11th World Conference of Science Journalists, July 1-5, 2019, in Lausanne, Switzerland.

At WoTS, thousands of visitors travelled through the booth navigating three sections named “Space,” “Ocean” and “Desert” to reflect environments that hold potential for human discovery and offer an interesting analogy to some of the issues faced in health care. In these exhibit areas, participants learned about the various stages of medical discovery and development, while hearing Celgene’s perspectives on the complexities of the health care environment. Visitors shared their feedback and interacted with features, including a short science-fiction film and several games.

“Celgene is proud to be part of this great health care expedition,” said Kevin Loth, Celgene Vice President, Corporate Affairs and Policy, Worldwide Markets. “We appreciate the opportunity we have to engage with members of the public about what we are doing as a company and industry. Through interactions like this, we can help people understand the critical roles pharmaceutical companies play in bringing new medicines to patients.”

Communication and Collaboration

Visitors began their tour with Space, the area of the booth focused on communication and collaboration. There, they viewed This is Axiom, a story of an astronaut lost in space and fighting for a chance to return home. Her story and the stories of those who help her along the way serve as a metaphor for the complex health care environment and the urgent need for collaboration among health care stakeholders when making decisions about patient care.

The booth also allowed stakeholders to contribute ideas regarding sustainability in health care. According to at least one attendee, rising medical costs are not the fault of any one individual stakeholder.

“It is too easy to point at other stakeholders; that will not lead us to a solution,” Arnt Wolter, strategic health care contractor from the Diakonessenhuis Hospital in Utrecht, said. “Every stakeholder can take responsibility for a part of the solution.”

Through interactions like this, we can help people understand the critical roles pharmaceutical companies play in bringing new medicines to patients.

Discovery and Exploration

The second area, Ocean, focused on the high-risk nature of medical discovery and the vast areas of exploration still open. An interactive game gave visitors the opportunity to develop an investigational medicine from its discovery; through clinical trials, regulatory approval and manufacturing; and finally launch. This exercise highlighted the many difficulties in real-world medical discovery and development. This part of the exhibit provided greater detail about the complex discovery process and the high rates of failure in pharmaceutical development.

At the end of Ocean, visitors were asked what they thought was the greatest challenge to discovering new treatment options. Answers included difficult science, challenging government policies, technological challenges, high costs and high risks. Most participants chose difficult science, followed by high costs and high risks.

Access to Medicines

Finally, visitors arrived at Desert, where access to medicines was the theme. An interactive pricing simulation game allowed participants to step into the role of a pharmaceutical company leader. In this simulation, they evaluated decisions on pricing and future investments. When asked at the end of this area to identify the primary driver of the cost of medicines, most visitors chose research and development.

Next Steps for Healthcare’s Great Expedition

The display enabled valuable conversations from diverse perspectives regarding the many facets of health care, including investments, research and development, value and innovation and the future. Celgene looks forward to gathering further perspectives at additional events with the Expedition in the coming year.

When Dr. John Marshall first started treating patients with pancreatic cancer nearly 30 years ago, the goal of treatment was to extend a patient’s life; the quality of the patient’s life was a secondary consideration. Despite some gains in survival for pancreatic cancer, the survival rate is still in the single digits; currently 8.5 percent of patients are alive five years after diagnosis. While research to improve treatment options continues, quality of life has become increasingly important.

“Patients want to be tough and compliant with their treatment because they want their cancer to go away,” said Marshall, Director, The Ruesch Center for the Cure of Gastrointestinal Cancers, at Lombardi Comprehensive Cancer Center, Georgetown University Medical Center. “But if their treatments are preventing them from enjoying life, they may need to reconsider their options.”

DR. JOHN MARSHALL

DR. JOHN MARSHALL, DIRECTOR OF THE RUESCH CENTER FOR THE CURE OF GI CANCERS, HAS SEEN QUALITY OF LIFE BECOME INCREASINGLY MORE IMPORTANT IN MAKING PANCREATIC CANCER TREATMENT DECISIONS.

The management of cancer is a continuum, and priorities can change between a focus mostly on treatment effectiveness to a greater emphasis on quality of life, according to Marshall. “Doctors aren’t good at documenting quality of life,” Marshall said. “We need to be better at focusing on the specific aspects that are most relevant to patients with pancreatic cancer.”

Doctors struggle for a couple of reasons. First, while survival can be objectively measured in months and years, quality of life is determined by subjective perceptions of physical, emotional, social and cognitive aspects of a patient’s life. Many doctors do not collect this sort of data because it is difficult to measure, especially in a busy medical practice.

Additionally, according to Dr. Marshall, each patient has a unique set of priorities. Some patients want to live longer, no matter what it takes; others may prioritize the quality of the remaining time they have.

Multiple factors affect quality of life, including symptoms and comorbidities associated with the disease. Sometimes it can be difficult to tell the difference, according to Dr. Michael Pishvaian, an assistant professor in the hematology/oncology division at MedStar Georgetown University Hospital and Lombardi Comprehensive Cancer Center.

“Pancreatic cancer is a miserable disease that causes a tremendous number of symptoms,”  Pishvaian said. Symptoms include loss of appetite, weight loss, back or belly pain, nausea, vomiting, diabetes and more.

Many patients expect pain or discomfort when being treated for cancer and sometimes suffer in silence. But when doctors know about their patients’ issues, they can often provide solutions. Doctors should be diligent about asking the right questions and encouraging their patients to respond honestly, reporting how they feel on both good and bad days throughout their treatment, according to Pishvaian.

Experts suggest there are a few ways patients and physicians can ensure that quality of life will be a consideration during treatment. First, communication is key to maintaining quality of life.

“As patients go through their diagnosis and treatment for pancreatic cancer, things continually change, making the need for information that much greater,” said Julie Fleshman, JD, MBA, president and chief executive officer of the Pancreatic Cancer Action Network. “Patients need to not only communicate their needs with the people who are supporting them, but also advocate for themselves by asking for the latest information on treatment options, clinical trials and support resources.”

Secondly, patients who feel that their doctors respect them and are treating them as “whole people” report a higher quality of life. Finally, doctors do not need to care for their patients alone; they can help their patients assemble a team that includes a nutritionist, a psychiatrist and others to provide comprehensive support for their patients.

Given the extent to which pancreatic cancer affects quality of life for patients, doctors need to be proactive in managing their disease, according to Marshall. “That might mean seeing them more often than you would see patients with other cancers. It might mean seeing them every other week or more, depending on how that patient is responding to treatment.”

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More people are being diagnosed with—and dying from—pancreatic cancer, due to an aging population and rising rates of risk factors such as obesity. As a result, costs for treating this disease are on the rise.

Incremental improvements in treatment have been made, but progress remains slow, so pancreatic cancer still carries a poor prognosis. Dr. Hani Babiker, a pancreatic cancer specialist at The University of Arizona Cancer Center, offers his thoughts on the meaning of value in pancreatic cancer care – for which survival rates are low – and how the value of care could be improved.

How do you define value in cancer care?

“I define value as pushing ourselves to deliver more comprehensive approaches to providing care for our patients. That means addressing all patient issues and concerns related to their cancer.

With my patients, I discuss not only treatment options and goals but also what foods, vitamins and supplements can help them manage their disease. I refer my patients to social workers who can help them deal with the stress that can accompany a cancer diagnosis. A palliative care physician in our clinic helps patients to control symptoms such as pain, nausea and vomiting. We focus on treating the whole patient. That’s how I seek to provide the best value.”

DR. HANI BABIKER

DR. HANI BABIKER, A PANCREATIC CANCER SPECIALIST AT THE UNIVERSITY OF ARIZONA CANCER CENTER, BELIEVES DOCTORS AND PATIENTS SHOULD FOCUS ON THE OVERALL VALUE THAT TREATMENTS PROVIDE.

Does the definition of value change for cancers with low survival rates and few treatment options, such as pancreatic cancer?

“Absolutely. It is difficult to compare the value of treatments across all cancer patients; the options and goals are not the same.

We should not deprive pancreatic cancer patients of the most effective treatment options for their particular disease because their prognosis is not as good as patients with other cancers. But when the quantity of life we can offer patients is short, quality of life becomes an increasingly important factor to the value we provide.

Most people with pancreatic cancer experience pain, and I’ve seen firsthand how chemotherapy has helped patients manage that pain.”

Why has progress in pancreatic cancer lagged behind other cancers?

“It is an inherently unique disease. The microenvironment surrounding pancreatic cancer tumors creates a barrier that is difficult for therapies to penetrate. It also suppresses the body’s immune cells that would typically hunt down and eliminate cancer cells. We still have a ways to go in understanding and treating this disease better.”

If we can identify pancreatic cancer earlier, by screening people who are at high risk because of their family or medical history or genetic predisposition, we can potentially treat more patients using the Whipple procedure. We may provide better outcomes and, therefore, may deliver better value, even though hospitalizations and surgeries are expensive.

So how can we continue to improve the value of pancreatic cancer care?

“Pancreatic cancer is tough to diagnose. We usually see patients who are in advanced stages, when the disease has spread beyond the pancreas. As a result, only 20 percent of patients diagnosed are eligible for a surgery known as the Whipple procedure, in which doctors remove the cancerous part of the pancreas. Surgery gives appropriate patients the best chance for a cure.

If we can identify pancreatic cancer earlier, by screening people who are at high risk because of their family or medical history or genetic predisposition, we can potentially treat more patients using the Whipple procedure. We may provide better outcomes and, therefore, may deliver better value, even though hospitalizations and surgeries are expensive.”

What role do innovative therapies play in reducing hospitalizations and improving outcomes?

“Surgery gives patients the best chance for a cure, but less than 20 percent of patients live at least five years after their operation. This statistic highlights the fact that most pancreatic cancers have spread and cannot be cured through surgery alone.

Doctors are now using chemotherapy before and after surgery to improve outcomes in pancreatic cancer. And we are seeing more therapies being developed that will continue to improve survival and, one day, reduce the need for costly hospitalizations and surgeries. In the future, the best way to add value for pancreatic cancer patients will be to invest in better screening and to continue funding research into more treatment options.”

Learn more about how screening in individuals at risk for pancreatic cancer can help to save lives: read “Family History Helped This Survivor Catch Pancreatic Cancer Early.”

Approximately 3.5 million women in the U.S. are living with breast cancer, including more than 154,000 with disease that has spread beyond the breast to other parts of the body, known as metastatic breast cancer (stage IV). The outlook for non-metastatic breast cancer patients has overall improved, with an average five-year survival rate reaching close to 100 percent for people with stage 0 or I breast cancer, and 93 percent for people with stage II breast cancer.

The prognosis for those women diagnosed with metastatic breast cancer is not as promising. But, as research continues, progress is emerging. The percentage of women surviving five years with metastatic breast cancer (aged 15-49) doubled from 18 to 36 percent between 1994 and 2012.

“Sometimes metastatic breast cancer can be considered much more of a chronic disease,” said Denise A. Yardley, M.D., a senior investigator at the Sarah Cannon Research Institute in Nashville, TN. “I’ve seen a positive impact on patients who continue relatively normal lives despite their disease and treatment.”

DENISE A. YARDLEY, M.D.

DENISE A. YARDLEY, M.D., FROM THE SARAH CANNON RESEARCH INSTITUTE BELIEVES WE ARE SEEING PROGRESS IN THE TREATMENT AND PROGNOSIS OF METASTATIC BREAST CANCER.

Treatment Advances Are Constantly Occuring

There are many forms of metastatic breast cancer. Patients’ tumors can be either positive or negative for growth receptors, signaling the presence or absence of the known drivers of the disease. And they may or may not have disease driven by HER2 (human epidermal growth factor receptor 2) receptors. That complexity and the cross signaling from the HER2 receptor to other growth factor receptors, as well as the multitude of treatments available to treat metastatic breast cancer are part of the reason it has been a particularly difficult disease to treat. But translational researchers are making significant strides to further understand tumor biology and the genomics behind breast cancer subtypes.

The end result is a more tailored treatment approach based on a patient’s specific tumor biology and other clinical factors. With an expansion in targeted therapies, there is greater value in having patients’ tumors thoroughly examined so treatments are better selected. “We’re continuing to try to improve our precision medicine and really tailor treatments to what’s going on in that specific patient’s tumor,” Yardley said.

Doctors also have a better understanding of how to use the growing array of treatments. While combination therapy is used in early stages of breast cancer, recent studies have provided additional evidence on how to  sequence treatment options for their patients. We now also focus  on balancing symptom control with quality of life and partnering with our patients to make appropriate treatment selections at any given time.”

There’s every reason to be optimistic for patients facing the diagnosis and challenges of metastatic breast cancer today.

Preventing Metastasis from the Start

About 30 percent of women with early stage breast cancer eventually develop metastatic disease. So in addition to improving the treatment of metastatic breast cancer, researchers are trying to continue to improve the cure rate and thus prevent breast cancer from becoming metastatic in the first place.

Metastatic Breast Cancer TreatmentMost women with early stage breast cancer will have surgery during the course of their treatment. Now, many are also candidates for  a variety of systemic therapies, either before or after surgery, to reduce the number of potentially microscopic cancer cells left behind and prevent the disease from coming back. Chemotherapy  is usually reserved for patients at higher risk for a recurrence or metastasis.

“We want to make sure we’re appropriately recommending specific therapies but sparing patients who have a lower risk of disease recurring and becoming metastatic,” Yardley said.

By administering these medications earlier, when the disease is still operable, researchers aim to increase the cure rate and prevent—or at least delay—recurrence and metastasis in breast cancer patients.

More Work to Be Done

Admittedly, much work remains to be done, according to Yardley. Over the last 60 years, breast cancer survival rates have tripled, but metastatic survival rates have a long way to go before they reach that level.

Clinical trials play an essential role in exploring new treatments and approaches in metastatic breast cancer, and these trials continue to become more targeted as researchers learn more about the disease subtypes. For instance, while immunotherapies have not proven effective in studies for metastatic breast cancer in general, trials investigating immunotherapy in specific breast cancer subtypes such as triple negative breast cancer have shown promising activity. Thus, targeted treatments in combination with chemotherapy continue to demonstrate great promise.

“The science has become astounding, allowing us to manipulate the biology of metastatic breast cancer through very tailored approaches,” Yardley added. “There’s every reason to be optimistic for patients facing the challenges of metastatic breast cancer today.”

To learn more about a patient’s experience with metastatic breast cancer, read “How This Metastatic Breast Cancer Survivor Told Her Family About Her Diagnosis.”

When treating a chronic disease such as psoriasis or psoriatic arthritis, time is of the essence: every day that a patient goes without an effective treatment is another day of suffering. Unfortunately, three-quarters of large employers offer their employees insurance plans that use step therapy policies, which can often delay patients from getting access to the medications prescribed by their doctors.

“You could be looking at a nine-month process before you get access to the doctor’s recommended treatment,” said Patrick Stone, vice president of government relations and advocacy at the National Psoriasis Foundation (NPF). “Insurance plans need to get patients access to medications their doctors determined were right for them sooner than that. They deserve better.”

Many states have stepped up to protect patients from step therapy procedures by enacting legislation that limits the use of step therapy, with Minnesota and New Mexico being two of the latest examples.

The Problems of Step Therapy

PATRICK STONE

PATRICK STONE FROM THE NATIONAL PSORIASIS FOUNDATION SAYS THAT THE ORGANIZATION IS NOT STOPPING UNTIL LEGISLATION PROTECTING PATIENTS FROM STEP THERAPY IS PASSED IN ALL 50 STATES AND ON THE FEDERAL LEVEL.

If a prescribed treatment isn’t on the insurer’s preferred medications list, the insurer may deny it until a patient tries and “fails” on one or more of the preferred options. This process, called step therapy, is commonly practiced among major private insurance plans.

Step therapy is based on a one-size-fits-all approach, assuming that patients respond similarly to treatments. But in reality, patients with chronic diseases such as psoriasis and psoriatic arthritis can have very different responses to the same medication.

Step therapy is not unusual in rheumatology and dermatology despite the fact that many of these chronic diseases are associated with serious comorbidities. Psoriasis and psoriatic arthritis patients can suffer from other ailments, making it even more important to address the disease effectively and promptly with appropriate therapies.

4 The Average Number of Treatments Psoriasis Patients Try

“Step therapy reform is a high priority for the psoriasis and psoriatic arthritis community,” Stone said. “If you’re not treating psoriatic arthritis in a timely and appropriate manner as determined by your doctor, it can certainly become a disabling disease.”

Reigning in Step Therapy

Recently passed step therapy reform legislation does not stop insurance carriers from enacting cost control measures. Instead, the laws are intended to protect patients by providing a timely exemption process to override step therapy procedure.

Over the past four years, the NPF has led a number of campaigns at the state and federal levels with other patient and provider groups across the country.

In 2018, New Mexico passed step therapy reform legislation. “As a result of step therapy legislation, people living with a psoriatic disease in New Mexico have better access to prescribed treatments,” Stone said. The total number of states that have enacted step therapy legislation is now up to 19.

The most effective spokespeople for step therapy legislation have been the patients, according to Stone. When legislation was being considered in Texas last year, a 16-year-old with psoriatic arthritis named Michael from San Antonio met with state legislators. In a room filled mostly with lobbyists, the Speaker of the House only wanted to hear from one person: Michael, who shared how step therapy delayed his treatment and the trouble that caused him. “Michael did a better job than any lobbyist could in articulating the issue,” Stone said.

As a result of step therapy legislation in Minnesota and New Mexico, the more than 190,000 Americans living with a psoriatic disease in those two states have better access to prescribed treatments.

Next Steps

With most state legislatures having already adjourned for the year, Stone and the NPF are already planning for 2019. “In the upcoming year, we plan to renew efforts in Florida, Georgia, Washington and Maine, while also exploring options in other states with no prior legislative attempts,” said Stone. “Meanwhile, states like Pennsylvania, Virginia and Oregon are considering folding step therapy regulations into larger bills aimed at protecting patients from insurance practices.”

“The momentum is behind us going into the 2019 legislative sessions,” Stone said. “We have a game plan in place already. We know what states we’ll be in, and we’re excited about the potential for large amounts of legislative victories during the next couple years regarding step therapy reform.”

Meanwhile, at the federal level, the administration recently announced that it would allow step therapy in Medicare Advantage plans for Part B medications, which are typically administered in a hospital or clinic setting. Step therapy is already allowed in Medicare Part D plans, which covers at-home prescription medications.

“Medicare Advantage holders are senior citizens and those who are permanently disabled,” Stone said. “That makes it even more difficult for them to understand how the mediation process works and how to appeal it. So we’ve still got plenty of work to do to protect them.”

To learn more about why the one-size-fits-all approach doesn’t work in psoriasis, read “Psoriasis Patients Deserve Their Prescribed Therapy Without Delay.”

More people are being diagnosed with Crohn’s disease and ulcerative colitis than ever before, but researchers aren’t exactly sure why. A variety of factors including genetics, weakened immune systems and the environment may be at play.

In this podcast produced for this year’s Crohn’s and Colitis Awareness Week, Cathy Ferrone, director of patient advocacy at Celgene, and Laura Wingate, senior vice president of Education, Support and Advocacy at the Crohn’s and Colitis Foundation, discuss the rise in worldwide incidence rates of inflammatory bowel disease and why research in this area remains so important.

 

 


CATHY FERRONE FROM CELGENE AND LAURA WINGATE FROM CROHN’S AND COLITIS FOUNDATION DISCUSS THE RISE IN INFLAMMATORY BOWEL DISEASE.

 

To learn more about the lifelong struggle of having an inflammatory bowel disease, read “What It’s Really Like to Live with Ulcerative Colitis.”

To explore the Crohn’s & Colitis Foundation’s resources available to patients, caregivers and health care professionals, visit their website at http://www.crohnscolitisfoundation.org/ or call 1-888-My-Gut-Pain.
 

In patients with myelodysplastic syndromes (MDS), a cancer in which the bone marrow does not make enough healthy blood cells, red blood cells may not mature and function properly. As a result, about 85 percent of patients with MDS develop serious anemia. Since roughly half of patients do not respond to current therapies aimed at increasing red blood cell production, many end up relying on frequent transfusions to treat the symptoms associated with anemia.

Sandra Kurtin, Ph.D., ANP-C, AOCN, University of Arizona Cancer Center, believes MDS will be in the spotlight at this year’s American Society of Hematology (ASH) annual meeting. In a Q&A, Kurtin discusses current research to uncover why red blood cells don’t mature properly in MDS and how targeting different stages of their maturation may lead to much-needed new therapeutic options.

Anemia Treatment MDS

What advances in the research of MDS will hematologists learn about at this year’s ASH meeting?

“The International Working Group for the Prognosis of MDS will be meeting to look at the molecular underpinnings of MDS and to identify groups of patients by risk. We’ll be looking at some molecular targets for potential new therapies in MDS.

During the meeting, I look forward to seeing data about some of the novel pathways implicated in the development of blood cancer in certain groups of patients. Researchers now believe that certain mutations within these pathways may help predict survival chances for some patients with MDS.”

Why has developing effective therapies for MDS been challenging?

“MDS is complicated. It’s been difficult to pinpoint a single pathway that overcomes the abnormalities inherent in MDS. For instance, the tumor cells’ surroundings, known as the microenvironment, may play a role. Current therapies target cancerous cells, but may not be targeting the microenvironment. Additionally, molecular abnormalities and abnormalities in the spliceosome are known to contribute to the pathobiology of MDS. Today, the only potential cure is a stem cell transplant, but many patients may not be eligible for one.”

Why is a stem cell transplant not an option for most patients?

“While some patients may not warrant immediate treatment, the only way to overcome the abnormalities of MDS is to replace the genetic profile through an allogeneic bone marrow transplant. But that is associated with several risks. Transplantation is typically considered for patients with higher-risk MDS.

Usually, patients have to be younger than a certain age and otherwise healthy. Some centers are now allowing transplants up to the age of 75. The median age of diagnosis for MDS patients is 76.”

What other treatment strategies are being explored for MDS?

“Treatment for lower-risk MDS is aimed at improving cytopenias, including anemia and reducing the need for transfusions. Meanwhile, the primary treatment goal for higher-risk MDS is to extend survival.

Doctors use treatments such as erythropoietin to stimulate the red blood cell production in some patients with lower-risk MDS. An unmet medical need remains in patients that do not respond or do not maintain response to supportive treatment.”

Now we’re coming to realize that transcription factors and other molecular attributes also regulate the production of these cells.

How are researchers working to understand the biological mechanisms that lead to MDS?

“Pathologists are still exploring the ‘normal’ process by which blood stem cells commit and differentiate to form all the different types of blood cells within the bone marrow. We used to think that the process was primarily driven by growth factors. Now we’re coming to realize that transcription factors and other molecular attributes also regulate the production of these cells.”

What are the most important unanswered questions in MDS research?

“We’re trying to understand why MDS is such a heterogeneous disease. Why do some patients have a slow-growing disease while others have a more aggressive malignancy? What are the mutations that make it different? We’re trying to identify targets for drugs within those mutations.

Among the therapies highlighted at ASH will be those aimed at novel targets. Some new therapies are combination regimens using different classes of drugs, and some are novel single-agent therapies that may be combined with other treatments in the future. Therapies approved for acute myeloid leukemia, which evolves from MDS, are also being explored for MDS. There’s finally a lot of exciting research after all these years.”

To learn more about these rare cancers are negatively impact the daily lives of patients, read “New Survey Reveals Myelodysplastic Syndromes Leave Patients Feeling Fatigued.”

Beta-thalassemia is a blood disorder, with more than 60,000 infants born worldwide with the disease each year. However, unless you live in Asia, India, the Middle East or the Mediterranean where beta-thalassemia is most prevalent, you may never have heard of this inherited blood disorder that disrupts the body’s ability to make hemoglobin.

But that may be changing as doctors in Europe and North America are increasingly encountering patients with beta-thalassemia. “We’re only now starting to deal with it, and there is a lot of work that needs to be done,” explained Dr. Maria Domenica Cappellini, a hematologist at the University of Milan in Italy, whose research focuses on the disease.

As hematologists gather for this year’s American Society of Hematology Annual Meeting in San Diego, Cappellini and other experts are sounding the alarm about the expanding prevalence of beta-thalassemia so that physicians and health systems everywhere can better prepare for an increasing number of patients with this genetic disorder.
 

Spreading Faster Than Ever

Beta-thalassemia is an inherited disease caused by mutations in a gene required for making a component of hemoglobin – a protein that carries oxygen in the blood. Those mutations either prevent or reduce the production of hemoglobin, which can cause a shortage of mature red blood cells and lead to anemia.

Dr. Maria Domenica Cappellini

DR. MARIA DOMENICA CAPPELLINI, A HEMATOLOGIST AT THE UNIVERSITY OF MILAN IN ITALY, BELIEVES MORE COUNTRIES NEED TO PREPARE FOR THE EXPANDING PREVALENCE OF BETA-THALASSEMIA.

Often times, children inherit the gene mutation from parents who are carriers but do not show any symptoms of the disease. In this scenario, the child has a 25 percent chance of developing beta-thalassemia and a 50 percent chance of being an asymptomatic carrier like their parents.

The mutations that cause beta-thalassemia are more common in Asia, India, the Middle East and the Mediterranean where they are found in up to 20 percent of the population. But the increase in modern migration means that cases are now cropping up more often in other regions.

Southern Mediterranean countries recognize the rise in patients with beta-thalassemia and have increased resources to meet the growing demand appropriately. While in Northern and Western Europe, health professionals and policymakers acknowledge this trend, they lack reliable data on the frequency and patterns of the disease just yet. Without data, it’s difficult to make the case for investing in programs to address the issue, which means patients struggle to find the right doctors.
 

Bracing for Beta-thalassemia

Many patients with beta-thalassemia require life-long regular blood transfusions and medication to reduce the levels of iron in their body. “These patients cannot produce enough mature red blood cells to transport oxygen throughout their bodies,” Cappellini said. “So transfusions are necessary for their survival.”

Understandably, beta-thalassemia treatment requires significant expertise and resources, including safe blood donations. Many countries are preparing their health care systems by improving resources for blood transfusions. “As migration throughout the world continues to change, health care systems will need to evolve rapidly to meet the needs of a more diverse population,” Cappellini said.

“I get daily emails from colleagues in other parts of Europe asking how to treat patients with beta-thalassemia,” Cappellini said. “We’re trying to make this information as accessible and comprehensive as possible.”

Beta-thalassemia is something that hematologists in North America and Europe need to get up to speed on and quickly.

Longer Term Solutions

We’re still a long way from being able to correct or erase the mutation that causes beta-thalassemia. Currently, the only available cure is a stem cell transplant, but many patients may not be eligible. Fewer than 10 percent of patients eligible for a stem cell transplant actually receive one, often due to high costs or a lack of a donor.

Meanwhile, researchers are constantly looking at other ways to improve treatment options and patients’ quality of life, including therapies that could reduce the need for red blood cell transfusions. Transfusion frequencies vary based on patient needs, sometimes requiring them to spend hours receiving treatment every couple of weeks.

Another long-term solution is prevention through carrier screening and education, and some countries have found success with this approach. For instance, Cyprus, Italy and Greece enacted mandatory premarital screening and genetic counselling in the 1970s and have subsequently achieved almost a 100 percent reduction in at-risk births.

Without mandatory screenings, health care systems rely on education to reduce the incidence of beta-thalassemia. “If we don’t educate more populations about how this disease is transmitted, we will continue to see an increasing number of carriers and an expanding prevalence of beta-thalassemia around the world,” said Cappellini. “Beta-thalassemia is something that hematologists in North America and Europe need to get up to speed on, and quickly.”

To learn more about beta-thalassemia, read “The Need for Safe Blood Donations for Beta-thalassemia Patients.”

New research has helped scientists better understand the more than 60 different molecular subtypes of non-Hodgkin’s lymphoma (NHL), revealing just how diverse and complex this group of blood cancers is. And with better understanding comes the potential for different treatment pathways, which clinicians anticipate seeing at this year’s American Society of Hematology (ASH) Annual Meeting.

“From a biological standpoint, molecular lymphoma subtypes are very different from one another,” said Dr. Georg Lenz, Director of the Department of Hematology, Oncology and Pneumology at the University Hospital in Muenster, Germany. “These complexities potentially warrant different treatment approaches.”

Expanding our knowledge of these unique molecular drivers may open the door for targeted treatment approaches. Dr. Lenz expects we will learn more about these approaches during the ASH congress.

Dr. Georg Lenz

DR. GEORG LENZ FROM THE UNIVERSITY HOSPITAL IN MUENSTER, GERMANY BELIEVES THAT UNDERSTANDING THE SUBTYPES OF NON-HODGKIN’S LYMPHOMA IS HELPING RESEARCHERS TO IMPROVE TREATMENT.

A Tale of Two Characterizations

NHL subtypes can be divided into two broad categories based on how quickly the disease progresses: they can either be indolent or aggressive. Indolent lymphomas, such as follicular lymphoma (FL), are usually slow-growing and represent up to 40 percent of all NHL cases. Aggressive lymphomas grow at a much faster rate, such as one of the most frequent subtypes, diffuse large B-cell lymphoma (DLBCL).
 

Fast Progress in Slow-Moving NHL

The treatment landscape for indolent NHL, which includes follicular and marginal zone lymphomas, has been evolving quickly. While many patients still receive chemotherapy, targeted therapies have been making headway. At this year’s ASH Annual Meeting, Lenz expects to see more data from trials of these investigational therapies in combination with—or instead of—chemotherapy, opening the possibility for chemotherapy-free treatment.

“While we see increased interest in chemotherapy-free treatments, these approaches still have serious side effects,” Lenz said. “We still have a lot of work to do in managing these toxicities.”

Fortunately, most patients with indolent NHL respond well to treatment, but about 20 percent of FL patients relapse rather early after initiation of therapy. Since each patient responds to treatment differently, clinicians need to adapt treatment approaches accordingly, particularly for patients who do not initially respond well.

“This could be the next step for patients,” Lenz said. “It’ll be a challenge for years to come, but hopefully, we’ll see some progress in predicting treatment responses at this year’s ASH.”

It has been challenging to translate the biological knowledge of NHL into clinical practice.

New Avenues in Aggressive NHL Subtypes

Meanwhile, researchers are still grappling with the genetic complexity of aggressive lymphomas. Even previously identified subtypes are evolving into a collection of unique subtypes. For instance, DLBCL can be further broken down into additional subtypes, including activated B cell-like (ABC) DLBCL and germinal center B cell-like (GCB) DLBCL.  Different treatment approaches for the two are being tested in clinical trials.

“DLBCL encompasses unique molecular subtypes that behave differently, both biologically and clinically,” Lenz said. “It has been challenging to translate the biological knowledge of NHL into clinical practice.”

One investigational option being studied in patients with relapsed or refractory DLBCL and other aggressive NHL subtypes is chimeric antigen receptor (CAR) T cell therapy.

“Results on CAR T cell therapy are encouraging. However, we need longer follow-up to correctly assess its efficacy,” Lenz said.

To learn how patients and doctors are partnering to make treatment decisions in lymphoma, read “Developing Confidence in Lymphoma Treatment Decisions.”

Over the past few decades, scientists have come to understand that the loss of brain tissue—categorized into grey and white matter—in people with multiple sclerosis (MS) is linked with disease worsening. But research is revealing that grey matter loss, in particular, may be closely associated with disability and cognitive impairment.

“Grey matter loss is one of the best predictors of disease progression in people with MS,” said Dr. John DeLuca, senior vice president for research and training at the Kessler Foundation. “Finally, we’re seeing data that may help us better understand the mechanisms that drive this disease.”

DeLuca is calling attention to the value of assessing grey matter and cognitive impairment in MS and what implications these findings may have in understanding the disease.

JOHN DELUCA, Ph.D.

JOHN DELUCA, Ph.D., FROM THE KESSLER FOUNDATION BELIEVES GREY MATTER LOSS IN THE THALAMUS CAN PREDICT COGNITIVE DECLINE IN PEOPLE WITH MS.

Grey Matter Loss Associated With MS Disability Progression

While researchers have known that grey matter loss is associated with long-term disability, a study published earlier this year has provided a more detailed picture of that connection.

The researchers looked at how specific patterns of grey matter loss were associated with disability progression in patients with MS using a standard MS disability scale (EDSS).

The researchers found the strongest relationship between disability progression and the loss of brain tissue in the thalamus, the largest area of deep grey matter, which transmits sensory information to other areas of the brain. In a post-hoc analysis of MRIs from 1,214 MS patients and 203 healthy controls, baseline thalamic volume loss increased risk of disability progression by 37 percent in relapsing MS.

“The research continues to provide more evidence that loss of grey matter is associated with increased disability,” he said. “And grey matter loss is seen most intensely in the thalamus of patients with MS.”

Probability of Disability Progression Due to Volume

Cognitive Impairment Shouldn’t Take a Backseat to Disability

The new study makes the case for grey matter loss as a predictor of disability progression in MS, but it did not look at cognitive function—which can also worsen as MS progresses. DeLuca wasn’t surprised. “Cognitive impairment just doesn’t get the same attention as disability in MS studies,” he said. “But it really should.”

Many patients agree. In a recent survey conducted by the Multiple Sclerosis Association of America and sponsored by Celgene, 27 percent of respondents said maintaining cognitive function was the most important consideration in the management of their MS. Only the prevention of disability progression was reported by more respondents (45 percent).

While cognitive impairment has been recognized in MS for more than a century, a test to measure cognitive function wasn’t developed until 2001. Researchers know that up to 65 percent of people with MS experience some level of cognitive impairment, and the National MS Society recently announced new recommendations for managing cognitive care for people with MS.

So could the same patterns of grey matter loss associated with disability also be related to cognitive impairment? It’s quite possible, according to DeLuca. “Thalamic damage already has an established relationship with cognitive decline,” he pointed out. “And grey matter loss is seen most intensely in the thalamus of patients with MS. So it’s probable.”

The more specific we can be regarding the role of grey matter loss, the better we can care for patients with MS.

Measuring Grey Matter Loss in Practice

So far, doctors don’t routinely use grey matter loss as a predictor of disability or cognitive impairment when caring for patients, but DeLuca believes that maybe they should consider it. “Grey matter loss could be a trigger for clinicians to watch their patients over time and monitor for potentially related problems,” he explained.

Given current evidence, DeLuca would like to see more trials differentiating grey matter loss from brain volume loss in general. But the main goal, of course, is to identify new ways to look at cognitive impairment. DeLuca is hopeful.

“I think we’ll start to see further research that show the correlation between grey matter and cognitive impairment as well as physical disability,” DeLuca said. “The more specific we can be regarding the role of grey matter loss, the better we can care for patients with MS.”

To learn more about how brain volume loss can affect patients with MS decades later, read “How Multiple Sclerosis Affects the Brain and CNS.”

Over the past year, doctors have seen promising results from studies investigating new treatment approaches using chemotherapy for patients with pancreatic cancer, a disease that remains among the deadliest of cancers. Yet 38 percent of pancreatic cancer patients received no treatment at all within one year of diagnosis, according to study findings.

“Those results are not surprising as therapy for pancreatic cancer is rarely curative,” said Gabriela Chiorean, MD, a gastrointestinal oncologist and researcher at the University of Washington. “Most pancreatic cancers are diagnosed at a stage where the goal is to prolong survival—not to cure the disease. Some physicians and patients may be less willing to choose treatment because of that.”

Chiorean believes that more patients with pancreatic cancer could benefit from and should be offered treatment for their disease. During this year’s Pancreatic Cancer Awareness Month, Chiorean is raising awareness of both this issue and the progress that’s been made in pancreatic cancer treatment.

The Harsh Reality of Pancreatic Cancer

Gabriela Chiorean, MD

GABRIELA CHIOREAN, MD, FROM THE UNIVERSITY OF WASHINGTON BELIEVES THAT DOCTORS SHOULD NOT GIVE UP ON PATIENTS WITH PANCREATIC CANCER BY NOT DISCUSSING THEIR TREATMENT OPTIONS.

While the statistics may seem dismal, they are improving. From 1993 to 2013, while the median overall survival for metastatic pancreatic cancer patients remained steady, more patients achieved long-term survival—defined as a year or longer. According to Chiorean, these survivors were diagnosed at a younger age and may have been more likely to receive treatment.

Chiorean believes that survival rates would further improve if more patients were offered treatment. But as research point out—many patients do not receive treatment. Chiorean’s personal experience backs the study findings; she frequently sees patients who were not offered treatment in other centers and are looking for a second opinion.

Another reason patients may not receive treatment is that pancreatic cancer is difficult to diagnose. As a result, 80 percent of patients are diagnosed at an advanced stage when curative treatments are not an option. By the time the cancer is detected, oncologists may be hesitant to offer treatment because they fear their patients are too fatigued or ill, and unable to tolerate treatment regimens, according to Chiorean. It may be the physician’s intent to relieve stress on both the patients and their caregivers.

Even more challenging, pancreatic cancers can only be removed less than 15 percent of the time. “If you can’t take it out of the body, eventually it will start spreading unless it has already spread,” Chiorean said.

Managing treatment toxicities and a patient’s quality of life can also make a difference, according to Chiorean. She tries to prevent side effects by adjusting treatment dosing as needed and continuously asks patients how they are feeling before each treatment.

“That’s where the art of medicine comes into play,” she said. “We’re not treating everyone the same.”

Early Screening for Pancreatic Cancer

Pancreatic cancer can present with broad gastrointestinal symptoms that can be diagnosed as peptic ulcer disease or irritable bowel syndrome, and sometimes it presents with new diabetes. “A clinician might treat patients for indigestion for a year, and then ultimately diagnose them with late-stage pancreatic cancer,” Chiorean said. “If a patient is losing weight and has new onset diabetes, they should be screened with an ultrasound or CT scan for pancreatic cancer.”

Researchers are working on ways to catch pancreatic cancer earlier. Imaging techniques to detect premalignant cystic neoplasms, and other benign conditions that may be precancerous, are being explored, as are biopsies followed by regular ultrasound screening for high-risk patients, including those with a family history of pancreatic cancer.

We’re learning more about the disease and can offer treatment options that allow patients to feel comfortable for as long as possible.

Pancreatic Cancer Care Is an Uphill Battle

Improving care for pancreatic cancer remains a struggle. The pancreas has limited blood supply, making it difficult for medications to penetrate it. But new treatment strategies are making in-roads. New therapy combinations are being used before and after surgery for patients with pancreatic cancer, and new approaches are being explored in clinical trials to make chemotherapy less intensive for some patients. Chiorean recommends clinical trials of pancreatic cancer treatments for eligible patients.

“Despite the statistics, there’s definitely hope in the future of pancreatic cancer treatment,” said Chiorean. “We’re learning more about the disease and can offer treatment options that allow patients to feel comfortable for as long as possible.”

To learn more about the patient experience with pancreatic cancer, read “Facing Each Day with Pancreatic Cancer, Hand-in-Hand.”

Celgene is a company built on a foundation of bold innovation to address areas of significant need for patients with cancer and other debilitating diseases. This unyielding drive has led the company to developments that have transformed the care of diseases like multiple myeloma and pancreatic cancer and has provided important new options for patients with psoriatic diseases.

As the company has grown, so has its commitment to innovation. In fact, the company has increased its investment in research and development by more than 36 percent per year on average since 2006, when its lead therapy for multiple hematologic diseases was approved. During this time, the company also built what would be a defining part of its research efforts, the distributed research model.

By coupling Celgene’s internal research and traditional business development efforts with a program focused on identifying and nurturing disruptive science outside the company, Celgene was able to option promising candidates for rare and debilitating diseases. The program currently features more than 50 collaborations with 21 unique compounds in clinical development.

Robert Hershberg

ROBERT HERSHBERG, M.D., PH.D., CELGENE’S EXECUTIVE VICE PRESIDENT AND HEAD OF BUSINESS DEVELOPMENT AND GLOBAL ALLIANCES.

“We are focused on great science, first and foremost,” said Robert Hershberg, M.D., Ph.D., Celgene’s Executive Vice President and Head of Business Development and Global Alliances. “We seek in our partners what we seek in our own scientific and development teams. Notably, we seek passion, a commitment to excellence, and a strong desire to bring novel treatments to improve patients’ lives.”

“The distributed research model—in place at Celgene for almost a decade—recognizes and embraces the fact that contributions to progress in human health can be readily seen in academic institutions, private foundations, small and large biotechnology companies, and in the pharmaceutical sector,” he continued. “Importantly, no single institution, company or entity can do this alone and there is an increasing interdependence on a range of efforts to bring this promise to patients. We seek partners in our core areas of scientific interest (Protein Homeostasis, Immuno-Oncology, Epigenetics, Immunology/Inflammation, and Neuroscience) and clinical interest (Hematology, Oncology, Inflammatory diseases) and hope to identify programs in adjacent, novel areas as well.”

One of the keys to the growth of the programs was to identify and incentivize this innovation early on.

“As products increase in both their complexity and their precision, intense support early in the development process is critical. The establishment of relevant pre-clinical models and deep interrogation of novel pathways provide the appropriate roadmap for moving early science forward towards the clinic,” continued Hershberg. “In early studies of these novel therapies in patients, an intense focus on ‘translational’ medicine—developing tools to gather as much data as possible in early clinical trials. These early efforts both improve the likelihood of clinical success and can dramatically reduce the timelines required to bring novel therapies to patients that desperately need them.”

Along with groundbreaking research, these partnerships also provide the opportunity to learn critical lessons in discovery research through new platforms for Celgene, and a chance to advance programs alongside an industry veteran for the partner companies.

An important example of this was the partnership with Agios Pharmaceuticals, Inc. The long-term research partnership provided the opportunity to evaluate multiple programs, to adjust the terms to fit each company, and most importantly, to deliver the first FDA-approved product to come out of Celgene’s distributed research model.

“Close collaboration from the very start has been key to the success of our relationship with Celgene” said David Schenkein, M.D., Chief Executive Officer of Agios. “By focusing on each other’s strengths, we were able to pursue the science and develop a new medicine, less than four years from the first in human study to approval in a blood cancer that had not seen a new medicine in nearly 40 years.”

Hershberg has a unique perspective on partnering with Celgene as he has now served on both sides of the model, in his current role as head of the company’s business development efforts, and as a partner during his time as CEO of VentiRx.

“Celgene has been on the leading edge of business development to engage external partners to extend our Research and Development footprint. Importantly, many of these partnerships allow and facilitate a partner’s ability to do what they do best—and to advance programs into early and mid-stage clinical development. The business structures are flexible and ideally designed to meet both Celgene’s and the partner’s unique needs,” said Hershberg.

Another long-term partner was recently in the news as well. Acceleron Pharma, alongside Celgene, announced top-line results from multiple pivotal studies of a collaboration that had been ongoing for more than ten years.

“Our collaboration is focused on developing and delivering transformational therapies to patients in areas of disease with few options,” said Habib Dable, President and Chief Executive Officer of Acceleron. “Because of our mutual commitment to this focus, we have advanced to the point where we are preparing for potential approval. Our shared experience has kept the combined team energized and on mission throughout.”

The close collaborations that make up Celgene’s distributed research model have bolstered a leading biopharmaceutical pipeline for Celgene, provided vital, early support for its partners’ promising programs and even delivered a new therapy to patients in need.

“The opportunity to partner early and leverage our research platform to identify potentially disruptive therapies has led to the opportunity to expand our collaboration twice already to encompass new targets and new areas of disease,” said Werner Lanthaler, Chief Executive Officer of Evotec AG. “We believe our work together across these multiple platforms have the potential to make meaningful impacts on patients’ lives and we continue to partner closely to make this happen.”

Celgene will continue to look for transformational science both within and outside of its walls as it seeks to deliver on its mission to improve the lives of patients worldwide. Successful partnerships, like those it has fostered already, will be essential in that effort.

In August 2005, Amelia List celebrated her first birthday, and her mother Julie breathed a sigh of relief. She had feared Amelia would develop severe food allergies, as her five-year-old sister Autumn had, by the time she turned one. But it was so far, so good.

Unfortunately, everything went downhill from there, Julie recalls. A month later, the entire family got the stomach flu. Everyone recovered well except for Amelia, whose vomiting and diarrhea continued. Six weeks later, she had lost 20 percent of her body weight. Julie and her husband took their daughter to a gastroenterologist, who diagnosed Amelia with eosinophilic esophagitis.

Having already joined several food allergy forums online, Julie knew what that meant. Eosinophilic esophagitis was not your typical allergic reaction to food. She turned to her husband. “We’re going to be one of those people whose kid can only eat one or two foods,” she told him.

More than 150,000 children and adults in the United States live with eosinophilic esophagitis, a relatively new disease that was only first recognized in the 1990s. There are currently no FDA-approved pharmaceuticals to treat EoE. Symptoms may be managed with elimination diets and other methods. Julie is sharing their story to raise awareness of the disease, with the hope that more can be done to improve their daily struggle.

Identifying Triggers

Julie knew eosinophilic esophagitis was not a typical food allergy, but she was stunned when their local gastroenterologist told them that he could only diagnose but not treat the condition. In fact, at that time, there was no doctor near their home in South Carolina who treated this rare disease. For the next four years, the family traveled eight hours to Cincinnati to see a specialist whenever necessary.

The doctor explained that proteins in the foods Amelia was eating were triggering a type of white blood cell called eosinophils to inflame her esophagus. This inflammation led to her vomiting, difficulty swallowing and recurring stomach pain.

Amelia underwent food trials to identify her food triggers, eating one or more foods at a time for two months to see whether they made her sick. If they didn’t, she’d get an endoscopy to check her upper digestive tract for inflammation. Her doctor would put her under anesthesia and insert a flexible tube with a camera into Amelia’s upper digestive tract.

Her doctor also took six to ten biopsies throughout the esophagus to determine if eosinophils were present. “The biopsies can reveal if eosinophils are present and causing damage that is not visible to the eye,” Julie said. “We never knew if food was safe until the biopsy results were returned.”

She went through this process with a dozen foods. To get her required nutrition, Amelia was given an amino-acid based formula through a gastric feeding tube. “The formula had no proteins that would trigger allergies, but it tasted terrible,” Julie said.

We never tell them they can’t do anything.
We’ve tube-fed Amelia while hiking.

Overcoming Setbacks

In 2007, the List family received more devastating news when their middle child, Abby, was diagnosed with eosinophilic esophagitis at the age of six. Studies have shown that siblings of someone with eosinophilic esophagitis are at increased risk for the disease, suggesting a role for genetic factors. Environmental factors are also thought to have a role.

Then in 2016, Amelia had a severe, life-threatening allergic reaction to white rice, her first allergic reaction unrelated to her eosinophilic esophagitis. Her immune system reacted, and Amelia soon found she could no longer eat any of the foods she previously tolerated.

Amelia resumed consuming formula through the gastric tube but then started reacting to that, too. To help tolerate the formula, she takes medication twice a day. She once again began food-testing with endoscopies to validate new safe foods. Since her initial diagnosis, Amelia has had 32 endoscopies and counting.

Today, she continues to consume formula, but it is not her sole source of nutrition. She can eat seven foods: apples, sweet potatoes, kidney beans, soy, millet flour, turkey and black olives. “For sure, you get sick of them, but I just have to keep eating them,” Amelia said. “I really don’t have any other choice.”

Growing Up Quickly

Amelia has been administering her own tube-feedings since she was a kid. Now 14, she has it down to a science. She eats this way three times a day, and she takes her equipment with her everywhere, plus the formula and water to mix.

Amelia reminds herself not to let eosinophilic esophagitis hold her back from living the life she’s dreamed of living. “It’s part of you, but it doesn’t control you. It’s not who you are,” Amelia said.

Going out—whether to school, on a field trip, to a friend’s house or on vacation—requires planning. If she goes to a party or sleepover, she brings a can of olives or a sweet potato in case she gets hungry. Sometimes, of course, she chooses to forgo events if she decides that they’re not worth the effort.

“We never tell them they can’t do anything,” Julie said. “We’ve tube-fed Amelia while hiking. But they definitely analyze situations ahead of time, which most kids don’t have to think about. These kids are very responsible and have to grow up quickly.”

To learn more about how Celgene is committed to supporting research for rare diseases, read Supporting Research to Find Cures for Rare Diseases.

While understanding the impact of myelodysplastic syndromes (MDS) on the lives of people diagnosed with these blood cancers can help inform their care, assessments of quality of life in MDS have been, for the most part, lacking.

But a recent survey commissioned by the MDS Foundation, Inc. is helping to fill that gap, to shed some much-needed light on the experience of patients and their caregivers. According to the results, many people with MDS surveyed said that fatigue had a significant impact on their daily lives.

“The number one complaint that we hear, by far, from patients with MDS is that they don’t have the energy to do the things that make them feel like they’re living,” said Tracey Iraca, executive director of the MDS Foundation, which is raising awareness of quality-of-life issues during this year’s MDS World Awareness Day. “There are so many little things we take for granted that these people struggle with.”

A Closer Look at the MDS Symptom of Fatigue

Tracey Iraca

TRACEY IRACA, EXECUTIVE DIRECTOR OF THE MDS FOUNDATION, INC., BELIEVES A BETTER UNDERSTANDING OF HOW MYELODYSPLASTIC SYNDROMES AFFECT PATIENT LIVES IS THE FIRST STEP TOWARDS IMPROVING THE MANAGEMENT OF THE DISEASE.

The fatigue and tiredness that people with MDS experience interferes with their daily activities, according to the survey. Patients surveyed reported that they often struggled with tasks such as cooking, cleaning, shopping, climbing stairs and taking care of their pets. Several respondents said that they relied on other people to complete many of those chores.

“My house is not nearly as clean since [I was diagnosed with] MDS,” one person responded. “I am exhausted a lot of the time. I can only work short times, and I have to sit down—then I usually fall asleep.”

Some people reported feeling tired all day, every day, while others only experienced exhaustion in the afternoon. Some also said that naps in the afternoon had become a necessity in their everyday life.

Fatigue drains people with MDS not only physically but emotionally, according to the survey. People with MDS reported losing patience with themselves and worrying about their loss of independence. Several also said they experienced feelings of isolation and loneliness from not being able to visit their family and friends.

Why Fatigue Is a Symptom of Myelodysplastic Syndrome

It didn’t surprise Iraca that fatigue was of significant concern for people with MDS. She has heard it dozens of times over the past decade at the Foundation. And she understands why.

In MDS, the bone marrow doesn’t produce enough healthy red blood cells, which transport oxygen to different cells and tissues. Young red blood cells are then inhibited from properly maturing, caused by what is known as erythroid maturation defects.

“The normal development of all blood cells is a complex process that relies on both stem cells and the environment within the bone marrow,” explained Sandra Kurtin, board member of the MDS Foundation, assistant professor of clinical medicine and assistant professor of nursing, The University of Arizona Cancer Center. “This process goes awry in MDS due to a variety of issues.”

As a result of this ineffective development of red blood cells, up to 90 percent of people with MDS have low red blood cell counts, a condition known as anemia. Without enough healthy red blood cells to transport oxygen, it leaves people feeling continuously tired and weak throughout the day.

“Patients are becoming much more aware of what’s happening—they are learning to talk with their doctor about their fatigue and other symptoms of anemia.”

Managing MDS Linked Anemia

Understanding how MDS affects the daily lives of people is the first step toward improving care for the thousands living with this disease. When people with MDS, doctors and caregivers discuss the complete patient experience, they can address what matters most.

“Patients are becoming much more aware of what’s happening—they are learning to talk with their doctor about their fatigue and other symptoms of anemia,” Iraca noted. “We want to educate people to identify these symptoms earlier on so that they can get treatment sooner for anemia.”

People with MDS may receive red transfusions to raise their low blood counts and antibiotics to prevent or fight infections. Some people may also receive a bone marrow transplant, chemotherapy, or other treatment options. Iraca is hopeful that research will help us learn more about the disease and how to treat it.

“Researchers are working to identify genetic defects in MDS so that they can develop therapies to target them,” Iraca said. “The research that’s happening now makes us hopeful.”

To learn about the high unmet need for people with MDS, read “Why I Advocate for People with Myelodysplastic Syndromes.”

One of Celgene’s defining characteristics has been a unique distributed research model, supporting promising programs from cutting-edge partners to advance new medicines for patients. In this model, Celgene and its partners mutually benefit from the resources, expertise and innovation from each entity.

One of the longest-standing examples of this collaborative strategy at work is the partnership between Celgene and Agios Pharmaceuticals, Inc., which began in 2010. The Celgene and Agios collaboration has encompassed research and development across a range of candidates in metabolic immuno-oncology and, importantly, led to the 2017 U.S. FDA approval of IDHIFA® (enasidenib) –the first approved medicine from the Celgene distributed research model.

Now, IDHIFA has earned another distinction – Prix Galien winner. IDHIFA was named the 2018 Best Pharmaceutical Product at the 12th annual Prix Galien Awards Gala on October 25 in New York City.

Presented by the Galien Foundation, the Prix Galien is an international award that recognizes outstanding achievements in improving the human condition through the development of innovative therapies and is regarded as the equivalent of the Nobel Prize in biopharmaceutical and medical technology research.

“All winners have made crucial contributions to the advancement of scientific understanding and improved outcomes for humankind,” said Bruno Cohen, Chairman of the Galien Foundation in a statement. “We are proud to honor their tireless work and unrelenting spirit.”

The road to this signature award reflects the shared commitment to patients at both Celgene and Agios. Working closely from the discovery stage, the companies evaluated potential candidates, conducted pre-clinical and clinical research, and ultimately achieved a U.S. approval and launch – less than four years from the first patient ever being dosed with IDHIFA. The companies share commercialization of IDHIFA.

“For all of us at Agios, it is truly an incredible honor to receive the 2018 Prix Galien Award with Celgene for IDHIFA for Best Pharmaceutical Product. As one of the highest accolades for pharmaceutical research and development, this award recognizes the extraordinary, collaborative effort that led to the creation of this important medicine,” said David Schenkein, M.D., Chief Executive Officer of Agios. “This feat would not be possible without the support of our partner Celgene, the patients who participated in our clinical trial, and the Agios scientists who made the IDH discovery and created the IDHIFA molecule.”

“The Celgene and Agios teams have spent years working together toward a common goal,” said Nadim Ahmed, President of Hematology/Oncology for Celgene. “The Prix Galien award is a recognition that our shared commitment has a meaningful impact on the lives of patients.”

“The Celgene and Agios team represent the very best attributes of collaboration,” said Krishnan Viswanadhan, Vice President of Global Alliances for Celgene, who accepted the Prix Galien on behalf of the team alongside Dr. Schenkein. “The combination of Agios’ pioneering science and Celgene’s deep experience in blood cancer research created tremendous potential for innovation, and the drive to help patients constantly pushed us to deliver.”

The Prix Galien award for IDHIFA recognizes, first and foremost, the significant potential to help change outcomes for patients, but also the value and importance of great partnerships.

Please see full Prescribing Information, including Boxed WARNING, for IDHIFA®

The current benchmark for efficacy in clinical trials of new plaque psoriasis treatments is a 75 percent reduction in the Psoriasis Area and Severity Index (PASI), which is a measure of the area of skin affected along with the skin’s appearance. But the most bothersome symptom, according to a survey of patients with plaque psoriasis, was not skin appearance, but itching.

As thousands of dermatologists gather at the 27th European Academy of Dermatology and Venereology Congress in Paris to discuss the latest data from ongoing clinical trials in psoriasis, Dr. Colby Evans, M.D., a dermatologist in Austin, TX, and also the immediate past chair of the Board of Directors of the National Psoriasis Foundation, discusses how researchers measure the effectiveness of treatments in clinical trials and how those measurements could be more comprehensive.

How do researchers measure the effectiveness of new plaque psoriasis treatments in clinical trials?

“Efficacy of plaque psoriasis treatments has immensely improved over the last 20 years, making more ambitious endpoints realistic. While a 75 percent reduction in PASI score has been the Food and Drug Administration’s benchmark, we see a growing interest in PASI 90 or 100 in more current trials. We want to get patients as close as we can to PASI 100. That being said, achieving a PASI 50 along with an improvement to quality of life is still clinically beneficial.”

DR. COLBY EVANS, M.D.

DR. COLBY EVANS, M.D., FROM THE NATIONAL PSORIASIS FOUNDATION BELIEVES THAT THERE IS ROOM FOR IMPROVEMENT IN THE MEASUREMENT AND GOALS OF EFFICACY IN PSORIASIS CLINICAL TRIALS.

Does PASI comprehensively capture all the clinical benefits of psoriasis treatments?

“PASI measures the size, as well as the level of scale, redness and thickness of psoriasis plaques. It does not capture other debilitating symptoms associated with psoriasis, such as psoriatic arthritis, itching or social stigma.”

How impactful are those symptoms on the lives of patients with plaque psoriasis?

“Itching can be debilitating and miserable, and can interfere with daily functioning. Survey data showed that people with psoriasis reported having a higher level of sleep-related problems compared to the general population.

Psoriatic arthritis is also of high concern among patients with psoriasis. Approximately 30 percent of plaque psoriasis patients develop psoriatic arthritis. It’s painful, can distort the joints and can be permanently disabling if untreated.”

What are the potential consequences of not capturing improvements in these symptoms?

“It’s important to take full measure of the patient and their life before you decide on treatment. PASI is a reasonable place to start, but it is more complicated than just the extent and thickness of their plaques. If you’re ignoring specific symptoms, patients can get left behind, and treatment decisions may be made without factoring in critical information.”

The more safe and effective treatment options we have, the better for patients with plaque psoriasis.

Do you measure PASI to make treatment decisions for patients who are not in clinical trials?

“In the real world, outside of trials, we don’t use a strict algorithmic treatment of psoriasis. In the regular clinical setting, I am more interested in knowing if the patient can live their life socially, occupationally and recreationally, and without feeling limited by their psoriasis. If they are, I know we’re on the path to success.”

How can clinical trials evolve to more comprehensively measure meaningful improvements for patients with plaque psoriasis?

“It’s becoming fairly common to have secondary endpoints that include itching and quality of life, which is progress. We’re seeing better management of psoriasis sub-types, such as patients with severe hand and foot psoriasis or patients who have severe arthritis without a lot of skin disease. Since the condition is quite diverse, there’s no one treatment for every patient. So the more safe and effective treatment options we have, the better for patients with plaque psoriasis.

With that in mind, many people who have had plaque psoriasis for decades aren’t aware of some of the new treatments. So a tremendous amount of patient education needs to be done.”

To learn more about treatment challenges that patients with psoriasis continue to face, read “Moderate Psoriasis Patient Needs Should Not Be Overlooked.”

Most patients diagnosed with lymphoma discuss the possibility of chemotherapy with a healthcare professional at some point. Chemotherapy is a standard of care for many forms of lymphoma, but most patients will experience multiple relapses.

Chemotherapy is a broad spectrum treatment that stops cell growth and division throughout the body, which can lead to side effects. Chemotherapies cannot differentiate between cancer cells and normal cells, so they also attack fast-growing but healthy cells, such as hair follicles and the cells lining the gut. That damage can lead to both short and long-term side effects, such as fatigue, nausea, a compromised immune system, fertility loss and an increased risk of infection or a second primary cancer.

While the benefits of chemotherapy often outweigh the risks, patients are eager for alternative solutions. Thankfully, research continues to look at different treatment pathways.

Meghan Gutierrez

MEGHAN GUTIERREZ, CHIEF EXECUTIVE OFFICER OF THE LYMPHOMA RESEARCH FOUNDATION (LRF), EXPLAINS WHY RESEARCHERS ARE EXPLORING CHEMOTHERAPY-FREE TREATMENTS

“We are learning a great deal about lymphoma subtypes and making progress in the discovery and development of new approaches that may improve quality of life,” Meghan Gutierrez, chief executive officer of the Lymphoma Research Foundation (LRF), said. “There is meaningful interest in exploring potential new treatments and combinations, many of which are chemotherapy-free.”

Lymphomas are caused by changes in immune cells called lymphocytes. In patients with lymphoma, the body makes many of these defective lymphoma cells that may not be detected by normal immune cells, which can properly fight infection and disease, including cancer. Restoring the immune system’s ability to fight cancer is a growing trend that has led to the development of immunomodulatory therapies, which can boost the tumor-killing cells of the immune system.

Investigators continue to explore new approaches focused on stimulating the immune system for patients with lymphoma.

The inherent ability of some types of immune cells to attack tumors relies on “tags” called antibodies on the surface of cancer cells. This killing process is known as antibody-dependent cell-mediated cytotoxicity (ADCC). In fact, several approved lymphoma therapies are antibodies that attach to cancer cells, leading the immune system to better identify and attack them. Researchers are now studying whether combining these antibody therapies with immunomodulatory therapies might further enhance cancer-killing ADCC, without the need for chemotherapy.

With further understanding of how both of these types of treatment work, separately and in combination, there is a potential to improve outcomes.

“Investigators continue to explore new approaches focused on stimulating the immune system for patients with lymphoma,” Gutierrez said. “It’s an incredibly exciting time as research is constantly evolving.”