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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.”

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.