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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.”

While about 10,590 children in the United States are diagnosed with cancer each year, more than 80 percent of them survive 5 years or more. But in sub-Saharan Africa, the statistic is vastly different – 90 percent of the 100,000 children diagnosed with cancer annually die from their disease.

At Celgene, we believe that cancer treatment should be available to patients no matter who they are or where they live. That is why Celgene recently supported expanded care capacity in resource-constrained countries through  $1 million in grants as part of the Celgene Cancer Care Links™ program.

In recognition of World Cancer Day 2019, Dr. David Poplack, M.D., director of the Global HOPE program at Texas Children’s Hospital’s Cancer and Hematology Centers and Baylor College of Medicine, explains how two grants received by Baylor College of Medicine and Texas Children’s Hospital from the program are being used to improve cancer care in sub-Saharan Africa.

This year marks the launch of the “I Am and I Will” World Cancer Day campaign, which encourages people to make a personal commitment to reduce the impact of cancer. So let’s start there. Can you describe what you’re doing to help fight cancer?

“Our goal at Global HOPE is to improve the survival rate for children with cancer in sub-Saharan Africa by increasing their capacity in this underserved region. To do so, we are establishing centers of excellence in six countries where we train pediatric doctors and nurses to become experts in diagnosing and treating children with cancer and blood diseases.”

What challenges are clinicians facing in sub-Saharan Africa?

“In my frequent travels to the region, there’s a noticeable lack of understanding of cancer. Many children lose their lives without ever being diagnosed. In some regions, they don’t even have a name for cancer.

A major reason so many children go undiagnosed is because of the inadequate health care infrastructure in most of these countries. For example, in most African countries, there are very few pediatric oncologists, the experts in the diagnosis and treatment of childhood cancer. In addition, many of the needed effective cancer therapies are simply not readily available.

We need to better educate the public to raise the awareness of cancer and train pediatricians to become specialists in how to properly diagnose and treat children with cancer. There’s also a tremendous need for adequate resources, like well-equipped medical centers that have the expertise, infrastructure and therapies to treat childhood cancer.”

PATRICK STONE

DAVID POPLACK, MEDICAL DIRECTOR OF TEXAS CHILDREN’S HOSPITAL’S CANCER AND HEMATOLOGY CENTERS HUGS A CANCER SURVIVOR AT THE GROUNDBREAKING FOR A CHILDREN’S CANCER AND HEMATOLOGY CENTER OF EXCELLENCE IN BOTSWANA IN 2017. SOURCE: BAYLOR COLLEGE OF MEDICINE/SMILEY POOL

Why is cancer becoming an important health care priority in sub-Saharan Africa today?

“In recent years, we have developed a better understanding of the burden of non-communicable diseases in low-resource countries. Of the 56.9 million global deaths in 2016, 71 percent were due to non-communicable diseases.

As a result, people have become more aware that cancer is a significant killer in sub-Saharan Africa – a region with some of the most resource-scarce countries. It’s fair to say that we are behind in improving cancer care in these countries. So, support for the Global HOPE program is critical to get closer to where we need to be.”

One of the grants you received focuses on pediatric Kaposi sarcoma in Malawi. What are the challenges that this program seeks to address?

“Kaposi sarcoma is a malignancy that affects both adults and children and is epidemic in sub-Saharan Africa, with especially high incidence in people with HIV. Our project seeks to better understand this disease and to develop an effective treatment.

Specifically, we are developing an approach known as risk-based treatment. For low-risk patients, the goal is to treat them with effective medication that has a lower toxicity. For those with high-risk disease, we must give them a more intensive treatment.”

We cannot achieve a world free from cancer unless we fund innovative approaches to address the problem of the disease in under-resourced countries.

The other grant is focused on pediatric Burkitt lymphoma in sub-Saharan Africa. How does the program propose to address this problem?

“Burkitt lymphoma is associated with Epstein–Barr virus and the endemic type originates in East Africa and the sub-Saharan countries that fall into the so-called ‘malaria belt.’ It presents frequently in children, and is universally fatal if not diagnosed and treated quickly.

A variety of therapies have been used to treat Burkitt lymphoma over the years, and our program is aimed at developing a treatment approach based on disease stage and risk factors. This approach requires making sure we can accurately diagnose the disease, using sophisticated forms of clinical diagnosis, including approaches such as flow cytometry, immunohistochemistry and biopsy.”

How did the Texas Children’s Hospital’s Cancer and Hematology Centers get involved in these global health programs?

“The Texas Children’s Hospital’s Cancer and Hematology Centers has the largest, and one of the most effective, training programs for pediatric oncologists in the United States. So, we have looked at where else in the world we could bring our expertise to impact childhood cancer.

In 1999, Baylor International Pediatric AIDS Initiative and Texas Children’s Hospital, supported by the Bristol-Myers Squibb Foundation’s (BMSF) Secure the Future Program, developed a program and built centers of excellence in African countries to help deal with the problem of pediatric HIV/AIDS. That program, directed by Dr. Mark Kline, is highly successful and treats more than 300,000 children in over a dozen countries. The Global HOPE program for managing childhood cancer developed from additional BMSF funding and leveraged the infrastructure we built for the original.”

What makes the funding that Global HOPE has received from Celgene so unique?

“There are many grant opportunities out there to help improve research for cancers that affect children in the United States, but few speak to the tremendous burden of pediatric cancer in under-resourced countries such as those in sub-Saharan Africa. We cannot achieve a world free from cancer unless we fund innovative approaches to address the problem of the disease in under-resourced countries. With this additional, extraordinary support from Celgene, we will specifically be able to study the biology and improve the care of two cancers that often strike children in sub-Saharan Africa.”

To learn more about Celgene Cancer Care Links™ program, read “Improving Cancer Care in Resource-Constrained Countries.”

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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.”

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Today, Celgene announced it will combine with Bristol-Myers Squibb, creating an innovative global biopharma leader. You can read the full announcement in the press release here.

This combination will enable Celgene to create greater impact for the patients who rely on our therapies, opportunities for our people, and value for our shareholders. Together with Bristol-Myers Squibb, Celgene will have leading franchises and a deep and broad pipeline—driving sustainable growth and delivering new options for patients with cancer, inflammatory and immunologic disease and cardiovascular disease.

What does that all mean? In short: we will be able to help more patients and explore new opportunities as part of an even stronger innovative biopharma leader.

“For more than 30 years, Celgene’s commitment to leading innovation has allowed us to deliver life-changing treatments to patients in areas of high unmet need. Combining with Bristol-Myers Squibb, we are delivering immediate and substantial value to Celgene shareholders and providing them meaningful participation in the long-term growth opportunities created by the combined company,” said Mark Alles, Celgene’s Chief Executive Officer.

Together we’ll have a broad portfolio of leading in-line products, and an expanded pipeline of early stage programs and near-term product launches to build a sustainable platform for future growth.

The combined company will have nine marketed products with more than $1 billion in annual sales, enabling us to create:

  • Leading oncology franchises in both solid tumors and hematologic malignancies led by OPDIVO and YERVOY as well as REVLIMID and POMALYST;
  • A top five immunology/inflammation franchise led by ORENCIA and OTEZLA; and
  • The #1 cardiovascular franchise led by ELIQUIS.

In addition to six expected near-term product launches (representing greater than $15 billion in revenue potential), the combined company will have a deep and diverse early and mid-stage pipeline across solid tumors and hematologic malignancies, immunology and inflammation, cardiovascular disease and fibrotic disease leveraging combined strengths in innovation.

The early-stage pipeline includes 50 high potential programs, many with important data readouts in the near-term.

patient and doctorTogether, we’ll also further our cutting-edge technologies and discovery platforms to sustain innovation leadership over time.

For example, combining with Bristol-Myers Squibb will expand our innovation capabilities in small molecule design, biologics/synthetic biologics, protein degradation, antibody engineering and cell therapy.

Consistent with Celgene’s long history of cultivating partnerships to benefit patients worldwide, the combined company will also have strong external partnerships with access to additional scientific platforms.

Today’s announcement represents the right strategic step for Celgene to continue to secure our long-term future—and deliver on our purpose to change the course of human health through bold pursuits in science with a promise to always put patients first.

“Our employees should be incredibly proud of what we have accomplished together and excited for the opportunities ahead of us as we join with Bristol-Myers Squibb, where we can further advance our mission for patients,” continued Mr. Alles. “We look forward to working with the Bristol-Myers Squibb team as we bring our two companies together.”

Important Information For Investors And Stockholders

This communication does not constitute an offer to sell or the solicitation of an offer to buy any securities or a solicitation of any vote or approval. It does not constitute a prospectus or prospectus equivalent document. No offering of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the U.S. Securities Act of 1933, as amended.

In connection with the proposed transaction between Bristol-Myers Squibb Company (“Bristol-Myers Squibb”) and Celgene Corporation (“Celgene”), Bristol-Myers Squibb and Celgene will file relevant materials with the Securities and Exchange Commission (the “SEC”), including a Bristol-Myers Squibb registration statement on Form S-4 that will include a joint proxy statement of Bristol-Myers Squibb and Celgene that also constitutes a prospectus of Bristol-Myers Squibb, and a definitive joint proxy statement/prospectus will be mailed to stockholders of Bristol-Myers Squibb and Celgene. INVESTORS AND SECURITY HOLDERS OF Bristol-Myers Squibb AND Celgene ARE URGED TO READ THE JOINT PROXY STATEMENT/PROSPECTUS AND OTHER DOCUMENTS THAT WILL BE FILED WITH THE SEC CAREFULLY AND IN THEIR ENTIRETY WHEN THEY BECOME AVAILABLE BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION.  Investors and security holders will be able to obtain free copies of the registration statement and the joint proxy statement/prospectus (when available) and other documents filed with the SEC by Bristol-Myers Squibb or Celgene through the website maintained by the SEC at http://www.sec.gov.  Copies of the documents filed with the SEC by Bristol-Myers Squibb will be available free of charge on Bristol-Myers Squibb’s internet website at http://www.bms.com under the tab, “Investors” and under the heading “Financial Reporting” and subheading “SEC Filings” or by contacting Bristol-Myers Squibb’s Investor Relations Department through https://www.bms.com/investors/investor-contacts.html.  Copies of the documents filed with the SEC by Celgene will be available free of charge on Celgene’s internet website at http://www.celgene.com under the tab “Investors” and under the heading “Financial Information” and subheading “SEC Filings” or by contacting Celgene’s Investor Relations Department at ir@celgene.com.

Certain Information Regarding Participants

Bristol-Myers Squibb, Celgene, and their respective directors and executive officers may be considered participants in the solicitation of proxies in connection with the proposed transaction.  Information about the directors and executive officers of Bristol-Myers Squibb is set forth in its Annual Report on Form 10-K for the year ended December 31, 2017, which was filed with the SEC on February 13, 2018, its proxy statement for its 2018 annual meeting of stockholders, which was filed with the SEC on March 22, 2018, and its Current Report on Form 8-K, which was filed with the SEC on August 28, 2018. Information about the directors and executive officers of Celgene is set forth in its Annual Report on Form 10-K for the year ended December 31, 2017, which was filed with the SEC on February 7, 2018, its proxy statement for its 2018 annual meeting of stockholders, which was filed with the SEC on April 30, 2018, and its Current Reports on Form 8-K, which were filed with the SEC on June 1, 2018, June 19, 2018 and November 2, 2018. Other information regarding the participants in the proxy solicitations and a description of their direct and indirect interests, by security holdings or otherwise, will be contained in the joint proxy statement/prospectus and other relevant materials to be filed with the SEC regarding the proposed transaction when they become available. You may obtain these documents (when they become available) free of charge through the website maintained by the SEC at http://www.sec.gov and from Investor Relations at Bristol-Myers Squibb or Celgene as described above.

Cautionary Statement Regarding Forward-Looking Statements

This communication contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended.  You can generally identify forward-looking statements by the use of forward-looking terminology such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “explore,” “evaluate,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” or “will,” or the negative thereof or other variations thereon or comparable terminology.  These forward-looking statements are only predictions and involve known and unknown risks and uncertainties, many of which are beyond Bristol-Myers Squibb’s and Celgene’s control.

Statements in this communication regarding Bristol-Myers Squibb, Celgene and the combined company that are forward-looking, including projections as to the anticipated benefits of the proposed transaction, the impact of the proposed transaction on Bristol-Myers Squibb’s and Celgene’s business and future financial and operating results, the amount and timing of synergies from the proposed transaction, the terms and scope of the expected financing for the proposed transaction, the aggregate amount of indebtedness of the combined company following the closing of the proposed transaction, expectations regarding cash flow generation, accretion to non-GAAP earnings per share, capital structure, debt repayment, adjusted leverage ratio and credit ratings following the closing of the proposed transaction, Bristol-Myers Squibb’s ability and intent to conduct a share repurchase program and declare future dividend payments, the combined company’s pipeline, intellectual property protection and R&D spend, the timing and probability of a payment pursuant to the contingent value right consideration, and the closing date for the proposed transaction, are based on management’s estimates, assumptions and projections, and are subject to significant uncertainties and other factors, many of which are beyond Bristol-Myers Squibb’s and Celgene’s control. These factors include, among other things, effects of the continuing implementation of governmental laws and regulations related to Medicare, Medicaid, Medicaid managed care organizations and entities under the Public Health Service 340B program, pharmaceutical rebates and reimbursement, market factors, competitive product development and approvals, pricing controls and pressures (including changes in rules and practices of managed care groups and institutional and governmental purchasers), economic conditions such as interest rate and currency exchange rate fluctuations, judicial decisions, claims and concerns that may arise regarding the safety and efficacy of in-line products and product candidates, changes to wholesaler inventory levels, variability in data provided by third parties, changes in, and interpretation of, governmental regulations and legislation affecting domestic or foreign operations, including tax obligations, changes to business or tax planning strategies, difficulties and delays in product development, manufacturing or sales including any potential future recalls, patent positions and the ultimate outcome of any litigation matter. These factors also include the combined company’s ability to execute successfully its strategic plans, including its business development strategy, the expiration of patents or data protection on certain products, including assumptions about the combined company’s ability to retain patent exclusivity of certain products, the impact and result of governmental investigations, the combined company’s ability to obtain necessary regulatory approvals or obtaining these without delay, the risk that the combined company’s products prove to be commercially successful or that contractual milestones will be achieved. Similarly, there are uncertainties relating to a number of other important factors, including: results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; the ability to enroll patients in planned clinical trials; unplanned cash requirements and expenditures; competitive factors; the ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates; the ability to maintain key collaborations; and general economic and market conditions. Additional information concerning these risks, uncertainties and assumptions can be found in Bristol-Myers Squibb’s and Celgene’s respective filings with the SEC, including the risk factors discussed in Bristol-Myers Squibb’s and Celgene’s most recent Annual Reports on Form 10-K, as updated by their Quarterly Reports on Form 10-Q and future filings with the SEC.

It should also be noted that projected financial information for the combined businesses of Bristol-Myers Squibb and Celgene is based on management’s estimates, assumptions and projections and has not been prepared in conformance with the applicable accounting requirements of Regulation S-X relating to pro forma financial information, and the required pro forma adjustments have not been applied and are not reflected therein. None of this information should be considered in isolation from, or as a substitute for, the historical financial statements of Bristol-Myers Squibb or Celgene. Important risk factors could cause actual future results and other future events to differ materially from those currently estimated by management, including, but not limited to, the risks that: a condition to the closing of the proposed acquisition may not be satisfied; a regulatory approval that may be required for the proposed acquisition is delayed, is not obtained or is obtained subject to conditions that are not anticipated; Bristol-Myers Squibb is unable to achieve the synergies and value creation contemplated by the proposed acquisition; Bristol-Myers Squibb is unable to promptly and effectively integrate Celgene’s businesses; management’s time and attention is diverted on transaction related issues; disruption from the transaction makes it more difficult to maintain business, contractual and operational relationships; the credit ratings of the combined company declines following the proposed acquisition; legal proceedings are instituted against Bristol-Myers Squibb, Celgene or the combined company; Bristol-Myers Squibb, Celgene or the combined company is unable to retain key personnel; and the announcement or the consummation of the proposed acquisition has a negative effect on the market price of the capital stock of Bristol-Myers Squibb and Celgene or on Bristol-Myers Squibb’s and Celgene’s operating results.

No assurances can be given that any of the events anticipated by the forward-looking statements will transpire or occur, or if any of them do occur, what impact they will have on the results of operations, financial condition or cash flows of Bristol-Myers Squibb or Celgene. Should any risks and uncertainties develop into actual events, these developments could have a material adverse effect on the proposed transaction and/or Bristol-Myers Squibb or Celgene, Bristol-Myers Squibb’s ability to successfully complete the proposed transaction and/or realize the expected benefits from the proposed transaction. You are cautioned not to rely on Bristol-Myers Squibb’s and Celgene’s forward-looking statements. These forward-looking statements are and will be based upon management’s then-current views and assumptions regarding future events and operating performance, and are applicable only as of the dates of such statements. Neither Bristol-Myers Squibb nor Celgene assumes any duty to update or revise forward-looking statements, whether as a result of new information, future events or otherwise, as of any future date.

Millions of Americans who rely on Medicare Part D may be facing sticker shock in 2020 if solutions that address out-of-pocket costs and access are not enacted quickly, based on a report from the Lupus Foundation of America.

Medicare Part D provides prescription drug coverage to more than 44 million Medicare recipients. While prescription drug coverage under part D hasn’t been around for as long as people may think, its popularity stems from its far reaching importance to the overall health of its millions of beneficiaries.

“Medicare did not offer a prescription drug benefit until Part D was enacted in 2003,” said Patrick Wildman, vice president of Advocacy & Government Relations for the Lupus Foundation of America. “Since then, it has greatly improved the health of so many people.” Beneficiaries include lupus patients, who incur an average of $12,643 per year in direct health care costs.

While Part D has been a big step, Wildman believes policymakers need to address looming challenges, including the impending “out-of-pocket” cliff, specialty tiers and more. The right policies will protect prescription medication coverage for America’s seniors, but Wildman notes we need the political will to make them happen.

The “Out-of-Pocket” Cliff

In 2005, the Lupus Foundation of America established the Medicare Access for Patients Rx (MAPRx) coalition — a group of more than 55 patient advocacy organizations representing seniors who depend on Medicare Part D for their prescription medications. The coalition recently published a report highlighting some of the challenges faced by Part D beneficiaries.

The most pressing issue, according to the report, is a steep increase in the “out-of-pocket” threshold, the amount patients pay before entering catastrophic coverage where their payment responsibility for their medicines drops to five percent for the remainder of the plan year. The Affordable Care Act capped how much that limit could increase each year. But the limit on growth of the threshold is set to expire in 2020, adding $1,250 to threshold in just one year, unless Congress acts.

“It is a looming crisis,” Wildman said. “As a coalition, we are worried that with that big of a spike, people may have challenges accessing medication they need.”

Infographic of Medicare Part D, Out of Pocket Spending

Specialty Tiers on the Rise

Part D plans also have been increasingly placing more medications on specialty tiers, which force seniors to pay up to a third of the costs for certain particularly expensive treatments. A study found that Part D beneficiaries pay an average of $3,949 annually out of pocket for specialty treatments for rheumatoid arthritis, $5,238 for multiple sclerosis and $6,322 for chronic myeloid leukemia. These chronic illnesses require several treatments that are frequently listed on specialty tiers.

Part D beneficiaries have no recourse to request exceptions to specialty tier pricing, either.9 The MAPRx coalition is fighting to change that.

“I think we need to fundamentally address the role of specialty tiers in Part D,” Wildman said. “Placing a medication on a specialty tier may stop some patients from filling their prescriptions and getting the treatment that they need.”

There is a lot of work still to be done to improve Medicare Part D for our seniors.

Improving Part D with an Out-of-Pocket Cap

While Medicare Part B beneficiaries can enroll in additional coverage that limits their annual out-of-pocket spending, most Part D beneficiaries must pay 5 percent of their medication costs after they hit the catastrophic phase without limit. Depending on what medications they have been prescribed, this can amount to thousands of dollars each month. But by putting an out-of-pocket cap in place, however, policymakers could help Part D beneficiaries better manage the financial burden of their conditions and illnesses, according to Wildman.

Strengthening and protecting access to medications is critical for millions of seniors with chronic diseases. The first step in this process, according to Wildman, is to convince policymakers how important the benefit is for seniors and people with disabilities, as well as others who depend on it. The coalition also wants to ensure that existing patient protections within the program stay in place and that recently introduced pricing policies do not restrict access.

“There is a lot of work still to be done to improve Medicare Part D for our seniors,” Wildman said. “We are looking forward to strengthening the program in the years to come.”

To learn more about Medicare Part D, read “Medicare Part D: 10 Years of Successfully Meeting Seniors’ Needs

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.”

At Celgene, bold science that benefits patients is at the core of our values and our business. Each year, Celgene’s cutting-edge medical research helps more patients around the world. While patients are the focus of our mission, we also take very seriously our responsibilities to our employees, our communities and our environment—responsibilities that we continue to live up to. Celgene’s 2018 Corporate Responsibility Report, released this week, highlights how we are working to make a difference in the lives of patients, communities and economies around the world.

“We are at a very important time in our company’s journey to help patients and create increased value for our stockholders,” Mark J. Alles, Chairman and Chief Executive Officer of Celgene Corporation, said. “Over the next two years, we expect to advance five late-stage products toward regulatory approval. These therapies represent the promise of our industry-leading investments in research and the beginning of our next wave of innovation.”

Patients First

Celgene’s commitment to changing the course of human health includes helping patients living in developing parts of the world. In 2017, Celgene joined 24 biopharmaceutical companies in the Access Accelerated partnership to improve access to treatment and care for non-communicable diseases — such as cancer — in low- and middle-income countries.

2018 Corporate Responsibility Report

During its first year, the partnership moved toward its goal by advancing 62 programs, including the Celgene Cancer Care Links™ grant program1 and the Academic Model Providing Access to Healthcare (AMPATH) Oncology Partnership, which Celgene joined in 2011 to increase access to cancer diagnosis and treatment in Kenya.

Celgene introduced the Cancer Care Links™ in December 2017 to improve cancer care in resource-constrained countries by giving organizations with existing infrastructure an opportunity to apply for grants to support oncology training, cancer prevention and detection, nursing programs, general medical support, pharmacy programs, and awareness and education initiatives. The first grant recipients were announced in December 2018.

This past March, Access Accelerated hosted a forum in Kenya, where Zeba Khan, Ph.D., Vice President of Corporate Responsibility for Celgene, took part in a panel to discuss the AMPATH Oncology Partnership. “Our partnership with AMPATH works within the current healthcare system to address a local need and to improve multiple myeloma care in Kenya,” Khan said.

ENGAGING ACROSS SECTORS AND DISCIPLINES TO ADDRESS NCDs

THE “ENGAGING ACROSS SECTORS AND DISCIPLINES TO ADDRESS NCDs” PANEL INCLUDED PATIENTS, HEALTHCARE PROFESSIONALS, BIOPHARMACEUTICAL COMPANIES, NON-PROFIT ORGANIZATIONS AND KENYA’S MINISTRY OF HEALTH.

Supporting Communities and the Planet

At Celgene, we’re committed to making a positive impact on the communities where we work and live through giving and volunteerism, and to the health of the planet through environmental stewardship and resource conservation.

Last year, more than 2,000 Celgene employees ran, cycled or walked in races and events to support finding a cure for serious diseases, including blood cancers, pancreatic cancer, psoriasis and psoriatic arthritis. In 2017, more than 1,000 Celgene employees helped raise $700,000 to support the Leukemia & Lymphoma Society’s Light The Night® events, making Celgene its top biopharmaceutical fundraising partner. Celgene was also the first National Presenting Sponsor of PurpleStride®, the Walk to End Pancreatic Cancer, in 2017. More than 280 employees participated in 52 PurpleStride walks and runs, raising $57,000.

Meanwhile, Celgene is making strong progress to meeting our 2020 environmental goals, which focus on reducing our carbon footprint, investing more in renewable electricity, reducing water withdrawal and reducing waste generation. This year, Celgene expanded its portfolio of LEED®-certified buildings and continued our trend of purchasing renewable energy. Renewable sources now make up 50 percent of all power used by Celgene worldwide, and wind-powered electricity fuels 73 percent of U.S. facilities’ energy. This achievement has resulted in an invitation to join the U.S. Environmental Protection Agency’s Green Power Partnership.

Built on Integrity

Celgene’s business culture is built on integrity, ethics, sound decision-making and behaviors that reflect our values and focus on patients. This year, Celgene joined the Pharmaceutical Supply Chain Initiative (PSCI) to enhance sustainability within our supply chain and was recognized for excellence in responsible clinical trial data sharing in the Bioethics International Good Pharma Scorecard, ranking in the top five for Clinical Trial Transparency.

Whether it’s sharing clinical trial data or providing grants to organizations in resource-constrained countries to improve the cancer care infrastructure, the common thread that binds our employees and all our activities is our focus on putting patients first. “We remain steadfastly committed to our mission, as we work to discover, develop and deliver to patients new and even more effective ways to improve and extend the lives of patients around the world,” Alles said.

To learn more about Celgene’s Corporate Responsibility activities, read the 2018 Celgene Corporate Responsibility Report.

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.”

Lung cancer remains the leading cause of cancer-related death in both men and women in the United States. And for America’s veterans, that risk is even higher as they are 25 percent more likely to be diagnosed with lung cancer than those who did not serve in the military.

As we recognize this year’s Veterans Day, Laurie Fenton Ambrose, President and CEO of Lung Cancer Alliance, explains how early detection and the treatment of lung cancer is evolving in and benefiting the veteran community, and what still needs to be done to improve the care for the men and women who have served our country.

Why are veterans disproportionately affected by lung cancer?

“Veterans are disproportionately affected for two key reasons: their smoking history and their occupational exposures. First, veterans have a higher prevalence of smoking than the civilian population. Many use smoking as a way to cope with the stress of their occupation and cigarettes were readily available to them while in service. At the same time, veterans were also exposed to a variety of chemicals that are linked with an increased risk of lung cancer, such as asbestos, Agent Orange, burn pits and chemical weapons.”

Why is treating veterans with lung cancer challenging?

LAURIE FENTON AMBROSE

LAURIE FENTON AMBROSE, PRESIDENT AND CEO OF LUNG CANCER ALLIANCE, IS HELPING TO LEAD THE CHARGE IN IMPROVING PREVENTION, SCREENING AND TREATMENT OF LUNG CANCER IN THE VETERAN COMMUNITY.

“Research has shown that many Veterans’ Affairs facilities are not prepared to implement comprehensive lung cancer screening programs. More work needs to be done to support the investment in infrastructure and resources to offer more coordinated care to American veterans with lung cancer as quickly as possible.

We also need to address the stigma associated with having lung cancer—which affects how the disease is both resourced and advocated for — and the comorbidities that many veterans face in addition to lung cancer.”

How is Lung Cancer Alliance working to improve care for veterans with lung cancer?

“We are working with military facilities and programs to reduce tobacco use and exposure. We are also working on awareness campaigns to alert veterans to their elevated risk for lung cancer as well as directing them to responsible screening and care so that we catch and manage the disease early.”

Why is early detection so important?

“Because it can save your life. Just like other diseases with approved screenings, you see higher survival rates in those whose disease has been caught early by screening. Now lung cancer can join this fold. Simply put, if you find lung cancer in its earliest form, you improve your treatment options and your quality of life. But right now, approximately 75 percent of lung cancers in the general population are diagnosed at late stage when there are fewer effective treatment options.

We need to highlight the benefits of screening and make sure that it is made available to our veterans. Studies have shown that low-dose CT screenings can decrease lung cancer death rates if we provide adequate resources and infrastructure. It’s key that we make sure everyone at risk—including our military community—is aware that these screenings are available to them.”

We’re at a pivotal moment right now with lung cancer screening and treatment advances, and we are seeing more lung cancer survivors than ever.

How are the efforts to improve screenings going so far?

“Screening is a fairly new preventive service. The federal government gave a green light just four years ago for coverage. So, we are now working hard to “ramp-up” this service in communities across the country. This involves bringing national awareness to the issue as well as educating providers and those at risk of the benefits and risks. We are also committed to making sure screening and care is being provided in the most responsible way in medical centers and considering how we collect information to help further improve early detection and treatment options. It is not easy. It takes time. But we are focused on moving this forward as rapidly as possible.

The challenge I see is that there isn’t the same sense of urgency and focus around lung cancer screening as we have seen with other cancers such as breast and colon. That’s likely related to the stigma around this disease. We really need more national attention on this issue.”

How are new treatment options helping patients with lung cancer?

“New treatment options for patients with lung cancer are improving care and bringing hope. For example, we have more targeted therapies and immunotherapies being paired with chemotherapy to treat lung cancer today than we have had in decades.

When you combine these new therapies with the ability to detect lung cancer earlier, outcomes can be more favorable for early stage patients. This one-two punch is making a dent in the high mortality rates. We’re seeing five-year survival rates rise to 19 percent; a leap forward from where we were in the 1980s, when five-year survival was only 13 percent. We’re at a pivotal moment right now with lung cancer screening and treatment advances.”

What else should veterans know about lung cancer?

“No veteran should ever feel alone. We are ready to support them. That’s what Lung Cancer Alliance is here to do. Veterans should know that there’s a place where they can go to feel part of the community and find information. We are honoring their service by making sure our service is there for them.”

To learn more about how researchers are developing new treatments strategies for lung cancer, read “Hitting Moving Targets in Lung Cancer Subtypes.”

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.

Celgene has long maintained a dual focus on not only the patients that its therapies treat but the employees at Celgene that work to develop those therapies and get them to market. In October, Celgene was ranked #9 on the Forbes’ World’s Best Employers 2018 list. This year Forbes also gave Celgene placements on the 2018 Global Growth Champions list and America’s Best Midsize Employers 2018.

Celgene Values WallTogether with Statista, Forbes World’s Best Employers list was compiled from an analysis of more than 430,000 global employee recommendations. Along with 30 detailed questions, respondents were asked to rate their own employer and the likelihood they would recommend their company to a friend or family member. They also listed other companies they admired.

Among the qualifying factors, Celgene has continued expansion of Diversity and Inclusion initiatives which likely contributed to its high ranking. Parallel with these efforts, Celgene recently announced its participation as a founding member of the Healthcare Businesswomen’s Association’s Gender Parity Collaborative where several Celgene leaders now sit on the collaborative’s Gender Parity Council.

“Our ranking as one of the best employers in the world highlights Celgene special culture and the direct connection every colleague has to our patients,” said Joe Hand, EVP, Global HR and Corporate Services. “As our global organization evolves, we continue to look for innovative and genuine approaches to enhance our employee experience and attract and retain talented people.”

Celgene Change Makers - Boudry 2017Celgene leadership also places a heavy focus on mentorship, volunteerism and employee inclusion groups. Women Advancing Leadership at Celgene and Celgene Pride Alliance are employee resource groups that offer employees opportunities to connect and network. Supported by Executive Sponsors who are members of the Celgene Executive Committee, these resource groups host events and campaigns that bring together Celgene employees in an inclusive and safe environment to advance the dialog of social awareness and acceptance and drive necessary change.

In 2018 Celgene employees also saw enhancements to many U.S. employee benefits including Work-Life benefits like increased paid parental leave, a “bridge back to work” policy that allows new parents to transition back to work on a part-time basis but with full pay, and a policy for flexible work arrangements, to name a few.

“We felt it was important that our policies matched our commitment to creating an inclusive environment for our employee base,” said Joe Hand. “We’re proud to provide more balance between employees’ personal and professional lives to enable our workforce to bring their best self to work at Celgene and to their families and loved ones at home.”

Celgene Named Among World’s Best Employers 2018 - Run

To learn more about Celgene continued progress toward Diversity; read our official Diversity Mission Statement.

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®