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World Cancer Day 2017

When a colleague in Mali was diagnosed with cancer last year, Joseph Camardo, M.D., senior vice president of Global Health at Celgene, wasn’t surprised that she flew to Tunisia for treatment. Having visited the West African country twice in the past two years, Camardo was familiar with its limited health care resources.

“The most advanced cancer care isn’t available routinely in Mali or most low- to middle-income countries,” he said. “So many other diseases, including HIV, malaria, and other infections, presented such need that not many people anticipated the burden of cancer in the developing world, so the infrastructure needed to treat cancer has not caught up.”

While the death rate from all cancers fell 25 percent between 1991 and 2014 in the United States, the same is not true in some low- and middle-income countries. World Cancer Day is an opportunity to reflect on how we collectively and individually can help improve cancer care in the developed world and beyond, according to Camardo.

JOSEPH CAMARDO, SENIOR VICE PRESIDENT OF CELGENE GLOBAL HEALTH AND CORPORATE AFFAIRS MEDICAL STRATETY, BELIEVES FIGHTING CANCER IN DEVELOPING COUNTRIES WILL REQUIRE A LONG-TERM COMMITMENT.

JOSEPH CAMARDO, SENIOR VICE PRESIDENT OF CELGENE GLOBAL HEALTH AND CORPORATE AFFAIRS MEDICAL STRATEGY, BELIEVES FIGHTING CANCER IN DEVELOPING COUNTRIES WILL REQUIRE A LONG-TERM COMMITMENT.

Taking place each year on February 4, Word Cancer Day aims to raise awareness about the disease and to encourage governments and individuals to take action in the fight against cancer. The ‘We can. I can.’ theme for 2016 to 2018 campaign takes a look at the steps that everyone—both together and individually—can help reduce the burden of cancer.

According to a report from the American Cancer Society (ACS), 57 percent of the estimated 14 million new cancer cases in 2012 occurred in low- and middle-income countries. The burden of the disease is expected to continue shifting to the developing world as the population there grows and ages.

This shifting burden is not just a concern for developing countries. Worldwide, the economic impact of cancer in 2008 was $895 billion, according to estimates from the ACS. And that’s not even including medical costs.

Helping developing countries fight cancer is not only the humane thing to do but also beneficial to the global economy.

Meanwhile, the potential economic gains from improving cancer care could be substantial. According to one study, a 1 percent reduction in cancer mortality would provide an estimated $500 billion benefit to the United States alone.

“Helping developing countries fight cancer is not just the humane thing to do but also beneficial to the global economy,” Camardo said. “If you don’t treat people in these regions, their workforces are not going to be as strong, their economies will not grow, and they will remain a burden on the global economy.”

Patients in these countries often lack access to screening, diagnosis and treatment. These health care systems were designed to fight infectious diseases, such as malaria, tuberculosis and AIDS, ensuring that vaccines and treatments are available across the country. As success has been achieved and deaths from infections have fallen, however, these same systems are revealing themselves as unprepared for the subsequent rise in cancer diagnoses, according to Camardo.

A BABY RECEIVES A VACCINE AT A MEDICAL CLINIC IN KENYA. MOST HEALTH CARE SYSTEMS IN LOW- AND MIDDLE-INCOME COUNTRIES WERE SET UP TO FIGHT INFECTIOUS DISEASE RATHER THAN NONCOMMUNICABLE DISEASES SUCH AS CANCER

A BABY RECEIVES A VACCINE AT A MEDICAL CLINIC IN KENYA. MOST HEALTH CARE SYSTEMS IN LOW- AND MIDDLE-INCOME COUNTRIES WERE SET UP TO FIGHT INFECTIOUS DISEASE RATHER THAN NONCOMMUNICABLE DISEASES SUCH AS CANCER

Improving cancer care in developing countries will certainly be a formidable challenge. “When you try to solve one problem, you find another that has to be solved first,” Camardo said. “For example, before you can bring doctors and diagnostic equipment to people in rural regions, you need to build roads. Many cancer therapies require refrigeration, but electricity is not available in some areas.”

Despite the challenges, Camardo is optimistic that differences can be made through public-private partnerships—such as the Academic Model Providing Access to Healthcare (AMPATH) initiative. Recognizing that the growing burden of cancer is reaching a crisis point in developing countries, Celgene and other partners have helped expand the services offered by the AMPATH-Oncology Institute. The effort provides cancer care for 20 million people in western Kenya, and nearly 20,000 patient visits to AMPATH-Oncology are made each year.

Camardo hopes that tangible results like these will show governments that they have an opportunity to move their countries forward with a long-term commitment to improving the lives of their citizens, building cancer care competence and structure while maintaining the fight against HIV, malaria, tuberculosis, and other infections.

“Flying cancer patients out of developing countries such as Mali is not a sustainable solution,” Camardo said. “It’s going to take a long time to build the infrastructure for cancer care in developing countries, but we have to start somewhere, and we have to start today.”

To learn more about the goals and vision for the partnership, visit the Accelerated Access website.

While there is much hope in the science of cancer research, critical policy reforms in Washington must be implemented to turn that optimism into cures. Non-scientific barriers to advancing innovation include knowledge-sharing among researchers, industry and caregivers, and current funding models, all of which can and should be addressed through political action.

Fortunately, the 21st Century Cures Act — which was signed into law by President Barack Obama this month — outlines several common-sense policies that offer solutions that give us the greatest chances of reaching a world free from death and suffering from cancers.

Prioritizing Patients

One critical part of the Cures proposal is a provision to include greater patient feedback data on the impact of therapies and treatments under review by the FDA. By incorporating self-reported symptom measurements as a regular part of clinical trials, patients can provide first-hand knowledge of quality of life measures like pain, nausea, fatigue, depression, or ability to carry out normal daily activities. This unique perspective on the benefits and risks of particular therapies is critically important as FDA evaluates the harms and benefits of new lines of treatment.

21st Century Cures will also support the FDA in working closely with trial sponsors to develop smarter, faster clinical studies with better-defined endpoints that can gauge patient responses to new therapies more quickly and guide regulatory decisions based on outcomes that are most meaningful to patients. These changes are designed to help promote more targeted and effective therapies for patients, increased competition among industry and lower associated costs.

Inspiring Cross-Sector Collaboration

No one organization alone will bring forth a world free from death and suffering from cancer on their own. 21st Century Cures will break down barriers among cancer researchers, physicians and patients to offer essential insights from the massive amount of health care data being generated today. A partnership formed earlier this year between Celgene and the National Cancer Institute serves as a working model for the types of collaborations needed for the Moonshot initiative to reach its potential. Similar programs will be supported by 21st Century Cures, which offers $5 million in funding for collaborative research programs that will collect and analyze data on diseases, with a particular focus on rare diseases.

Investing Today for Cures Tomorrow

While basic research sets the foundation for the development of new therapies, our scientists too often lack the resources needed to capitalize on the scientific opportunities of today. Compared to their 2003 levels (and adjusted for inflation), federally funded budgets for critical research centers such as the National Institutes of Health (NIH) and National Cancer Institute (NCI) are down 18 and 21 percent, respectively. The 21st Century Cures Act will provide the NIH with additional funding on top of its annual appropriations to support its strategic initiatives. Such innovative federal research funding models are essential to producing research that has the potential to revolutionize cancer treatment.

Policy Can Help Cure Cancer

In September, more than 25,000 lanterns with the names of cancer survivors and those who have died from disease surrounded the Capitol Reflecting Pool in Washington, D.C. during the American Cancer Society Cancer Action Network’s Lights of Hope ceremony. The annual event is designed to serve as a reminder of the integral role legislators in Washington play in fostering the dramatic breakthroughs we need to cure cancers that continue to kill our loved ones.

A supportive environment for medical innovation provided by 21st Century Cures will help determine whether we achieve our goal of eliminating all cancers and fulfill the hope of the patients we serve. The enactment of this law has set a critical bar and momentum for the 115th session that is vital to keeping a world free of suffering from cancers within reach.

At ASH this year, Celgene has unveiled a new campaign that underscores its ongoing commitment to lymphoma research. Celgene has multiple ongoing phase III programs across a number of aggressive and indolent diseases.

The campaign slogan speaks to “making lymphoma a distant memory” and features a dinosaur skeleton whose bones spell out the word “lymphoma”. This reflects the commitment Celgene researchers have, now and in the future, to develop treatments and advance care in lymphoma. Their vision is for a future in which lymphoma is more than treatable, it’s a distant memory.

This year’s ASH meeting will feature nearly 70 studies that include the investigational use of Celgene agents in lymphoma and leukemia – further exemplifying Celgene’s commitment to lymphoma research.

To learn more about Celgene and its lymphoma programs – visit http://www.researchoncology.com/areas-of-research/lymphoma/science/

While progress made in immunotherapy is bringing hope to many patients, chemotherapy remains the backbone of treatment for the majority of patients living with non-small cell lung cancer. In some ways, researchers continue to evaluate the full potential of this foundational therapeutic class.

UPAL BASU ROY, DIRECTOR OF RESEARCH AND POLICY AT THE LUNGEVITY FOUNDATION EXPLAINS THAT CHEMOTHERAPY AND IMMUNOTHERAPY MAY MAKE A POWERFUL COMBINATION IN THE FUTURE.

UPAL BASU ROY, DIRECTOR OF RESEARCH AND POLICY AT THE LUNGEVITY FOUNDATION EXPLAINS THAT CHEMOTHERAPY AND IMMUNOTHERAPY MAY MAKE A POWERFUL COMBINATION IN THE FUTURE.

“We’re still learning how best to use chemotherapy and personalize it to help lung cancer patients live longer, better lives,” Upal Basu Roy, director of research and policy at the LUNGevity Foundation, said. “The role of chemotherapy in lung cancer treatment is still evolving and improving every day. I don’t see it ever really going away anytime soon.”

Lung cancer remains one of the deadliest forms of cancer, with an approximate 18 percent five-year survival rate. While certain subtypes of non-small cell lung cancer can be treated with targeted therapies, the majority are still treated with chemotherapy.

Fortunately, the experience of chemotherapy today is considerably less challenging than it was decades ago.

“When my grandmother was treated with chemotherapy for cancer in the 1980s, she suffered just as much from the side effects as she did from the cancer,” Basu Roy said. “But when my uncle was treated in the 2000s, his doctors prescribed him anti-nausea medications to help minimize that side effect.” Other medicines used along with chemotherapy aim to battle fatigue and loss of appetite.

By making chemotherapy more tolerable, doctors have expanded its use to earlier stages, when the disease has not spread (“metastasized”) beyond the lungs, so surgeons can attempt to remove the tumor. Chemotherapy before surgery can help shrink the tumor, making it easier to be removed. Giving chemotherapy soon after surgery is meant to kill any cancer cells left behind and has increased the five-year survival rate for patients with non-small cell lung cancer by 5 percent. When the tumor cannot be removed surgically, a combination of chemotherapy and radiation, given at the same time, has helped patients live longer.

We’re figuring out not only which treatment approaches are most effective but also how to combine them and in which order

With immunotherapies, which work with your body’s immune cells to fight cancer, the potential for innovative combination treatments with chemotherapy is growing. These new therapies have significantly reduced, or in rare case even eliminated, the tumor burden of metastatic disease in patients. But they don’t work for everyone. For instance, PD1 inhibitors work for 20 to 30 percent of patients with non-small cell lung cancer, meaning the remainder needs other options. Some studies are combining immunotherapy with chemotherapy to determine whether more patients respond.

With a rapidly changing treatment landscape, community oncologists who treat many types of cancer may not be up to date on the latest advances specific to lung cancer. So patients should learn all they can, ask plenty of questions and seek out second opinions from lung cancer specialists.

To provide the lung cancer community with the information needed to ask the right questions and make the best treatment decisions with their doctors, Celgene has worked with Cancer Support Community and LUNGEVITY to publish an educational resource called “Treatments for Advanced and Metastatic Lung Cancer” as part of the Frankly Speaking About Cancer® series. This guide is designed to help patients feel empowered to take control of their cancer—and their lives – and provides a comprehensive overview of treatment options for metastatic lung cancer, as well as a tear-out discussion guide to help patients understand what questions to ask and the type of information they need to work with their doctor to decide the best treatment options.

“We’re figuring out not only which treatment approaches are most effective but also how to combine them and in which order,” Basu Roy said. “And as we continue to learn, we need to make sure this information gets to the front lines, to the community oncologists who see the majority of lung cancer patients.”

To learn more about the treatment options for metastatic lung cancer, read Treatments for Advanced and Metastatic Lung Cancer at the Cancer Support Community website.

Patients are Waiting: Supporting the Pancreatic Cancer Community

People who have been diagnosed with pancreatic cancer are waiting for information about their treatment options, according to a 2013 survey. Such findings underscore the importance of raising awareness of this deadly disease during Pancreatic Cancer Awareness Month.

Seventy percent of patients and caregivers surveyed said doctors did not provide treatment options when they were initially diagnosed, and 14 percent said they were never given the information. And although current treatment guidelines for pancreatic cancer recommend participation in clinical trials, half of patients surveyed reported their doctors never discussed the option with them.

Education plays an essential role in empowering patients and families. The pancreatic cancer community must continue investing in resources that help patients better cope with their anxieties and improve their overall experience. When patients understand their disease and the options available, they are more likely to stick with their treatment.

“At Celgene, we understand the personal tragedy and the toll pancreatic cancer takes on families and loved ones,” Mark Alles, chief executive officer of Celgene, said. “The 7,000 employees at Celgene are working every day on behalf of thousands of people suffering with this terrible disease.”

Celgene understands that patients are waiting for better treatment options and looking for information that will help them and their doctors guide their treatment choices. Celgene employees are focused on bringing new therapies and educating the pancreatic cancer community about the latest advances in the treatment of this deadly disease.

Many Celgene employees have experienced the devastation and feeling of helplessness caused by a pancreatic cancer diagnosis, through family members and friends. James Lajeunesse, hematology oncology consultant at Celgene, lost his father to pancreatic cancer. Because of his personal experience with his father’s pancreatic cancer journey, Lajeunesse said that he is “inspired to help healthcare providers understand the different treatment options.”

We’re making progress, and we will stay committed until we can find a cure because, after all, we know that patients are waiting.

Celgene has been at the forefront in the fight against pancreatic cancer and is dedicated to supporting patients and those who care for them. In partnership with 29 international patient groups, we launched the first-ever World Pancreatic Cancer Day in 2014. We were the first national sponsor for the Pancreatic Cancer Action Network’s PurpleStride events, which help to raise disease awareness as well as funds that support patients and advance research. Other initiatives we have supported over the years include the European Multi-Stakeholder platform on Pancreatic Cancer, the Pancreatic Cancer Forum in Milan and the “CancerCare Get You There” transportation assistance program.

Beyond supporting those patient group events and programs, Celgene has developed a personal information center for pancreatic cancer patients and caregivers at NavigatePanc.com. The site allows patients and caregivers to create their own personalized online libraries with information about the disease, clinical trials and support groups.

Above all else, however, pancreatic cancer patients are waiting for new treatment options. While researchers have made much progress, pancreatic cancer remains deadly, with less than 8 percent of patients living five years after being diagnosed.

Working with the pancreatic cancer community, Celgene is committed to improving the lives of those with pancreatic cancer by leveraging the latest science. Today, Celgene is conducting three global clinical trials for patients with various stages of pancreatic cancer and supporting more than 50 researcher-led studies in early stage and locally advanced disease.

“We’re making progress, and we will stay committed until we can find a cure because, after all, we know that patients are waiting,” Alles said.

To learn more about our commitment to the pancreatic cancer community, watch our “Patients are Waiting” video now.

Meghan Buzby, former senior director of advocacy at the International Myeloma Foundation, believes federal oral parity legislation is sorely needed.

Meghan Buzby, former senior director of advocacy at the International Myeloma Foundation, believes federal oral parity legislation is sorely needed.

Forty-two states have now passed laws that require insurers to provide parity in cost-sharing between what a patient pays to receive an intravenously administered cancer treatment and an orally administered cancer treatment. However, federal action is needed to protect the 93 million Americans whose employer-sponsored health plans are not state-regulated.

While states regulate health plans funded through insurers, the federal government regulates those where the employer assumes the financial risk for employee health benefits, otherwise known as self-funded plans. Today 63 percent of the 147 million covered American workers are enrolled in self-funded plans.

The state laws prevent cancer patients from paying more for oral medications than they would for therapies given intravenously at a treatment center. Cancer patients who face higher costs for their oral therapies are less likely to adhere to their treatment regimen, according to one study.

“Oral therapies are the future of cancer care, and we need to ensure that every patient has affordable access to those treatments,” Meghan Buzby, former senior director of advocacy at the International Myeloma Foundation, said. “We need oral cancer therapy parity laws in all 50 states and at the federal level.”

Oral cancer therapies are the future of cancer care, and we need to ensure that every cancer patient has affordable access to those treatments.

Last year, the House of Representatives and the Senate introduced bills that, if passed, would protect those 93 million Americans from outdated insurance practices. So far, a bipartisan group of 117 representatives and 20 senators have co-sponsored the Cancer Drug Coverage Parity Act.

“It’s a bipartisan effort. That’s something we’ve never seen before in our oral parity efforts,” Buzby said. The bill is positioned with strong bipartisan support to potentially be included in future health care legislation. “We’re making real strides in Congress this year.”

Why We Need Federal Oral Parity Legislation

Cancer patients and advocates can help by telling their senators and representatives to support H.R. 2739 and S. 1566. The International Myeloma Foundation has set up a   website to make it easy and has organized the Patient Equal Access Coalition where advocates can learn more about the bills.

Even if a federal oral parity law is enacted, additional action would be needed to provide the 56 million seniors covered under Medicare from unequal access. “We’ve been operating our advocacy efforts under the hope that if we pass this for commercial plans, then that would prove it could work in the Medicare population as well,” Buzby said.

Visit the International Myeloma Foundation’s Action Center today to ask your federal legislators to support federal cancer therapy coverage parity legislation.

By Ed Svec

Lymphoma survivor Ed Svec and his wife Susan have participated in Light The Night walks for the past seven years.

Lymphoma survivor Ed Svec and his wife Susan have participated in Light The Night walks for the past seven years.

For seven years, I have been participating in the Leukemia & Lymphoma Society’s Light The® Night walks in Long Island, New York. Sometimes I’m asked why I walk. I say, “I don’t do it for myself; I walk for future blood cancer patients.”

There’s no screening. There’s no known prevention. Our best hope today rests in supporting research to find cures and making sure all patients get the best support and care possible.

My hope is that the treatment for those diagnosed with blood cancer in the future won’t be as invasive, debilitating or emotionally draining as what I went through.

My lymphoma was a journey to hell and back again. After being diagnosed with non-Hodgkin’s B-cell follicular lymphoma in 2009, I underwent eight sessions of chemotherapy in just four months.

During that time, I wasn’t allowed to drive. And I couldn’t go into public places because my immune system was compromised; it was like being under house arrest.

I lost 55 pounds and every hair that I owned, and my toenails started to peel. I felt like a soft shell crab.

In the end, though, my lymphoma went into remission, and I survived. So I’m grateful to the people who were participating in these walks years before I got sick, raising funds and supporting research in blood cancer. It was partly through their efforts that my odds of surviving lymphoma were as good as they were.

What I went through pales in comparison to what my friend experienced when he was diagnosed with the same disease 15 years prior.

Like the people who walked a decade before I was diagnosed, now it’s my turn to pay it forward by raising funds to improve treatment for blood cancer in the future.

You can see how far we’ve come in the treatment of blood cancer at the Light The Night events. The sheer number that participate in these walks today is inspiring. Survivors of every age and background come to share their journeys and to carry their white lanterns with pride. Hearing those stories and seeing those white lights is one of the most uplifting things that someone currently struggling with blood cancer can experience.

That’s why I decided to share my story in front of a large crowd of patients and survivors at my local walk in Nassau County on Long Island, NY last year. Afterward, a couple introduced me to their six-year-old daughter. She was being treated for lymphoma at the time but didn’t want to talk about it with her parents or anyone else. I understand; I’m 63, and it’s tough for me to talk about it.

I knelt down beside her and asked her if she was okay. She said “yeah” and hugged me. It’s small interactions like this at these walks that can make big differences to patients who need to know they are not alone in their journey. We are fighting against this disease right along side them.

Ed Svec gave a speed to current patients and survivors during last year’s Light the Night walk in Nassau County, New York.

ED SVEC GAVE A SPEECH TO CURRENT PATIENTS AND SURVIVORS DURING LAST YEAR’S LIGHT THE NIGHT WALK IN NASSAU COUNTY, NEW YORK.

By supporting continued research, we can improve the lives of patients like that little girl and stop losing loved ones to blood cancers. In the meantime, we remember those we have lost during the Light The Night walks. Participants write down and display the names of people who have lost their battles in the Remembrance Tent. This year, I’m up to three names. I don’t want to add anybody else.

Like the people who walked a decade before I was diagnosed, now it’s my turn to pay it forward by raising funds to improve treatment for blood cancer in the future. I hope that you will join us for a walk this year and make a difference in the lives of blood cancer patients.

Discover how you can participate in an upcoming walk near you by visiting the Leukemia & Lymphoma Society’s Light The Night website

Discovering a new therapy is no easy task, and Esther Martinborough, executive director of research at Celgene, knows this firsthand. In 2008, she saw Receptos’ two therapeutic candidates for cancer fail before her team rallied to identify a potential new treatment option for multiple sclerosis (MS). Aided by the 2015 acquisition of Receptos by Celgene, her team is committed to bringing this potential new treatment option to MS patients.

In this Q&A, Martinborough explains that the company’s quick turnaround was due to two factors: the right team and a smarter way to screen therapeutic candidates.

Our Commitment to Multiple Sclerosis

ESTHER MARTINBOROUGH (FAR LEFT) EXECUTIVE DIRECTOR OF RESEARCH AT CELGENE< AND HER COLLEAGUES REVIEW DATA REGARDING THE COMPANY’S THERAPEUTIC CANDIDATE FOR MS.

What was Receptos’ original focus?

When the company was founded in 2008, we were called Apoptos and were focused on several exciting opportunities to develop anticancer therapies. After six months of preclinical research, it became apparent that we could not move forward. The scientific rationale was not holding together, and we explained this to our investors. Fortunately, our investors recognized we had a very qualified team with multiple individuals who had brought treatments to the market successfully. Based on that expertise, our investors put their trust in us and gave us another chance, so we worked as a team to carefully make sure our next hypotheses had the best chances of getting to the clinic. We were thorough, looking closely at multiple compounds and how they worked. We looked at a wide variety of candidates that were scientifically interesting.

Why did you choose to focus on a sphingosine-1-phosphate (S1P) modulator for MS?

There was a good biological rationale. Other S1P modulators were already in the clinic for MS, so we knew it could work. While the S1P compounds we evaluated weren’t quite ready for the clinic, we saw an opportunity to get them there quickly.

The S1P modulators that were ahead of us in clinical development, for the most part, had similar structures. We decided not to go down that same path.

How long has the development process taken?

We started working on our S1P modulator in March 2009 and were in the clinic less than two years later, which is quite simply astounding. When we began developing S1P modulators, about 20 other companies were ahead of us. Today, we’re one of the leaders in the pack.

How did you accelerate the development?

The difference was our screening approach. Most companies move step-by-step in screening candidates: they test the efficacy and then move onto studying the side effects and toxicology. We consolidated these steps into just one experiment. Using a biomarker for S1P as a preclinical measure of efficacy and lung weight in an animal model to measure safety, we were able to pick the therapeutic candidates with the right potential balance of efficacy and safety quickly.

ESTHER MARTINBOROUGH (RIGHT) AND A COLLEAGUE AT CELGENE DISCUSS THE CHEMICAL STRUCTURE OF THE COMPANY’S S1P MODULATOR THAT IS CURRENTLY IN CLINICAL DEVELOPMENT FOR MS.

ESTHER MARTINBOROUGH (RIGHT) AND A COLLEAGUE AT CELGENE DISCUSS THE CHEMICAL STRUCTURE OF THE COMPANY’S S1P MODULATOR THAT IS CURRENTLY IN CLINICAL DEVELOPMENT FOR MS.

What made this S1P modulator stand out from the others being developed?

The S1P modulators that were ahead of us in clinical development, for the most part, had similar structures. We decided not to go down that same path because that would likely only lead to the same safety and efficacy profiles. Instead, we designed chemical structures in our S1P modulator that were different from those already reported. We looked to map out a unique chemical space that could benefit MS patients.

Learn more about the role of S1P signaling molecules in MS and other immune-inflammatory diseases, read “Corralling White Blood Cells to Rein in Multiple Sclerosis.”

When recruiting patients for a clinical trial, it is important that researchers select the right mixture of people—in terms of age, gender and ethnicity—who they’re seeking to treat with a new therapy or treatment intervention. This variety is critical for researchers to understand how treatments impact patients differently. Unfortunately, achieving diversity in clinical trials continues to be a challenge.

While African Americans make up over 20 percent of newly diagnosed multiple myeloma cases in the United States, they represent only 8 percent of cancer clinical trial participants. When it comes to multiple myeloma, a cancer of a type of white blood cells called plasma cells, African Americans are twice as likely to be diagnosed with the disease, which makes it even more important that they are better represented in myeloma clinical trials.

Meanwhile, African Americans are more likely than any other racial group to be diagnosed with and die from cancer. African Americans have also benefited less from advances in myeloma treatment over the past two decades than whites, suggesting unequal access to or response to treatments.

African Americans & Myeloma Clinical Research

“Without a doubt, African Americans are underrepresented in myeloma trials,” Brendan Weiss, an assistant professor of medicine at the University of Pennsylvania who specializes in myeloma, said. “Now that new tools like next-generation DNA sequencing can shed some light on the genetic diversity in myeloma, studies should absolutely do the best they can to try to get diverse representation.”

Understanding the genetic differences between African American patients and other patients with myeloma could go a long way in explaining the differences among different patient groups in how the disease progresses and responds to treatment. As a result, doctors may get more insight into how to treat the disease most effectively for each group.

Alfiee Breland-Noble, an associate professor of psychiatry at Georgetown University, explains why minorities have a mistrust of the American medical system.

Alfiee Breland-Noble, an associate professor of psychiatry at Georgetown University, explains why minorities have a mistrust of the American medical system.

But recruiting African Americans for trials has been difficult. In one 2013 survey, African Americans were more likely than any other racial group to cite lack of trust as a reason not to participate in a clinical trial and to believe that patients are sometimes enrolled in trials without their consent.

“African Americans have a historical mistrust of the medical system,” said Alfiee Breland-Noble, an associate professor of psychiatry at Georgetown University who studies disparities in clinical trials. “A lot of that mistrust comes from people feeling like they’re not treated equally in the health care system.”

To boost enrollment of African Americans in clinical trials, including those of myeloma therapies, clinicians have to build an environment of trust. Clinics must be proactive in communicating the value of clinical trials consistently—with pamphlets in the waiting room, for example, not just when recruiting patients—so people can begin to learn why participation is so vital to improving the understanding of disease and treatments for everyone.

The message we want clinicians to get across to African American patients is: you are needed, you are important, and we need to know how these medications impact people like you.

For instance, one study found that providing patient navigators to help guide patients and answer their questions about the process raised the enrollment rate of eligible African American cancer patients to about 80 percent. Another group found that African Americans who participated in a clinical trials education program through their local church were almost three times as likely to enroll in a clinical trials registry.

“The message we want clinicians to get across to patients is: you are needed, you are important, and we need to know how these medications impact people like you so that we can help other people like you,” said Breland-Noble.

Diversity among researchers, clinicians and clinic staff can also help patients of various backgrounds feel more comfortable throughout their care. It’s a sentiment that Celgene’s Standing in the Gaap initiative is already embracing, with efforts to boost the numbers of students pursuing cancer research and medicine at historically black colleges and universities.

By making the field of myeloma care and research more diverse, the campaign aims to make African American patients feel more included and more likely to participate in clinical trials. It’s only then that researchers can study the full links between genetics and myeloma.

Discover more information about how myeloma affects African Americans on the Standing in the Gaap Facebook page.

After 56 hours of running, New York City businessman and ultramarathoner Eric Gelber realized he was barely moving. It was his second attempt to run 200 miles around Central Park to raise awareness and funds for research towards a myeloma cure, and the last 2.5 miles took four hours to complete. At that pace, he’d be running for another 24 hours.

After 176 miles in two and a half days, he was done. Although the effort beat the 164 miles he ran during his first attempt the previous year and raised $240,000 for myeloma research, Gelber felt defeated. “I feel like I failed,” Gelber admitted. “But I know that no one else feels that way.”

So to commemorate this year’s Blood Cancer Awareness Month, Gelber is making one last attempt to run 200 miles around Central Park and raise an additional $250,000 for myeloma research during September 16-18, 2016. Hitting that goal will put him past $1 million in total money raised on behalf of the Multiple Myeloma Research Foundation (MMRF), an organization whose sole mission is finding a myeloma cure, since his efforts began nearly a decade ago.

In 2007, Gelber’s long-time family friend Anita Sorrell received a stem cell transplant as part of her treatment for multiple myeloma, a rare cancer of plasma cells that help our bodies fight infection. The disease can lead to low blood counts, bone and kidney damage and infections. While advances in our understanding of the disease have lead to progress in treating myeloma over the past decade, a cure has remained elusive. “Although the disease affected her physically, it never dampened her spirit,” Gelber recalled.

Gelber wanted to support his friend during her journey with myeloma. He signed up for the New York City Marathon that year to raise money for the MMRF. He raised over $6,000 in that first effort.

She told me that when she wore the medal, she felt the meds going straight into her veins. That motivated me to do more.

After the marathon, he gave Sorrell his medal. She carried it around everywhere in her purse and wore it during her chemotherapy treatments. “She told me that when she wore the medal, she felt the meds going straight into her veins. That motivated me to do more.”

He realized that by running ultramarathons—races longer than the traditional marathon length of 26.2 miles—more people donated, and previous donors gave more. A 2011 155-mile solo run in the Catskill Mountains in New York raised $35,000. A year later, he competed in the Badwater Ultramarathon, a 135-mile race from Death Valley to Mount Whitney in California, and raised $65,000.

With every mile, Eric has taken strides to raise greater awareness and funds in the hopes of finding a cure for myeloma. By fundraising that drives research through the MMRF, Eric feels he is doing all he can to support patients and find the quickest way to a cure.

In 2012, Sorrell lost her battle with myeloma. Devastated by the loss, Gelber’s resolve to help find a cure was strengthened. He decided to bring his efforts to New York City where more people could see and participate. That’s when the idea for “The Journey Towards a Cure,” 200-mile ultramarathon, was born.

Myeloma patient Pam gives Eric Gelber a hug during his 2014 attempt to run 200 miles around Central Park. She flew from the Midwest to New York to support the event that raised funds towards a myeloma cure. Source: Multiple Myeloma Research Foundation

MYELOMA PATIENT PAM GIVES ERIC GELBER A HUG DURING HIS 2014 ATTEMPT TO RUN 200 MILES AROUND CENTRAL PARK. SHE FLEW FROM THE MIDWEST TO NEW YORK TO SUPPORT THE EVENT THAT RAISED FUNDS TOWARDS A MYELOMA CURE. SOURCE: MULTIPLE MYELOMA RESEARCH FOUNDATION

While exhausting, the first two attempts have been very rewarding for Gelber beyond the funds and awareness raised. During the runs, he has met people living with myeloma who come out just to shake his hand, thank him or run a lap with him. They tell him that his endeavors give them hope.

Gelber is looking forward to seeing their familiar faces and new ones during his third and final attempt this month. He knows that it may be his most challenging yet. It will be his first ultramarathon after undergoing hip surgery last December.

“People ask me about the parallels between what I do and those fighting cancer. That’s one I struggle with because I have a choice. They don’t,” he said. “People have good and bad days. You have to get up and fight.”

To find out how you can support Gelber’s final attempt by signing up to run a mile with him, cheering him on or donating to the cause, visit the Multiple Myeloma Research Foundation’s website.

University of Southern California professor Dana Goldman believes that health care today suffers from short-term thinking.

University of Southern California professor Dana Goldman believes that health care today suffers from short-term thinking.

In the United States, over 14 million people are living with cancer, and that number is expected to increase to 18 million by 2020. Thanks to many decades of advances in therapies, someone diagnosed with cancer today now has a 67 percent chance of living five years or longer. With the promise of immunotherapies and cancer moonshot efforts, a sense of optimism that we can defeat cancer is growing.

With cures on the horizon, now is the time to invest in preparing the health care system for the economic impact. While reimbursement strategies currently focus on costs and benefits of a treatment in the short-term, a year or two, we will need a long-term view to realize the true benefits of medical advances.

“We grossly underinvest in disease prevention and cures,” Dana Goldman, director of University of Southern California’s Schaeffer Center for Health Policy and Economics, said. “When viewed through a long-term lens, these treatments may be well worth the money, but people are focused on the cost per pill, which is hardly the right metric.”

This focus on immediate costs is what Goldman calls “short-termism” in health care. In a Harvard Business Review article, he and co-author Amitabh Chandra, the director of health policy research at the Harvard Kennedy School of Government, argue that the problem stems from the way patients, employers, health care providers and even the government are overly concerned with immediate costs and take a shortsighted view of benefits.

When viewed through a long-term lens, these treatments may be well worth the money, but people are focused on the cost per pill, which is hardly the right metric.

The reaction to breakthrough hepatitis C treatments is an excellent example of short-termism, according to Goldman. The newest treatments not only cure infected patients but also prevent the virus but being spread, reducing long-term medical costs for both the treated patient and everyone protected from future infection.

Health economists believe a value-driven system is needed to address this issue. For cancer, such a system would recognize the value of a cure while also taking into account the price of the therapy.

Recognizing the Long-term Value of Good Cancer Care

One way to move toward a value-driven system may be to lengthen insurance contracts. “There is nothing magical about a one-year contract; perhaps we could go to three years to start,” Goldman said. “To protect patients, we could provide a way for patients to change plans sooner if they believe they are being harmed by theirs.”

Other suggestions offered by Goldman involve side payments between plans. If a patient with cancer moved between plans after an expensive treatment, for example, the new insurer would pay the original for a portion of the cost of that treatment. Conversely, if the original plan denied the patient access to an expensive but valuable therapy, they would be required to compensate the new insurer.

Another idea involves insurers making a series of payments to pharmaceutical companies for as long as a patient remains in remission. “This annuitizes the high upfront cost and makes price dependent on performance,” Goldman said. “We are seeing the private sector take steps in these directions.” For instance, insurers Cigna and Aetna have both made similar deals for a new heart-failure treatment.

Regulatory issues regarding the length of insurance contracts and payments between insurers as well as the current inability of insurers to track treatment results over time are major roadblocks to revamping the health care system but can be overcome, according to Goldman. A far greater obstacle may be changing how we think and process information—and overcoming our tendency to favor emotionally driven short-term gains over more reasonable long-term goals.

“While we are starting to understand the biology behind decision-making, we can’t do much about it yet,” Goldman explained. “My rational brain—like everyone else’s—tells me that I shouldn’t eat doughnuts, but I always end up at the doughnut shop.”

Myeloma doctors, patients and advocates are speaking out about a new report being developed by the Institute for Clinical and Economic Review (ICER) to determine whether innovative myeloma therapies are worth the money. The institute claims the report will help insurers make more cost-driven reimbursement decisions, but critics say that it will only limit treatment options and shorten lives.

Rafael Fonseca, Chair, Department of Medicine at Mayo Clinic in Arizona, believes that the Institute of Clinical and Economic Review’s report on innovative myeloma therapies is misguided.

Rafael Fonseca, Chair, Department of Medicine at Mayo Clinic in Arizona, believes that the Institute of Clinical and Economic Review’s report on innovative myeloma therapies is misguided.

Rafael Fonseca, Chair, Department of Medicine at Mayo Clinic in Arizona, believes that the Institute of Clinical and Economic Review’s report on innovative myeloma therapies is misguided.

The American Society of Hematology, the Cancer Support Community and the Multiple Myeloma Research Foundation (MMRF) are among the many organizations in the community that have voiced their concerns about the ICER report since the initial draft was released in April and at a public meeting held to discuss the report last month. A subsequent version was issued in May, and the final report is expected to be published in June.

Rafael Fonseca, M.D., Chair, Department of Medicine at Mayo Clinic in Arizona has written a formal critique of the report. “The important question here is, ‘Value for whom?’ It’s certainly not the patient,” he said. “The institute has framed the report around the notion of how insurance companies can get the best value for treatments, so the intent here is to ultimately limit treatment options.”

The institute claims its report will incorporate perspectives from all health care stakeholders, but whether they will follow through remains to be seen. The MMRF, for example, expressed “serious concerns” with the institute’s efforts back in March—about a month prior to the report’s release, in response to a direct inquiry from ICER for feedback. The draft version of the report did not address these concerns, and the MMRF has gone out of its way to clarify its limited involvement.

While ICER revised its report to clarify some stakeholder input and data sources, many concerns have largely been disregarded. The institute claims that patients, doctors and payers must make decisions today based on the evidence at hand. But for doctors, that evidence includes not just published data but also ongoing trials and their own clinical experience.

ICER & the True Value of Innovative Myeloma Therapies

“Overall, the report in its current state includes minimal input from stakeholders within the myeloma medical or patient communities,” Fonseca said. “By no means does this report accurately reflect current clinical practices in myeloma today.”

Over the past decade, new therapies for myeloma have helped patients live longer, but doctors are still trying to figure out how to best treat patients with all the options now available, including testing new combinations.

“Anyone would be really challenged to assess the value of myeloma individual therapies at this point in time,” Fonseca said. “We are still learning about these treatments on the medical side, so pretending that we could begin factoring in economics into the equation is pure nonsense.”

We are still learning about these treatments on the medical side, so pretending that we could begin factoring in economics into the equation is pure nonsense.

Brian Durie, a multiple myeloma specialist at the Cedars-Sinai Medical Center and chairman of the board of the International Myeloma Foundation, believes that myeloma medical experts—not ICER—should be providing treatment recommendations. He notes that the International Myeloma Working Group, which consists of over 200 experts, will meet this June to develop guidelines that will allow for an individualized treatment approach, based on a patient’s unique characteristics and preferences, that evolves as the disease progresses.

Adaptation and adjustment are essential in myeloma treatment today. It’s a complex disease with multiple genetic factors that make each patient’s case unique, and it requires an individualized, malleable approach. Attempting to generalize the effectiveness—let alone cost-effectiveness—of a therapy across the board for all myeloma patients doesn’t account for the realities of the disease.

The ICER assessment also fails to capture the total cost of care and the economic benefits of these medicines. New therapies can reduce costs for doctor’s visits and hospitalizations and allow patients and caregivers to be more productive and to contribute to society. Last year, a study found that providing the most appropriate myeloma treatment option delayed relapses and the accompanying spikes in costs, making those therapies cost-effective in the long run.

“Total cost of care is almost never looked at,” said Fonseca. “We need to start defining value in other ways than just costs. We need to focus on the value of myeloma therapies to individual patients.”

Learn more about our take on the method used by ICER to assess the value of innovative treatments in our related story.

Health care costs are on the rise, and the reason isn’t complicated. People today are living longer, and older people use more health care resources. People aged 65 and older already make up 8.5 percent of the world’s population, and that number is expected to reach nearly 17 percent by 2050.

Michael Mandel, Chief Economic Strategist of the Progressive Policy Institute, says health spending has increased mainly because we have more health care workers today.

Michael Mandel, Chief Economic Strategist of the Progressive Policy Institute, says health spending has increased mainly because we have more health care workers today.

As a result, more and more health care workers will be needed. The demand will be so great, in fact, that we may not be able to keep up; just last month, the Association of American Medical Colleges projected that the United States could face a shortage of up to 94,700 physicians by 2025. To solve that problem, medical innovation will play a vital role by making people healthier and bringing down health care costs in the long-term.

However, when U.S. policymakers talk about reining in health care costs, all too often, they focus on prescription medications, although the biggest driver of spending has been and continues to be labor.

“When you look at the data, the cost increases have more to do with the hiring of health care workers than excess spending on either equipment or medications,” Michael Mandel, chief economic strategist at the Progressive Policy Institute. “Health care is labor-intensive and becoming increasingly so.”

The cost of labor represents more than 40 percent of the increase in government and private health care spending since 2007, according to the Institute’s estimates. Prescription medicines, on the other hand, represent just 10 percent of the increase. In fact, the Centers for Medicare and Medicaid Services reported that how much people pay for prescription drugs has dropped by 14 percent since 2007.

Labor: The Biggest Driver of Health Care Spending

“It’s easy to propose cuts in prescription medications to control costs, but that’s short-sighted,” said Mandel. “Policymakers need to realize that would be moving in the wrong direction.”

A good example can be found in the new hepatitis C therapies, which are forecasted to reduce the number of costly liver transplants by up to 26 percent. The problem is private insurance companies can’t see that far ahead; they don’t know whether a hep C patient taking a medication today will still be on their plan years from now when that transplant is avoided.

It’s not just private insurers that are missing the big picture; state-run Medicaid programs have also been withholding treatments from patients.

Policy makers should invest now in solutions that end up saving money, not to mention improving lives. The people running the federal Medicare program understand this imperative. While prescription medications account for just 10 percent of the program’s budget, they are responsible for 60 percent of the slowdown in Medicare spending since 2011.

In 2015, the program spent $9 billion to ensure access to hepatitis C treatments. “If you think about it, these are drugs that can really prevent liver disease,” Sean Cavanaugh, deputy director for Medicare with the Centers for Medicare and Medicaid Services, told the Associated Press. “We felt it was most appropriate to allow patients to have them earlier.”

If we don’t find a way to support innovation, we will have an enormous problem in health care

The millions of Americans who are not on Medicare deserve the same. Policy makers should focus on the long-term picture and consider ways to subsidize private and state insurers for therapies that offer better outcomes. That investment would pay off when more people on Medicare require fewer visits to the doctor’s office. Considering we may not have enough doctors to go around soon, the investment seems like a sound decision.

“If we don’t find a way to support innovation, we will have an enormous problem in health care,” said Mandel. “We need to think differently than we are today; we need to start thinking long-term.”

As the large baby-boomer generation continues to age, cancer cases are on the rise. Add to that the increased success of cancer treatments, and more people are living with cancer instead of dying from it.

It makes sense then that people are concerned that cancer care could be driving up overall health care spending, especially as insurers are asking patients to pay a higher percentage of the bill than ever before. But cancer care costs have been increasing at the same rate as other health care spending, according to a new study published by the Community Oncology Alliance (COA).

Community Oncology Alliance executive director Ted Okon says that many are surprised that spending for cancer is not rising faster than for overall health care. 

Community Oncology Alliance executive director Ted Okon says that many are surprised that spending for cancer is not rising faster than for overall health care.

Actuarial firm Milliman conducted the study, which looked at Medicare and commercial insurance data from 2004 to 2014. The analysts found that the per-patient costs increased at similar rates for those who did and those who did not receive cancer treatment.

Medicare costs rose 36 percent over the 11-year period for patients treated for cancer, and 35 percent for those not treated for cancer. For commercial insurers, the increases were 63 percent and 61 percent, respectively.

“The results have startled not only the oncologists involved in the study but also outside physicians who have seen the results,” Ted Okon, executive director of the COA, said. “None of us could have predicted this going into the study.”

Cancer Costs Not Outpacing Other Health Spending

The study authors further divided up cancer care costs by treatment type, treatment location and other factors. They found that while spending on biologics rose 335 percent for Medicare patients, these therapies still represented only 9 percent of total cancer care costs in 2014. At the same time, the percentage of cancer care costs that went to surgery and hospitalizations fell 27 percent and 11 percent, respectively. The opposing trends are likely no coincidence; previous studies have shown that new therapies can decrease spending in other health care expenses and reduce overall cancer care costs.

And obviously, these new therapies are also helping patients. Debra Patt, a practicing oncologist at Texas Oncology and a study team member, has noticed the positive impact on her patients firsthand. A decade ago, she told patients with myeloma that they had less than three years to live on average. But with new treatments options, half of patients diagnosed with this blood cancer today are living six years or longer, and some are living up to 10 and 20 years.

They’re expensive therapies, but you have to understand them in context

Oncologist Debra Patt believes using more cancer therapies provides significant economic value and improves patient lives.

Oncologist Debra Patt believes using more cancer therapies provides significant economic value and improves patient lives.

The better prognoses also mean patients may be taking some medications longer. That increases short-term costs, but it does offer a return on investment in terms of patient productivity and reduced hospital and physician fees. “They’re expensive therapies, but you have to understand them in context,” Patt said.

In the context of the COA study, total care spending has actually increased at a slower rate than the number of people who have been diagnosed with cancer. Cancer diagnoses in the Medicare population have increased 16 percent, while spending has only increased 7 percent. In the commercially insured population, the difference is even starker: 39 percent versus 14 percent, respectively.

Cancer Cases Rising Faster Than Costs

For Patt, the take-home message is that cancer care costs are increasing proportionately to other health spending. “We need to look at cancer data continually to better understand where we’re spending our money, and how to work together to design programs to give us better value.”

As the demand for health care increases, so does the need for smart, evidence-based decision making in health care. Yet a cost-effectiveness measure that has several shortcomings is the basis for a reimbursement decision-making model proposed by the Institute for Clinical and Economic Review (ICER).

While that method—called quality-adjusted life years (QALY)—is one useful tool for guiding health care decisions, it becomes problematic when we focus solely on this factor and generalize its applicability across disease states. If insurers and policymakers continue to make these missteps, patients will lose access to life-changing medicines.

Ariel Beresniak, founder and health economist at Data Mining International, believes quality-adjusted life years (QALYs) should not be the only measure used for health care decision-making.

Ariel Beresniak, founder and health economist at Data Mining International, believes the quality-adjusted life years (QALYs) should be abandoned for health care decision-making.

“Using QALYs for cost-effectiveness analyses of new medicines is problematic,” Ariel Beresniak, founder and health economist at Data Mining International, said. “This approach is quite unfair and discriminatory.”

Beresniak is not alone in this indictment. Health economists have been pointing out the drawbacks of QALYs ever since the formula was introduced in the 1980s. Over the years, QALYs have been shown to be too narrowly focused on patients’ physical health, ignoring benefits to their emotional and mental health, their careers and families and other societal benefits. QALYs also discriminate against the elderly, patients with chronic diseases and those who have limited treatment options. Finally, the approach is widely inconsistent when used to make health decisions.

The inconsistencies stem from how QALYs are calculated. The formula considers two factors: the length and quality of the extra life that a treatment offers. So if a treatment allowed a patient to live two years longer in perfect health, the benefit would be two QALYs. But if those two years were spent in a wheelchair, that might be reduced to only one QALY.

The formula is simple, maybe a little too simple, according to Beresniak. It’s based on the assumption that people generally agree about how they would prefer to live. But health economists have questioned whether two years in a wheelchair is actually equal to one year in perfect health in everyone’s eyes.

It’s impossible to get a consensus on patient preferences to justify using QALYs.”

To address this issue, Beresniak led a three-year, 1 million euro project funded by the European Union Commission. After interviewing 1,300 people in Belgium, France, Italy and the United Kingdom, the team found their preferences could not be simplified in the QALY approach.

“It’s impossible to get a consensus on patient preferences to justify using a QALY approach,” Beresniak said. “Countries who use this approach to make health care decisions are only delaying patients access to innovative medicines. It is anything but scientific.”

Beresniak likens the QALY approach to equating two 40-degree days with one 80-degree day. The relative enjoyment of the two days is a personal preference, not something we can come to a consensus about, he notes.

Looking at QALYs Is Not Enough

Beresniak believes that instead of relying on QALY assessments, insurers and policymakers should be more flexible with how they measure the value of new medicines by considering cases on an individual basis. As a result, health care decisions may better align with how patients and society actually value treatments for these specific conditions.

For instance, clinical trials have developed a variety of meaningful measures of outcomes and health-related quality of life for people with psoriasis and psoriatic arthritis, including the Psoriasis Severity Index and the American College Rheumatology Response Criteria. Yet many of these measures are not typically incorporated into cost-effectiveness analyses.

“We need to be more precise when making reimbursement decisions,” Beresniak said. “We should look at relevant clinical outcomes, and we should be disease-specific. We cannot only rely on QALYs to make health care decisions. It is time to use a number of alternative approaches which are much more robust to express the real value of innovative products.”

Neurologist Bruce Cree at the University of California, San Francisco Medical Center believes that more therapies are needed to help MS patients remain independent.  

NEUROLOGIST BRUCE CREE AT THE UNIVERSITY OF CALIFORNIA, SAN FRANCISCO MEDICAL CENTER BELIEVES THAT MORE THERAPIES ARE NEEDED TO HELP MS PATIENTS REMAIN INDEPENDENT.

As the theme of this year’s World MS Day is “independence,” it’s fitting to remember that for the more than 2 million people around the world with multiple sclerosis (MS), small gains in treatment effectiveness can mean big gains in quality of life and independence.

MS is a chronic neurological disorder that’s usually diagnosed in early adulthood—between the ages of 20 and 40—and that often gets progressively worse over the following decades. Symptoms vary over time and among patients but include fatigue, vision and speech problems and impaired coordination—all of which can make doing everyday activities like eating, bathing and dressing challenging.

In one survey, 65 percent of people with MS said mobility was the greatest challenge related to the disease. Others ranked fatigue, pain and muscle spasms as the most debilitating aspects. Indeed, 80 percent of MS patients will experience some degree of impaired mobility within 10 to 15 years of being diagnosed with the disease.

“MS tends to affect people at the prime of their lives when they are busy working and raising families,” Dr. Bruce Cree, a neurologist at of the University of California, San Francisco Medical Center, said. “As a consequence of the neurological impairments of the disease, people often lose their ability to work.”

World MS Day 2016: Toward a Greater Sense of Independence

Indeed, 55 percent of patients in one study were unable to stay employed following diagnosis. The economic costs of the disease—including medical care and lost productivity—are huge, estimated at $10 billion per year in the United States alone.

Over time, patients become reliant on family and caregivers to help them carry out even the most basic tasks, like going to the bathroom. Depression, social isolation, strained family relationships and divorce are all common in MS patients, according to Dr. Cree.

There is no cure for MS, but a growing number of therapies help prevent symptom flare-ups and slow the progression of the disease. While most previous MS drugs were injections, some newer therapies come in convenient pill form.

New therapies can certainly improve the quality of life for many patients.

“New therapies can certainly improve the quality of life for many patients,” said Cree. “Not only have studies found that patients greatly prefer pills to injections.”

Still, no existing treatment addresses the greatest burdens of the disease for patients with advanced MS—fatigue and cognitive impairment.

“An MS patient might look like everyone else,” said Dr. Cree. “There’s not always something on the surface that tells you they have MS. But their quality of life can be greatly impacted by aspects of the disease that are not obvious.”

To improve the quality of life, future therapies to treat MS must not only slow the progression of disease but also enable patients to be more independent and boost quality of life.

For many years, inflammatory bowel disease (IBD) was a disease of the Western world—most diagnoses occurred in Europe, North America and Australia. Today, however, IBD incidence is on the rise on nearly every continent.

Some 5 million people around the world have IBD, and in the United States alone, the disease is estimated to cost the healthcare system more than $6 billion a year. In the fast-growing populations of China and India, rates of the two main subtypes of IBD—ulcerative colitis and Crohn’s disease—are beginning to rise.

“In countries where these diseases have been ignored in the past, we’re now seeing them appear for the first time,” Dr. Jean-Frederic Colombel, director of the Susan and Leonard Feinstein IBD Clinical Center at Icahn School of Medicine at Mount Sinai, said. “It’s becoming a worldwide epidemic.”

Inflammatory Bowel Disease: A Growing Global Problem

IBD is characterized by inflammation of the digestive system that can cause chronic nausea, diarrhea, cramping and weight loss. The disease often surfaces when patients are in their teens, twenties or thirties, leading to many years of pain and disability as well as costly monitoring.

First described in the 1930s,IBD has recently become much more common. Most studies suggest a significant rise in the number of cases of Crohn’s disease and ulcerative colitis worldwide since 1960. Between 2000 and 2009, the number of children hospitalized with IBD in the United States jumped by 64 percent.

The current hypothesis is that changes in lifestyle are making people more susceptible to IBD. These changes could include diet, cleanliness, and pollution exposure.

Scientists don’t fully understand what causes IBD, although studies suggest that genetics and bacteria that live in the human gut may be involved. “The current hypothesis is that changes in lifestyle are making people more susceptible to IBD,” explained Colombel. “These changes could include diet, cleanliness and pollution exposure.” These factors can cause changes in the gut bacteria, increasing the risk for IBD.

While the causes remain unclear, a cure has been even more elusive. Although surgery is an option, colectomy for ulcerative colitis is associated with significant morbidity, and Crohn’s disease may recur even after surgical resection of the small bowel or colon. As a result, most patients turn toward medicines to manage their disease, and new treatment options are being explored in clinical trials.

Gastroenterologist Dr. Jean-Frederic Colombel has witnessed the global rise of inflammatory bowel disease first hand at Mount Sinai in New York City.

Gastroenterologist Dr. Jean-Frederic Colombel has witnessed the global rise of inflammatory bowel disease first hand at Mount Sinai in New York City.

“We have made a lot of progress with the treatments we have now,” Colombel said. “We can heal some patients and block the progression of the disease towards bowel damage and disability.”

But Colombel stresses that early detection is critical to improving outcomes in IBD.

“There is a window of opportunity for treatment; when you miss this window, it becomes much more difficult to manage,” Colombel said. “Unfortunately, there is often a long delay between the onset of symptoms and when a patient is actually diagnosed.”

In fact, it usually takes more than a year—and sometimes a few years—for people to be diagnosed after their symptoms appear. In countries where IBD is a more recent phenomenon, this delay may be even longer.

Patients and primary care physicians need to be aware of the signs of IBD, so a proper diagnosis can be made. Gastroenterologists around the world must also be more aware and better equipped to treat the growing and complex burden of IBD.

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Beta-thalassemia patient Louis Pericleous has raised awareness of the disease by speaking at conferences and events.

Beta-thalassemia patient Louis Pericleous has raised awareness of the disease by speaking at conferences and events.

For the past 50 years, Louis Pericleous, who lives in Cyprus, has been in and out of the hospital nearly every week to receive life-saving blood transfusions. He is one of hundreds of thousands of people worldwide living with beta-thalassemia, a genetic disorder that causes profound anemia, in which the body does not make enough red blood cells to carry oxygen to support the body. Even with blood transfusions, patients may develop hypothyroidism, diabetes, lung problems and cancer as a result of their disease and the transfusions used to treat it. Beta-thalassemia can lead to an early death for some patients, and there is a clear need for new therapies.

There is no approved drug to treat the anemia of beta-thalassemia. The only cure for beta-thalassemia is a bone marrow transplant, but finding a match is typically limited to a small percentage of patients. So most patients live like Pericleous—relying on transfusions, typically every two to four weeks, which provide healthy red blood cells from a donor, and constantly keeping a close eye on their health.

“We really depend on the kindness and love of other humans,” Pericleous, a former Board Member of Thalassaemia International Federation and former President of the Cyprus Thalassaemia Association, said. “If people didn’t donate their blood, we couldn’t survive.”

Beta Thalassemia: Current Treatments Not Enough

Without treatment, affected individuals may die early due to diseases such as heart failure. And while regular transfusions lengthen lifespan and ease some symptoms, the constant blood transfusions can cause iron overload (too much iron in the body), which can lead to heart and liver problems and hormone imbalances unless patients also take iron-chelation therapy.

In developing countries, the standard of care is often sub-optimal. In lower socio-economic regions, fewer people donate blood, and the donations that are made are often not thoroughly screened for infectious diseases such as HIV and hepatitis or antigens that prod the recipient’s immune system to attack the new red blood cells.

“It’s amazing that in this day and age people cannot access or afford quality treatments,” Pericleous said. “In areas other than the United States, Canada and Europe, thalassemia remains a deadly disease.”

Thomas Coates, a hematologist at the Children’s Hospital of Los Angeles, says access to appropriate care is essential for beta thalassemia patients.

Thomas Coates, a hematologist at the Children’s Hospital of Los Angeles, says access to appropriate care is essential for beta thalassemia patients.

Physicians who have little experience with thalassemia may also give subpar treatment, raising the risk for complications. “To protect patients from iron overload, doctors sometimes don’t transfuse as often enough,” Thomas Coates, a hematologist at the Children’s Hospital of Los Angeles, said. “Inadequate transfusion increases the risk of certain complications of thalassemia and results in chronic fatigue.” Beyond fatigue, under-treatment of anemia can hinder growth and even lead to heart failure.

Beta thalassemia also affects patients on a psychological and social level, according to a 2014 study. For instance, patients may be of short stature or have bone deformities or impaired fertility—all of which could affect how they think of themselves and how they think others see them.

Managing this disease without blood transfusions and iron chelation remains a dream for most of us.

For Pericleous and others like him, improving their lives means freeing them from the constraints and complications of transfusions. “It would be ideal to find a substitute such as a pill or a tablet instead of a blood transfusion,” Pericleous said. “But managing this disease without blood transfusions and iron chelation remains a dream for most of us.” Now it’s up to researchers and clinicians to help them achieve that dream.

To do their part, Celgene and Acceleron have initiated a global clinical study for patients with beta-thalassemia. More information about this study as well as others in beta-thalassemia can be found at ClinicalTrials.gov.