Blog Archives

Measuring the effectiveness of treatments for multiple sclerosis (MS) is complicated; the disease biology is not entirely understood, and symptoms vary from person to person. While doctors and researchers continue to explore new endpoints for clinical trials that evaluate MS therapies, measuring relapse rates remains one of the most common.

Neurologist Enrique Alvarez, M.D., Ph.D., at the University of Colorado, Denver, discusses the importance of continuing to evaluate potential new therapies based on their ability to reduce relapse rates, along with newer measures that are bolstering these evaluations.

How do researchers measure the efficacy of new treatments for MS?

NEUROLOGIST ENRIQUE ALVAREZ, M.D., PH.D., AT THE UNIVERSITY OF COLORADO, DENVER, EXPLAINS WHY RELAPSE RATES HAVE REMAINED A BENCHMARK IN MS TRIALS.

NEUROLOGIST ENRIQUE ALVAREZ, M.D., PH.D., AT THE UNIVERSITY OF COLORADO, DENVER, EXPLAINS WHY RELAPSE RATES HAVE REMAINED A BENCHMARK IN MS TRIALS.

“Relapse rates are a benchmark for measuring the efficacy of new MS treatments in clinical trials. A relapse occurs when a new neurological symptom emerges or an old symptom gets worse for at least 24 hours. MS can cause a variety of symptoms such as vision loss, pain, fatigue or impaired coordination.”

“Signs of a relapse can be reported by the patient, and may have an immediate impact on the quality of a patient’s life. Relapse rates also tend to go hand-in-hand with disability rates; relapses are often associated with some lasting disability.”

How else are researchers measuring efficacy in MS?

“We have set criteria about how to determine a relapse, but these assessments are often subjective and can be ‘noisy.’ This noise can be associated with a pseudo-relapse, which is the return of an old symptom because of factors unrelated to MS, such as stress, fever or an infection. MS symptoms can also fluctuate throughout the day, affecting a patient’s assessment.” 

“The challenge is finding a way to measure clinical events across all the patients in a study. For example, how do you compare a patient who might have bladder function issues with a patient who has vision loss? That type of comparison remains a challenge.”

These newer measurements are valuable, but we still need to look at relapse rates.

How are researchers overcoming the challenges of measuring efficacy in MS trials?

“We have been including more objective, less noisy measures such as MRI [magnetic resonance imaging] metrics in trials. MRI measurements can serve as a substitute for clinical outcomes reported by patients.”

“In MS, the body’s immune system causes inflammation and damage in the brain, resulting in scar tissue, which we call a lesion. MRI can measure new and growing lesions in the brain. The more lesions a patient has, the more likely he or she will experience worsening symptoms and future relapses.” 

SECONDARY MEASUREMENTS SUCH AS MRI BRAINS SCANS CAN HELP MEASURE THE EFFICACY OF NEW MS TREATMENTS.

SECONDARY MEASUREMENTS SUCH AS MRI BRAINS SCANS CAN HELP MEASURE THE EFFICACY OF NEW MS TREATMENTS.

What are some newer measures that are being explored?

“Newer measures are exploding. We’re starting to see a host of cognitive testing measures. Balance is being evaluated more. There are kinetic measures, like those you can find on a smartphone app, to see how much the patient is walking. We’re trying to get a sense of how the patient feels their life is changing on the tested therapy. You lose a little of the objectivity that you get with a measurement like MRI, but you gain a better sense of how the patient feels they’re doing.”

Could these newer measurements replace relapse rates as a benchmark?

“These newer measurements are valuable, but we still need to look at relapse rates. Rather than replace them, we’ll be more likely to see combinations of multiple endpoints used to determine the efficacy of tested therapies in trials. The more measurements, the better.”  

To learn about how relapses can affect the lives of people living with MS, read “World MS Day: MS Doesn’t Stop Me from Living a Life I Love.”

Many of the 1.6 million Americans living with inflammatory bowel disease (IBD) struggle to find an effective treatment, leaving them with pain, fatigue and other symptoms that directly affect their lives but may not be obvious to others. Although more than 380 active clinical trials are exploring investigational treatment options for the two most common forms of IBD—Crohn’s disease and ulcerative colitis—many patients either don’t know of these studies or don’t understand the possible benefits of participating.

As patients and advocates work to bring visibility to this disease during this year’s Crohn’s and Colitis Awareness Week (December 1-7), Dr. Bruce Sands, a gastroenterologist at Mount Sinai Hospital and the Icahn School of Medicine at Mount Sinai in New York, explains why some people living with IBD could benefit from discussing clinical trials with their doctors.

DR. BRUCE SANDS, A GASTROENTEROLOGIST AT MOUNT SINAI Hospital and the Icahn School of Medicine at Mount Sinai IN NEW YORK, EXPLAINS WHY SOME PEOPLE LIVING WITH IBD COULD BENEFIT FROM DISCUSSING CLINICAL TRIALS WITH THEIR DOCTORS.

DR. BRUCE SANDS, A GASTROENTEROLOGIST AT MOUNT SINAI Hospital and the Icahn School of Medicine at Mount Sinai IN NEW YORK, EXPLAINS WHY SOME PEOPLE LIVING WITH IBD COULD BENEFIT FROM DISCUSSING CLINICAL TRIALS WITH THEIR DOCTORS.

Why are some people living with IBD unaware that there are clinical trials for their disease?

“IBD clinical trials usually recruit patients who have active, flaring disease. But many patients on existing therapies who may still be flaring are not being cared for by doctors involved with trials. So these doctors may not be prepared to discuss clinical trials as an option. These patients can consider getting another opinion from a doctor at a medical center that offers IBD clinical trials. A good resource for IBD patients to learn more is ClinicalTrials.gov.”

How do you address patients’ concerns about enrolling in a trial?

“Patients feel more comfortable about enrolling when they understand the process. I try to explain the different stages to them. Such as, in Phase 1, researchers focus on evaluating the safety of a new treatment. A treatment doesn’t get to Phase 2 or Phase 3 until we know more about the medication’s safety. At each later stage, more patients take part, and researchers gain better knowledge of the treatment’s safety and efficacy profile.”

“Sometimes, patients hesitate to take part because they worry they will receive a placebo and be left untreated. I tell them that in almost every IBD trial, the investigational therapy is added on top of their existing medication. So they will not be left untreated. Furthermore, most studies allow all participating patients access to the investigational medication after eight to 12 weeks.”

We continue to see progress by studying investigational medications that may provide patients with better symptom relief and disease control.

What do you tell patients who qualify for a clinical trial?

“I explain that there are two big reasons why they should consider enrolling in a clinical trial. First, a clinical study may give them access to a medication that works for them.”

“Second, if people with Crohn’s disease and ulcerative colitis don’t enroll in trials, we will never see advances in the treatment of these diseases. Voluntary patient participation has been essential to the development of new treatment options over the last two decades and will continue to be so in the future.”

Why is it important that we continue to explore new treatments for Crohn’s and ulcerative colitis?

“We have yet to find a medication that works for every person with IBD. And while some existing treatments may work for some patients at first, their effectiveness can wear off over time. Meanwhile, the incidence of IBD is rising across the globe—in the developed world and also countries such as India and China.”

“Over time, we hope to understand which patients do better with which medications through clinical trials. But at this point, we simply need more treatment options.”

How hopeful are you about the future of IBD treatment?

“More than 200 genes are thought to contribute to the risk of Crohn’s disease and ulcerative colitis. Given that complexity, and how the biology differs from person to person, achieving a cure may be very difficult. While we all hope for a cure someday, we continue to see progress by studying investigational medications that may provide patients with better symptom relief and disease control.”

To learn why targeted therapies could be an important therapeutic option for IBD patients, read “Interest Grows in Targeted IBD Research.”

The public often only sees the outside symptoms of plaque psoriasis: raised, red patches of skin covered with silvery scales. But the “Psoriasis Inside Out” theme of this year’s World Psoriasis Day (October 29) implores us to look at the “less visible” aspects of the disease.

New research is shining a light on one of those hidden characteristics of psoriasis: the increased risk of developing other diseases. Comorbidities associated with psoriasis include psoriatic arthritis, depression, diabetes, cardiovascular disease and metabolic disease.

Dr. Steven Feldman, a dermatologist practicing at Wake Forest University, explains that the presence of psoriasis comorbidities can affect a patient's health and their care.

Dr. Steven Feldman, a dermatologist practicing at Wake Forest University, explains that the presence of psoriasis comorbidities can affect a patient’s health and their care.

The presence of these comorbidities can not only impact a patient’s health but also affect their care. “For example, if a patient has a comorbidity of diabetes or liver disease, certain medicines may not be the most appropriate choice of treatment because they could increase the risk of liver damage,” explained Dr. Steven Feldman, a dermatologist practicing at Wake Forest University.

Although physicians who treat patients with psoriasis may be aware of comorbidities, dermatologists often focus on the skin, and other specialists may not pay attention to psoriasis as they focus on the particular disease or condition that they have expertise in. As a result, the medical community has struggled to understand the full extent of comorbidities in psoriasis patients.

To paint a clearer picture, Dr. Feldman and his colleagues analyzed insurance claims data from over 460,000 Americans diagnosed with psoriasis from 2008 to 2014. They found that 46 percent of those psoriasis patients were also diagnosed with high cholesterol, 42 percent with high blood pressure and 18 percent with depression. Other common comorbidities in this psoriasis population included diabetes, obesity and heart disease.

“While this approach is good, it’s not perfect,” Dr. Feldman explained. “For instance, if people don’t go to the doctor, then their psoriasis or their comorbidities would not be detected in studies.”

People with psoriasis should have regular health exams and screening tests to monitor their weight, blood pressure and cholesterol.

While the study provides a clearer picture of the burden of comorbidities, the relationship between psoriasis and these coexisting diseases remains less clear. So far, researchers have identified some potential contributing factors including common inflammatory pathways, cellular mediators and genetic susceptibility.

“Also, people living with psoriasis can make lifestyle choices that could reduce their risk of comorbidities,” Dr. Feldman said. “For instance, exercise and a healthy diet may help to prevent cardiovascular disease for people with psoriasis.”

The Most Prevalent Comorbidities in Psoriasis Patients

REFERENCE: SHAH KAMAL, MILLAR’S LILLIAN, CHANGOLKAR ARUN, FELDMAN STEVEN R.. REAL-WORLD BURDEN OF COMORBIDITIES IN US PATIENTS WITH PSORIASIS. JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY. 2017;77.

While dermatologists commonly screen for comorbidities such as psoriatic arthritis and depression, screening for other comorbidities such as cardiovascular disease is often done by the patients’ primary care providers.  Even though we do not understand the underlying factors that link these diseases, the fact remains that it’s important for patients and physicians to be aware of these comorbidities. Increasing awareness can help these psoriatic disease comorbidities and their risk factors from being overlooked and could potentially lead to earlier diagnosis and management.

“We don’t have enough research to know fully how comorbidities should affect our treatment,” Dr. Feldman said. “But given the increased risk of cardiovascular disease, people with psoriasis should have regular health exams and screening tests to track their weight, blood pressure and cholesterol.”

To learn why it’s important for psoriasis patients to obtain access to their recommended medications immediately, read “Psoriasis Patients Deserve Their Prescribed Therapy Without Delay.”

On the wall next to her computer, Tracey Iraca, Executive Director of the Myelodysplastic Syndromes (MDS) Foundation, Inc., keeps a photo of former board member Bob Weinberg and his dog, Milkshake.

EARLIER THIS YEAR, TRACEY IRACA WAS NAMED THE NEW EXECUTIVE DIRECTOR OF THE MYELODYSPLASTIC SYNDROMES FOUNDATION.

EARLIER THIS YEAR, TRACEY IRACA WAS NAMED THE NEW EXECUTIVE DIRECTOR OF THE MYELODYSPLASTIC SYNDROMES FOUNDATION.

When Weinberg was diagnosed with MDS in 1998, doctors offered him a stem cell transplant, a risky procedure that could have ended or extended his life. Like many, he struggled with finding a suitable match.  Weinberg decided against the procedure because there was no guarantee he would be able to live his life “without limits” and he did not want to risk the opportunity to see his daughter grow up.

Fifteen years later, when Weinberg’s condition deteriorated, he tried to get a transplant but was too sick to qualify. He passed away shortly thereafter.

“Deciding whether to get a transplant is a difficult decision for MDS patients,” Iraca said. “I think of Bob every time I talk with someone who is struggling with that decision.”

With limited treatment options, many of the estimated 60,000 MDS patients in the United States today still face difficult choices. Iraca is hoping to raise greater awareness of this issue during this year’s MDS World Awareness Day and through her new role at the foundation.

Unaware and Underdiagnosed

Iraca was first hired by the foundation in 2004, as a patient coordinator to write thank you notes to donors and send requested information to patients. Like many of the patients she sent information, Iraca knew little about this rare disease in which the bone marrow does not make enough healthy blood cells. But as she took on more responsibilities, she began to understand the unique challenges that MDS patients face.

One of those challenges is that the disease is difficult to diagnose, often leading to treatment delays. Not all primary care physicians are aware of this rare disease, so they may not recognize low blood counts as a reason to send patients to a hematologist for a bone marrow biopsy, which is necessary to diagnose MDS.

“The biopsy needs to be examined by a pathologist who is a specialist,” she explained. “Patients need to understand how difficult the diagnosis is to make and that it’s ok to ask for a second opinion. It’s the patient’s right to get confirmation on a diagnosis of MDS.”

AS EXECUTIVE DIRECTOR OF THE MYELODYSPLASTIC SYNDROMES FOUNDATION, IRACA (FAR RIGHT) HAS BEEN MEETING PATIENTS AND LEARNING MORE ABOUT THEIR NEEDS FOR OVER 13 YEARS.

AS EXECUTIVE DIRECTOR OF THE MYELODYSPLASTIC SYNDROMES FOUNDATION, IRACA (FAR RIGHT) HAS BEEN MEETING PATIENTS AND LEARNING MORE ABOUT THEIR NEEDS FOR OVER 13 YEARS.

As the MDS Foundation grew from its humble roots in a carriage house in Crosswicks, NJ, it began referring patients to over 175 MDS Centers of Excellence around the world to get proper diagnosis and treatment from specialists. These centers all have appropriately trained medical staff and meet other quality measures.

A Matter of Time

Since joining the foundation, Iraca has seen considerable improvements in the medical understanding of MDS. The number of annual references to the disease in scientific publications has increased 33 percent over the past decade, from 739 in 2007 to 980 in 2016. That research has helped uncover the role that the immune system plays in the development of MDS.

Despite that progress, treatments advances for MDS have been few and far between over the past decade. Stem cell transplants remain the only potential cure. But, as Weinberg’s experience exemplified, that approach comes with risks that some patients don’t want to take, and many other patients are ineligible because of their health.

The field is changing, and so much research is happening. We have many reasons to be hopeful for the future of MDS.

Most patients rely on supportive therapy such a transfusions to raise their low blood counts and treatments for infections. As a result, the median survival for high-risk MDS is still just two years.

“The good news is we’ve raised awareness of the need for new treatments through MDS World Awareness Day and throughout the year,” Iraca said. “So now there are more than 1,700 MDS ongoing clinical trials today. We’re extremely hopeful that there will be new options in the not-so-distant future. It’s only a matter of time at this point.”

Standing Room Only

Given the rise in MDS research, the foundation realized the need to educate patients and professionals alike. So they began hosting patient forums, support groups and international conferences focused specifically on the disease.

It’s at these events that Iraca continues to find inspiration from patients and family members who gather to learn more about MDS. She sees their excitement when meeting researchers and asking them questions. They learn coping mechanisms and ways to manage treatment side effects from other patients.

IRACA (SECOND FROM LEFT) AND COLLEAGUES HAVE TRAVELED AROUND THE WORLD HIGHLIGHTING THE NEED FOR MORE MDS RESEARCH AT MEDICAL CONFERENCES, INCLUDING THE 21st CONGRESS OF THE EUROPEAN HEMATOLOGY ASSOCIATION IN COPENHAGEN.

IRACA (SECOND FROM LEFT) AND COLLEAGUES HAVE TRAVELED AROUND THE WORLD HIGHLIGHTING THE NEED FOR MORE MDS RESEARCH AT MEDICAL CONFERENCES, INCLUDING THE 21st CONGRESS OF THE EUROPEAN HEMATOLOGY ASSOCIATION IN COPENHAGEN.

“So many newly diagnosed patients and families are scared,” she said. “It’s hard to sit through a program without getting emotional sometimes. But finding someone who is going through the same thing you are is so helpful to patients. You can see them starting to feel better at these events as they learn more about the disease and build relationships with other patients and caregivers.”

As for working with professionals, the MDS Foundation is expanding its educational efforts. They sponsor an international conference every other year and are looking to adding more regionally based professional events in the off-years in countries such as Australia, Brazil and Israel.

They have also established MDS-specific sessions during general medical conferences, including the American Society of Hematology annual meeting. Iraca has been surprised by the interest in and attendance at these sessions, which are often standing room only.

“They’re being trained on the most up-to-date research, which will trickle down to the patients,” said Iraca. “The field is changing, and so much research is happening. We have many reasons to be hopeful for the future of MDS.”

To learn how research is leading to new, more personalized treatment options for MDS patients, read “Hope through New Research into Myelodysplastic Syndromes.”

Many people living with moderately to severely active inflammatory bowel disease (IBD) are looking for additional treatment options to help them to cope with the physical and emotional burdens of their disease. Therapies called biologics that target a protein relevant to the immune system called tumor necrosis factor (TNF) are effective for many IBD patients. However, not everyone responds to these treatments. Now, investigational therapies that target other immune pathways are showing promise in clinical trials.

Dr. Brian G. Feagan, director of clinical trials at the Robarts Research Institute, SAYS the Inflammatory bowel disease medical community is increasingly interested in therapies that target sites of inflammation.

Dr. Brian G. Feagan, director of clinical trials at the Robarts Research Institute, SAYS the Inflammatory bowel disease medical community is increasingly interested in therapies that target sites of inflammation.

As more data on these IBD therapies come out of this year’s World Congress of Gastroenterology at ACG2017, Dr. Brian G. Feagan, director of clinical trials at the Robarts Research Institute, explains why the medical community is increasingly interested in therapies that target pathways associated with inflammation in the two most common forms of IBD, ulcerative colitis and Crohn’s disease.

Why is it important to develop targeted therapies for patients with IBD?

“Before biologic therapies were approved for IBD, we relied on steroids and immunosuppressive agents that broadly suppressed the immune system. We didn’t know exactly how these treatments worked but did know that they hit many different pathways. They were not very selective. For some patients whose ulcerative colitis or Crohn’s disease is caused by a particular pathway, these broad-spectrum agents may or may not hit that pathway, leaving some IBD patients without an effective treatment.”

People feel like they cannot plan their lives with the disease, but the continued investment in research is giving them hope.

How did the biologics change IBD treatment for patients?

“The biologics target a single protein that plays a role in the development of IBD, called TNF. Before the success of these anti-TNF therapies, the medical community didn’t think that blocking a single molecule or pathway would be effective. They believed that a combination of pathways was responsible for disease and that broad-spectrum therapy was needed. Clinical trials proved that theory wrong, at least for some patients. We have learned a lot about TNF blockers in the last 20  years.”

To learn why researchers must continue to explore new treatment options for IBD, read the “World IBD Day: Current Treatments for IBD Not Meeting Patient Needs” infographic.

To learn why researchers must continue to explore new treatment options for IBD, read the “World IBD Day: Current Treatments for IBD Not Meeting Patient Needs” infographic.


How have advances in understanding IBD opened the door for additional targeted therapies?

“Now that we know a single pathway can make a difference, as with TNF, researchers have started to look for other specific pathways associated with IBD. We are learning more about how these pathways control the immune response, interact with bacteria in our gut and are associated with complications of the disease, such as blockages in the intestine (strictures) and inflammatory tracts between the bowel and other organs, most commonly the skin (fistulas). This focus on specific pathways has evolved out of oncology, where researchers look for disease-related pathways and then use therapies that target specific pathways in individual patients. We haven’t quite gotten there in IBD, but that is the goal.”

Why is new research important for patients?

“People with ulcerative colitis and Crohn’s disease deal with substantial mental and social disabilities. The embarrassment of having IBD can negatively affect their lives. People feel like they cannot plan their lives with the disease, but the continued investment in research is giving them hope.”

To learn why researchers must continue to explore new treatment options for IBD, read the “World IBD Day: Current Treatments for IBD Not Meeting Patient Needs” infographic.

In 2009, a patient with acute myeloid leukemia (AML) was the first person with cancer to have his or her whole genome sequenced, helping scientists to learn more about the molecular drivers of the disease. Despite the knowledge gained, researchers have struggled to develop therapies that specifically shut down those drivers.

But this year brings hope for patients with AML, with the approvals of several new treatment options, including therapies that target specific molecular mutations. Dr. Gwen Nichols, chief medical officer for the Leukemia & Lymphoma Society (LLS), believes that these targeted therapies are helping to usher in the era of precision medicine in AML. As we recognize Blood Cancer Awareness Month, Dr. Nichols explains the challenges of translating knowledge into treatments and why she is excited about the future of precision medicine in AML.

Dr. Gwen Nichols, chief medical officer for The LLS, is hopeful about the future of precision medicine in AML.

Dr. Gwen Nichols, chief medical officer for The LLS, is hopeful about the future of precision medicine in AML.

Why has treating AML remained a challenge?

“AML is a complex and dynamic disease that really needs a precision medicine approach to treat appropriate patients. Some patients diagnosed with AML will respond to standard chemotherapy regimens, but most will relapse. Chemotherapy targets highly proliferating cells but may be missing the cells that initiated the AML. Those cells remain behind, recover and can cause the disease to come back in AML patients. This is one reason why the five-year survival rate for AML patients remains low at just 27 percent.

Why has it been challenging to develop targeted therapies for AML?

“When the AML genome was sequenced, researchers thought they were going to find single mutations that drive the disease. They believed that if you got rid of this single molecular abnormality, you could get rid of the disease. We have found a few of these mutations in other cancers, such as in the Bcr-Abl tyrosine kinase in chronic myeloid leukemia. But over the last decade, we’ve learned that some cancers, including AML, are more complex and driven by multiple factors. So an effective therapy targeting one mutation won’t be the end of the story because it’s only one piece of the puzzle. As we work toward the future of precision medicine, we need to look at multiple targeted therapies in combination.”

 AML is a complex and dynamic disease that really needs a precision medicine approach to treat appropriate patients.


What type of diagnostics would you like to see to facilitate precision medicine in AML?

“In a perfect world where it costs nothing and can be done rapidly, you would sequence a patient’s genome as frequently and as completely as possible. The targeted sequencing that doctors are doing for AML patients today makes the most sense because that information can help determine diagnosis and prognosis. But I fear that we may be missing valuable information by not sequencing more of our patients’ genomes. We also need to sequence at intervals to make sure the disease has gone away and again when there’s evidence that the disease is coming back. We can’t assume that it’s the same [form of the] disease when it returns.”

How do the clinical trial designs need to change for precision medicine?

“In diseases such as AML, it’s clear that there are subsequent mutations as the disease progresses and that the disease becomes more complex as it evolves. Most therapies are first tested in patients with relapsed or refractory disease, but you cannot expect a targeted agent to be effective when other driving mutations have arisen. This is a recipe for failure. We may be throwing out therapies that could benefit patients because we are testing them at a time when the disease is so complex that there’s little hope for a single therapy to be effective. That’s why the LLS’ Beat AML Master Trial is focused on newly diagnosed AML patients.”

What needs to happen to truly enable precision medicine in AML?

“The last couple of months have been exciting with several new therapies introduced for AML. We are seeing real progress toward that now with this first wave of targeted therapies. With over 700 clinical trials active or recruiting in AML, there is certainly more to come. But the hope would be to have several different therapies available that target all the drivers of AML. These therapies will not be developed on their own. We need to think about the best way to help facilitate the future of precision medicine through novel trial design and combinations.”

For more information on the progress of precision medicine, read “Getting Patients Access to ‘Precision’ Medicine Is Crucial.”

Multiple myeloma is a growing problem. In Europe, the number of people who will be diagnosed with this blood cancer is projected to increase 17 percent by 2025, according to the World Health Organization based on 2012 figures. With these rising numbers comes a greater burden on healthcare systems and society, not to mention patients.

MICHAEL ZAIAC, VICE PRESIDENT OF CELGENE EMEA, EXPLAINS WHY MULTIPLE MYELOMA CASES ARE ON THE RISE AND WHAT THE FUTURE HOLDS FOR TREATING THE DISEASE.

MICHAEL ZAIAC, VICE PRESIDENT OF CELGENE EMEA, EXPLAINS WHY MULTIPLE MYELOMA CASES ARE ON THE RISE AND WHAT THE FUTURE HOLDS FOR TREATING THE DISEASE.

Fortunately, treatment advances over the past 20 years are helping people with multiple myeloma live longer lives. The number of clinical trials in multiple myeloma has approximately tripled since the early 2000s, and in fact, this cancer stands out as a striking example of what medical innovation can accomplish. Between 1990 and 2011, five-year relative survival rates for patients with multiple myeloma increased more than 60 percent, compared with 15 percent for all cancers. Some innovative therapies may also help patient quality of life for those with multiple myeloma.

As we commemorate Blood Cancer Awareness Month, Dr. Michael Zaiac, vice president Medical Affairs, Hematology/Oncology, at Celgene EMEA, discusses past successes and explains why it’s critical that innovation continues in the search for a cure.

What is driving the increase in multiple myeloma in Europe?

“People aged  65 and older are more likely to get multiple myeloma, and there are more older adults in the world than ever before. This is especially true in the Western world, where multiple myeloma is currently most prevalent. These trends mean we will likely see more and more cases of multiple myeloma.

39K new cases of multiple myeloma are diagnosed in Europe each year.“But patients may be able to have a different experience today than they had 20 years ago. Treatment options introduced since then are extending their lives and helping their quality of life.”

What impact does multiple myeloma have in Europe?

“Nearly 39,000 new cases of multiple myeloma are diagnosed in Europe each year. While most patients are elderly, 30 percent are between the age of 15 and 64 at diagnosis. Over 40 percent of multiple myeloma patients cannot work because of their disease, and another 23 percent were forced to retire early. Those are direct impacts on productivity. But the disease also has an indirect impact on productivity as elderly patients rely on family and friends for care, forcing those caregivers to miss days of work. We have had a real opportunity to reduce this burden with new and effective treatments over the past couple decades.”

How have treatment advances changed patient lives?

“Patients are living longer lives because of advances in the treatment of multiple myeloma. The five-year relative survival rates for multiple myeloma has increased more than 60 percent since 1990, which is a bigger improvement than we have seen for all cancers on average.”

Treatment advances have led to longer remissions for patients, suggesting a cure may one day be possible.

1 out 3 people who are diagnosed with multiple myeloma are working age.What is driving the rapid progress in multiple myeloma treatment?

“Innovation, without a doubt. And that cannot stop now. Even with the innovations over the past few decades, we have more to do. For example, the five-year survival rate (based on 2007-2013 data) for people with breast cancer is 90 percent; for multiple myeloma, it’s 50 percent. We cannot yet guarantee a cure for a single multiple myeloma patient, so medical innovation is a crucial, ongoing task.”

What new treatment approaches are being explored?

“At least 50 new treatment options for multiple myeloma are being studied in clinical trials, which is significantly more than there were a decade ago. We know the immune system plays a role in this blood cancer, and we have seen the success of certain therapies that act on the immune system and have built on it. Researchers are also looking at harnessing the power of the immune system through novel investigational immunotherapies, including CAR T-cells and bispecific antibodies.

“Additionally, combining different treatments allows for the targeting of different cancer processes simultaneously, and there are ongoing studies investigating various combinations of therapies. Finally, researchers are studying the benefits of different types of stem cell transplants.”

17% — The increase of worldwide incidence of multiple myeloma projected by 2025.How hopeful are you that there will one day be a cure?

“I’m very hopeful for a cure. Treatment advances have led to longer remissions for patients, suggesting that a cure may one day be possible. Multiple myeloma cells are not all the same, so treatment combinations may be needed to make sure we eliminate all the cancer cells and do not give the disease a chance to come back. We are hopeful that, as research continues to advance our understanding of disease and the development of new approaches to treatment, the future may bring multiple myeloma patients a cure or may allow them to live very close to their natural lifespan.”

To learn more about how treatment advances are giving patients a reason to be hopeful, read “A Decade of Progress in Myeloma, and More To Come.”

Alan F. List, MD, president and CEO of the Moffitt Cancer Center in Tampa, has made many contributions to hematology. List remains focused on what he has to offer the hematology field, so, last December, when he was recognized with the 2016 Celgene Career Achievement Award, he was humbly grateful.

Alan F. List, MD, president and CEO of the Moffitt Cancer Center in Tampa, was recognized with the 2016 Celgene Career Achievement Award.

Alan F. List, MD, president and CEO of the Moffitt Cancer Center in Tampa, was recognized with the 2016 Celgene Career Achievement Award.

Celgene is committed to supporting investigators who conduct hematology research and has established the Celgene Awards, comprised of the Career Achievement Award, Young Investigator Award, and Future Leaders in Hematology Award to recognize those investigators who have made significant contributions to hematology research. In addition to acknowledging the winners, the recipients’ institutions receive a grant from Celgene to continue efforts in hematology research and education.  An independent selection committee selects the institution based on the submissions received.

As nominations are being accepted for the 2017 Celgene Awards for Clinical Research in Hematology, Dr. List shares what continues to excite him about the hematology field, what he attributes his success to and where he thinks research is headed.

2016 Young Investigator AwardWhat did the recognition of your research through the Celgene 2016 Award mean to you?

“This award is a recognition of all the work that my collaborators and I have done together. Nothing that I have achieved thus far has been the result of one individual. It’s always been a collaborative effort.”

Which of your contributions are you most proud of?

“Three things come to mind. The first is my work in developing a treatment option for MDS. In 2001, I was investigating the role of angiogenesis — the formation of blood vessels — in the bone marrow of MDS patients. That research led me to explore whether existing therapies that slow the growth of blood vessels could stop the disease from getting worse. I applied for a grant and conducted a clinical trial that led to a new treatment option.

“The second is my work on multidrug resistance in MDS and high-risk acute myeloid leukemia (AML). We tested a potential therapy and took it to a phase III trial. That study remains the only one to show a survival benefit in high-risk AML patients.

“The third is my work to help speed up findings for the next generation. At the Moffitt Center, I’ve mentored some very bright researchers.”

2017 Celgene Awards: Nominate a Colleague Today!What inspired you to pursue a career in hematology?

“It seemed that there was so much potential for research in bone marrow-based malignancies such as MDS and AML. Researchers can access the disease directly through a bone marrow aspirate or by simply drawing blood from patients and studying the cells. That’s difficult to do in solid tumors.”

“Also the notion that the hematologist serves as both physician and pathologist creates an ideal opportunity to optimize insight into the disease pathology. You understand the case better than anyone as the physician, so you are more likely to have insights when reading the bone marrow.”

This award is a recognition of all the work that my collaborators and I have done together. Nothing that I have achieved thus far has been the result of one individual.

Celgene 2016 Future Leaders in Hematology AwardsWhat were your biggest career challenges?

“For everyone in research, funding is the greatest challenge. It became an even greater challenge for physician researchers like me in the ‘80s and ‘90s. At that time, the National Institutes of Health (NIH) began prioritizing Ph.D.’s for basic research grants and physicians only for trials. It is a challenge to be a successful physician researcher because you have the demands of patient care on the clinical side, but I’ve always enjoyed both aspects. I’ve been fortunate to have great collaborators that have allowed me to explore my research interests and still care for patients.”

What impact do you hope to make as President and CEO of Moffitt Center?

“We haven’t seen any significant rise in NIH funding for nearly a decade. That has been challenging for research institutions like the Moffitt Cancer Center. My responsibility is to make sure that the institution is financially solvent to pursue our mission of improving cancer care. One way we’re doing that is by partnering with insurers and the Center for Medicare and Medicaid Services as they explore new payment models. We’ve also partnered with pharmaceutical companies that have helped sponsor novel laboratory research.”

What is the future of the hematology field?

“In the past, we have been very linear in our view of science and biology. Research focused on gene mutations over the last ten years has been successful, but we know other factors also play significant roles in cancer. The disease is dynamic and complex, and it’s difficult to understand when we look at one part of the whole system.

“We’ll treat and prevent cancer better when we take a systems biology approach and look at the complete picture of how cancer affects the body, including metabolic and cell signaling networks. Systems biology is going to be critical in furthering our understanding of cancer’s complexities.

“At the same time, for many cancers, we’ve also been treating patients the same way for the past 40 years. If a therapy works, we give patients the highest dose that they can tolerate until it stops working. Then we move onto the next therapy, linearly. In the future, we’re going to see more adaptive therapeutic approaches that are flexible and can change in response to a patient’s tumor at a particular time.”

To learn how to nominate a colleague for the 2017 awards, visit the Celgene Awards for Clinical Research in Hematology website.

2017 Celgene Awards: Nominate a Colleague Today!

Although blood cancers are relatively rare, their root causes may not be so different from those of other cancers. And since blood cancer samples may be more easily accessible than solid tumor samples, it can help research efforts and make excellent models for research.

DR. LOUIS DEGENNARO, PRESIDENT AND CHIEF EXECUTIVE OFFICER OF THE LEUKEMIA & LYMPHOMA SOCIETY, EXPLAINS HOW ADVANCES IN BLOOD CANCER HAVE IMPROVED TREATMENT IN OTHER CANCERS.

DR. LOUIS DEGENNARO, PRESIDENT AND CHIEF EXECUTIVE OFFICER OF THE LEUKEMIA & LYMPHOMA SOCIETY, EXPLAINS HOW ADVANCES IN BLOOD CANCER HAVE IMPROVED TREATMENT IN OTHER CANCERS.

“We’re able to make faster progress in blood cancer because we’re studying the real cancer in the patient,” said Louis DeGennaro, president and CEO of the Leukemia & Lymphoma Society. “When you draw a leukemia patient’s blood sample, you have an exact representation of their cancer. That’s not the case in solid tumors, where you’d have to manipulate and grow the cells in a lab to study them.”

That’s why the LLS is especially focused during Blood Cancer Awareness Month on raising awareness of this second leading cause of cancer death in the United States (behind only lung cancer).

Blood cancer is actually a family of over 140 distinct diseases that affect blood cells, acting as “bullies” within the circulatory and immune systems. These abnormal blood cancer cells typically grow more quickly and survive longer than normal, interfering with the production and functioning of healthy cells.

Like all families, though, blood cancers have their differences. Lymphoma, leukemia, multiple myeloma and myelodysplastic syndromes (MDS), four major types of blood disorders, affect specific cell types in particular neighborhoods of the body. For instance, lymphoma alters white blood cells in the lymphatic system, while multiple myeloma disturbs plasma cells in the bone marrow.

These four diseases branch out into further subtypes. Leukemia comprises acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myeloid leukemia (CML) and chronic lymphocytic leukemia (CLL); lymphoma includes over 60 different forms. The distinction between all those subtypes lies partly in the specific cell type impacted. But scientists are learning that commonalities are more critical than distinctions.

“When it comes to treating cancer, we’re beginning to understand that the kind of cell affected is not the most important aspect,” DeGennaro said. “It’s the underlying mutations and mechanisms—which are shared by many types of cancer—that matter more.”

For instance, researchers have found mutations in a tumor-suppressor gene called TET2 in multiple blood cancers, suggesting it plays a role in how blood cells are regulated in the body. But even beyond blood cancer, the TET gene family has also been linked to breast, liver, lung, pancreatic and prostate cancers.

Blood Cancer's Family Tree

Their commonalities may mean medical advances in one blood cancer could provide insights into others.

An example is a class of therapies called tyrosine kinase inhibitors (TKIs), which have nearly doubled the five-year survival rate for CML since they were introduced 15 years ago. Today, TKIs have been approved to treat many other diseases, including lung, thyroid, kidney and breast cancers.

Roots of Care: Multiple Myeloma

“Blood cancer research is paying dividends to other cancers and diseases, and that will only continue as we learn more about these diseases,” said DeGennaro.

To learn how you can help the Leukemia & Lymphoma Society in supporting blood cancer research, read “Why I’m Walking in Light The Night® This Year.”

Cancer survives and thrives by working around the body’s natural defenses and turning off the immune system’s roadblocks before it can attack the disease. One way tumor cells flourish is by using the programmed death-1 (PD-1) receptor and programmed death-ligand 1 (PD-L1) pathway to dampen the immune system. Innovative new therapies are now tackling this pathway in an attempt to slow the progression of certain tumors

PD-1 is a “checkpoint,” which immune cells use to determine whether they should attack an enemy, such as a tumor cell or a cell infected with a virus, or shut themselves down. Cancers, though, have found ways to manipulate PD-1. For example, they make high levels of its ligand, PD-L1. So when immune cells approach tumors, they become anesthetized by the PD-L1 and lose their ability to attack.

New immunotherapy research is examining whether antibodies that block the PD-1/PD-L1 pathway can awaken and reactivate immune cells so they can once again kill tumor cells.

PD-1 and PD-L1 antibodies release the brakes on the immune system and can restore its natural antitumor response

There are other therapies designed to work with the immune system to combat cancer, but PD-1 and PD-L1 inhibitors may hold unique potential for some hard-to-treat cancers.

“PD-1 and PD-L1 antibodies release the brakes on the immune system and can restore its natural antitumor response,” said Robert Hershberg, Executive Vice President, Head of Business Development and Global Alliances, at Celgene Corporation,former Chief Scientific Officer and leader of the Immuno-Oncology Center of Excellence. “I think there’s very little doubt now that the future of oncology is inextricably linked to the immune system.”

While targeted therapies effectively shut down just one target within cancer cells, immunotherapy has more widespread effects — working with the body’s immune system as a whole to make it more difficult for the cancer to survive. Early clinical research suggests that a range of solid tumor cancers, including melanoma, lung cancer, bladder cancer, head and neck cancer (among others), respond to immunotherapy. Using sophisticated immune monitoring techniques to determine which patients respond to these immune-targeting agents remains a crucial endeavor at Celgene.

Disrupting the PD-1 checkpoint may also result in an unchecked immune response that may lead to adverse effects for some patients. Researchers are learning how to engineer these therapies to not only be more effective but also minimize molecular interactions that may have undesirable consequences.

Down the road, combination therapy with PD-1 and PD-L1 antibodies could be even more advantageous. “It’s a breakthrough and revolutionary, but really the tip of the iceberg,” Hershberg said.

PETER SCHAFER, EXECUTIVE DIRECTOR OF TRANSLATIONAL MEDICINE AT CELGENE, BELIEVES WE ARE AT A TIPPING POINT WHERE THE COLLECTION OF LUPUS GENETIC RESEARCH IS ABOUT TO GIVE WAY TO NEW THERAPEUTIC APPROACHES.

PETER SCHAFER, EXECUTIVE DIRECTOR OF TRANSLATIONAL MEDICINE AT CELGENE, BELIEVES WE ARE AT A TIPPING POINT WHERE THE COLLECTION OF LUPUS GENETIC RESEARCH IS ABOUT TO GIVE WAY TO NEW THERAPEUTIC APPROACHES.

In the past 50 years, only one new therapy has been developed exclusively for lupus. Meanwhile, patients with this inflammatory disease — in which the body’s immune system attacks its own tissue — continue to combat symptoms like fatigue, joint pain and rashes.

But Peter Schafer, executive director of Translational Medicine at Celgene, believes new genetic research in lupus is finally giving way to new investigational approaches that target mutations driving the disease.

As this year’s Annual European Congress for Rheumatology (EULAR) 2017 gets underway in Madrid, Schafer explains how genetic research is opening up new avenues of research for this difficult-to-treat disease.

How is our growing understanding of the genetic changes underlying lupus influencing the development of new treatments options?

We’re living in an era that’s beyond the sequencing of the human genome. Over the past decade, researchers have identified a multitude of genetic variants linked with particular diseases. Companies are beginning to realize the value of targeting the proteins that are encoded by those genes.

Managing Lupus Remains a Challenge

How has our understanding of genetics evolved over the past decade?

Three years ago, if I told someone that there was a change in a part of a gene that did not include the “recipe” for a protein, they would dismiss that mutation. Now we know that you shouldn’t necessarily ignore it. We’re realizing that other parts of the gene may be doing something else—for instance, controlling the quantity of protein being produced. Sometimes, the most obvious experiments go undone. One example is comparing the amount of a protein being produced in people with a given disease and those without. You may never bother to look if you dismiss that genetic change.

Have researchers found such variants that are believed to contribute to lupus?

Yes. For instance, there are variants in the genes for two proteins, called Ikaros and Aiolos, that help with the development of the immune cells that can cause inflammation. Lupus patients have twice the normal Ikaros levels and four times the normal Aiolos levels.

“Until this year, there had not been a program to evaluate a treatment that is designed specifically to target the genetic drivers of lupus.”

Have therapies that target such mutations linked with lupus been developed?

A few medications exist that target some of the proteins of the identified genes, but they are not approved for treating lupus. Their use in lupus will have to be investigated. Until this year, there had not been a program to evaluate a treatment that is designed specifically to target these potential genetic drivers of lupus.

What makes developing targeted therapies for lupus more challenging than for diseases such as cancer?

Lupus can be a debilitating disease, but the mortality rate isn’t as high as in cancer. As a result, a patient is less likely to be willing to deal with side effects, and their expectation is much higher.

Also, you’re not trying to kill a tumor. Instead, you’re trying to maintain some degree of suppression of the immune system over the long term. Lupus patients don’t use a high dose of treatment for a short period and then stop; they have to keep using their therapy. You want to know how low the dose can be and still be effective, so you don’t put the patient at risk unnecessarily.

High Levels of Ikaros & Aiolos in Lupus

How challenging is it to test the efficacy of treatments for lupus?

Lupus affects so many different tissues, joints and organs. Clinical efficacy measures of a new lupus treatment should evaluate the effectiveness of that treatment across the various organ systems involved in lupus. You’re looking for an improvement in at least some of those symptoms without worsening others.

At this time, lupus patients are being classified by which tissues are affected. But now that we’re looking at the disease from a genetic perspective, we have seen genetic variants in lupus patients regardless of the tissue affected. This finding is shifting our thinking about how to approach a disease like lupus, because if all lupus cases have similar root causes, genetic evaluation is important. Classifying patients solely by their affected organs might not be as useful for treatment purposes.

To learn about how targeted therapies are being explored for other inflammatory diseases, read “Accelerating MS Research Through Teamwork, New Approaches.”

Last month, James Fitzgerald celebrated his wedding day. It was an event that he never thought he would see when he was diagnosed with acute myeloid leukemia (AML) six years ago. He was immediately hospitalized, and his liver and kidneys failed within 24 hours. His doctors put him into a medically-induced coma for a week before starting his treatment, which included an intensive combination of chemotherapy and radiation. It was one of the only treatment options for AML at that time.

But that’s changing for AML patients like Fitzgerald. Therapies that target some of the specific molecular genetic changes in AML are being developed, and that’s making mutational profiling more important than ever. That’s good news for Fitzgerald who is currently in remission but is concerned that his disease may one day come back.

“When I was diagnosed, I didn’t know to ask my doctors about the different molecular mutations or whether that would even make a difference in my treatment,” Fitzgerald said. “It was only after my treatment that I started asking questions and learning about the genetics of AML.”

Spot the Difference in AML

Enabled by breakthroughs in genome sequencing and analysis over the past decade, researchers have learned more about the genetics that drive AML. In 2008, an AML patient was the first cancer patient to have her genome sequenced, enabling researchers to discover 10 molecular mutations related to the disease.

Since then, scientists have identified as many as 80 potentially disease-associated mutations in many genes, including NPM1, FLT3-ITD, IDH2, DNTM3A, KIT, IDH1, and CEBPA. They have now found genetic differences between the 34 subgroups of AML, confirming the idea that not every AML case is the same.

This diversity can make treatment a challenge. But scientists and patients are hopeful that a better understanding of these molecular changes will improve outcomes. That’s particularly good news for a disease that has not seen substantial improvements in 20 years.

I hope doctors start learning more about the mutations in AML and start talking with their patients about the genetics of the disease.

The current standard of treatment — an intense combination of chemotherapy — is not particularly easy for patients to tolerate, something Fitzgerald can attest to. Eventually, he received a bone marrow transplant and has been in remission ever since.

“It certainly wasn’t fun,” said Fitzgerald. “I had a 106-degree fever at one point, and I was in the hospital for three months. It was definitely tough for me, but at least I had that option.”

Given the severity of treatment-related adverse events, the standard treatment regimen is often not prescribed for older or unhealthy patients. So while half of AML patients under age 60 years live five years or longer, only up to 15 percent of older patients, who more often cannot receive intense chemotherapy, reach the same milestone.

Mutational Profiling May Help Inform Therapeutic Decisions

Researchers are turning their focus to develop therapies that target the specific molecular mutations that drive AML. A recent analysis of 200 patients showed that more than 99 percent of patients had at least one mutation associated with the disease.

As new targeted therapies are being explored, an understanding of the underlying mutations in each patient becomes increasingly important for doctors and patients to decide on an appropriate treatment plan. The Leukemia & Lymphoma Society is exploring the possibility of bringing a personalized approach to AML treatment through its Beat AML clinical trial. With such initiatives in progress, molecular profiling is on its way to becoming a standard part of disease classification and treatment for AML patients.

“I hope doctors start learning more about the mutations in AML and start talking with their patients about the genetics of the disease,” said Fitzgerald. “I can only hope AML patients don’t have to go through what I faced in my treatment.”

To learn more about how clinical trials are bringing a precision medicine approach to AML, read “Bring Patients Closer to Personalized AML Treatments.”

 

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DR. RAFAEL BEJAR, AN ASSISTANT PROFESSOR AT THE UNIVERSITY OF CALIFORNIA, SAN DIEGO, BELIEVES THAT RESEARCHERS WILL MAKE SIGNIFICANT PROGRESS IN THE TREATMENT OF MYELODYSPLASTIC SYNDROMES PATIENTS.

DR. RAFAEL BEJAR, AN ASSISTANT PROFESSOR AT THE UNIVERSITY OF CALIFORNIA, SAN DIEGO, BELIEVES THAT RESEARCHERS WILL MAKE SIGNIFICANT PROGRESS IN THE TREATMENT OF MYELODYSPLASTIC SYNDROMES PATIENTS.

For patients with myelodysplastic syndromes (MDS) — bone marrow disorders that affect blood cell production — the early 2000s were a time of advancement, when several new treatment options were approved. But since then, research and treatment advances have been minimal.

Now, as researchers gather for the 2017 International Symposium for Myelodysplastic Syndromes this week, there is renewed hope. With a better understanding of the how the immune system and genetic mutations contribute to the disease, experts believe that new treatments may be around the corner. Dr. Rafael Bejar, an Assistant Professor at the University of California, San Diego, explains why he’s looking forward to this year’s event.

What are some of the challenges in treating MDS?

We’ve been relying on three medications that were approved over 10 years ago. These medications have freed some patients from the need for blood transfusions, improved their blood counts and likely extended their lives. But not everybody responds to these therapies. We need more therapeutic options to treat the disease in different ways. When you have multiple medications to choose from, you can also start profiling patients to predict which treatment is going to be best for them allowing you to further personalize care.

What has made medical advances in MDS so difficult?

MDS is very different than many other cancers. With its elderly and often frail patient population, we have to find medications that are not only effective but also tolerable and safe.

To do this, we must understand the disease better than we do today. MDS cells don’t grow well in the laboratory, making them difficult to study.

What recent advances will be highlighted at this year’s symposium?

One of the fascinating new discoveries in the last few years involves how the innate immune system may be involved in the development of MDS. We have learned that MDS cells create inflammation and thrive in this environment. We may be able to target this particular type of inflammation with medications and therapeutic antibodies in order to make MDS cells more vulnerable. This approach has yet to be thoroughly tested clinically, but the research in this area could provide a rich set of therapeutic targets.

There is also growing interest in how B and T cells, which form our adaptive immune system, might play a role in MDS. Researchers are now exploring various types of immune approaches for the treatment of MDs. This exciting research involves understanding how the immune system reacts to MDS cells.

If we can make this disease more manageable, patients will end up living better lives and dying with MDS and not of MDS.

What are researchers learning about the biology of MDS? How are those insights helping us to treat patients?

We’re using mutations to help identify patients who are more likely to respond to a given therapy. Genetics has begun to tease apart different subsets of MDS with distinct patterns of mutations and important differences in clinical outcomes. For example, patients who have mutations in a gene called SF3B1 tend to do relatively well. But patients who have mutations in TP53 usually have more aggressive disease and an overall poorer prognosis. We‘ve always tailored our treatments to our perceptions of how patients are likely to do with their disease, but now we can use more accurate methods to help predict that prognosis.

Some of the new science is also informing the diagnosis of MDS, correct?

Right now, many people who have unexplained low blood counts are left without a diagnosis. They don’t meet the current diagnostic criteria for MDS. But we’re learning that about 40 percent of these patients have mutations associated with MDS. This data supports adding DNA sequencing and mutation tests to our diagnostic workup for these patients. We may need to expand the umbrella of what we consider MDS to include patients with an MDS-like condition that is likely to progress. And, we may be able to tell those patients without these mutations that they’re likely to do well without treatment.

How optimistic are you that we will see more treatment advances soon?

MDS is certainly an area where there’s a lot in development and a lot to feel hopeful about. However, stem cell transplants currently remain our only potential cure. While advances are making transplants safer and more effective, we’ll still need other treatment options for the majority of patients who cannot receive a stem cell transplant. There are several new targets to explore, including inflammatory cascades, immune checkpoint inhibitors, apoptotic pathways and mutated splicing factors. When we succeed we’ll be attacking the disease from many different angles. With these potential new targets, we are looking to make tremendous progress in how we treat patients. If we can make this disease more manageable, patients will end up living better lives and dying with MDS and not of MDS.

To learn more about this blood disorder, read “What Are Myelodysplastic Syndromes?”

When Michael Tuohy was diagnosed with multiple myeloma in 2000, the only thing he could think about was living long enough to walk his then 7-year-old daughter Ally down the aisle and seeing his 2-year-old son Mikey graduate from college. At the time, multiple myeloma patients had just a 35 percent chance of living five years. But 17 years later, Tuohy is about to see his wishes come true; his daughter is planning her wedding for next year, and his son is now a freshman in college majoring in biomolecular science.

Since his diagnosis, Tuohy and his wife, Robin, have seen a lot of positive advances in multiple myeloma treatment and support. During this year’s Multiple Myeloma Action Month, they are giving back to the community by sharing what they’ve learned.

How did you become involved with the multiple myeloma community?

Michael: After my diagnosis, we searched out the best resources and found the International Myeloma Foundation (IMF) website. Geraldine Ferraro had just announced she had multiple myeloma. The IMF was asking patients to write letters to Congress about battling multiple myeloma and the importance of funding research. Susie Novis Durie, the founder of the IMF, saw our letter and asked us to share our story in Washington that year. We both went, and it was the first time we met any other multiple myeloma patients. I had gone about six months without meeting another patient. We became empowered through the IMF and decided it was time to take action to help others.

MULTIPLE MYELOMA SURVIVOR MICHAEL TOUHY (SECOND FROM THE LEFT) AND HIS WIFE ROBIN (FAR RIGHT) FEEL FORTUNATE THAT HE HAS LIVED LONG ENOUGH TO SEE HIS DAUGHTER ALLY (RIGHT) GRADUATE FROM COLLEGE. SINCE THEN, SHE HAS STARTED HER CAREER AS AN ELEMENTARY SCHOOL TEACHER, AND HIS SON MIKEY (FAR LEFT) HAS BEGUN HIS COLLEGE STUDIES AS A BIOMOLECULAR SCIENCE MAJOR.

MULTIPLE MYELOMA SURVIVOR MICHAEL TOUHY (SECOND FROM THE LEFT) AND HIS WIFE ROBIN (FAR RIGHT) FEEL FORTUNATE THAT HE HAS LIVED LONG ENOUGH TO SEE HIS DAUGHTER ALLY (RIGHT) GRADUATE FROM COLLEGE. SINCE THEN, SHE HAS STARTED HER CAREER AS AN ELEMENTARY SCHOOL TEACHER, AND HIS SON MIKEY (FAR LEFT) HAS BEGUN HIS COLLEGE STUDIES AS A BIOMOLECULAR SCIENCE MAJOR.

Why did you start a multiple myeloma support group?

Robin: Connecticut did not have a multiple myeloma support group, and Susie suggested that we start one. But we weren’t the kind of people who sit around and whine. So we decided our group — the Connecticut Multiple Myeloma Fighters’ Information Group — would focus on empowering multiple myeloma patients and caregivers to have better-informed conversations with their doctors and to learn about treatment, side effects, clinical trials and the importance of seeking out the opinion of a myeloma expert.

In 2005, the IMF hired me to help with its growing Support Group Program. Today, there is a network of over 150 myeloma specific groups in the United States and over 300 worldwide. The IMF has numerous programs specifically geared to ensure support and information are available at the local level. The IMF applauds the efforts and commitment of these groups and conducts an annual summit to offer help, training, and encouragement to support group leaders.

How does your support group help new multiple myeloma patients?

Michael: The first thing we tell people is to see a multiple myeloma specialist. Their local oncologists are treating many different cancers. Form a team with your local oncologist to work with a myeloma specialist. Myeloma is 1 percent of all cancers and a very individual disease; what works for one may not work for another. You still need a local oncologist, someone who can do your blood testing and know your history in case you need hospitalization close to home. But you want a multiple myeloma specialist as well. You want someone who is conducting clinical trials and knows the latest treatments and research. It should be a collaboration between your multiple myeloma specialist, local oncologist and other experts. You have a lot of people working for you that you need to coordinate — you’re the captain of the ship.

When Michael was diagnosed, his treatment options were limited. Now that there are so many more options, people should feel encouraged to talk with their doctors about what’s best for them.

How does your attitude make a difference in living with multiple myeloma?

Robin: We’ve never lived our lives waiting for the other shoe to drop. We’ve remained positive. You have to be confident in your doctor and the treatment that you’ve decided on together. Knowledge is power. Patients should learn as much as they can so they can have conversations with their doctors and remain engaged.

How else are you giving back to the community?

Robin: Our whole family has gotten involved in advocating for increased funding for multiple myeloma research, Food and Drug Administration approval of new therapies and oral parity laws. Those laws push for equal insurance reimbursement for anti-cancer treatments regardless of the delivery method. Michael was on an oral therapy at one point, so we know the benefits; it allowed him to go home, take a pill and get on with his normal routine.

A silver lining to the multiple myeloma journey is seeing and appreciating our children become advocates and doing good things in the world. Our son Mikey is now in his first year of college, studying biomolecular sciences. Our daughter is an elementary teacher.

SINCE MICHAEL TOUHY’S MULTIPLE MYELOMA DIAGNOSIS, HE AND HIS WIFE ROBIN HAVE ATTENDED MEDICAL MEETINGS TO LEARN ABOUT THE LATEST TREATMENTS AND SHARE THAT INFORMATION WITH OTHER PATIENTS.

SINCE MICHAEL TOUHY’S MULTIPLE MYELOMA DIAGNOSIS, HE AND HIS WIFE ROBIN HAVE ATTENDED MEDICAL MEETINGS TO LEARN ABOUT THE LATEST TREATMENTS AND SHARE THAT INFORMATION WITH OTHER PATIENTS.

What has changed in the 17 years since Michael’s diagnosis?

Robin: When Michael was diagnosed, his treatment options were limited. Now that there are so many more options, people should feel encouraged to talk with their doctors about what’s best for them. I also stress to people to stay fit and get all their check-ups. You have to keep your body in good shape. As long as you can stay healthy and take care of yourself outside of your multiple myeloma, those treatment options are your future. Don’t be afraid of your options.

Michael: We’ve been going to the American Society of Hematology annual meeting for a few years now. Lately, it’s been standing room only. Thousands of participants are attending the presentations to hear about the latest developments. It blows my mind and gives me hope for the future of multiple myeloma.

BRUCE BEBO, PH.D., EXECUTIVE VICE PRESIDENT OF RESEARCH AT THE NATIONAL MS SOCIETY, BELIEVES THAT ADVANCES IN MS TREATMENT ARE ENCOURAGING.

BRUCE BEBO, PH.D., EXECUTIVE VICE PRESIDENT OF RESEARCH AT THE NATIONAL MS SOCIETY, BELIEVES THAT ADVANCES IN MS TREATMENT ARE ENCOURAGING.

While treatment for multiple sclerosis (MS) has improved over the past 20 years, there’s still no cure. Most people living with MS have a form of the disease called relapsing-remitting MS (RRMS) with cycles of relapses (when symptoms flare up) followed by periods of remission (times of little or no symptoms). But researchers now better understand the pathways that contribute to MS relapses, and that knowledge is driving the development of new treatments.

During this year’s MS Awareness Month (March), we spoke with Bruce Bebo, Ph.D., executive vice president of Research at the National MS Society, to understand the questions driving MS research today and the progress being made.

The National MS Society is funding more than 300 research projects and invests about $50 million in MS research each year. What are some of the most exciting recent advances?

Advances in repairing the myelin sheath, which protects nerve cells and is destroyed by MS, are encouraging. This approach has tremendous promise to work with immunotherapies to stop the disease from getting worse. MS is an autoimmune disorder in which the body’s immune system attacks and destroys nerve cells; so immunotherapies may ultimately slow down or halt the neurodegeneration in MS. A better understanding of the role of B cells (a type of immune cell) play in this destruction will likely lead to new and improved treatments for this type of MS.

Most of the 2.3 million people worldwide with MS have a type called relapsing-remitting MS (RRMS). What are the big questions that are driving research in RRMS? 

We still don’t know the precise targets that the immune system recognizes in RRMS, the environmental risk factors and triggers or how the estimated 200 genes that have been associated with MS actually contribute to the disease.

How will answering these questions help lead to more effective treatment options for patients with RRMS?

Immunotherapies today often affect immune cells that fight infection as well as those that cause MS. Knowing the targets will allow us to develop more precise therapies that will prevent the immune system from attacking nerve cells but not interfere with fighting infections. Understanding the environmental lifestyle factors and genetic factors will also reveal pathways and strategies for treatment and prevention.

There will always be some people who respond and others who do not. Having more options will allow doctors to personalize therapies for people living with RRMS.

With over a dozen available treatment options, why do we need more for RRMS?

There will always be some people who respond to specific treatments and others who do not. Having more options will allow doctors to personalize therapies for people with RRMS. Another reason is that our best treatments today only inhibit relapses by about 50 percent. We can do better than that! And more targeted immunotherapies could help stop the progression of RRMS without leaving a person with MS vulnerable to infections.

How important is a person with MS’s role in RRMS research and clinical trials?

The only way we can make progress in MS is by studying people with the disease. Patient-centered research efforts are gaining momentum. One of those efforts is the iConquerMS program, which is collecting data from 20,000 people about their lives with MS and their treatment responses. This will help answer questions about how environmental and lifestyle factors influence MS. People with MS can also take part in clinical trials of new MS therapies. They can find out about these at the National MS Society website.

The vision of National MS Society-funded research is to move us closer to a “World Free of MS.” While we are still searching for a cure, what does freedom from MS mean for people with the disease in real-life terms?

Freedom means something different to every individual living with MS. To some it simply means having the energy to enjoy dinner with their family at the end of the day. For others, it means being able to enjoy activities such as hiking, biking or painting.

CAROLYNN KIEL’S PANCREATIC CANCER WAS DETECTED EARLY BECAUSE SHE WAS AWARE OF HER FAMILY’S HISTORY WITH THE DISEASE AND HAD ANNUAL SCREENINGS.

CAROLYNN KIEL’S PANCREATIC CANCER WAS DETECTED EARLY BECAUSE SHE WAS AWARE OF HER FAMILY’S HISTORY WITH THE DISEASE AND HAD ANNUAL SCREENINGS.

After Carolynn Kiel lost her mother and sister to pancreatic cancer within the same year, her doctor recommended she get screened annually for an early detection marker for the disease. Even though she thought her physician was being overly cautious, she decided it was better to be safe than sorry. One year later, that screening resulted in an early diagnosis of pancreatic cancer that she believes helped save her life.

What many people don’t realize is that 10 percent of pancreatic cancer patients have a family history of the disease. In fact, the risk of developing pancreatic cancer more than doubles if a person’s mother, father or sibling had the cancer. Now, 13 years after her diagnosis, Carolynn wants to help raise awareness of how screening in high-risk individuals can help save lives, by sharing her family’s journey with pancreatic cancer.

When did pancreatic cancer first impact your family?

My sister Marilyn was diagnosed with pancreatic cancer in 2000. She was 58 years old, 11 months younger than me, and had flu-like symptoms that wouldn’t clear up. At the time, pancreatic cancer was a mystery to our family. The only person we knew who had pancreatic cancer was Michael Landon of Little House on the Prairie. Then my mother was diagnosed with pancreatic cancer about a year later after we noticed she was losing significant weight. My mom’s cancer was more advanced than my sister’s when she was diagnosed, so she lived only about two months after diagnosis. Then, just a few weeks later, my sister died as well.

Did that family history with pancreatic cancer change how you managed your health?

I thought our family was done with pancreatic cancer, but my primary care doctor said I should start getting tested annually for an early marker of pancreatic cancer called CA19-9. So I first got a blood test done looking for early signs in April 2003. That test came back negative. A year later, the CA19-9 marker came back positive, and a follow-up endoscopy led to my diagnosis of pancreatic cancer. I was mad. I said, “This isn’t fair!” I couldn’t believe that this disease affected the lives of so many people in one family.

CAROLYNN KIEL’S MOTHER (PICTURED ABOVE) WAS DIAGNOSED WITH PANCREATIC CANCER IN 2001 AND PASSED AWAY ABOUT TWO MONTHS LATER. HER DEATH SPURRED KIEL TO BEGIN ANNUAL PANCREATIC CANCER SCREENING, WHICH RESULTED IN AN EARLY DIAGNOSIS.

CAROLYNN KIEL’S MOTHER (PICTURED ABOVE) WAS DIAGNOSED WITH PANCREATIC CANCER IN 2001 AND PASSED AWAY ABOUT TWO MONTHS LATER. HER DEATH SPURRED CAROLYNN TO BEGIN ANNUAL PANCREATIC CANCER SCREENING, WHICH RESULTED IN AN EARLY DIAGNOSIS.

How did that early diagnosis affect your treatment?

We caught the disease early, so I was lucky to be a candidate for surgery. It is called the Whipple procedure. Only 20 percent of patients are eligible for the surgery. I was “lucky,” but that doesn’t mean the treatment was easy. The Whipple procedure is like going to hell, but you get to come back. It’s brutal. Getting through it requires a positive attitude and determination. I needed people to talk to, people who were going through the same thing as I was, and I found a pancreatic cancer support group. It made a significant difference in my life. So now, 13 years later, having survived my battle with pancreatic cancer, I make the time to talk with any pancreatic cancer patient who needs someone.

Do your other family members get screened for pancreatic cancer annually?

I worry about my daughter and sister. They’re tested once a year with the CA19-9 blood test and get annual checkups. With any cancer, early detection is the most important thing. Pancreatic cancer is so sneaky that by the time you have the symptoms, you’re already far along. The CA19-9 test isn’t something that’s included with regular blood work, so it’s important to check for it if you have a family history of pancreatic cancer. The CA19-9 marker is not perfect, but it is elevated in most people with pancreatic cancer. It’s one of the best things that we have right now for early detection.

CAROLYNN KIEL (FAR LEFT) AND HER FAMILY HAVE BEEN IMPACTED BY PANCREATIC CANCER. HER SISTER MARILYNN (LEFT) AND HER MOTHER (RIGHT) BOTH PASSED AWAY FROM THE DISEASE IN 2002. HER DAUGHTER STACY (FAR RIGHT) GETS ANNUAL SCREENING FOR THE DISEASE.

CAROLYNN KIEL (FAR LEFT) AND HER FAMILY HAVE BEEN IMPACTED BY PANCREATIC CANCER. HER SISTER MARILYNN (LEFT) AND HER MOTHER (RIGHT) BOTH PASSED AWAY FROM THE DISEASE IN 2002. HER DAUGHTER STACY (FAR RIGHT) GETS ANNUAL SCREENING FOR THE DISEASE.

There are ongoing studies and registries for individuals with a family history of pancreatic cancer to understand the genetic components of this disease better. Have you participated in any of these?

My daughter, sister and I had blood work sent to the National Familial Pancreatic Tumor Registry at Johns Hopkins probably 12 years ago. The information and blood work will help researchers determine the genetic and non-genetic risks for pancreatic cancer. It may help improve early detection, so more people can have a better shot of surviving like I did. I want to do anything that helps others. My attitude is that if there’s a slight chance it will help, then I will do it. What would stop anyone from helping out if they could?

What advice do you give people who are newly diagnosed with pancreatic cancer?

I tell people who are newly diagnosed to get a second opinion about their proposed treatments and even about their diagnosis. I also pass along advice given to me by my surgeon, which is not to go to a neighborhood hospital or a general surgeon for a Whipple procedure. Go to a surgeon who specializes in Whipple procedures. I also tell those same people to make sure their children and siblings get annual screenings. Early detection helps a lot with this disease.

To learn more about how patients cope with pancreatic cancer, read “Facing Each Day with Pancreatic Cancer, Hand-in-Hand.”

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.