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Last year, chimeric antigen receptor (CAR) T cell therapy was recognized as the Advance of the Year in the American Society of Clinical Oncology (ASCO) Annual Report for its demonstrated benefits in certain blood cancers and its potential in many other tumor types. At this year’s ASCO annual meeting, the interest in CAR T cell therapy remains strong with the availability of more data for approved and investigational therapies.

“So far, the oncology community has greeted CAR T cell therapy with extraordinary enthusiasm,” said Dr. Jeremy Abramson, clinical director for the Center for Lymphoma at Massachusetts General Hospital. “We’ve had few effective treatment options for difficult-to-treat blood cancers like diffuse large B-cell lymphoma [DLBCL]. The newest data from ASCO continues to suggest that CAR T cell therapy may represent an important advance for some patients.”

DR. JEREMY ABRAMSON FROM MASSACHUSETTS GENERAL HOSPITAL BELIEVES THAT MANY QUESTIONS ABOUT CAR T CELL THERAPIES WILL BE ADDRESSED AT THIS YEAR’S ASCO ANNUAL MEETING.

DR. JEREMY ABRAMSON FROM MASSACHUSETTS GENERAL HOSPITAL BELIEVES THAT MANY QUESTIONS ABOUT CAR T CELL THERAPIES WILL BE ADDRESSED AT THIS YEAR’S ASCO ANNUAL MEETING.

A Long Time in the Making

The first investigational CAR T cells were developed over 30 years ago, using genetic engineering advances with the goal to reprogram a patient’s immune system to recognize and attack cancer cells. Early attempts were lackluster; the engineered T cells were slow to reproduce, died quickly and produced weak immune responses that weren’t effective at killing tumor cells.

Advances in genetic engineering tools and techniques, as well as a far better understanding of the human genome, have advanced this type of technology. Over the past five years, the number of clinical trials involving CAR T cell therapies has skyrocketed from just a handful to more than 180.

In 2017, the U.S. Food and Drug Administration approved the first two CAR T cell therapies — one for children with relapsed or refractory acute lymphoblastic leukemia and another for non-Hodgkin lymphoma in adults who have failed at least two other kinds of treatment.

These are just two examples of diseases for which CAR T cell therapy represents a radically different therapeutic approach.

“CAR T cells are specifically engineered to recognize, go after and attack the cancer cells,” Abramson said.

How CAR T Cell Therapy Works

CAR T CELL THERAPY BEGINS BY REMOVING A PATIENT’S T CELLS, WHICH FIGHT INFECTIONS IN THE BODY, THROUGH A BLOOD DRAW. THOSE CELLS ARE THEN SENT TO A MANUFACTURING SITE WHERE THEY ARE GENETICALLY ENGINEERED TO RECOGNIZE AND ATTACH TO ANTIGENS EXPRESSED ON CANCER CELLS AND SOME NORMAL CELLS. PATIENTS THEN RECEIVE CHEMOTHERAPY BEFORE THESE PROGRAMMED CELLS ARE RETURNED TO THEIR BODIES TO SEEK AND ATTACK CANCER CELLS. PATIENTS ARE MONITORED FOR SIDE EFFECTS AFTER CAR T CELL THERAPY.

Exploring Questions in Blood Cancers

While the first two CAR T therapies have been approved, Abramson notes that many questions remain, including CAR T cell therapy production, safety and longevity.

Scientists are still fine-tuning the process of creating CAR T cell therapies. For instance, studies continue around different ratios of two subtypes of T cells—CD4+ and CD8+ T cells— that may behave differently. That’s because CD8+ T cells have a cancer-killing effect, while CD4+ T cells produce chemical messages that boost T cell production. Finding the right ratio of CAR T cells created from these subtypes may impact the efficacy and safety of these treatments, according to Abramson. But the clinical significance of CD4:CD8 ratio remains unknown.

“Even the most effective therapies can only be administered if the toxicities can be identified and successfully treated and reversed,” Abramson explained. “We’re continuing to learn about potential toxicities with the different approved and investigational CAR T cell therapies, and how to optimally manage and prevent them.”

We are studying ways to make these therapies work better, designing more effective CAR T cells that may target different or multiple cancer proteins and combining them with other medications.

Adverse events that have been noted in trials of CAR T cell therapy include cytokine release syndrome (CRS) and neurotoxicity. CRS symptoms include fever, nausea, or headaches, and neurotoxicity symptoms include delirium, headaches and problems speaking. ASCO attendees will get a better understanding of the severity and timing of these and other potential safety issues as well as insight into paths for their prevention and treatment.

As for the durability of CAR T cell therapies, Abramson believes there’s work to be done.

“Less than half of patients with DLBCL who receive CAR T cell therapy are still in remission a year later,” he said. “We are studying ways to design more effective CAR T cells that may target different or multiple cancer proteins and learn how to combine them with other medications like checkpoint inhibitors or immunomodulators to see if we can enhance CAR T cell activity.”

Beyond the Blood

So far, CAR T cell therapies have only been approved for the treatment of blood cancers. The major challenge in solid tumors, such as lung and breast cancers, has been identifying a target that’s restricted to the tumor, so the CAR T cells don’t also attack the patient’s healthy cells.

CAR T cells kill healthy immune cells called B lymphocytes, for instance, as well as lymphoma cells, but patients can often do without those particular cells. But a treatment that attacked an entire organ — or organs — would have catastrophic effects.

According to Abramson, one potential way to get around the problem may be to create CAR T cells that attack only when they encounter a specific combination of targets. While a single protein might be shared by cancer and healthy cells, researchers are searching for patterns of multiple targets only found on cancer cells. Whether or not this tactic succeeds, Abramson is optimistic that scientists can find a way.

To learn more about the advances that will be discussed at ASCO 2018, read “ASCO 2018 Preview: Precision Medicine, CAR T Cells and Immunomodulators.”

Dr. Abramson is a lead principal investigator for Juno and has consultant/advisory roles with Celgene.

The cost and value of medical innovation in oncology is one of the hot topics on the agenda for this year’s American Society for Clinical Oncology Annual Meeting (ASCO). One cancer that has seen an increase in relative survival rates over the past decade is multiple myeloma. So it makes sense that multiple myeloma is at the center of a debate at ASCO on the cost and value of new therapies.

As one of the participants in that debate, Dr. Rafael Fonseca, a hematologist, oncologist and chair of the Department of Internal Medicine at Mayo Clinic in Arizona, will argue that society can’t afford not to provide patients with multiple myeloma access to the best care possible. In this Q&A, Fonseca shares his views about the affordability of cancer therapies, why many doctors hold onto the notion that medications are too expensive and the implications for future multiple myeloma treatment.

DR. RAFAEL FONSECA FROM THE MAYO CLINIC IN ARIZONA BELIEVES THAT SOCIETY CAN’T AFFORD NOT TO PROVIDE PATIENTS WITH MULTIPLE MYELOMA ACCESS TO THE BEST CARE POSSIBLE.

DR. RAFAEL FONSECA FROM THE MAYO CLINIC IN ARIZONA BELIEVES THAT SOCIETY CAN’T AFFORD NOT TO PROVIDE PATIENTS WITH MULTIPLE MYELOMA ACCESS TO THE BEST CARE POSSIBLE.

In the debate, you will argue that we can’t afford not to provide patients with access to the right treatment for them. How did you come to that conclusion?

“Over the past decade, I’ve witnessed so many patients with multiple myeloma increasingly beating the odds for survival. So I wanted to know what exactly was responsible for this. After some research, I concluded—as many of my colleagues have also—that it was the new medications. These innovative therapies provide tremendous value to our patients and society.”

When you discuss this topic with your fellow oncologists, what is the most compelling evidence supporting your position?

“I usually take a stepped approach to presenting my point of view. I ask them to consider the progress that we’ve made in survival in cancers like multiple myeloma and what they have seen in their own patients. I help them understand the value by walking them through all the new therapies that those improvements are attributed to. You can’t just look at the price tag of a specific medication, which they often focus on most.”

The opposing position is that patients and society cannot afford multiple myeloma therapies. Why don’t you believe that argument?

“The data don’t support the argument. One study found that 98 percent of patients paid $50 or less to fill their prescriptions in 2017. While that could be a hardship for some people, it is far different than the list prices of thousands of dollars that make headlines. So we need to talk about what patients are paying in the real world and what’s best for our patients.”

“Beyond that, there are the ethical considerations. Doctors should prescribe the medications they believe will benefit their patients the most.”

Why do doctors continue to say that new cancer therapies are unaffordable if the data suggest they aren’t for most patients?

“I feel like most cancer doctors are concerned about the cost of prescription medications out of their sense of compassion and responsibility for their patients. They see their role as treating patients responsibly. Prescribing a therapy that may cost them thousands seems inconsistent with that mission.”

“But they are so busy caring for patients that they don’t have the time to research the real world data about what patients actually pay for their prescriptions. As we see in other areas of discourse, facts matter. I believe that misunderstood empathy and baseless rhetoric can have real-world consequences for patients.”

Everyone agrees that today’s cancer treatments are simply not good enough and that innovation is key to improving cancer care.

Have you ever had a patient —or many—who could not access treatments that you prescribed?

“I cannot think of a single patient who could not access a medication due to financial reasons. I’ve had patients who have chosen other treatment options but for other reasons—never financial. In a few instances, we have had to go above and beyond to get them financial help from the manufacturer or non-profit groups. But those cases are the exceptions, not the rule.”

What do those on the other side suggest should be done about the affordability of multiple myeloma care and what are the potential consequences?

“When we say that new therapies are too expensive, what we’re doing is calling for price regulations. But without a doubt in my mind, those regulations will kill innovation. Medical innovation is a high-risk, high-reward endeavor. We should not fool ourselves into thinking that there won’t be consequences; we’ll have fewer new treatments for our patients as a result.”

Is there common ground in this debate over the value of multiple myeloma care that can be used to move forward?

“There is plenty of common ground. I think today’s cancer treatments are simply not good enough and that innovation is key to improving cancer care. So we should make sure that we do not hinder that innovation.”

“Most cancer doctors also agree that clinical trials should be as fast as possible without sacrificing safety, so they cost less and lead to faster approvals. And I think that we all think patients should have access to the best treatment options but have different ideas on how to provide that access.”

To learn more about the how medical innovation has improved the treatment of multiple myeloma, read “A Decade of Progress in Multiple Myeloma, and More to Come.”

Dr. Fonseca has received speaker fees, advisory board fees, travel support in connection with consulting services, and research support from Celgene.

Last year, Americans took more prescription medicines than ever before due in part to efforts to improve adherence. In addition, the number of new medications approved more than doubled from 2016. But if you think the country is paying substantially more at the pharmacy as a result, you may be surprised.

Prescription medication spending increased just 0.6 percent last year, less than the rate of inflation and the lowest growth rate since 2012, according to a report published by the IQVIA Institute for Human Data Science. Murray Aitken, executive director of the IQVIA Institute, explains why prescription spending growth has remained in check, why growth in prescription spending isn’t necessarily a bad thing and what we can expect in the near future.

MURRAY AITKEN, EXECUTIVE DIRECTOR OF THE IQVIA INSTITUTE, BELIEVES HIGH GROWTH IN PRESCRIPTION SPENDING CAN REPRESENT GOOD VALUE IN TERMS OF LONGER, HEALTHIER LIVES.

MURRAY AITKEN, EXECUTIVE DIRECTOR OF THE IQVIA INSTITUTE, BELIEVES HIGH GROWTH IN PRESCRIPTION SPENDING CAN REPRESENT GOOD VALUE IN TERMS OF LONGER, HEALTHIER LIVES.

Why was the growth rate for prescription medication spending just 0.6 percent in 2017?

“That relatively low rate is due to patent expirations and lower cost generics, which are offsetting the growth from new therapies and price increases. In fact, a record-setting 1,027 generic medications were approved in 2017. If we look at retail and mail-order prescriptions, spending actually declined 2.1 percent last year, as we had more new injectable and infusible treatments than oral treatments last year. The prescription spending growth rate in 2017 is significantly lower than the 9 to 10 percent that we saw in 2014 and 2015, which were historically high levels of growth.”

What happened in 2014 and 2015 that caused those high rates of growth in prescription spending?

“Between 2014 and 2015, we saw the rise and fall of hepatitis C spending, as therapies that cured a significant number of patients were introduced. Since that wave of innovation, spending has grown more slowly.

“When innovative therapies are introduced that address an unmet need for many patients, spending will go up as we saw in hepatitis C. That is not a bad thing. That growth is supported by the value that innovative therapies provide in the form of longer and better lives. When we get an effective therapy for Alzheimer’s, for instance, we will see spending increase as we treat the millions of Americans to improve their lives and reduce other healthcare costs of caring for those with the disease.”

Prescription Drug Spending Increased Just 0.6% in 2017

How are the prescription spending growth rates in your report different from those that we see elsewhere?

“First, we are not basing our analysis on list price. We are looking at the revenue manufacturers receive after all the rebates, discounts, coupons and vouchers have been accounted for. Secondly, we are reporting on the total use of all medicines through all distribution channels, not just individual medications. Individual medication costs tend to make headlines but aren’t necessarily reflective of the overall market.”

Are patients paying more for prescription medications because of price increases?

“Over the past five years, patient out-of-pocket costs have actually declined by 15 percent to $8.69 per prescription on average in 2017. These lower out-of-pocket costs are due to higher usage of generics and manufacturers’ coupons.

“But patients’ out-of-pocket costs remain high for a small number of prescriptions. Patients paid more than $500 for about 0.2 percent of all prescriptions in 2017. These patients are usually those in the coverage gap of Medicare plans or the deductible phase of their insurance plans. They may get the same medicine for less at a different time of year.”

Patient Out-of-Pocket Costs Down 15% Since 2013

What is the outlook for prescription spending and patient costs over the next few years?

“While there are many uncertainties, including government policy, we don’t foresee any major disruptions over the next five years. So we’re forecasting spending growth to average between 2 and 5 percent per year. Growth will continue to be driven by innovation in various disease areas, but especially in oncology. That growth will continue to be offset by expirations of patents, which are designed to keep spending in check over the long term.”

To learn how medical innovation saves lives and has reduced health expenditures, read “When It Comes to Healthcare Costs, New Medicines Are the Solution, Not the Problem.”

More than 32,000 oncology professionals will soon gather in Chicago for the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting. This year’s meeting, with a theme of “Delivering Discoveries: Expanding the Reach of Precision Medicine,” promises to be the most exciting yet because researchers are further unraveling the different mechanisms by which cancer starts, grows and metastasizes, as well as how the immune system responds to it.

This progress is ushering in a new era of precision medicine, according to Wim Souverijns, Corporate Vice President of Global Marketing for Hematology & Oncology at Celgene. Much of the research Celgene will present at this year’s meeting includes updates on its chimeric antigen receptor (CAR) T cell therapy platforms, as well new data related its investigational pipeline across hematologic malignancies and solid tumors.

CAR T Cell Data Continue to Roll In

One area of personalized medicine with incredible potential in cancer is CAR T cell technology. This process involves a patient’s immune cells being collected, modified in a laboratory to recognize cancer cells and reinfused to attack the cancer.

At this year’s ASCO meeting, Souverijns is looking forward to seeing longer-term safety and outcome data for several clinical trials involving investigational CAR T cell therapies in relapsed or refractory patients with difficult to treat blood cancers like multiple myeloma and diffuse large B-cell lymphoma.

While these therapies come with great promise, their safety profile needs to be properly characterized to ensure appropriate use of these therapies, Souverijns explains. At ASCO he expects further insights about particular side effects such as cytokine release syndrome (CRS) and neurotoxicity.

“These longer-term data will help us to better understand these effects and how to manage them,” he said. “This will be crucial to realize the promise of these investigational treatments for patients.”

Three years ago, most patients were getting doublets, and people were questioning the need for triplets. Today, people are talking about quadruplets.

CAR T beyond Blood Cancers

The big hope is that CAR T cell therapies will be able to go beyond hematological malignancies and effectively target solid tumors as well. Preclinical data of CAR T cell therapies in solid tumors are emerging. The challenge, though, is that solid tumors often aren’t responsive to treatment simply because therapies physically can’t reach the tumor.

“CAR T also holds promise in solid tumors, but it’s going to be a much harder nut to crack from a technological and scientific perspective than with blood cancers,” Souverijns said. “The integration of Juno’s recently acquired CAR T cell science powerhouse with Celgene’s deep disease and cellular therapy expertise provides a great opportunity with these new investigational technologies for cancer patients.”

AT THIS YEAR’S AMERICAN SOCIETY OF CLINICAL ONCOLOGY MEETING, ATTENDEES WILL DISCUSS ADVANCES IN CANCER RESEARCH, INCLUDING ADVANCES IN CAR T CELL THERAPY AND COMBINATION THERAPIES BUILT ON THE FOUNDATION OF IMMUNOMODULATORS.

AT THIS YEAR’S AMERICAN SOCIETY OF CLINICAL ONCOLOGY MEETING, ATTENDEES WILL DISCUSS ADVANCES IN CANCER RESEARCH, INCLUDING ADVANCES IN CAR T CELL THERAPY AND COMBINATION THERAPIES BUILT ON THE FOUNDATION OF IMMUNOMODULATORS.

Triplets Becoming More Common in Multiple Myeloma

At last year’s ASCO conference, clinicians saw data combining immunomodulators with other therapies to treat diseases like multiple myeloma. Souverijns expects to see even more data from trials this year testing combination therapies. While these combinations used to focus on later lines of treatment when patients have exhausted other options, the new data are showing options for earlier patient segments as well.

More seems like it may be better as more triplet therapies are being approved and utilized, while even quadruplets are being tested now. This is something he expects will be discussed at length at the conference as doctors are realizing that adding new therapies to the foundation of an immunomodulator may drive better outcomes.

“Three years ago, most patients were getting doublets, and people were questioning the need for triplets,” Souverijns said. “Today, people are talking about quadruplet therapy. It’s amazing how quickly cancer care is progressing, making each ASCO more extraordinary than the last.”

For all the potential advancements, Celgene’s Vice President, U.S. Medical Affairs Teng Jin Ong, M.D., pointed to precision medicine at this year’s conference.

“Our increasing insight into the biology of cancer drives the discovery of new therapies that are targeting very specific cancer mutations and allow for greater improvements in outcomes for patients with such mutations,” said Dr. Ong. “We’ll remember 2018 as the tipping point for precision medicine and showing how it could work in practice.

To learn more about how CAR T cells may help immune cells identify cancer cells, read “Revealing Cancer Cells to the Immune System.”

 

When Ralph Hills was diagnosed with an aggressive blood cancer called acute myeloid leukemia (AML) more than three years ago, his doctor told him to get his affairs in order. He interpreted that statement as a polite way of saying that he didn’t have long to live. But a last-minute phone call from his doctor led him to the right treatment for his disease.

AML is driven by as many as 76 gene mutations. Approved and investigational therapies that target those mutations are opening up the possibility to tailor treatments for individual patients. As a preview to a Facebook Live event hosted by Celgene on Wednesday, April 18 at 9:30 a.m. EDT in recognition of the third annual AML World Awareness Day on April 21, Hills discusses how precision medicine is helping to save lives by sharing his journey with AML.

How did you learn that you had AML?

“In December 2014, I was 70 years old and experiencing back pain. I thought my doctor was going to schedule surgery, but instead, he told me to make an appointment with our local cancer center in Connecticut. A couple days later, my wife and I were sitting in a room with an oncologist who told me I had AML. He said that he was going to prescribe chemotherapy and that I should get my affairs in order. He also suggested that we get a second opinion.”

How did you react to learning you had AML?

“That meeting left me in a haze. I felt like I lost control of everything. We cried. I knew that the prognosis for AML was not good for someone my age, and I lost hope. But a few days later, we took my doctor’s advice about getting a second opinion and went to the leukemia department of Weill Cornell Medicine in New York City.

“Targeted therapy inhibits the activity of specific genes, proteins or antibodies that have mutated and are helping cancer cells to grow. This inhibition can slow or stop the growth of cancer cells.”

Targeted therapy inhibits the activity of specific genes, proteins or antibodies that have mutated and are helping cancer cells to grow. This inhibition can slow or stop the growth of cancer cells.

Targeted therapy inhibits the activity of specific genes, proteins or antibodies that have mutated and are helping cancer cells to grow. This inhibition can slow or stop the growth of cancer cells.

Did your treatment plan change after receiving the second opinion?

“It didn’t, at first. My new oncologist recommended the same decades-old standard of care for AML, which is an aggressive chemotherapy regimen. I knew the treatment would have harsh side effects and would not cure my AML.”

“The week before my treatment was scheduled to start, I visited a friend who was being treated with chemotherapy for his blood cancer. He told me that he was tired of fighting and ready to give up. I imagined that I was going to be in his position in a matter of months.”

What happened next?

“The night before my chemotherapy was going to start, my doctor called during her ski vacation. She asked me if I would consider changing my treatment plan. Apparently, I had a molecular mutation in a gene that might respond to a targeted therapy that was in clinical trials. So I had a choice of being treated with chemotherapy or an unknown treatment. I trusted my doctor and knew that she wouldn’t have offered it to me unless she thought it was the right treatment for me at that time.”

AML Awareness: A Patient's Perspective

Did you know that your doctor had ordered molecular profiling of your disease?

“No, we didn’t know at the time. She took my blood and bone marrow samples when we first met with her, but I thought that was just pretty standard routine tests. I didn’t know that she went the extra mile and ordered molecular profiling to see if I had any mutations. But that test was what qualified me for this trial.”

What was your treatment experience like?

“For six months, I could hardly move. I was in my bed unable to eat regular foods, getting all my sustenance from nutritional supplements. I lost 45 pounds.”

“After that, the bad leukemia cells began to disappear, leaving room for the healthy blood cells and platelets. Every two weeks, I went in for testing and watched the number of bad blood cells drop. Then I was told that I have indiscernible leukemia. The doctor told me to go home because she had sick people to see. That moment was the start of my second life.”

The idea to target the bad guys and avoid affecting other healthy cells makes sense to me.

What kept you going during your treatment?

“My dedicated wife, Dorcas, handled much of the physical and emotional load. She made phone calls and wrote newsletters to keep my friends and family up to date. They sent me get-well cards and flowers. I played card games with my grandchildren for five minutes at a time, which was all I could manage. But little things like that kept me going.”

How do you feel now?

“My life has changed in many, many ways. Like all cancer survivors, I visit my doctor for regular medical check-ups. I’m eliminating all the stuff that wasn’t important and enjoying every moment more. I’m semi-retired now. This summer, I spent a couple of months in Canada. In September, I will celebrate my third birthday after my diagnosis. That’s the way I’m treating all this — as a complete reset on my life.”

What do you hope the future has in store for AML patients?

“The idea to target the bad guys and avoid affecting other healthy cells makes sense to me. I’m delighted that my doctor did the molecular profiling to qualify me for the right clinical trial among the 7,000 that are ongoing in the United States so I could get the right treatment. I hope every person, young and old, rich and poor, gets the right treatment at the right time like I did. I feel like I am one of the very, very lucky ones.”

To learn more about the progress that is being made in the treatment of AML, join us for a special Facebook Live event with AML patient Ralph Hill on Wednesday, April 18, 2018 at 9:30 a.m. EDT.

When he started his career in medicine over 25 years ago, Dr. Jack Burks had no treatments for his patients with multiple sclerosis (MS). So when the first MS treatment was approved in 1993, he was grateful to finally have something — anything — to prescribe for one of his patients who had a severe form of the disease. Twenty years later, that patient is still doing well on that same medication and has yet to suffer a relapse.

Stories such as this one help demonstrate the impact that progress in research can make on patients’ lives. But Dr. Burks, the chief medical consultant at the Multiple Sclerosis Association of America (MSAA), knows that there are still many unmet needs. Although 15 therapies have been approved for the treatment of MS, none offer a cure, and, as with all treatments, there are potential risks to consider. In this Q&A, Dr. Burks provides his perspectives on the current state of MS treatment and why he remains optimistic about the future.

DR. JACK BURKS, THE CHIEF MEDICAL CONSULTANT AT THE MULTIPLE SCLEROSIS ASSOCIATION OF AMERICA, KNOWS THAT THERE ARE STILL MANY UNMET NEEDS IN MULTIPLE SCLEROSIS.

DR. JACK BURKS, THE CHIEF MEDICAL CONSULTANT AT THE MULTIPLE SCLEROSIS ASSOCIATION OF AMERICA, KNOWS THAT THERE ARE STILL MANY UNMET NEEDS IN MULTIPLE SCLEROSIS.

What is the treatment journey like for people with MS?

“The MS treatment journey for patients can fall into a pretty broad spectrum. For some patients, treatment goes smoothly. The response is excellent, and everything goes well. But for others, it can be a struggle.

“While we have several therapies available, we cannot guarantee that they will work for a particular patient. So we go with what we think will work best, and if it doesn’t work out, we may recommend switching therapies. That change, of course, can be stressful and confusing for patients. But without treatment, the disease will worsen, and patients will only experience further impairment.”

When should a patient and their doctor consider switching therapies?

“Doctors usually recommend changing therapies for one of two reasons: safety or efficacy. Either the patient cannot tolerate the treatment because of side effects, or the treatment is no longer controlling the disease.

“Patients tend to focus more on the side effects. They might not see the brain lesions in magnetic resonance imaging, but they do know if they get sick every time they take a medication. Side effects scare them.

“But switching therapies should not be an immediate reaction to those side effects. If someone has mild injection site reactions, the doctor can recommend applying a warm compress. If they experience gastrointestinal problems, their doctor might suggest taking the medication at a different time of the day.”

What can help smooth out the journey for people with MS?

“A good doctor-patient relationship is essential to managing treatment expectations and side effects alike. Patients need to know and feel that their doctor is looking out for their best interests. Doctors need to understand a patient’s priorities and communicate in a way that builds trust.

“Patients need to become knowledgeable about their disease before their first appointment. They should prepare a list of questions to discuss with their doctor. There are no wrong questions. Doctors and patients should share treatment decisions, which helps patients own their treatment and can improve adherence.”

We’re doing pretty well even though we don’t know the cause of the disease. The future looks very bright for new MS treatments.

How does patient preference factor into MS treatment decisions?

“Doctors need to talk with their patients about their preferences. Some patients do not mind if their treatment is a pill or an injection, while others have stronger opinions.

“Doctors need to understand a patient’s specific concerns because if not, that’s an equation for poor adherence.”

When should doctors bring up MS clinical trials with their patients?

“One of the first questions my patients ask me is ‘What’s new?’ They want to know about the new options that are approved or being developed. So doctors need to know about the trials that are ongoing. I try to focus on trial results, side effects and contraindications with other medications.

“Patients might not be interested in enrolling in a trial, especially if their current treatment is working for them, but often they just want to know what is in the future of MS treatments and how it may affect their treatment down the line.”

What is your view of the future of MS treatment?

“I’m pretty optimistic. We’re doing pretty well even though we don’t know the cause of the disease. The future looks very bright for new MS treatments.

“When I look at these new medications, I consider not only how well a therapy reduces relapse rates, but also the safety profile. Side effects, as I mentioned, are one of the reasons why patients stop taking their medication. Right now, I am reviewing over 20 new therapies that are in trials and have the potential to help so many more people than we already are.”

To learn more about how researchers are studying new MS treatments, read “Relapse Rates: A Benchmark for Measuring MS Treatment Efficacy.”

February 28, 2018 marks the 11th annual Rare Disease Day, dedicated to raising awareness of the impact of rare diseases worldwide. This year’s theme – research – highlights the many advances that have been made in the treatment of rare diseases, while emphasizing remaining research gaps.

This year, and every year, Celgene joins with the many supporters of Rare Disease Day as we all strive toward the ultimate goal: cures.

A rare disease is one that affects fewer than 200,000 individuals in the United States and less than one in every 2,000 in Europe. As a whole, though, rare diseases are hardly insignificant; there are approximately 7,000 different rare conditions affecting more than 300 million people globally.

Over the decades, regulatory initiatives have helped to encourage more innovation and the generation of much-needed therapies for these patients. The U.S. Food and Drug Administration’s (FDA) Orphan Drug Act of 1983 spurred significant progress by creating incentives for research and allowing for expedited approval of new therapies. While fewer than 10 treatments for rare diseases were approved from 1973 to 1983, more than 400 medicines and biologic products for rare diseases have been approved since the Act was passed.

More recently, provisions of the 21st Century Cures Act of 2016 have helped to streamline FDA review of rare disease therapies. And in December 2017, the Rare 2030 pilot program was adopted by the European Commission to research sustainable policies, address challenges and identify opportunities in the field of rare diseases from 2020-2030.

And yet despite these efforts, approved therapies are available for only five percent of rare diseases. Clearly, we still have a long way to go for these patients.

With clinical trials in more than 45 rare cancers and immuno-inflammatory diseases, Celgene recognizes the significance of medical innovation to address these often overlooked diseases. Celgene’s research efforts center on patient need, including rare diseases.

Clinical trials are key to making progress, but for rare diseases, enrolling a clinical trial can be a challenge, given the limited number of patients. To find the most patients that can participate in a trial, Celgene believes a critical first step involves carefully examining and engaging the appropriate trial locations, for instance, by establishing a presence in countries where a rare disease is more common.

Driven by patient and physician insights, Celgene also leads research efforts that go beyond traditional trial programs. For instance, the Journey Pro app, launched by Sage Bionetworks with financial support from Celgene, uses patient-reported data collected through mobile and wearable technologies to help quantify the daily burden of chronic anemia, which affects patients with rare diseases such as myelodysplastic syndromes, myelofibrosis and beta-thalassemia. The app provides direct and immediate information to research participants to help them manage their health, with a long-term goal of developing a tool to evaluate new treatments.

In multiple myeloma, Celgene has collaborated with the UAMS Myeloma Institute and Dana-Farber Cancer Institute to create the Myeloma Genome Project, a global initiative compiling the largest set of genetic profiling data associated with clinical outcomes. The Project aims to develop a genetic classification system, and eventually, relevant tests for genetic mutations that could improve the diagnosis and prognosis of multiple myeloma while driving more personalized, targeted approaches to treatment.

In addition to these and other research endeavors, Celgene’s collaborative efforts with patient and professional advocacy organizations through the Patients’ Partners program, launched in 2011, explore new ways to strengthen patient support from diagnosis through treatment.

As research efforts in rare diseases continue to expand across the healthcare ecosystem, the progress being made drives Celgene to keep pushing forward in an effort to deliver new solutions that may improve patients’ lives.

When doctors evaluate if a treatment is working for one of their patients with ulcerative colitis (UC), an inflammatory bowel disease that causes damage to the mucosal layer of the digestive tract, they will ask their patient about symptoms, such as bleeding, diarrhea and pain. But when researchers are evaluating the effectiveness of potential new treatments in a clinical trial, they need to include an objective assessment of disease activity as well.

So researchers are increasingly using endoscopy, a procedure using a flexible tube equipped with a video camera to look at patients’ digestive tracts, and examining tissue samples removed during a biopsy under a microscope to determine how well the mucosa is responding to an investigational therapy. In this Q&A, Dr. Keith Usiskin, executive director at Celgene, explains how by combining these two measurements, an assessment of mucosal healing can be made.

DR. KEITH USISKIN, EXECUTIVE DIRECTOR AT CELGENE, BELIEVES THAT COMBINING ENDOSCOPIC AND HISTOLOGIC MEASUREMENTS PROVIDES A DETAILED VIEW OF MUCOSAL HEALING IN ULCERATIVE COLITIS.

DR. KEITH USISKIN, EXECUTIVE DIRECTOR AT CELGENE, BELIEVES THAT COMBINING ENDOSCOPIC AND HISTOLOGIC MEASUREMENTS PROVIDES A DETAILED VIEW OF MUCOSAL HEALING IN ULCERATIVE COLITIS.

Why is mucosal healing important?

“Ulcerative colitis causes massive damage to the mucosa, weakening blood vessels and, eventually, leading to ulcers. Doctors and researchers are finding that achieving mucosal healing correlates with a better quality of life and other measurable benefits for patients with UC.

“For instance, studies have found that patients with UC in remission and with no signs of microscopic inflammation are less likely to be hospitalized or to experience relapse, in which their UC symptoms return. The data isn’t as strong as we’d like just yet, but we see a definite trend beginning to take shape.”

How is mucosal healing assessed in UC trials?

“Organizations conducting UC clinical trials assess mucosal healing often use both endoscopic appearance — what a gastroenterologist sees regarding redness, inflammation and ulcers during an endoscopy — and histological appearance — what a pathologist sees under a microscope regarding inflammation when they examine a tissue sample from a biopsy.”

Why are both endoscopies and biopsies needed to assess mucosal healing?

“While UC causes mucosal damage continuously, not every part of the mucosa is affected to the same extent. So endoscopies provide researchers with a bird’s eye view of the mucosa throughout the entire rectum and large intestine, including regions that are very inflamed and those that are less so.

“But endoscopic appearance doesn’t tell you everything. Studies have found that up to 24 percent of patients whose mucosa looks good in endoscopic assessments still have evidence of microscopic inflammation when a pathologist looks at a biopsy taken from the mucosa. That inflammation suggests the mucosa still is not fully healed and that the patient is at higher risk for relapses.”

Mucosal Healing: An Increasingly Accepted Endpoint in Studies of Ulcerative Colitis Treatments

What constitutes mucosal healing in these assessments?

“Defining mucosal healing remains one of the biggest challenges in UC clinical studies. The medical community and regulatory agencies have not come to a clear consensus on what constitutes mucosal healing in UC.

“Several scoring systems have been proposed for endoscopic and histologic assessments, but researchers have not decided which should be used. In Celgene’s studies, we use a widely used index for disease activity by endoscopic assessment in UC developed by researchers at the Mayo Clinic and a grading scale for histological assessment in UC developed by Dr. Karel Geboes at the University Hospitals Leuven in Belgium. We classify mucosal healing as a Mayo score less than or equal to one and a Geboes histologic score of less than two.”

What are the challenges in assessing mucosal healing?

“Clinician bias has been one of the most significant limitations in assessing mucosal healing. If the patient says they’re doing great, the clinician is more likely to report that the mucosa looks better, even if it seems the same as before starting treatment. The inverse can be true as well. We use central readers who do not know the patient’s health status to eliminate that bias. They can grade the endoscopies and biopsies based solely on their best judgment and experience.”

“The combination of endoscopy and pathology assessment of biopsies is key to the assessment of mucosal healing.”

Does the invasive nature of this assessment affect patient retention?

“Some patients are hesitant to enter a clinical trial if there are too many colonoscopies or endoscopies. Clinical researchers try to limit that when designing protocols for studies to minimize procedures that patients may find uncomfortable.

“We try to make it clear to patients what is expected of them when they sign up for a clinical trial and limit the burdens as much as possible. But we still have to make these assessments to determine the efficacy of potential new treatment options for UC.”

What could improve the assessment of mucosal healing?

“In the future, we may identify biomarkers that correlate with clinical symptoms, benefits and outcomes for patients with UC. Celgene is participating in an initiative to identify such biomarkers, and one day, noninvasive biomarkers may eliminate the need for endoscopy or biopsies. Imaging techniques such as MRIs and CT scans also may prove useful to assessing mucosal healing in future clinical trials and in the clinic. But right now, the combination of endoscopy and pathology assessment of biopsies is key to the assessment of mucosal healing for both scientific and regulatory purposes.”

To learn more about clinical trials for ulcerative colitis and other inflammatory bowel diseases, read “The Importance of Clinical Trials for Inflammatory Bowel Disease.”


The immune system is the body’s main level of defense against a hostile world. From viruses and bacteria to the bodies’ own sick and dying cells, immune cells search out and destroy the trouble-makers.

But cancer cells can be tricky; they have ways of hiding from the immune system. The ability of tumor cells to evade the immune response is a key reason why cancers can be so difficult to treat. That’s why researchers are creating tools to help patients’ immune cells better detect and then kill tumor cells.

One method that’s recently become available for childhood leukemia is to engineer a patient’s own T cells – a type of white blood cell – to recognize proteins found on the surface of the cancer cells. This type of therapy is called chimeric antigen receptor (CAR) T cell therapy.

With CAR T cell technology, doctors remove some T cells from a patient and genetically modify them with a homing beacon for proteins made by tumors. The newly armed T cells are then multiplied into the billions and infused back into the patient to hunt down the cancer.

The type of CAR engineered for a patient depends on his or her cancer. For example, the special receptor might target a protein called CD19, which sits on the surface of many leukemias and lymphomas, including diffuse large B cell lymphoma. Or the CAR might be designed to draw T cells to a protein called B-cell maturation antigen, which is found on tumor cells in up to 70 percent of patients with multiple myeloma.

Researchers are currently investigating ways to enhance this technology by finely tuning the ratio of different kinds of T cells given back to the patient. The idea is to give patients just the right combination of the cells that carry out the killing (called CD8 T cells) and the cells (CD4 T cells) that are thought to help make the assassins more mobile and recruit additional immune cells.[i]

While CAR T cell therapies use markers on the outside of cancer cells to fight them, another immune strategy in development looks inside. In this case, the patient’s immune cells are given an engineered T cell receptor (TCR) that helps it recognize pieces of proteins from within cancer cells. When these protein bits find their way to the surface of the tumor cell, the engineered TCR helps the T cell latch on tightly to the cancer cell.

Whether from within or without, cancer cells make proteins that reveal them as unwanted guests in the body. Harnessing the power of the immune system to better detect these flags may one day offer new hope to patients who currently have few options.



[i] Abbas AK, Lichtman AH, Pillai S. Cellular and Molecular Immunology. 8th ed. Philadelphia, PA: Saunders Elsevier; 2012.

New Jersey is well-positioned for strong economic growth, and biopharmaceutical innovation is a significant driver of that potential. In 2011, the biopharmaceutical industry supported 322,049 direct and indirect jobs and created $87 billion in economic output in the Garden State. To ensure a high-growth N.J. economy for the future, the biopharmaceutical industry is helping to strengthen the state’s ecosystem of innovation by supporting emerging companies, job growth and pro-innovation policies.

“Historically, New Jersey has been a powerhouse in the biopharmaceutical industry,” said Debbie Hart, president and CEO of BioNJ, a non-profit that promotes the state’s biotechnology industry. “Because of its strong foundation in life sciences, increasing early-stage medical innovation is one of the greatest growth opportunities in New Jersey.”

One reason for New Jersey’s historical and continued leadership in the global biopharma industry is its highly skilled and educated workforce. The state has skilled biopharmaceutical talent and more scientists and engineers per square mile than anywhere else in the world. This workforce is supported by the five research universities,13 teaching hospitals and four medical schools that call New Jersey home.

Location also makes New Jersey attractive to biotech companies, given its proximity to important collaborators, which include the many established pharmaceutical companies within the state, the U.S. Food and Drug Administration and the National Institutes of Health a train ride away in Maryland, and Wall Street and venture capital across the river in New York.

Startups are particularly important in driving medical innovation in New Jersey’s biopharmaceutical industry. The majority of treatments approved in recent years originated in companies outside of the 30 largest biopharmaceutical firms; 2016 followed that trend, with more than 60 percent of approved therapies coming from companies with a significant New Jersey footprint.

Incubators serve an important role in helping startups establish themselves and grow, providing resources that would otherwise be unavailable

While New Jersey has the right ingredients to attract biopharmaceutical startups, its entrepreneurial ecosystem has the opportunity to offer even better support to home-grown startups, through incubators and business accelerators that offer resources such as funding, mentoring, workspace or equipment to young companies. Currently, New Jersey has 15 business incubators and accelerator programs compared with 375 for California and 179 for New York.

DEBBIE HART, CEO AND PRESIDENT OF BIONJ, BELIEVES NEW JERSEY’S BIOPHARMACEUTICAL INDUSTRY HAS THE POTENTIAL TO DRIVE THE STATE’S ECONOMIC GROWTH.

DEBBIE HART, CEO AND PRESIDENT OF BIONJ, BELIEVES NEW JERSEY’S BIOPHARMACEUTICAL INDUSTRY HAS THE POTENTIAL TO DRIVE THE STATE’S ECONOMIC GROWTH.

“Incubators serve an important role in helping startups establish themselves and grow, providing resources that would otherwise be unavailable to such young companies,” Hart said. “Looking at the biopharma industry in New Jersey, more incubators can certainly help increase early-stage innovation.”

Recognizing this opportunity, Celgene has launched the Thomas O. Daniel Research and Collaboration Center on its campus in Summit, New Jersey. The new center will provide state-of-the-art facilities and resources for high-potential scientists to build on their preclinical research in the important effort to discover innovative therapies for patients with unmet medical needs.

“With its iconic brand and cutting-edge science, the Thomas O. Daniel Research Incubator and Collaboration Center has the potential to attract, create and support companies that will produce the world’s next generation of therapies and cures,” Hart said. “Those treatments will help cut the overall cost of health care, which will benefit the economy, society and—most importantly—our patients.”

Prospective researchers, entrepreneurs and companies interested in joining the Incubator will submit applications for residency on the webpage within the Collaboration Center, which will be reviewed by a Celgene selection committee.

“The HealthCare Institute of New Jersey (HINJ) congratulates Celgene on the launch of its incubator, which will enhance New Jersey’s expanding innovation ecosystem,” said Dean J. Paranicas, President and Chief Executive Officer of HINJ.  “We look for this exciting initiative to create opportunities for new life sciences companies to develop novel treatments and cures that will benefit patients everywhere.”

To learn more about  the Thomas O. Daniel Research Incubator and Collaboration Center  and find out how you can apply, visit CelgeneIncubator.com.

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!