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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.”