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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.”