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Top 10 Clinical Research Achievement Awards - Top 10 Awardees

Friday, January 31, 2020   (0 Comments)
Posted by: Kimberly Durante
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CR Forum Announces 2020 Top Ten Clinical Research Achievement Awards

Washington, D.C. – January 31, 2020 – The Clinical Research (CR) Forum will honor ten outstanding clinical research studies at the 2020 Top Ten Clinical Research Achievement Awards at the National Press Club on April 14, 2020. At the event, one study will receive the prestigious Herbert Pardes Clinical Research Excellence Award for the research study that best shows a high degree of innovation and creativity, advances science, and has an impact upon human disease. 

These award-winning studies exemplify major advances resulting from the nation’s investment in research to benefit the health and welfare of its citizens, and reflect the influential work being conducted by investigators at nearly 60 research institutions and hospitals across the United States, as well as at partner institutions from around the world. All nominated studies were published in peer-reviewed journals during 2019.

The Top 10 were selected based on the degree of innovation and novelty involved in the advancement of science; contribution to the understanding of human disease and/or physiology; and potential impact upon the diagnosis, prevention, and/or treatment of disease.

The 2020 Top 10 Clinical Research Achievement Awardees are (in alphabetical order according to title):

· A041202: A Randomized Phase III Study of Bendamustine Plus Rituximab Versus Ibrutinib Plus Rituximab Versus Ibrutinib Alone in Untreated Older Patients (65 Years of Age) with Chronic Lymphocytic Leukemia (CLL), nominated by The Ohio State University, and represented by Dr. Jennifer Woyach, Associate Professor of Medicine. This study compared standard chemotherapy (bendamustine plus rituximab) to two targeted treatment regimens (ibrutinib given alone or in combination with rituximab) for the initial treatment of patients age 65 or older with chronic lymphocytic leukemia (CLL).  CLL is the most prevalent adult leukemia, and the average age at diagnosis is about 70, however, this is one of the first trials specifically targeting this older patient population.  The results of this study established ibrutinib as a standard of care for the initial treatment of older patients with CLL, and changed the paradigm of initial treatment away from chemotherapy and toward targeted therapy.

 

· Breakthrough Discovery and Development of Innovative Precision-Based Therapy in Complex Lymphatic Anomalies, nominated by the Perelman School of Medicine at the University of Pennsylvania, and represented by Dr. Hakon Hakonarson, Professor of Pediatrics. Lymphatic vessels are part of the human body’s lymphatic system, which transports a clear fluid containing white blood cells called lymph around the body to help clear toxins and waste. Lymphatic anomalies, such as central conducting lymphatic anomaly (CCLA) are life-threatening rare diseases that disrupt circulation of lymphatic fluid resulting in swelling of multiple organs.  The study team performed whole-exome sequencing on DNA from a severe CCLA patient, a 12-year old boy, which identified a previously undiscovered mutation in the ARAF gene.  The researchers then worked with zebrafish models to explore how the responsible gene mutation disrupted lymphatic channels. Next, they demonstrated that a drug called a MEK inhibitor, known to act on biological pathways affected by ARAF, rescued the structural defect in the zebrafish, causing them to develop normal lymphatic vessels. These results supported the patient’s medical team request to use a MEK inhibitor called trametinib (a chemotherapy drug) in the patient. The patient had been on palliative care and was predicted to have only months to live. After three months of treatment, the patient showed significant improvements in his breathing and reduced fluid retention, and an MRI showed that his lymphatic vessels were remodeling themselves. This remodeling resulted in marked improvement of the patient’s pulmonary function tests, greatly reduced his reliance on supplemental oxygen, and enhanced his ability to return to near normal daily activities. The results are a clear example of how knowledge of a genetic mutation and its mechanisms can guide new opportunities for existing medical treatments, which in this case was life-saving.

 

· Canagliflozin and Renal Outcomes in Type 2 Diabetes and Nephropathy, nominated by Stanford University, and represented by Dr. Kenneth Mahaffey, Professor of Medicine (Cardiovascular Medicine). This landmark clinical trial showed that an approved drug, canagliflozin, lowered the risk of kidney failure by 30% in people with Type 2 diabetes and kidney disease. In addition, the trial demonstrated that canagliflozin reduced the risk of major cardiovascular events such as death from heart causes, heart attacks and strokes, as well as hospitalization for heart failure. It is the first time that there is a therapy for patients with Type 2 diabetes and chronic kidney disease that decreases kidney failure. This double-blind, randomized trial involved 4,401 participants in 34 countries.

 

· Development of CAR T Cell Therapy for Myeloma, nominated by the Center for Cancer Research, National Cancer Institute, and represented by Dr. James Kochenderfer, Investigator, Surgery Branch. Multiple myeloma is almost always an incurable cancer. While there are many treatment options available for myeloma, in most cases the cancer will relapse. This study focused on developing a new therapy for myeloma that uses the body’s own immune system to treat cancer. By reprogramming a patient’s immune cells to attack their myeloma cancer cells, patients with relapsed multiple myeloma were able to be treated. While still preliminary, this study adds a new tool for doctors to use to treat myeloma patients with relapsing disease.

 

· Hematopoietic Stem Cell Transplantation for Frequently Relapsing Multiple Sclerosis, nominated by Northwestern University Feinberg School of Medicine, and represented by Dr. Richard Burt, Chief of Immunotherapy and Autoimmune Diseases in the Department of Medicine. The goal of autologous hematopoietic stem cell transplantation (HSCT) is to reboot a faulty immune system. Hematopoietic stem cells are taken from the patient’s blood, then the immune system is wiped clean with immune specific drugs. Next, stem cells are reintroduced to the body, where they facilitate an immune system recovery. During this process, in which there are no disease-causing immune cells or inflammation, the new immune cells reset to self-tolerance and no longer attack the body. Dr. Burt pioneered the development of HSCT, and was the first to treat multiple sclerosis patients with this treatment. The randomized trial demonstrated that that HSCT reverses neurologic disability, patients continue to improve for over 2 years after transplantation, and most patients show no further progressive disability or evidence of new disease activity over 5 years. HSCT is markedly superior to the current, ongoing drug therapies in preventing relapses, slowing disease progression, decreasing the burden of disease in the brain, and improving a patient’s quality of life.  HSCT also leads to significant cost savings for both private insurance companies and public health: it carries a one-time cost of roughly $98,000, while MS drugs cost roughly $80,000 per year and are generally given indefinitely. More important, in reversing MS and stopping new disease activity, HSCT is a win for the patient because it accomplishes what no drug has done before. 

 

· Large-Scale Assessment of a Smartwatch to Identify Atrial Fibrillation, nominated by Stanford University, and represented by Dr. Marco Perez, Associate Professor, Medicine. Atrial fibrillation is a condition of the heart that is characterized by an irregular and often very fast heartbeat. It is the most common recognized cause of stroke, and accounts for 130,000 deaths and 750,000 hospitalizations in the United States every year. Since symptoms are not always experienced, wearable devices, like smartwatches, are able to measure the heartbeat using light sensors, which detect irregularities consistent with atrial fibrillation. The goal of the study was to see how well a smartwatch could identify atrial fibrillation using the light sensor on the Apple Watch. 400,000 people from around the United States were enrolled in this virtual study in just eight months.  Once one downloaded the app and enrolled in the study, their smartwatch was used to detect irregularities in their heartbeats, which then prompted them to use their smartphones to connect with a study doctor. Heart monitors were mailed to the participants to see if we could pick up atrial fibrillation. Very few, only 5 out of 1,000 people, had an irregular pulse detected.  However, if they did have an irregular pulse, there was an 84% chance that they were having atrial fibrillation at that time.  If they wore a monitor 2 weeks later, 34% of them were still having episodes of atrial fibrillation. The exciting thing about this study is that it represents a new way of doing very large clinical studies.  The results show that the smartwatch could safely identify atrial fibrillation.  Doctors will be better informed when they manage patients with irregular heartbeats detected on wearable devices.

  

· Prevention of Sudden Cardiac Death in High-Risk Patients With Hypertrophic Cardiomyopathy, nominated by Tufts Medical Center, and represented by Dr. Martin Maron, Director, Hypertrophic Cardiomyopathy Center; Co-Director, Cardiac CT and MRI; Assistant Professor, Tufts University School of Medicine. Hypertrophic cardiomyopathy (HCM) is a relatively common genetic heart disease that causes the walls of the heart to abnormally thicken. HCM affects over 700,000 Americans (1 in 200-500 people in the general population) and remains the most common cause of sudden death in young people. Implantable cardioverter defibrillators (ICDs) are devices that can continually monitor the heart’s beating and respond to a dangerous arrhythmia by jolting the heart back to normal rhythm, essentially saving the life of the patient who otherwise would have likely suffered a sudden death event.  Much research supports the use of ICDs as a preventative strategy in high-risk HCM patients. However, deciding which patients are the highest risk and therefore the most deserving of ICD therapy has been challenging, and a clear consensus on appropriate methods for identifying patients most in need of an ICD has been elusive. This study provides strong evidence that will help make these decisions easier for doctors and patients. Researchers followed more than 2,000 HCM patients treated over a 17-year period to test a novel risk assessment strategy.  The results showed the strategy is highly effective for predicting which individuals really are at risk of sudden death. For 82 of the 500 who received an ICD, the device successfully responded to at least one critical event within 5 years.  Since the average age of these patients is 45, many additional life-saving interventions could be expected to occur.

 

· Skin-like Devices for Wireless Monitoring of Vital Signs in Neonatal Intensive Care, nominated by Northwestern University and Represented by Dr. Steve Xu, Medical Director of the Querrey Simpson Institute for Bioelectronics. This study reports on the development and preliminary validation of a new NICU monitoring technology, embodied as a pair of skin-like wireless devices that, when used in a time-synchronized fashion, can reconstruct full vital signs information with clinical-grade precision. One device mounts on the chest to capture electrocardiograms (ECGs); the other rests on the base of the foot to simultaneously record photoplethysmograms (PPGs). This binodal system captures and continuously transmits ECG, PPG, and (from each device) skin temperature data, yielding measurements of heart rate, heart rate variability, respiration rate, blood oxygenation, and pulse arrival time as a surrogate of systolic blood pressure. Through successful tests on neonates with gestational ages ranging from 28 weeks to full term, the team demonstrated the technology’s full range of functions in two level III NICUs. Their technology not only reproduces capabilities currently provided by invasive, wired systems, the present standard of care, but also offers multipoint temperature sensing and continuous blood pressure tracking. The devices soft, flexible, and thin nature dramatically reduces the risk of skin injury on fragile neonates and allows for greater compatibility with medical imaging. By eliminating wired connections, these platforms also facilitate therapeutic skin-to-skin contact between neonates and parents, which is known to stabilize vital signs, reduce morbidity, and promote emotional bonding. They also offer cost-effective capabilities and reusability with great relevance to low resource settings.

 

· Sustained outcomes in oral immunotherapy for peanut allergy (POISED study): a large, randomized, double-blind, placebo-controlled, phase 2 study, nominated by Stanford University, and represented by Dr. Rebecca Sharon Chinthrajah, Clinical Associate Professor, Medicine. Food allergies affect an estimated 220 million people, including more than 9 million adults and 6 million children in the U.S. Management of food allergies currently focuses on avoidance of exposure to triggering foods, though many such foods such as peanuts are common ingredients in food products and therefore difficult to avoid. Many people with a peanut allergy are accidentally exposed and experience potentially life-threatening reactions, including anaphylaxis, each year. Unfortunately, there are no FDA-approved therapies for any food allergy, an area of tremendous unmet medical need. This important clinical trial evaluated the sustained effects of peanut allergy oral immunotherapy in a randomized long-term study in adults and children. Participants in the trial ingested a dose of a peanut protein each day, conditioning their immune systems to tolerate peanuts. The goal was to dampen the immune response “which can result in swelling, itching and difficulty breathing” so it would no longer be life threatening. During a two year study, 120 participants were randomly assigned to a no-peanut group (n=60), a 300 mg peanut-protein group (n=35), and a placebo group (n=25). The study showed that peanut oral immunotherapy can desensitize most individuals with peanut allergy to 4000 mg of peanut protein, but discontinuation, or even a reduction to 300 mg daily, increases the likelihood of regaining clinical reactivity to peanut. Over the entire study, the most common adverse events were mild gastrointestinal symptoms, which were seen in 90 of 120 patients and skin disorders, which were seen in 50 of 120 patients. Adverse events decreased over time in all groups.

 

· Systolic Blood Pressure Intervention Trial (SPRINT) MIND Study, nominated by Wake Forest School of Medicine, and represented by Dr. Jeff Williamson, Professor of Internal Medicine and Chief, Section on Gerontology and Geriatric Medicine. More than 50 million people suffer from dementia worldwide, with 10 million new cases each year. Alzheimer’s Disease, the most common type of dementia, is the sixth leading cause of death in the US. Unfortunately, there are no proven interventions to prevent dementia or Mild Cognitive Impairment (MCI), which is an early stage of dementia. Hypertension is a strong risk factor for dementia and is one of the most common illnesses, affecting 75% of persons older than 65. While studies that did not randomize patients found that blood pressure control may lower the risk of dementia, large randomized clinical trials to reduce blood pressure that also examined the occurrence of dementia did not find such a relationship. However, these earlier trials did not follow patients for a long enough period of time nor use state-of-the-art methods for identifying MCI or dementia. Thus, the current study was designed specifically to see if patients who were more intensely treated for high blood pressure with a goal of achieving a systolic blood pressure of 120 had a lower risk for developing dementia or MCI than patients receiving standard treatment with a goal of achieving a systolic blood pressure of 140. The study found more intensely treated patients had a 19% lower risk of suffering MCI and a 15% lower risk of suffering either MCI or dementia. Patients who intensely treated also had a 17% lower risk of developing dementia alone, although this lower risk was not statistically significant. Most important, this is the first study to clearly identify an intervention of intensive blood pressure control in patients with hypertension that can lower the risk of developing cognitive impairment. The study also shows that concerns that many physicians have about lowering blood pressure too much in older patients with hypertension may not be warranted.

 

At the awards ceremony on April 14, three of the Top 10 studies will receive additional recognition and cash prizes:

  • The Herbert Pardes Clinical Research Excellence Award, named in honor of CR Forum board member Herbert Pardes for his profound impact on clinical research and academic medicine; this award, which comes with a $7,500 cash prize, is for the research study that best shows a high degree of innovation and creativity, advances science, and has an impact upon human disease. 
  • The Distinguished Clinical Research Achievement Awards, presented to the top two studies that show creativity, innovation, or a novel approach that demonstrates an immediate impact on the health and well-being of patients. Each study will receive a $5,000 cash prize.

 

Awardees will have the opportunity to meet with policymakers the day after the ceremony to discuss their findings and the critical role of federal funding for clinical research.

 

About the Top Ten Clinical Research Achievement Awards

Recognizing the need to celebrate our nation's clinical research accomplishments that involve both innovation and impact on human disease, the Clinical Research Forum conducts an annual competition to determine the ten outstanding research accomplishments in the United States. These major research advances represent a portion of the annual return on the nation's investment in the health and future welfare of its citizens.

 

About the Clinical Research Forum and the Clinical Research Foundation

The mission of the Clinical Research Forum is to provide leadership to the national and clinical translational research enterprise and promote understanding and support for clinical research and its impact on health and healthcare. For more information, visit www.clinicalresearchforum.org.

 

The Clinical Research Foundation is the charitable arm of the Clinical Research Forum. As a 501(c)(3), gifts to the foundation support the Top Ten Clinical Research Achievement Awards and are tax deductible.

 

Media Contact: Andrea Van Hook, 202-367-2483, avanhook@clinicalresearchforum.org.