2020 Top 10 Clinical Research Achievement Awards

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April 15, 2020

The Clinical Research Forum is proud to announce the 2020 Top Ten Clinical Research Achievement Awards. All ten outstanding research studies were recognized and honored through our virtual event on April 15, 2020. In addition, three of the studies received further honors:

  • Skin-like Devices for Wireless Monitoring of Vital Signs in Neonatal Intensive Care, nominated by Northwestern University, received 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.

  • Canagliflozin and Renal Outcomes in Type 2 Diabetes and Nephropathy, nominated by Stanford University and represented by Dr. Kenneth Mahaffey, and Hematopoietic Stem Cell Transplantation for Frequently Relapsing Multiple Sclerosis, nominated by Northwestern University Feinberg School of Medicine and represented by Dr. Richard Burt both received 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 received a $5,000 cash prize.

The 2020 Top Ten Clinical Research Achievement Awardees are:

  • 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. 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 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. 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. 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. 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 Multiple 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. 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 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. 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. 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 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.



  • Skin-like Devices for Wireless Monitoring of Vital Signs in Neonatal Intensive Care, nominated by Northwestern University and Represented by John Rogers and 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. 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. 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.



  • 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. 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. The study found more intensely treated atients 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.

 

Related Links:

-Click here to view the 2020 Virtual Top 10 Clinical Research Achievement Awards

-A compilation video of all of the awardees can be found on our YouTube Channel here

-Please click here to view the 2020 Program

 

 

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