Ann Romney Center for Neurologic Diseases

Ann Romney Center for Neurologic Diseases


Howard L. Weiner, MD, co-director of the Ann Romney Center for Neurologic Diseases, discusses the Google Life Sciences study with research associate Bonnie Glanz, PhD, and Ann Romney, who will participate in the study.

Ann Romney Center for Neurologic Diseases

GROUNDBREAKING RESEARCH INITIATIVES

 
We are pleased to share with you highlights of a broad array of activities spanning five disease areas by the more than 250 faculty and researchers affiliated with the center.


PARTNERSHIP ANNOUNCED WITH GOOGLE LIFE SCIENCE


Howard L. Weiner, MDA team of researchers from the Ann Romney Center for Neurologic Diseases led by Howard Weiner, MD, and Tanuja Chitnis, MD, are partnering with Google Life Sciences to learn more about the biologic, physiological, environmental, and behavioral factors that influence a patient’s experience with multiple sclerosis (MS) as the disease progresses. Wearable devices that measure an individual’s activity have become mainstream. This technology has the potential to collect robust data that could inform research and clinical care for specific diseases, including MS. The study, named SysteMS, will pilot wearable sensors developed by the life sciences team at Google that are designed to continuously measure movement, activity, and vital signs. Building upon the renowned CLIMB study at Brigham and Women’s Hospital (BWH), the study will enroll approximately 2,000 patients. This novel technology has the potential to inform clinicians and researchers about the day-to-day abilities and challenges of their patients, and offer insights toward new treatment targets and improved patient care.


FROM MS TO BRAIN TUMORS: AN INTERDISCIPLINARY DISCOVERY


Dennis Selkoe, MDGlioblastomas, one of the most deadly forms of brain tumors, are known to suppress the immune system, making them exceptionally difficult to treat. The immune system also plays a role in MS, and MS researchers in Dr. Weiner’s lab have identified a regulatory cell that plays an important role in down regulating the immune system. They found that these regulatory cells, which are present in abnormally high quantities around glioblastomas in animal models, can be identified by the expression of a peptide called LAP (Latency Associated Peptide) on the surface of the cells. LAP-positive regulatory cells have been shown to promote cancer malignancy and immune suppression in other types of cancer. With this in mind, researchers at the center have developed a highly specific antibody that targets LAP-positive regulatory cells and removes them from the body. When Dr. Weiner and his colleagues used this antibody to treat glioblastomas in animal models, the results were significant: immune response was restored, tumor growth arrested, and survival time increased. These promising results have led the team at the center to plan a clinical trial with this antibody.


THE A4 STUDY: THE FIRST-EVER PREVENTIVE CLINICAL TRIAL FOR ALZHEIMER’S DISEASE


Ann RomneyReisa Sperling, MD, MMSc, director of the Center for Alzheimer Research and Treatment (CART) at BWH, is leading the groundbreaking Anti-Amyloid Treatment in Asymptomatic Alzheimer’s Disease (A4) Study, aimed at preventing memory loss due to Alzheimer’s. Using leading-edge brain scan technology to identify and track early amyloid buildup that can occur for decades before symptom onset, the study will test the use of solanezumab—an investigational anti-amyloid antibody—in patients who have no outward symptoms yet but show signs of amyloid accumulation in the brain. One thousand participants across more than 60 sites in the U.S., Canada, and Australia will be monitored for three and a half years to track the earliest stages of memory loss, allowing researchers to determine whether solanezumab can help the brain to clear the amyloid, thereby treating the disease before symptoms ever develop. This multicenter, collaborative effort will help deepen our understanding of how Alzheimer’s progresses and offer new hope for disease prevention.


A POTENTIAL NEW DRUG TARGET FOR ALS


A POTENTIAL NEW DRUG TARGET FOR ALSWorking toward potential treatment options for this devastating disease, Dr. Weiner and his team have identified a specific microRNA—a small RNA molecule that regulates gene expression—that is elevated in the blood and spinal fluid of patients with ALS and may play a key role in disease progression. Studies in the lab have shown that this particular microRNA—MiR-155—can be blocked by administering an anti-MiR-155 compound, prolonging life in animal models and showing promising results by lowering levels in human cells. Dr. Weiner and his team, which includes collaborators at Massachusetts General Hospital, Johns Hopkins University, and UMass Memorial Medical Center, are now working with a pharmaceutical company to develop a drug aimed at this unique and promising target.


WORKING TOWARD A VACCINE FOR ALZHEIMER’S DISEASE


Ann RomneyTogether with their teams, Dr. Weiner and Dennis Selkoe, MD, are currently investigating the development of a nasal spray aimed at clearing the brain of the amyloid proteins responsible for forming the characteristic plaques that impair memory and cognitive function in Alzheimer’s disease. Building upon a decade of research in which Dr. Weiner also called upon his expertise in MS to understand related biological processes and refine the vaccine, the team has honed in on a substance called protollin that enhances the body’s immune response. This research is showing promising results in the lab. Drs. Weiner and Selkoe are now working with pharmaceutical companies in Canada and the Netherlands to manufacture a protollin-based nasal vaccine and move it forward toward clinical trials.


A NOVEL HYPOTHESIS FOR PARKINSON’S DISEASE


Ann RomneyFor many years, researchers have been investigating the role of alpha-synuclein (αSyn) in Parkinson’s disease and its potential as a target for therapy. Working closely with Dr. Selkoe, Tim Bartels, PhD, and his colleagues have discovered that the typical structural form of αSyn is actually four αSyn proteins wound together, and that this structure resists disease-associated changes. Dr. Bartels hypothesizes that, in Parkinson’s, this normal four-part structure becomes more prone to disassembling into single units, which can then regroup into toxic assemblies that spread through the nervous system and initiate brain disease. The research team is now screening for drugs that stabilize the aggregated form of αSyn and prevent it from unfolding, as this could potentially prevent movement impairments and other symptoms experienced by those affected with Parkinson’s disease.


MOVING TOWARD A BLOOD TEST FOR PARKINSON’S DISEASE


Ann RomneyIn a paper published in the prominent journal, Brain, a multicenter study led by Clemens R. Scherzer, MD, pointed to the possibility of an effective biomarker for early Parkinson’s disease. The study, which looked at data from BWH patients as well as two large national study cohorts, found that reduced expression levels of the alpha-synuclein (SNCA) gene—related to the αSyn protein implicated in disease progression—are associated with Parkinson’s. This was true even for patients in the landmark Parkinson’s Progression Marker Initiative (PPMI), which enrolls patients very early on, before symptoms have met standard diagnosis criteria. SNCA levels can be obtained from a simple blood test, creating a pathway toward earlier, improved diagnosis, more effective clinical trials, and possibly one day a cure for Parkinson’s disease.


HARNESSING THE POWER OF ADULT-DERIVED STEM CELLS


Ann RomneyTracy Young-Pearse, PhD, a rising star with a new research lab at the center, has made tremendous progress in the use of adult-derived stem cells, which can be easily obtained from adult human tissues, to study mechanisms of brain disorders. In collaboration with center researcher Matthew J. LaVoie, PhD, she and her team have established a novel cellular model of Alzheimer’s disease, through which they can effectively study living human neurons cultured from Alzheimer’s patients. They are working to illuminate the mechanism by which the disease begins, and in collaboration with scientists at MIT, are developing a new technique for examining cell responses to new drugs that affect amyloid generation. Dr. Young-Pearse aims to apply these methodologies to other neurological diseases, further illuminating the underlying mechanisms of these diseases and opening doors to novel therapies. Dr. LaVoie is also currently using similar techniques to generate new cell models of Parkinson’s disease.


For Media
BWH Communication & Public Affairs
617-525-6370
bwhmediarelations@partners.org