Ann Romney Center for Neurologic Diseases

Ann Romney Center for Neurologic Diseases

Anna Krichevsky, PhD, Rohit Bakshi, MD, and Tracy Young-Pearse, PhD, are among the 250 brilliant researchers at the Ann Romney Center for Neurologic Diseases who are breaking ground on new treatments and cures

Ann Romney Center for Neurologic Diseases

RESEARCH AND DISCOVERY

 


BREAKING NEW GROUND IN BRAIN TUMOR RESEARCH


Widely recognized as a pioneer in the field, Anna Krichevsky, PhD, is spearheading innovative and encouraging research on malignant brain tumors. With only marginal improvements in survival rates over the past 25 years, new approaches are urgently needed to better treat this devastating disease. Dr. Krichevsky and her team are focused on the role of microRNA in brain tumor development—small RNA molecules that regulate gene expression—as these molecules also represent a promising class of targets for tumor treatment. The team is focused specifically on miR-10b, a microRNA abundant in most brain tumors, which promotes tumor growth and appears essential for the survival of tumor cells. The team has discovered a gene editing technique to eliminate miR-10b within the tumor, halting its activity and killing the cancer cells. They are now working to optimize this potentially life-saving therapeutic strategy, which stands to provide a common therapy with widespread impact for the thousands of patients diagnosed with brain tumors each year.


PROMISING ADVANCES TOWARD A BLOOD TEST FOR ALZHEIMER’S DISEASE


Ann RomneyDennis Selkoe, MD, continues to advance efforts to identify and validate Alzheimer’s disease (AD) biomarkers, then leverage those discoveries toward new tools for diagnosis, monitoring, and treatment. Using the most sophisticated technology available, Dr. Selkoe and his colleague, Dominic Walsh, PhD, are working to detect proteins in the blood or cerebrospinal fluid that could serve as biomarkers for AD, including tau, the protein that makes up the “tangles” that accumulate in the brain and impair cognitive function in AD. They were able to successfully measure elevated tau levels in blood samples, suggesting that it may become possible to detect AD with a simple blood test. Such a test offers the potential to detect AD even before symptoms develop, an extraordinary breakthrough that would allow for earlier intervention and improved outcomes in countless patients, as well as streamlined clinical trials.


MOVING TOWARD A NEW THERAPY FOR ALS


A POTENTIAL NEW DRUG TARGET FOR ALSWorking closely with collaborators at Massachusetts General Hospital, Oleg Butovsky, PhD, is studying microRNA to develop potential new treatments for ALS. Dr. Butovsky and his team have focused their attention on miR-155, a microRNA molecule that plays a major role in regulating inflammation, and may be involved in the progression of ALS and other neurodegenerative conditions. Building on their studies showing that inhibiting MiR-155 prolonged life in animal models and lowered levels in human cells, Dr. Butovsky and his team have partnered with miRagen Therapeutics—a biopharmaceutical company that specializes in developing microRNA-targeting therapies—to develop specific inhibitors to shut down MiR-155 in humans. In the past year, they have identified two promising blockers that will soon be ready for clinical trials.


EXPLORING THE GUT-BRAIN CONNECTION IN MULTIPLE SCLEROSIS


Ann RomneyExisting research has suggested a connection between the gut microbiome—the collection of microorganisms that live in the intestine—and brain inflammation. But how the two are linked and how diet may influence this connection has remained largely unknown, until now. In a new line of study led by Francisco Quintana, PhD, researchers have shown for the first time that changes in diet and the gut microbiome may influence the activity of astrocytes—star-shaped cells that reside in the brain and spinal cord—which play a key role in controlling inflammation and neurodegeneration, two processes closely tied to MS. The study, published in Nature Medicine in August 2016, may point to potential new therapeutic targets, as well as biomarkers for diagnosis and monitoring disease progression.


THE PARKINSON’S DISCOVERY ENGINE: UNDERSTANDING DISEASE PROGRESSION


Ann RomneyIn Parkinson’s disease, as in many neurodegenerative conditions, the pace of disease progression varies considerably between patients, but the underlying mechanisms driving these differences remain poorly understood. Clemens Scherzer, MD, is working to decode the genetic architecture controlling disease progression in Parkinson’s, which will lay the groundwork for precision therapies, improving clinical trial design and heralding a new era of tailored treatment for Parkinson’s. Dr. Scherzer is working to develop genetic biomarkers that are predictive of how the disease will progress, and is partnering with pharmaceutical companies to match drugs to the specific targets identified by these markers. More aggressive inherited forms of the disease can be blocked, and mutations that slow or halt the progression of Parkinson’s—protective genes—will be targets for drugs that aim to mimic that effect. The goal is to speed potential new therapies to clinical trial and, ultimately, prevent disease progression.


UNRAVELING THE MYSTERIES OF NEUROLOGIC DISEASE IN WOMEN


Ann RomneyAlthough sex differences are pervasive in neurologic diseases, they represent an underserved area of research where many key questions remain unanswered. Researchers at the center aim to help fill this critical knowledge gap through an exciting new focus that will build upon research already underway by our expert investigators. This new effort is a response to the unique challenges faced by women, who often find themselves at the intersection of patient and caregiver. For example, the new PREG-MS study led by Maria Houtchens, MD, aims to create the first evidence-based guidelines to improve how we care for pregnant women with MS and their babies. Research will center on several critical areas, including the underlying biological mechanisms behind the different risk profiles of women and men; identifying the most significant gaps in our understanding; and how to best capitalize on this emerging understanding to develop treatments and services specifically for women. Center investigators will collaborate closely with expert colleagues from across BWH and beyond, harnessing a wealth of multidisciplinary expertise and advancing a comprehensive approach to these complex issues.


NEW FRONTIERS IN BRAIN IMAGING


Ann RomneyWith the opening of the Building for Transformative Medicine, clinicians and researchers at the center now have access to advanced tools and opportunities to collaborate, pushing the boundaries of discovery in ways that have never been possible before. Research teams will be able to leverage state-of-the-art imaging technologies, including a 7 Tesla (7.0T) MRI scanner, the newest and most powerful MRI machine available and the first to be installed in a clinical setting in North America. Due to arrive in 2017, the 7.0T will allow clinicians and researchers to visualize structures and pathways not previously visible by MRI, offering new insight into otherwise unseen processes. The vastly improved signal will help differentiate between conditions with similar symptoms and allow doctors to choose the best treatment options for patients. The 7.0T will at first be used exclusively for clinical research, with the hope that it will soon be approved by the FDA for clinical use. A true game changer, this new instrument will help our research teams to propel work forward across all five disease areas, paving the way toward a new era in diagnosis, monitoring, and treatment for neurologic diseases.


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