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URMC neurologist Curt Benesch, M.D., M.P.H., and anesthesiologist Laurent Glance, M.D., lead the effort to develop new American Heart Association/American Stroke Association (AHA/ASA) recommendations to lower the risk of perioperative acute stroke.

The statement, which was published in the journal Circulation, focuses on the cerebrovascular complications of non-cardiac surgery and summarizes the current literature concerning the preoperative neurological risk stratification and management of patients before undergoing non-cardiac, non-neurological surgery; intraoperative strategies to mitigate the risk of stroke; and the identification and treatment of patients who experience a perioperative stroke.

Benesch and Glance served as chair and vice chair of a panel of surgeons, anesthesiologists, neurologists, and nurses convened by the AHA/ASA to draft the statement. Robert Holloway, M.D., M.P.H., chair of the Department of Neurology, also served on the panel. The group conducted a literature review that emphasized publications based on randomized, controlled trials, followed by those describing meta-analyses, very large administrative databases and quality registries, and relevant, smaller observational studies. The final scientific AHA/ASA statement was endorsed by the American Academy of Neurology and the American Association of Neurological Surgeons. Benesch and Glance also co-authored an earlier companion paper on the perioperative risks of stroke in patients undergoing cardiac surgery.

Perioperative stroke can be defined as any embolic, thrombotic, or hemorrhagic cerebrovascular event with motor, sensory, or cognitive dysfunction lasting at least 24 hours, occurring intraoperatively or within 30 days after surgery. The incidence of perioperative stroke in patients undergoing non-cardiac, non-neurological surgery is between 0.1% and 1.0%, a number that has risen since 2004 in both men and women and across races and ethnic groups. More than 60% of in-hospital strokes are likely perioperative, occurring on either a surgical service or in the angiography suite.

The new statement stratifies preoperative risk factors, provides guidance on stroke recognition in the perioperative setting, and details stroke prevention strategies, including management of medications, blood pressure, blood transfusion, ventilation, and anesthetic technique. The statement also recommends that large vessel occlusions (LVO) -- ischemic strokes that result from a blockage in one of the major arteries of the brain and represent 10% of perioperative strokes -- be treated via mechanical thrombectomy. In instances where clinical situations lack high-quality clinical trial evidence, recommendations reflect the best evidence available and the consensus of experts to provide pragmatic guidance to practitioners who must make real-world decisions every day in clinical practice.

AHA/ASA Scientific Statements:

Perioperative Neurological Evaluation and Management to Lower the Risk of Acute Stroke in Patients Undergoing Noncardiac, Nonneurological Surgery

Considerations for Reduction of Risk of Perioperative Stroke in Adult Patients Undergoing Cardiac and Thoracic Aortic Operations: A Scientific Statement From the American Heart Association

"Growing evidence says this is real," says Miriam Weber, an associate professor of neurology and of obstetrics and gynecology. "Multiple studies have shown declines in memory and attention," she says. "What we don't know is whether it persists. So far it seems like it may be temporary, just through the transition" from perimenopause through menopause.

University of Rochester Medical Center (URMC) neurologist Gretchen Birbeck, M.D., M.P.H., has received a $4.3 million award from the National Institutes of Neurological Disorders and Stroke (NINDS) to continue her research in sub-Saharan Africa on the neurological problems that arise in people recovering from malaria, HIV, and other infectious diseases, including COVID.

The NINDS Research Program Award, which uses the R35 funding mechanism, is given to investigators whose record of research achievement demonstrates an ability to make major contributions to the field of neuroscience. The eight-year award is intended to provide recipients the freedom to embark on ambitious, creative, and longer-term research projects, without the constraints of specific aims.

Birbeck has spent the last 25 years working in Zambia and Malawi in collaboration with local government ministries, medical schools, hospitals, and other U.S.-affiliated neuroscientists to identify the mechanisms of common neurological disorders and improve care through evidence-based interventions and clinical trials. Her research has focused on evaluating outcomes of cerebral malaria and other brain infections in children, and the neurological symptoms that arise from chronic HIV infection and treatments. These diseases -- which are prevalent in sub-Saharan Africa -- have broad effects on cognitive, behavioral, quality-of-life, and economic outcomes.

Birbeck will initially focus on two research projects:

• Nearly one-third of cerebral malaria survivors develop epilepsy or other neurological conditions soon after recovery. Previous research by Birbeck has demonstrated that improved seizure control and management of aggressive fever caused by malaria could provide the key to decreasing the risk of brain injury and developing epilepsy. Birbeck and her team will examine the role of neuroinflammation in structural injury and neurologic morbidity with laboratory assessments of acute inflammation, serial neuroimaging, and long-term neurological outcomes. Researchers will also investigate the effects of co-infection with COVID on children who have recovered from malaria.
• Given the widespread availability of HIV therapies, the next challenge in neuro-HIV care in Africa includes disorders associated with chronic low grade inflammation brought about by the virus and the toxicity of long-term use treatments such antiretroviral drugs. Specifically, studies have shown high rates of cerebrovascular disease in children with HIV, despite long-standing effective treatment of the virus. Utilizing a network of rural and urban HIV clinics, the team will study HIV-associated accelerated aging of the nervous system. Given its highly inflammatory state, the researchers will examine whether COVID could potentially contribute to this burden in children. The team will also see if COVID infection in adults with HIV contributes to cognitive impairment, psychiatric symptoms, strokes, neuropathies, and/or seizures.

The projects involve researchers, clinicians, and students and trainees from URMC, the University of Zambia's University Teaching Hospitals, Queen Elizabeth Central Hospital in Malawi, the University of Malawi College of Medicine, the Centre for Infectious Disease Research in Zambia, and a consortium of rural hospitals in Zambia led by Chikankata Hospital. The research program award will also help provide the infrastructure, mentorship and an environment for scholarship and training for both U.S. and African academics.

A new study observing the sleep patterns of nearly 8,000 adults in the United Kingdom goes against earlier beliefs about the connection between sleep duration and the possible development of dementia later in life.

"Previous studies have indicated people who sleep excessively, or long sleepers, tend to have an increased risk for dementia," said Dr. Alice Hoagland, a sleep specialist with Rochester Regional Health. "But this is the first study that indicated that people who biologically were short sleepers also had a higher increased risk for dementia."

The study followed people for 25 years, beginning when they were age 50. It found that people who slept six hours or less had a higher risk of being diagnosed with dementia in their 70s.

Doctors with both Rochester Regional Health and the University Rochester Medical Center had many questions about the study - and not all of them could be answered based on the findings.

"I would be very hard pressed to say that being a very short sleeper, sort of staying up late at night and getting up early in the morning and all that, is predictive of developing dementia because we simply don't know which way this goes," said Dr. Alice Hoagland, director of Rochester Regional Health's Insomnia Clinic.

"Whether these are people who just naturally get six hours of sleep or less - because there are people who are like that - or whether these are people who would like to sleep longer and they just can't because they don't have the opportunity, that's yet to be seen," said said Dr. Michael Yurcheshen, a professor of neurology and sleep medicine with URMC.

"We certainly do see patients in our practice here who do get six or fewer hours of sleep who do seem to function just fine," said Dr. Yurcheshen.

Asked about the future of Parkinson's disease in the US, Dr Ray Dorsey says, "We're on the tip of a very, very large iceberg."

Dorsey, a neurologist at the University of Rochester Medical Center and author of Ending Parkinson's Disease, believes a Parkinson's epidemic is on the horizon. Parkinson's is already the fastest-growing neurological disorder in the world; in the US, the number of people with Parkinson's has increased 35% the last 10 years, says Dorsey, and "We think over the next 25 years it will double again."

Most cases of Parkinson's disease are considered idiopathic -- they lack a clear cause. Yet researchers increasingly believe that one factor is environmental exposure to trichloroethylene (TCE), a chemical compound used in industrial degreasing, dry-cleaning and household products such as some shoe polishes and carpet cleaners.

After the pandemic forced thousands of trials to shut down, researchers found clever ways to conduct human studies remotely — while reaching more people, quickly and cheaply.

Remote trials are likely to persist in a post-pandemic era, researchers say. Cutting back on in-person visits could make recruiting patients easier and reduce dropout rates, leading to quicker, cheaper clinical trials, said Dr. Ray Dorsey, a neurologist at the University of Rochester who conducted remote research for years.

In fact, he noted, enrollment in one of his current virtual studies, which is tracking people with a genetic predisposition to Parkinson's, actually surged last spring. "While most clinical studies were paused or delayed, ours accelerated in the midst of the pandemic," he said.

Progressive vision loss, and eventually blindness, are the hallmarks of juvenile neuronal ceroid lipofuscinosis (JNCL) or CLN3-Batten disease. New research shows how the mutation associated with the disease could potentially lead to degeneration of light sensing photoreceptor cells in the retina, and subsequent vision loss.

"The prominence and early onset of retinal degeneration in JNCL makes it likely that cellular processes that are compromised in JNCL are critical for health and function of the retina," said Ruchira Singh, Ph.D., an associate professor in the Department of Ophthalmology and Center for Visual Science and lead author of the study which appears in the journal Communications Biology. "It is important to understand how vision loss is triggered in this disease, what is primary and what is secondary, and this will allow us to develop new therapeutic strategies."

Batten disease is caused by a mutation in the CLN3 gene, which is found on chromosome 16. Most children suffering from JNCL have a missing part in the gene which inhibits the production of certain proteins. Rapidly progressive vision loss can start in children as young as 4, who eventually go on to develop learning and behavior problems, slow cognitive decline, seizures, and loss of motor control. Most patients with the disease die between the ages of 15 and 30.

It has been well established that vision loss in JNCL is due to degeneration of the light-sensing tissue in the retina. The vision loss associated with JNCL can precede other neurological symptoms by many years in some instances, which often leads to patients being misdiagnosed with other more common retinal degenerations. However, one of the barriers to studying vision loss in Batten disease is that mouse models of CLN3 gene mutation do not produce the retinal degeneration or vision loss found in humans. Additionally, examination of eye tissue after death reveals extensive degeneration of retinal cells which does not allow researchers to understand the precise mechanisms that lead to vision loss.

URMC is a hub for Batten disease research. The Medical Center is home to the University of Rochester Batten Center (URBC), one of the nation's premier centers dedicated to the study and treatment of this condition. The URBC is led by pediatric neurologist Jonathan Mink, M.D., Ph.D., who is a co-author of the study. Batten disease is also one of the key research projects that will be undertaken by the National Institute of Child Health and Human Development-supported University of Rochester Intellectual and Development Diseases Research Center.

To study Batten disease in patient's own cells, the research team reengineered skin cells from patients and unaffected family members to create human-induced pluripotent stem cells. These cells, in turn, were used to create retinal cells which possessed the CLN3 mutation. Using this new human cell model of the disease, the new study shows for the first time that proper function of CLN3 is necessary for retinal pigment epithelium cell structure, the cell layer in the retina that nourishes light sensing photoreceptor cells in the retina and is critical for their survival and function and thereby vision.

Singh points out that understanding how RPE cell dysfunction contributes to photoreceptor cell loss in Batten disease is important first step, and it will enable researchers to target specific cell type in the eye using potential future gene therapies, cell transplantation, and drug-based interventions.

Additional co-authors of the study include Cynthia Tang, Jimin Han, Sonal Dalvi, Kannan Marian, Lauren Winschel, Celia Soto, Chad Galloway, Whitney Spencer, Michael Roll, Lisa Latchney, Erika Augustine, Vamsi Gullapalli, and Mina Chung with URMC, David Williams and Stephanie Volland with the University of California, Los Angeles, Vera Boniha with the Cleveland Clinic, and Tyler Johnson with Sanford Research. The research was supported with funding from the National Eye Institute BrightFocus Foundation, the David Bryant Trust, the Foundation of Fighting Blindness, the Knights Templar Eye Foundation, the Retina Research Foundation, and Research to Prevent Blindness.

We are pleased to announce that Ann Leonhardt Caprio, DNP, RN, ANP-BC, UR Comprehensive Stroke Center Program Coordinator, has recently been named as a Fellow of the American Heart Association (FAHA), Council of Cardiovascular and Stroke Nursing. Highly competitive, election as a FAHA recognizes the recipient's scientific accomplishments, volunteer leadership and service. Earning the FAHA credential demonstrates to colleagues and patients that the recipient has been welcomed into one of the world's most eminent organizations of cardiovascular and stroke professionals.

Fellowship recognizes leaders within the AHA who have made significant contributions to the field of cardiovascular and cerebrovascular science and medicine. FAHA demonstrate outstanding credentials and achievement, contribute to the AHA through involvement at the local, affiliate, and/or national level, and receive a strong recommendation from leaders in their field.

John Lueck

Joanne Ladolcetta, a freelance writer based out of San Francisco, wanted to put together information to help address circulating fears and misconceptions about the recently FDA approved COVID-19 mRNA vaccines. She reached out to friends Bryant Johnson (Cartoonist) and John Lueck (Researcher at the University of Rochester Medical Center) to tackle some of the most common vaccine questions. The collaboration resulted in an approachable informative write-up with creative illustrations and helpful links to provide readers the ability to do a deeper dive into the generation and application of the novel COVID-19 mRNA vaccines.

Courtesy of Bryant Johnson

Read More: Blog: Are the new mRNA vaccines safe?

The Department of Neurology would like to welcome residency applicants, interviewing on Monday, January 11^th.

Adult Neurology Applicants

George Cappos -- University of Florida
Avinash Kolli -- Loyola University Chicago Stritch School of Medicine
Abena Kwegyir-Aggrey -- Pennsylvania State University
Sloan Lynch -- Boston University
Benjamin Meyer -- Columbia University
Stephanie Reyes -- University of Michigan
William Signorile, III -- Stony Brook University

Child Neurology Applicants

Serina Bsales -- Rutgers, Robert Wood Johnson Medical School
Anthony Di Caro -- New York Institute of Technology College of Osteopathic Medicine
Shama Khan -- Drexel University College of Medicine
Rima Madan -- State University of New York Upstate Medical Center

The Department of Neurology would like to welcome residency applicants, interviewing on Monday, January 12^th.

Adult Neurology Applicants

Mary Abramczuk -- Drexel University
Tiffany Kyo -- Case Western Reserve University
Amanda Lin -- Wright State University
Chiedza Mupanomunda -- Northwestern University
Jennifer Purks -- Georgetown University
Ilana Selli -- University of Buffalo

Lockdowns and social distance have led to new digital health technologies becoming crucial tools in fighting the pandemic. "Digital health technologies such as telemedicine have been vital in addressing certain health needs that local health systems are not able to handle," Valerie Shelly, program manager, ACCESS Health shared.

According to Ray Dorsey, director of the Center for Health and Technology at the University of Rochester Medical Center, the majority of patient consultations in the U.S. are now happening virtually, and there's been at least a ten-fold increase in virtual checkups in recent weeks.

The question technology companies should ask themselves is how to sustain the use of these effective telehealth services after the pandemic subsides.

The principal project of the Intellectual and Developmental Disabilities Research Center (IDDRC) will focus on Batten disease, specifically CLN3 disease, also known as juvenile-onset Batten disease. Children with this rare genetic disorder start developing symptoms such as vision loss, impaired motor control, seizures, and dementia between 5-10 years of age. The University of Rochester Batten Center (URBC) is a recognized leader in research and treatment of this condition. With several potential gene therapies for Batten disease currently in advanced stages of development, URMC will focus on identifying biomarkers to evaluate the effectiveness of these experimental treatments.

"Even within individual families, we have learned that each diagnosed child may have a different disease experience in terms of the age of symptom onset, pattern of symptom progression, and the types of burdens experienced by them and their families," said Heather Adams, Ph.D., associate professor in the departments of Neurology and Pediatrics and coinvestigator of the UR IDDRC Principal Project. "We cannot miss any opportunity to learn from affected individuals."

Patients and families from across the U.S. come to URMC for care and to participate in research. While some visit labs in Rochester equipped to work with these patients and their families, in many instances a mobile URBC lab will travel to them. "Research is the main way that we feel like we contribute to the Batten community. We try to support research as much as we can. It is how we feel connected, doing what we can to help everyone's effort," said Bridget Patterson who lives in Virginia. Two of her four children, Nora and Gabriel, have Batten disease. "A disease like this really does affect the whole family. We have two other children that are not affected, but they feel the effects of the disease every day. I know that helping their lives be better too is one reason why we are involved in this research. If we can find a cure, a treatment, Nora and Gabriel would be the ones most dramatically impacted but it would help all of us."

There are 11 other currently known childhood-onset forms of Batten disease, genetically distinct from one another, and all have significant impacts on neurodevelopment. URBC is designated a Center of Excellence by the Batten Disease Support and Research Association.

Researchers at the University of Rochester Medical Center (URMC) and Duke University Medical Center will collaborate on a study that investigates why some older patients, who become severely ill from COVID-19, develop delirium that can lead to brain damage and a dementia diagnosis.

Nearly 30-percent of patients hospitalized with COVID-19 develop delirium -- a state of confusion and impaired awareness. For severely ill patients the likelihood of delirium is closer to 70-percent. "The initial delirium, and then the cognitive, behavioral, and emotional problems -- commonly known as dysexecutive syndrome -- were arguably the most notable things in older people that had managed to survive COVID-19," said Harris Gelbard, M.D., Ph.D. Professor and Director, Center for Neurotherapeutics Discovery, who is also the principal investigator at URMC on this study which is being funded by the National Institute of Aging. "Nobody knows whether that's permanent or not because of the advanced age of the people this is impacting."

Using a model for inhaled lipopolysaccharide-based acute lung injury in mice to mimic what happens in the lungs of a severally ill COVID-19 patient, Gelbard and his colleague Niccolo Terrando, Ph.D. at Duke University Medical Center will look for specific events in the neurovascular unit —brain endothelial cells and their blood-brain barrier (BBB) forming tight junctions that support the central nervous system -- that can be traced back to cognitive impairment. Part of the hypothesis Gelbard is investigating is that scarring in the lung may cause platelets and inflammatory white cells to migrate to blood vessels in the central nervous system with the white cells traversing the BBB to cause neurologic disease. "The goal is to establish what the prerequisites are for lung injury that will lead to brain injury. And at that point, then we can start asking more complicated questions."

The researchers will also investigate how the body responds to URMC-099, an anti-inflammatory and neuroprotective agent developed by Gelbard, to prevent these sequelae. The use of behavior analysis at Duke and in vivo brain imaging at URMC will determine delirium-like changes in the mice.

Gelbard and Terrando are confident that this study will lead to a larger and longer study of the impact COVID-19 has on the brain of an older population. "If you could do something to prevent that in the first place, chances are you are going to do better, down the road."

The University of Rochester Medical Center (URMC) in collaboration with the Parkinson's Foundation is developing a palliative care program that will be become the standard of care in the organization's 33 Centers of Excellence clinics across the U.S.

The initiative -- which is being funding with $1.6 million in support from the federal Patient-Centered Outcomes Research Institute (PCORI) -- is led by URMC neurologist Benzi Kluger, M.D., a professor of Neurology and Medicine and director of URMC's new Palliative Care Research Center.

Kluger and his current and former colleagues in Rochester and the University of Colorado have developed an innovative model of palliative care delivery consisting of interdisciplinary teams of providers that address the needs of Parkinson's patients and their families as they grapple with the physical, emotional, social, and spiritual symptoms of their disease. In a previous PCORI-funded study published earlier this year in JAMA Neurology, Kluger and his team demonstrated that outpatient palliative care improves quality of life and other outcomes, including reductions in caregiver burden and improvement in motor symptoms.

The Parkinson's Foundation Centers of Excellence program recognizes medical centers that have brought together teams of neurologists, movement disorder specialists, physical and occupational therapists, and mental health professionals, and provide high levels of support and care for Parkinson's patients.

Kluger and his team are developing virtual training programs tailored to the unique needs and circumstances of each clinic. The program will provide caregivers with the tools, resources, and skills to deliver a patient-centered model of care that effectively addresses psychosocial issues patients are experiencing, provides support to families and care partners, screens for non-motor symptoms like pain and depression, and encourages the development of advanced care roadmaps for individual patients.

Maiken Nedergaard, M.D., D.M.Sc. has been awarded the International Prize for Translational Neuroscience of the Gertrud Reemtsma Foundation for her research in the glymphatic system, the brain's unique waste removal process.

Nedergaard's research was recognized by the Foundation for findings that "offer new approaches for treatments and preventive measures for Alzheimer's and other neurodegenerative diseases."

First discovered by researchers in the URMC Center for Translational Neuromedicine in 2012, the glymphatic system piggybacks on blood vessels and pumps cerebrospinal fluid, washing waste from the brain. The accumulation of toxic proteins like beta amyloid are linked neurological disorders, including Alzheimer's disease. Nedergaard's lab has since gone on to show how sleep disruption, age, and injury can impair the brain's ability to effectively remove waste.

Nedergaard was presented the award at a ceremony at the Max Plank Society in Cologne, Germany on September 10.

The University of Rochester has been designated an Intellectual and Developmental Disabilities Research Center (IDDRC) by the National Institute of Child Health and Human Development (NICHD). The award recognizes the Medical Center's national leadership in research for conditions such Autism, Batten disease, and Rett syndrome, will translate scientific insights into new ways to diagnose and treat these conditions, and provide patients and families access to cutting edge care.

The IDDRC at the University of Rochester will be led by John Foxe, Ph.D., director of the Del Monte Institute for Neuroscience, and Jonathan Mink, M.D., Ph.D., chief of Child Neurology at Golisano Children's Hospital. The designation is accompanied with more than $6 million in funding from NICHD.

A new study hasfound that tanezumab, a monoclonal antibody that inhibits nerve activity, provides relief in patients with chronic low back pain, one of the leading reasons why people seek medical care and the number one cause of disability worldwide.

"This demonstration of efficacy is a major breakthrough in the global search to develop non-opioid treatments for chronic pain," said John Markman, M.D., director of the Translational Pain Research Program in the University of Rochester Medical Center (URMC) Department of Neurosurgery and lead author of the study which appears in the journal Pain. "There were also improvements in function linked to the reduction in pain severity."

This is the first study that shows long-term relief for chronic low back pain with a single dose of tanezumab delivered under the skin once every two months. The study was conducted in 191 sites across eight countries in North America, Europe, and Asia.

Researchers are increasingly finding that certain proteins circulating in the bloodstream heighten the sensitivity of cells in the nervous system to pain. One of these proteins, called nerve growth factor (NGF), may explain why some individuals experience more intense and chronic back pain. Tanezumab is an NGF inhibitor.

The patients with chronic low back pain enrolled in this study did not previously have relief with at least three different types of pain medication, including opioids, and were considered "difficult-to-treat." Patients with symptoms, signs, and x-ray evidence of moderate-to-severe osteoarthritis, a disorder commonly found in older patients with chronic low back pain, were excluded from the study.

A new study shows that when specific human brain cells are transplanted into animal models of multiple sclerosis and other white matter diseases, the cells repair damage and restore function. The study provides one of the final pieces of scientific evidence necessary to advance this treatment strategy to clinical trials.

"These findings demonstrate that through the transplantation of human glial cells, we can effectively achieve remyelination in the adult brain, " Steve Goldman, M.D., Ph.D., professor of Neurology and Neuroscience at the University of Rochester Medical Center (URMC), co-director of the Center for Translational Neuromedicine, and lead author of the study. "These findings have significant therapeutics implications and represent a proof-of-concept for future clinical trials for multiple sclerosis and potential other neurodegenerative diseases."

The findings, which appear in the journal Cell Reports, are the culmination of more than 15 years of research at URMC understanding support cells found in the brain called glia, how the cells develop and function, and their role in neurological disorders.

Goldman's lab has developed techniques to manipulate the chemical signaling of embryonic and induced pluripotent stem cells to create glia. A subtype of these, called glial progenitor cells, gives rise to the brain's main support cells, astrocytes and oligodendrocytes, which play important roles in the health and signaling function of nerve cells.

The professor of Neurosurgery and Neurology found that asking, "Is your pain tolerable" in conjunction with the traditional 0-10 scale can help doctors better understand whether treatment — including opioids — is necessary.

We have added a new link to the Neurology Intranet Page under the General Links to the left. Clicking on the COVID-19 Neurology link will take you to a BOX folder with access to files containing current COVID-19 information related to Neurology. If you are not logged into BOX, you will be prompted to log in. If you do not have a BOX account, please contact NeurologyIT at 5-0431 and they will create an account for you.

A new book titled "Ending Parkinson's Disease: A Prescription for Action" and authored by University of Rochester Medical Center neurologist Ray Dorsey, M.D. and his colleagues, lays out a new vision to prevent, advocate for, care for, and treat this major and growing global health threat.

"Parkinson's disease is a looming pandemic and we are woefully unprepared to meet this challenge -- many people remain undiagnosed and untreated, research funding for the disease has stagnated, and the most effective treatment is now a half century old," said Dorsey, the David M. Levy Professor of Neurology and director of the Center for Health + Technology (CHeT). "At least some cases of Parkinson's are man-made and, therefore, preventable."

Parkinson's disease is the fastest growing neurological disorder in the world, outpacing Alzheimer's.Over the past 25 years, the number of people with the condition has jumped from three million to more than six million, and by 2040, it is projected to double again.

First described in early 18^th century London at the height of the Industrial Revolution, Parkinson's and its rise have been fueled by environmental exposures to harmful chemicals. The two biggest culprits currently still in use are paraquat, a herbicide widely used in the U.S. despite being banned in 32 countries, and trichloroethylene (TCE), a solvent used in a wide range of industrial and consumer products. Not only are agricultural and industrial workers at risk of exposure, but these chemicals also enter the food chain, water supply, reside in the soil in brownfield sites, and impact indoor air quality.

The authors also advocate removing barriers to care. For example, over 40 percent of individuals with the disease do not see a neurologist soon after diagnosis, primarily because of where they live. Technologies like telemedicine have been shown to deliver effective specialized care to patients and improves their lives. However, Medicare policies often do not pay for these services.

Confronting the Parkinson's pandemic will require marshalling the same focus and resources employed with success to address other public health challenges, such as polio, HIV, and breast cancer. The authors label their course of action PACT: prevent the disease, advocate for policies and resources, care for all affected, and treat the condition with new and more effective therapies.

URMC has a long been a leader in the field of Parkinson's research and care. Medical Center researchers were instrumental in conducting pivotal clinical trials that led to at least four FDA-approved drugs currently treat the disease. CHeT is a leading center for the application of new technologies to study and assess Parkinson's. The themes detailed in the book will form the basis of a new initiative launched by URMC in the coming months that will focus on the research, grassroots advocacy, and public policy steps necessary to prevent the disease and expand access to care.

Additional co-authors of the book include Todd Sherer, Ph.D., CEO of the Michael J. Fox Foundation, Mike Okun, M.D., with the University of Florida, and Bastiaan R. Bloem, M.D., Ph.D., with Radboud University in the Netherlands. Proceeds from sales of the book will support Parkinson's research at URMC and other institutions.

Benzi Kluger, M.D., has been tapped to lead palliative care research across URMC. Kluger comes to URMC from the University of Colorado School of Medicine and started at the Medical Center on January 1.

Kluger, who has been appointed a professor of Neurology and Medicine, is the director of URMC's new Palliative Care Research Center within the Department of Medicine. In this role, he will develop resources and core infrastructure to enable researchers and clinicians from across the University to undertake palliative care research projects.

"Dr. Kluger has established himself as a leading researcher and scholar in both neurological disorders and palliative care," said Bob Holloway, M.D., M.P.H., chair of the Department of Neurology. "We are fortunate to have recruited him to Rochester and look forward to helping him have a major impact on the field of palliative care."

"In his young and blossoming career Benzi has already gained international recognition as a productive, creative scholar in the realm of innovative palliative care delivery models," said Robert Horowitz, MD, chief of the Palliative Care Division at URMC. "He is an ambitious, generative, and prolific scholar, clinician, teacher and human being, with an explicit commitment to building ties across UR schools, departments, and divisions."

Kluger's specific interest is in innovative models of palliative care delivery, in which an interdisciplinary team addresses the many needs of seriously ill patients and their families, as they grapple with the physical, emotional, social, and spiritual symptoms of their disease. Kluger is working with faculty in the Wilmot Cancer Center, Divisions of Palliative Care and Nephrology, the School of Nursing, and other Medical Center departments and divisions to develop and evaluate integrated models of outpatient palliative care. Kluger will also oversee the creation of a new Neuropalliative Care Division within the Department of Neurology.

With support from a PCORI grant, Kluger led a multisite randomized controlled trial in Colorado that compared the effectiveness of multidisciplinary outpatient palliative care integrated with standard care versus standard care alone for Parkinson's patients. The results of the study, which were published earlier this month in JAMA Neurology, showed that outpatient palliative care improves quality of life and other outcomes, including reductions in caregiver burden and improvement in motor symptoms.

"Palliative care provides a framework to address the multiple needs of patient populations from the time of diagnosis and is particularly beneficial when people reach more into more advanced stages of illness where our traditional care models have less to offer," said Kluger. "This effort is part of a wider movement to make palliative care a standard and expected part of care for persons living with serious diseases."

Kluger conducted his Medical and Neurology Residency training at the University of Colorado. He completed fellowship training in Behavioral Neurology and Movement Disorders at the University of Florida. He recently established the International Society of Neuropalliative Care (ISPN), which has members from US, Canada, Europe, Asia and Australia, and will be the organization's first president. Kluger and Holloway are co-editors of the book "Neuropalliative Care: A Guide to Improving the Lives of Patients and Families Affected by Neurological Disease."

"One hears a lot about malaria deaths, but the sad reality is that a third of survivors sustain a brain injury with associated neurological sequelae. More than 200,000 African children each year end up with neurodisabilities from malaria. This is low-hanging fruit for decreasing the global burden of neurological disease and increasing the human capacity in this region of the world."

~Gretchen Birbeck, M.D., M.P.H., Professor of Neurology, ANA International Outreach Committee Chair

To celebrate International Women's Day (March 8), we're highlighting ANA members who are doing impressive work both domestically and abroad. Keep reading for our interview with Gretchen Birbeck, M.D., M.P.H. Dr. Birbeck is a neurologist who divides her time between the U.S. and Africa. Her U.S. academic home is the University of Rochester, where she is the Rykenboer Professor of Neurology. She also serves as chair of the ANA's International Outreach Committee.

We spoke with her to learn more about her research, her work with the International Outreach Committee, and what the theme of this year's International Women's Day, "Each for Equal," means to her.

Can you give an overview of the initiatives you've been working on in Zambia?

My work in Zambia really mirrors what an academic neurologist does anywhere—I teach, provide clinical care and conduct research. And for me the split is about 70/30 with the largest proportion of my time being spent on research activities.

On the research front, I am the Principal Investigator for three NIH-funded R01s. The ChASE study is a Cohort Study of HIV-Associated Seizures and Epilepsy. One of the clinical challenges in providing neurological care in sub-Saharan Africa is knowing what to do for someone who is HIV infected presenting with new onset seizure. Epidemiological data that otherwise informs if and when to start a chronic antiseizure medication may not apply to persons with HIV especially if an HIV-associated drug reaction or opportunistic infection has precipitated the seizure. And unfortunately, the choice of seizure medications may be limited to enzyme-inducing agents that interact with antiretroviral medications. ChASE is providing some insights on what causes seizures in this population, who is at risk of long-term seizure disorders and what happens when the seizure medications available in HIV endemic regions are combined with the HIV medications used locally. This is an especially dynamic study as it involves adults and children in rural and urban populations. I spent the first two decades of my time in Zambia based in a rural area and I still have excellent research teams and colleagues there so it is wonderful to remain working and engaged with them even though I am now based primarily in Lusaka.

I am also working on the Malaria Fever study which is a randomized controlled trial (RCT) of aggressive antipyretic therapy using maximal dose ibuprofen and acetaminophen for fever control in pediatric malaria. My K23 project was a prospective cohort study of neurological outcomes in pediatric cerebral malaria survivors. Since completing my K23, I've been working down the list of potentially modifiable risk factors for brain injury in this population hoping to eventually conduct a multi-country RCT of a neuroprotective "package" of care to improve outcomes in child survivors of cerebral malaria. One hears a lot about malaria deaths, but the sad reality is that a third of survivors sustain a brain injury with associated neurological sequelae. More than 200,000 African children each year end up with neurodisabilities from malaria. This is low-hanging fruit for decreasing the global burden of neurological disease and increasing the human capacity in this region of the world. I also think what we learn about epileptogenesis from the cerebral malaria model may help us understand the process in general and this has implications for advances in neurological care everywhere.

Finally, the MRI Ancillary Grant is an imaging study of children enrolled in the Malaria Fever study. This is evaluating structural evidence of brain injury to potentially identify neuroprotective effects and/or side effects from the antipyretics that won't necessarily be mediated by fever control. This is really an important additional outcome for the RCT since it is quite possible that the anti-inflammatory benefits of the antipyretics might be neuroprotective yet not significantly reduce fever. But the addition of imaging may also help us better identify adverse effects from the antipyretics. One of the neurological phenomena that occurs in pediatric cerebral malaria is the development of brain microhemorrhages. These feature prominently among autopsy findings and, more recently, our research team identified microhemorrhages in children who survived cerebral malaria. So, one major concern is that children who receive ibuprofen (which is not standard of care for malaria fevers) may have an increased risk of microhemorrhages or frank bleeding. The MRI Ancillary Grant will allow us to see if this is occurring.

Clinically, I have an epilepsy clinic in rural Zambia that I staff weekly, I pinch hit for attending on child neurology consults when needed on the inpatient service and I read EEGs Zambia at the University Teaching Hospitals (UTH) Children's Hospital and Malawi for Queen Elizabeth Central Hospital's Pediatric service.

On the teaching front, there is a new postgraduate training program in neurology now at UTH and it is doing an amazing job of transforming care and education here. I can't take any credit for the program, which is led by Dr. Deanna Saylor (Johns Hopkins) and was co-founded by Dr. Omar Siddiqi (Beth Israel at Harvard), but I will proclaim myself to be the program's biggest cheerleader. And in this vibrant environment, I'm able to spend my medical education endeavors primarily as a mentor for young researchers, both American and African. My mentees study stigma, nutritional neuropathies, cognition in adolescents with HIV, TB meningitis, the impact of health system structure on neurological care delivery, CNS IRIS, and comorbid HIV and NCDs.

So, things are busy and chaotic, but never boring!

The Parkinson's foundation is putting on a charity hockey game Saturday February 8^th from 4-6pm. I will be participating in the event showing washed up NHL players how it's done along with raising money by selling tickets for a good foundation and cause so anyone that can make the game and support it would be amazing! They are asking players to sell tickets (in the "purchasing from a player" box (Here's the link)) and I thought who better to ask than the people that work hands on with Parkinson's disease. Thanks, everyone!

Justin Alves,
Human Subject Research Coordinator

The Huntington Study Group, which was founded in 1993, is a research organization that is devoted to finding new treatments for Huntington's disease. The group includes more than 400 investigators and coordinators from over 100 research sites. Amy has been Instrumentally involved with our Huntington's disease program for more than twenty years. Her commitment to the Huntington's disease community is unparalleled. She is compassionate, knowledgeable, reliable and engaged at all levels of care for patients and families: local, regional, national and international. It is an honor to continue to work with and learn from her. Congratulations, Amy!

The Clinical Materials Services Unit (CMSU) has been awarded the contract to provide drug supply and distribution services to the for a new clinical research initiative that seeks to rapidly evaluate new drug candidates for Amyotrophic Lateral Sclerosis (ALS).

The HEALEY ALS Platform Trial -- which will be conducted by the Sean M. Healey & AMG Center at Massachusetts General Hospital -- is a new clinical trial initiative in which studies of investigational ALS treatments are tested and evaluated simultaneously. New treatments can be added to the study as they become available. This approach has already proven successful in the cancer field and will greatly accelerate therapy development by allowing investigators to test more drugs, increase patient access to trials, and reduce the cost by quickly and efficiently evaluating the effectiveness of multiple therapies.

Three drugs developed by Biohaven Pharmaceuticals, RA Pharma, and Clene Nanomedicine will be the first to participate in the platform trial. CMSU will be responsible for providing clinical supply chain management, packaging, labeling, distribution, and return services for all the drugs used in the platform study.

CMSU is led by senior research associate Cornelia Kamp, M.B.A., and director of Clinical and Business Affairs Patrick Bolger, R.Ph., M.B.A., and is a core research unit of the Center for Health + Technology (CHeT). Over the past 11 years CMSU has provided clinical supply services to 60 multi-center clinical trials conducted in the US, Canada, New Zealand and Australia supported with funding from the NINDS, NCCAMS, NICHD, NEI, Michael J. Fox Foundation, DOD, FDA, and numerous pharmaceutical and biotech companies. At any given time, CMSU supports between 15-20 clinical trials. CMSU recently moved to its new location at 150 Metro Park in Rochester.

Living microglia, genetically marked to glow green, in the cerebral cortex with magenta colored blood vessels from a mouse treated with URMC-099.

A new study published in the Journal of Neuroinflammation found that prophylactic treatment with URMC-099 -- a "broad spectrum" mixed-lineage kinase 3 inhibitor -- prevents neuroinflammation-associated cognitive impairment in a mouse model of orthopedic surgery-induced perioperative neurocognitive disorders (PND).

PND, a new term that encompasses postoperative delirium, delayed neurocognitive recovery, and postoperative neurocognitive disorder, is the most common complication after routine surgical procedures, particularly in the elderly. Following surgery, such as hip replacement or fracture repair, up to 50 percent of patients experience cognitive disturbances like anxiety, irritability, hallucinations, or panic attacks, which can lead to more serious complications down the line. Currently, there are no FDA-approved therapies to treat it.

Developed in the laboratory of Harris A. "Handy" Gelbard, M.D., Ph.D., director of the Center for Neurotherapeutics Discovery at the University of Rochester Medical Center, URMC-099 inhibits damaging innate immune responses that lead to inflammation in the brain and accompanying cognitive problems. Using animal models of diseases like HIV-1-associated neurocognitive disorders, Alzheimer's disease and multiple sclerosis, Gelbard has shown that the compound blocks enzymes called kinases (such as mixed lineage kinase type 3, or MLK3) that respond to inflammatory stressors inside and outside cells.

Gelbard and Niccolò Terrando, Ph.D., director of the Neuroinflammation and Cognitive Outcomes laboratory in the Department of Anesthesiology at Duke University Medical Center, used an orthopedic surgery mouse model that recapitulates features of clinical procedures such as a fracture repair or hip replacement, which are often associated with PND in frail subjects. In a pilot experiment, they treated one group of these mice with URMC-099 before and after surgery, and another group prior to surgery only. Gelbard and Terrando's teams, including first author Patrick Miller-Rhodes, a senior pre-doctoral student in the Neuroscience Graduate Program working in the Gelbard lab at URMC, measured the following:

• How the surgery affected the central nervous system and the immune cells (microglia) that reside there was evaluated using stereology and microscopy.
• Surgery-induced memory impairment was assessed using the "What-Where-When" and Memory Load Object Discrimination tasks.
• The acute peripheral immune response to surgery was assessed by cytokine/chemokine profiling and flow cytometry.
• Long-term fracture healing was assessed in fracture callouses using micro-computerized tomography and histomorphometry analyses.
• For additional details see the "Materials and Methods" section of the study

The team found that the surgery disrupted the blood brain barrier and activated microglia (a first line immune responder present in the inflamed brain), which led to impaired object place and identity discrimination when the mice were subject to the "What-Where-When" and Memory Load Object Discrimination tasks. Both URMC-099 dosing methods prevented the surgery-induced microgliosis (increase in the number of activated microglia) and cognitive impairment without affecting fracture healing.

"A major concern regarding the use of anti-inflammatory drugs for PND is whether they will affect fracture healing. We found that our preventive, time-limited treatment with URMC-099 didn't influence bone healing or long-term bone repair," said Gelbard and Terrando, professor of Neurology, Neuroscience, Microbiology and Immunology, and Pediatrics at URMC and associate professor of Anesthesiology at Duke University Medical Center, respectively. "These findings of improvement in cognition and normal fracture healing provide compelling evidence for the advancement of URMC-099 as a therapeutic option for PND."

"Right now we have nothing to treat this condition," said Mark A. Oldham, M.D., assistant professor in the department of Psychiatry at URMC who treats patients with PND. "We work hard to provide good medical care, including helping people sleep at night and making sure they are walking, eating and drinking, but it isn't clear that these efforts have any meaningful long-term impact."

According to Oldham, recent studies that track patients following an episode of PND show that many of them don't resolve completely, and that they have a new cognitive baseline after delirium.

"It is increasingly an accepted fact that after delirium, people have suffered some kind of neurological insult, which leaves them cognitively or functionally worse off than before the incident," he noted.

Next steps for the research include identifying definitive mechanisms for pain modulation, immune cell trafficking and neuro-immune characterization in PND. Gelbard and Terrando are tackling some of these questions with funds from the National Institutes of Health (RO1 AG057525). The current study was also funded by multiple grants from the NIH (P01MH64570, RO1 MH104147, RO1 AG057525 and F31 MH113504). The University of Rochester has four issued U.S. patents and multiple issued patents in foreign countries covering URMC-099.

The URMC Clinical Materials Services Unit (CMSU) has relocated to a newly renovated space at 150 Metro Park in Rochester. CMSU is a unique academic-based organization that provides consulting and supply chain logistics to small and large multi-center clinical trials.

The CMSU will hold an open house at the new facility on Tuesday, September 10 from 3:00 to 6:00pm.

CMSU was founded in 2008 when clinical researchers at URMC determined the need for a dedicated, on-site facility to manage entire supply chains in support of clinical trials. The CMSU is a core research unit of the Center for Health + Technology (CHeT) which is directed by Ray Dorsey, M.D.

CHeT faculty have been involved in the conduct of clinical research for more than 30 years and have conducted 133 clinical trials, involving 48 different sponsors, 43,000 study participants, and have played a leading role in bringing seven new drugs to market -- five for Parkinson's disease and two in Huntington's disease.

CMSU provides a full array of investigational drug and device packaging, labeling, distribution, and accountability services that support academic medical centers, pharmaceutical and biotech companies, and contract research organizations. CMSU, which is led by executive director Cornelia Kamp, M.B.A, is staffed by 8 dedicated full time employees with more than 150 years of collective pharmaceutical industry experience.

Over the past 11 years CMSU has provided clinical supply services to 60 multi-center clinical trials conducted in the US, Canada, New Zealand and Australia supported with funding from the NINDS, NCCAMS, NICHD, NEI, Michael J. Fox Foundation, DOD, FDA, and numerous pharmaceutical and biotech companies. At any given time, CMSU supports between 15-20 clinical trials.

CMSU also manages the logistical drug supply operations of NeuroNEXT, a NINDS-funded national network of academic medical centers dedicated to accelerating clinical research for neurological disorders. To date, CMSU has been involved in determining the drug supply requirements for 36 of the 67 proposals approved for clinical trials.

The CMSU was previously located in the BioVenture Center in Henrietta. The new 8,800+ square foot facility operates under current Good Manufacturing Practices (cGMP) and is licensed by the New York State Board of Pharmacy. The new location consolidates warehouse, office, and processing space and allows for the more efficient coordination and distribution of research materials.

The UR Medicine Mobile Stroke Unit (MSU) is now being dispatched to provide stroke care to patients throughout Monroe County. The MSU had been operating on a pilot basis in the City of Rochester since its launch in October 2018.

The MSU, which is operated in partnership with American Medical Response (AMR) and is the only unit of its kind in upstate New York, serves as an "emergency department on wheels" and brings the medical expertise and technology necessary to diagnose and treat stroke directly to the patient. Immediate care is essential during a stroke, during which millions of brain cells die every minute. However, if caught early, many stroke victims can make a full recovery.

It is estimated that 3,000 people in Monroe County suffer from a stroke every year. Stroke is the fifth leading cause of death and the number one cause of long-term disability in the U.S.

The MSU is equipped with a portable CT scanner that scans the patient's brain to determine the type of stroke they are experiencing. These scans and results from a mobile lab on the unit are transmitted to stroke experts at UR Medicine's Comprehensive Stroke Center at Strong Memorial Hospital, who consult via an on board teleconferencing system with the EMS personnel and determine if treatment -- in the form of the clot busting drug tissue plasminogen activator (tPA) -- can be administered immediately on scene.

"The ability to diagnose and start care in a patient's driveway is a game changer for our region," said Tarun Bhalla, M.D., Ph.D., Chief of Stroke and Cerebrovascular Surgery at the UR Medicine Comprehensive Stroke Center and director of the Mobile Stroke Unit initiative. "We are grateful to our partners in the EMS community for their cooperation in making this lifesaving technology available to stroke patients across Monroe County."

"The sooner patients receive care, the more likely they are to return to their lives," said Curtis Benesch, M.D., M.P.H., Chief of Stroke and Medical Director of the UR Medicine Comprehensive Stroke Center. "The time saved by delivering care directly to a stroke patient on scene can mean the difference between recovery of function or a lifetime of disability."

The MSU is dispatched by the City of Rochester/Monroe County 9-1-1 Emergency Communications Center in coordination with the following EMS agencies:

• Brighton Volunteer Ambulance
• Churchville Fire Department Rescue Squad
• CHS Mobile Integrated Health Care (Chili, Henrietta, Scottsville, Caledonia)
• Gates Volunteer Ambulance
• Greece Volunteer Ambulance
• Hilton Fire Department Ambulance
• Honeoye Falls-Mendon Volunteer Ambulance
• Irondequoit Ambulance
• Monroe Ambulance
• Northeast Quadrant Advanced Life Support
• Penfield Volunteer Emergency Ambulance Service
• Perinton Ambulance
• Pittsford Volunteer Ambulance
• Point Pleasant Fire Department Ambulance
• Rush Fire Department Ambulance
• RIT Ambulance
• Seabreeze Fire Department Ambulance
• Union Hill Volunteer Ambulance
• Webster Emergency Medical Services

"AMR is proud to partner with the University of Rochester and Monroe County to expand the available care options in Monroe County," said Tim Frost, regional director for AMR Western New York. "We are focused on providing the best possible care for the communities we serve, and bringing this new technology to the area is a testament to that."

An article appearing in Neurology Today, a publication of the American Academy of Neurology, describes how University of Rochester Medical Center (URMC) researchers have painstakingly compiled decades of patient data and developed a highly sensitive rating scale that has provided a detailed picture of Batten disease. This tool, which was developed 17 years ago, has set a standard for how to conduct natural history research in rare childhood neurodegenerative diseases.

A common challenge in the treatment and study of neurological disorders is that these diseases are often poorly understood. The complex manifestation of these conditions -- in which the appearance, severity, and progression of symptoms can vary widely -- combined with the difficulty in recruiting study participants with rare neurological disorders often conspire to hamper efforts to precisely define the disease. This is a major problem when it comes to clinical trials, where rigorously defined outcome measures are required to determine if an experimental treatment is effective.

CLN3 disease is such an example. The disorder is one of a family of conditions called neuronal ceroid lipofuscinoses (NCL), more commonly referred to as Batten disease, which are characterized by vision loss, movement disorders, seizures, and dementia. It is estimated 2 to 4 out of every 100,000 children in the U.S. have NCL. CLN3 disease, a juvenile onset form of NCL, is the most prevalent form of the disease.

Initiated in 2001 by URMC neurologists Frederick Marshall, M.D. and Jonathan Mink, M.D., the Unified Batten Disease Rating Scale (UBDRS) includes physical, seizure, behavioral, and vision assessments. Over time, the scale has been refined based on clinical observations. To date, University of Rochester Batten Disease researchers have used the UBDRS to perform almost 500 evaluations in more than 200 patients in the U.S. and around the world.

The development of the UBDRS was followed by the creation of a registry of known cases and the formation of the University of Rochester Batten Center in 2005. The center is co-directed by Mink and Erika Augustine, M.D. and includes Heather Adams, Ph.D. and Amy Vierhile, D.N.P on the leadership team and has become a leading center internationally for clinical research on all forms of Batten disease.

Read more about the development of the UBDRS in Neurology Today.

Two new grants from the National Institute of Neurological Disorders and Stroke (NINDS) will pave the way for new treatments for neuronal ceroid lipofuscinoses and Charcot Marie Tooth diseases, two groups of rare neurological disorders. The funding, which totals $10 million, will support new research programs led by University of Rochester Medical Center (URMC) neurologists Erika Augustine, M.D., and David Herrmann, M.B.B.Ch., and involve an international team of scientists and clinicians.

The funding comes from the NINDS Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases program, which was created to support studies that lay the groundwork for the next generation of treatments -- including gene replacement therapies -- currently under development. URMC researchers play leading roles in three of the five NINDS clinical trial readiness programs. URMC neurologist Rabi Tawil, M.D., is a co-director of an existing program that focuses on facioscapulohumeral muscular dystrophy.

"For many neurological diseases, there is a lack of preparedness to run the highest quality clinical trials," said Herrmann. "This includes making sure that you have teams in place and ways to effectively measure the effect of a new drug. And because you're dealing with a rare disease, getting enough patients into a trial is often a challenge."

"As the pipeline of potential new treatments expands, we have to be ready," said Augustine. "This means not just understanding the natural history of these disease, but to have clinical trial tools that are fit for purpose, both to meet regulatory requirements and to measure what is important to patients and families."

The research program led by Augustine will focus on Juvenile neuronal ceroid lipofuscinosis (CLN3 disease) -- the most prevalent form of a family of neurological disorders commonly referred to as Batten diseases. The symptoms of CLN3 disease emerge in early childhood and involve vision loss, seizures, and impaired cognitive and motor function, all of which worsen as the disease progresses and youth typically die of disease complications by their twenties or thirties. There are currently no therapies available to modify the course of the disease.

The new funding will support a partnership between the University of Rochester Batten Center and the University of Hamburg in Germany to validate clinical outcomes and neuroimaging biomarkers that will precisely measure the symptoms and progression of CLN3 Disease and enable researchers to determine if new experimental therapies are effective. This funding builds upon more than 15-years of leading research by the Batten Center, which is directed by Jonathan Mink, M.D., Ph.D., in collaboration with Heather Adams, Ph.D. Frederick Marshall, M.D., Amy Vierhile, P.N.P., and Christopher Beck, Ph.D.

The program headed by Herrmann, who heads the URMC Neuromuscular Disease Program, will focus on Charcot Marie Tooth disease (CMT), a family of rare inherited peripheral neuropathies that is characterized by progressive weakness, imbalance, sensory loss, and gait abnormalities. While physical and occupational therapy, braces and other orthopedic devices, and surgery can help individuals cope with the disabling symptoms of the disease, there is currently no disease modifying treatment for CMT.

The project will involve researchers from URMC, the University of Iowa, the University of Pennsylvania, the University of Sydney in Australia, University College of London, and the C. Besta Neurological Institute in Milan, Italy. The team will be validating a clinical outcomes tool and new imaging technologies that measure the integrity and density of muscle tissue and nerve endings. The goal of the program is to generate a set of biomarkers and patient-centered measures of meaningful functional improvement that can ultimately be used in future clinical trials.

Gretchen L. Birbeck, MD, MPH, Professor, Department of Neurology, University of Rochester Medical Center, has dedicated much of her career to improving care for neurological disorders in sub-Saharan Africa. Her work in Zambia initially focused on understanding the burden of epilepsy and barriers to care for patients with epilepsy, and this early work led to studies addressing the pathophysiology and determinants of outcomes of pediatric cerebral malaria in both Malawi and Zambia. She has also contributed to our understanding of neurological complications of HIV and the overall burden of neurological diseases in sub-Saharan Africa.

Dr. Birbeck has worked to improve the neurologic training of healthcare workers and to develop research capacity in both Zambia and Malawi. Moreover, she has been a true pioneer in the field of global neurology as the first to demonstrate how one could have a viable academic neurology career while working in a low-resourced setting. In fact, she has maintained continuous NIH funding for her work in sub-Saharan Africa for the past 16 years. For her work, she has been named Fellow of the American Academy of Neurology, the American Neurologic Association, and the Royal Society of Tropical Medicine & Hygiene; and has received numerous awards. She is a U.S. Paul Rogers Society Ambassador for Global Health Research and was recognized by the International League Against Epilepsy as an Ambassador for Epilepsy.

Her trailblazing work serves as the foundation for the field of global neurology, and she has mentored the majority of neurologists in academic global neurology today. Birbeck's dedication to improving neurological care in sub-Saharan Africa and building the careers of junior African and U.S. neurologists and scientists is unparalleled.