New Location for Clinical Trial Support Services
Wednesday, September 4, 2019
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.
Mobile Stroke Unit Expands Operations to Monroe County
Wednesday, August 21, 2019
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.”Read More: Mobile Stroke Unit Expands Operations to Monroe County
URMC Research Tool Unlocks Natural History of Batten Disease
Wednesday, July 31, 2019
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.
New Grants will Accelerate Clinical Trials in Rare Neurological Disorders
Wednesday, June 26, 2019
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. Read More: New Grants will Accelerate Clinical Trials in Rare Neurological Disorders
Johns Hopkins School of Medicine 2019 Honors Gretchen Birbeck, MD, MPH, with their Global Achievement Award
Monday, June 10, 2019
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.Birbeckhas 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’sdedication to improving neurological care in sub-Saharan Africa and building the careers of junior African and U.S. neurologists and scientists is unparalleled.
New Gene Therapy Poised to Transform Care for Spinal Muscular Atrophy
Wednesday, June 5, 2019
The University of Rochester Medical Center (URMC) has been tapped as one of the first institutions in the U.S. to offer a new gene replacement therapy to treat spinal muscular atrophy (SMA). The new treatment can be delivered within weeks of birth and clinical trials have shown that it dramatically changes the course of the disease.
“SMA is a devastating disease and, until very recently, diagnosis was tantamount to either a death sentence or a lifetime of severe disability,” said URMC neurologist Emma Ciafaloni, M.D., the director of the URMC Pediatric Neuromuscular Medicine Program. “This new therapy, combined with the clinical expertise necessary to treat this complex disease, holds the promise to slow, if not completely halt, the progression of SMA.”
Ciafaloni served on a panel overseeing data collection and safety for clinical studies of the new treatment. She is also served as an advisor to Avexis, the company that developed the new gene therapy and was acquired by Novartis in 2018. The treatment is being marketed under the brand name Zolgensma.
The gene therapy, which is administered in the form of a one-time IV infusion, was recently approved by the Food and Drug Administration (FDA) under a “fast track” review process. URMC is one of only 17 medical centers in the U.S., and the only one in New York State, that will initially offer the treatment.
SMA – a rare hereditary genetic disease that is diagnosed in one out of every 10,000 children born in the U.S. each year – is caused by a mutation in the survival motor neuron gene (SMN1). This genetic flaw disrupts the production of a protein critical to the function of the nerves that control muscles.
In infants with SMA type 1, the most common and severe form of the disease, symptoms will typically appear within six months as they begin to gradually experience difficulty breathing, swallowing, speaking, and moving. Left untreated, the infant’s muscles will progressively deteriorate, eventually leading to paralysis and death, which in most cases occurs within two years. SMA is the number one genetic cause of death in infants.
The new treatment employs a genetically-engineered virus that enters the spinal cord and delivers fully functioning copies of the SMN1 gene directly to the patient’s motor neuron cells. These healthy genes rapidly replicate, enhance the cells’ protein production, and help re-establish muscle control.Read More: New Gene Therapy Poised to Transform Care for Spinal Muscular Atrophy
New Multi-Institutional Partnership to Focus on Stroke Rehabilitation
Monday, May 20, 2019
The University of Rochester Medical Center (URMC), Burke Neurological Institute, and Wadsworth Center of the New York State Department of Health (NYSDOH) have been awarded a $5 million grant from the Empire State Development Corporation to speed the development of ground-breaking neurological treatments for those disabled from stroke.
The project is a part of the NeuroCuresNY (NCNY) initiative, a new non-profit formed by the three institutions to accelerate the discovery of novel treatments for chronic neurological impairment and disability. The new state funding will support a two-year pilot study that will be launched in January 2020. This study design will be unique because it will test the efficacy of state-of-the-art robotic-assisted rehabilitation technology combined with drugs to improve the functional recovery of stroke patients.
Neurological conditions such as stroke, traumatic brain injury and spinal cord injury permanently disable more than one million people each year in the U.S., and stroke is the nation’s leading cause of disability. Clinical trials for neurological disabilities and impairments are usually passed over because of unclear results, high costs, and challenges in recruiting participants. NCNY will seek to lower the barriers to participation in clinical trials by assisting with travel funding for patients, while providing a supportive and guiding environment for patients and their families.
Clinical and research faculty from URMC Departments of Neurology, Neurosurgery, and Physical Medicine & Rehabilitation will collaborate with the UR Neurorestoration Institute during the pilot study.Read More: New Multi-Institutional Partnership to Focus on Stroke Rehabilitation
Mouse Study: Deep Sleep Helps the Brain Wash Away Toxic Proteins
Friday, March 1, 2019
Deep sleep allows the brain to wash away waste and toxic proteins more efficiently, according to a new mouse study published in the journal Science Advances. The new findings shed light on previous evidence linking Alzheimer’s disease with aging and sleep deprivation.
“Sleep is critical to the function of the brain’s waste removal system and this study shows that the deeper the sleep, the better,” said Maiken Nedergaard, MD, DMSc, co-director of the Center for Translational Neuromedicine at the University of Rochester Medical Center (URMC) and lead author of the study.
“These findings also add to the increasingly clear evidence that quality of sleep or sleep deprivation can predict the onset of Alzheimer’s and dementia.”
The study suggests that the slow and steady brain and cardiopulmonary activity linked to deep non-REM sleep are optimal for the function of the glymphatic system, the brain’s waste removal system. The findings may also explain why some forms of anesthesia can result in cognitive dysfunction in older adults.Read More: Mouse Study: Deep Sleep Helps the Brain Wash Away Toxic Proteins
Grant Marks Two Decades of NIH Support for Muscular Dystrophy Research
Tuesday, February 26, 2019
Deposits of toxic RNA (red) are seen here inside muscle cell nuclei (blue) from an individual with myotonic dystrophy
The University of Rochester Medical Center (URMC) has received $8 million from the National Institutes of Health (NIH) to support pioneering research on muscular dystrophy. The grant, which is a renewal of URMC’s Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, will fund ongoing work to investigate the genetic mechanisms and progression of this complex multi-system disease, research that has led scientists to the threshold of potential new therapies for myotonic dystrophy.
“The mission of the URMC Wellstone Center is to promote research that leads to effective treatments for muscular dystrophy,” said Charles Thornton, M.D., a professor in the URMC Department of Neurology and director of the URMC Wellstone Center. “This new funding will enable us to continue a research program that has been forged from a true partnership between bench scientists, clinical researchers, and patients and their families.”
URMC is home to one of six NIH-designated Wellstone Centers in the nation. URMC was selected in the first cycle of funding when the program was launched 16 years ago and is the only Wellstone Center that has been continuously funded since the program’s inception. With the current award, URMC has received a total of $29.8 million in NIH funding to study the disease since 2003.
The URMC Wellstone Center focuses on myotonic dystrophy, a disease that can be lethal in infants and adults and is characterized by progressive disability. Researchers at URMC have been studying myotonic dystrophy for more than 30 years and their work has transformed our understanding of the biological mechanisms of the disease. The new funding will support a long-standing collaboration between researchers at the University of Rochester and RNA scientists at the University of Florida.
Approximately 40,000 Americans have myotonic dystrophy, which is one of the most common forms of muscular dystrophy. People with the disease have muscle weakness and prolonged muscle tensing (myotonia), which makes it difficult to relax muscles after use. Eventually many patients have difficulty walking, swallowing, and breathing.Read More: Grant Marks Two Decades of NIH Support for Muscular Dystrophy Research
Possible Parkinson's 'Pandemic' Looms: Report
Wednesday, February 20, 2019
TUESDAY, Feb. 19, 2019 (HealthDay News) -- The number of people living with Parkinson's disease worldwide could double in the next two decades, experts project.
In a report warning of a possible Parkinson's "pandemic," researchers say the stage is set for cases to surge to 12 million or more by 2040.
What's to blame? In large part, trends that are generally positive: Older age is a major risk factor for Parkinson's, and with life expectancy rising worldwide, more people will develop the disease. At the same time, Parkinson's patients are surviving longer, which drives up the number of people living with the disease at any given time.
Then there's a less expected factor: Declining smoking rates. While the habit has many devastating effects, research suggests it protects against Parkinson's.
Those are obviously trends that no one wants to reverse, said report author Dr. Ray Dorsey.
There are, however, other ways to slow the projected rise in Parkinson's, said Dorsey, a professor of neurology at the University of Rochester Medical Center in New York.Read More: Possible Parkinson's 'Pandemic' Looms: Report
URMC Designated as a Duchenne Care Center
Monday, February 11, 2019
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization dedicated to advancing care and research for patients with Duchenne muscular dystrophy, has named the University of Rochester Medical Center (URMC) a Certified Duchenne Care Center. This program of leading centers, created in 2014 by PPMD, reviews and recognizes clinics nationwide for their outstanding neuromuscular programs.
“Our goal is to make a positive impact on the quality of life of children, adolescents, and adults affected by Duchenne by providing a team of experts in all specialties needed to best care for patients, a friendly and supportive atmosphere, and coordinated care that is highly accessible for families,” said Emma Ciafaloni, M.D., the director of the URMC Duchenne Muscular Dystrophy Clinic. “We are honored to be recognized by PPMD as a Certified Duchenne Care Center as we continue to provide the best care and support possible for Duchenne patients and their families.”Read More: URMC Designated as a Duchenne Care Center