Neuroscience | Volume 27 | 2025

NE UR OSCIENCE

University of Rochester | Ernest J. Del Monte Institute for Neuroscience Vol. 27 - 2025

John J. Foxe, PhD

Kilian J. and Caroline F. Schmitt Chair in Neuroscience

Director, Ernest J. Del Monte Institute for Neuroscience

Professor & Chair, Department of Neuroscience

Director, Golisano Intellectual and Development Disabilities Institute

Listen to Neuroscience Perspectives with Lisa Genova

ON THE COVER:

Clinical Materials Services Unit (CMSU) employees prepare shipment in warehouse.

FROM THE DIRECTOR’S DESK

As this year begins to wind down, time feels anything but slow. It is a challenge to reflect on where we are and where we are going with so much instability around us. However, as scientists, we must not be deterred by an unexpected outcome or the unknown; it is actually where we may be most comfortable, the space between, the quest.

At the Del Monte Institute, we are on the heels of hosting two national meetings. Leading experts in intellectual and developmental disabilities discussed groundbreaking research and the latest advancements in the field during our first co-sponsored symposium with the Golisano Intellectual and Developmental Disabilities Institute. Immediately following, we co-hosted, with the Rose Kennedy Center IDDRC, the directors from the IDDRCs from across the country for vibrant discussions and workshops to spark new collaborations and ideas. Since becoming an IDDRC five years ago, we have made investments in resources and scientific talent, and most recently, have begun to integrate clinical work through the Golisano IDD Institute.

During the University’s Meloria Weekend, I had the distinct honor of being the host at New York Times bestselling author Lisa Genova’s headline talk. Best known for her debut novel,

Still Alice, she is also a trained neuroscientist. We were delighted to have her as a guest on our podcast, Neuroscience Perspectives, while she was in Rochester. She provided insight into her writing process and how she has developed the ability to bridge science and storytelling, promoting empathy and shaping the public's understanding of neurological diseases and the people they affect.

John Foxe, PhD

Chair, Department of Neuroscience

Bradford Berk, MD, PhD

Professor of Medicine, Cardiology

Robert Dirksen, PhD

Chair, Department of Pharmacology & Physiology

Diane Dalecki, PhD

Chair, Department of Biomedical Engineering

Jennifer Harvey, MD

Chair, Department of Imaging Sciences

Robert Holloway, MD, MPH

Chair, Department of Neurology

Paige Lawrence, PhD

Chair, Department of Environmental Medicine

Hochang (Ben) Lee, MD

Chair, Department of Psychiatry

As we gather for the annual Society for Neuroscience conference, the largest neuroscience conference in the world, I am energized for URochester neuroscience to continue to make its mark on the field and society. But most importantly, we are an integral part of the larger community that this conference brings together. As scientists, we are stronger together; collaborations are vital to advancing the field, and creating strong connections across the country and around the world is pivotal to our future.

In Science,

John J. Foxe, PhD

Shawn Newlands, MD, PhD, MBA

Chair, Department of Otolaryngology

Webster Pilcher, MD, PhD

Chair, Department of Neurosurgery

Steven Silverstein, PhD

Professor, Department of Psychiatry

Duje Tadin, PhD

Chair, Department of Brain & Cognitive Sciences

Editor/Writer

Kelsie Smith Hayduk

Kelsie_Smith-Hayduk@ urmc.rochester.edu

Contributors

Mark Michaud, Maureen Malone

Feature Photography

John Schlia Photography, J. Adam Fenster

Design

Beth Carr

NEUROSCIENCE

Volume 27 | Winter 2025

2 LATEST RESEARCH

Researchers have discovered that we process sound at a fixed time, a new regimen could be a game-changer for a rare genetic muscle disorder, and an internationally recognized scientist receives a major honor.

4 FEATURE: RAISING THE BAR FOR NEUROLOGICAL DRUG DISCOVERY

CHeT has played an instrumental role in bringing 12 drugs and devices to market.

8 FACULTY Q&A

Sunday Francis, PhD, is an assistant professor of Neuroscience who studies transdiagnostic symptoms, such as compulsivity, in neurodevelopmental disorders. Her lab uses transcranial magnetic stimulation to understand how stimulation to the brain can impact these symptoms.

9 STUDENT SPOTLIGHT

Emma Strawderman is an MD/PhD candidate in the Medical Science Training Program and works in the Translational Brain Mapping Program. Her research investigates how the most common type of brain tumor in adults can rewire the brain as it grows and integrates into neural circuits.

Clinical Materials Services Unit (CMSU) employees label drugs prior to shipment. Read more about their work on page 4.

Millisecond Windows of Time May Be Key to How We Hear, Study Find

Researchers have long thought the brain adjusts to the speed of speech when, rather, the brain is processing information in 100-millisecond increments, even when the speed of speech changes. Neuroscientists discovered that the auditory part of the brain is “listening” or clocking in at a fixed time. Published in Nature Neuroscience, assistant professor Sam Norman-Haignere, PhD, measured the neuronal responses in epilepsy patients who had temporary electrodes implanted inside their brains so medical doctors could monitor their seizures. “The better we understand speech processing, the better we think we'll be able to understand what is causing deficits in speech processing,” said Norman-Haignere.

Study: Hormone Combination

Improved

Strength and Function in FSHD Patients

New research has demonstrated that a combined regimen of growth hormone and testosterone is safe, well-tolerated, and is associated with meaningful improvements in muscle mass, strength, and mobility for men living with facioscapulohumeral muscular dystrophy (FSHD). Researchers believe that this regime could benefit patients with other forms of muscular dystrophy. FSHD is a genetic muscle disorder that usually begins in adulthood. It causes gradual weakness of the face, shoulders, upper arms, and hips. Over time, many patients struggle to walk independently and perform everyday tasks. There are currently no treatments that can slow or reverse these losses.

“We’ve never seen a therapy in FSHD deliver both real gains in strength and lasting benefit after treatment stops,” said Chad Heatwole, MD, director of the University of Rochester Center for Health + Technology, and principal investigator and lead author of the study in Neurology Genetics “This hormone combination could mark the first treatment to not only slow this disease, but help patients regain function.”

Neurobiologist Suzanne Haber, PhD, Elected to the National Academy of Medicine

The internationally recognized scientist whose work has transformed our understanding of the brain networks that play a central role in many mental health disorders, including obsessive compulsive disorder and depression, has been elected to the National Academy of Medicine. Recognized as one of the highest accolades in health and medicine, the distinction acknowledges Haber’s significant contributions to the fields of neuroscience and psychiatry over the past 40 years.

Leader of the National Institutes of Health-funded Silvio O. Conte Center for Basic and Translational Mental Health Research at the University of Rochester, Haber partners with scientists and physicians from institutions across the country to turn her findings on circuit dysfunction into treatments that can improve people’s lives. She’s currently using circuitry models to determine which circuits physicians should target for deep brain stimulation and transcranial magnetic stimulation therapy to be most effective for individuals with OCD, depression, and addiction.

Brain-On-A-Chip Technology Reveals How Sepsis and Neurodegenerative Diseases Damage the Brain

Researchers are using state-of-the-art microchips with human tissue to better understand how the brain operates under healthy conditions and is damaged through neurodegenerative diseases or conditions like sepsis.

William R. Kenan Jr. Professor James McGrath, PhD, and his team used tissue chips to show what happens at the blood-brain barrier when the immune system creates an uncontrollable systemic inflammatory response known as a cytokinetic storm. Their experiments showed that with a high enough cytokine storm, the blood-brain barrier breaks down, leading to brain injury. They also discovered that pericytes, or support cells, fill in holes that are difficult for endothelial cells, the groups of cells that form blood vessels, to fill. This demonstration of the interaction between pericytes and endothelial cells opens the door to therapeutics that can preserve or introduce more pericytes to help keep the blood-brain barrier stable.

A photonic wafer with sensor chips used in microphysiological systems to simulate infection and treatment on human lungs and brains, is photographed at the University of Rochester Medical Center // photo by J. Adam Fenster / University of Rochester

TRANSLATING SCIENCE INTO HOPE

The CHeT Model for Neurological Drug Discovery

By Mark Michaud

The University of Rochester Center for Health + Technology (CHeT) stands as one of academic medicine’s most successful experiments in translating science into patient benefit. Over nearly four decades, CHeT has played an instrumental role in bringing 12 drugs and devices to market—including landmark treatments for Parkinson’s, Huntington’s, and neuromuscular diseases—while pioneering new ways to design, measure, and deliver clinical trials.

What began as a small coordination effort in the late 1980s has evolved into a global model for how an academic research center can run industry-grade clinical trials, invent new endpoints, and move therapies from lab to life faster and more equitably.

Today, under the leadership of Chad Heatwole, MD, MS-CI, CHeT is building on that legacy while charting an ambitious path forward, employing new tools like digital endpoints and predictive analytics to speed clinical trials in neurological and other diseases. The approach blends scientific rigor with empathy for the patient experience. “Every new technology or trial design we adopt,” Heatwole said, “should be focused on improving the lives of the people we’re striving to help.”

At its core, CHeT embodies a simple but radical idea: that rigorous, patient-centered science and operational

excellence can—and should—live within academia. From the analog coordination of early Parkinson’s trials to today’s decentralized, sensor-driven studies, CHeT’s evolution reflects a steady commitment to the principle that research should answer the questions that matter most to patients.

Building an academic powerhouse

CHeT’s origins at the University of Rochester date back to 1987, when neurologist Ira Shoulson, MD, launched the Coordination and Data Center to oversee DATATOP, an NIH study examining the effects of deprenyl (selegiline) and tocopherol (vitamin D) in Parkinson’s disease. The team

enrolled roughly 800 participants across 28 sites, coordinating logistics by phone, fax, and mail—and learning early that patient welfare must drive design.

When the Data Safety Monitoring Board looked at the data and said it was unethical to not treat the people on a placebo, the study was restructured so all participants received active treatment. “We were developing research principles before it was mandated,” says Cindy Casaceli, MBA, who later guided the center’s operational maturation. Those experiences not only shaped the ethics of trial design but also laid the foundation for the sophisticated research enterprise that would follow.

Shoulson’s work also helped knit durable international networks of researchers—the Parkinson Study Group and the Huntington Study Group—that standardized practices, aggregated registries, and created recruitment pipelines still central to modern trials. Renamed the Clinical Trials Coordination Center (CTCC), as the 1990s progressed, the center professionalized, hiring industry talent to document workflows, building standard operating procedures, and demonstrating its ability to meet the industry’s regulatory expectations. Those changes transformed ad hoc academic competence into audit-ready operations, opening the door to partnerships with pharmaceutical firms.

Expansion, integration, and the emergence of CHET

By the 2000s, the team confronted a familiar problem: fragmented vendors, high indirect costs, and fragile supply chains that stalled trials. The response was pragmatic—build what was missing. In 2008, Cornelia Kamp, MBA, established the Clinical Materials Services Unit (CMSU) to provide cGMP-compliant packaging, labeling, distribution, and returns management. “We were getting really lousy service from big contract research organizations,” Kamp said. “So we built our own.” CMSU handled large-scale shipments, complex

cold-chain logistics, and blinded-kit design, quickly attracting institutional and external business.

In 2009, Karl Kieburtz, MD, MPH, consolidated the CTCC, CMSU, and growing translational capabilities into the Center for Human Experimental Therapeutics (CHET)—a single entity to bridge “the valley of death” between discovery and first-in-human testing. Under Kieburtz, who had previously served as the first director of the CTCC, the entities moved out from under the Department of Neurology and into an independent center that brought these capabilities to the broader University and global academic community.

The unified center combined trial design, regulatory strategy, supply logistics, outcomes science, and analytics so investigators no longer had to stitch together vendors. With these capabilities in place, Rochester could now manage every phase of a trial—from design to delivery—under one roof. As Kieburtz put it, CHET offered industry “experience, expertise, and finesse.” That integrated model also became a training crucible, producing project managers, database programmers, and fellows who learned the full lifecycle of trials inside one system.

“Fifteen years of CHET/CHeT show how taking existing pieces of research infrastructure from within the University and positioning them in an independent center, along with new faculty and resources, can expand the mission and connectedness of research,” said Kieburtz.

The CHeT Era

Under the leadership of Ray Dorsey, MD, CHET evolved into CHeT—Center for Health + Technology—adding digital endpoints and decentralized methods to its toolkit while retaining the institutional engine that moves therapies toward patients.

Rebranding signaled more than a new name—it marked a shift from getting molecules into humans to reimagining how trials reach and measure patients. Under successive leaders— Kieburtz’s infrastructure build, Dorsey’s push into telemedicine and remote sensing, and now Heatwole’s emphasis on state-ofthe-art measurement technologies and rare diseases—CHeT became a hybrid engine of science, operations, and technology.

CHeT’s portfolio still favors late-translational, multi-center studies—its “sweet spot”—but couples that work with digital tools and analytic muscle. CHeT associate director Jamie Adams, MD, and her team have driven trials that use wearable sensors, smartphone apps, and decentralized visits to capture continuous, real-world data. Charles Venuto, PharmD, founded and leads CHeT’s Analytics division, which allows researchers to model, predict, and act on data and design more efficient clinical trials. Brett Kinsler, DC, leads CHeT’s Health, a division that works to bring CHeT’s discoveries and technologies to the greater community, and Melissa Kostrzebski, MS, MBA, now heads the CTCC.

Operationally, CHeT is rare in scope: trial coordination, outcomes measurement, clinical materials, analytics, and regulatory support operate in concert, enabling the center to take on complex international protocols and novel endpoints that many partners consider too risky.

Defining the “CHeT Difference”

If CHeT’s brand can be distilled to one idea, it is this: measure what matters to patients. “Historically, treatment assessments have been created through black-room discussions in the absence of any patient input. As a result, the patient’s voice and preferences are often missing from research priorities,” said Heatwole.

Building on the NIH-supported National Registry for Myotonic Dystrophy & Facioscapulohumeral Dystrophy, in 2010, Heatwole created the first patient-reported outcome (PRO) measure for myotonic dystrophy, type 1. His team has since developed more than 250 validated, disease-specific PROs now employed by academic, industry, and government researchers around the globe to measure patient-meaningful responses to therapeutic interventions during clinical trials.

The second pillar is operational breadth. While some centers have fragments of CMSU’s capabilities, few have the whole package—from packaging and cold-chain logistics to interactive response systems and global distribution. CHeT can design sensitive endpoints, recruit hard-to-find patients, manage complex supplies, and stand behind data through regulatory review.

Transforming trial design: from clinics to connected devices

CHeT began integrating wearables, smartphone apps, and remote assessments early—because richer, continuous data and decentralized participation solve real, practical problems. Wearables capture continuous, objective signals over longer periods of time rather than snapshots in the clinic; they also expand access for participants who live far from academic centers. “By leveraging wearables, we’ve expanded the boundaries of clinical research,” said Adams, who is leading studies in Parkinson’s and Huntington’s that are employing smartwatches to measure progression in the diseases.

When paired with CHeT’s registries and modeling expertise, sensor data help identify likely responders, shrink sample sizes, and accelerate decisions. Regulators are watching. Though digital endpoints still require validation, CHeT’s approach—pairing patient-centered measures with continuous

sensor streams—creates increasingly persuasive clinical trial dossiers. Connected devices have moved from experimental add-ons to core tools for modern trials.

Real-World Impact: Approvals, Rare Diseases, and Patients Reached

The model is proven by treatments that reached patients and diseases once ignored by the pharmaceutical industry. Rochester has helped lead pivotal trials for five Parkinson’s drugs, three Huntington’s drugs, and treatments for rare neuromuscular diseases, among others. “Every drug approved to treat Huntington’s disease was studied through the University of Rochester,” Kieburtz said.

“While new therapies are still desperately needed, our researchers were involved in the development of most frontline drugs used to treat Parkinson’s, which have alleviated motor symptoms and improved the lives of people suffering from this progressive disease,” said Adams.

Beyond approvals, CHeT turned risky projects into viable programs by building natural-history registries, validating meaningful outcomes, and running inspectionready operations. Casaceli captures the ethos: “We’re not just focused on finances…we’re focused on moving research forward and bringing hope to patients and families.”

As the center turns toward new frontiers—from braincomputer interfaces to AI-driven clinical trial modeling—it remains guided by the same principles that shaped its beginnings under Shoulson: scientific curiosity married to pragmatic compassion. That, ultimately, is the CHeT difference and the foundation for its next generation of innovation.

Q&A with Sunday Francis, PhD

Sunday Francis, PhD, is an assistant professor of Neuroscience. She completed her PhD in Computational Neuroscience at the University of Chicago. And received her undergraduate degree in Neuroscience at Brown University. She researches transdiagnostic symptoms, like compulsivity, of neurodevelopmental disorders (NDD), aiming to impact individuals regardless of their diagnosis. Her lab uses transcranial magnetic stimulation, or TMS, to understand how stimulation to the brain can impact associated symptoms.

Please summarize your research.

My lab studies pediatric neuromodulation and NDD. We use TMS, a non-invasive form of brain stimulation that uses magnetic pulses to induce electrical fields in the brain through the scalp. We ‘ping’ a system, either exciting or inhibiting the network, and then note how it changes. TMS can be used as a treatment for depression and OCD, and the FDA has cleared it for adolescent depression.

How did you become interested in your field?

I do not like to be bored, and science does not let me get bored. If you can keep me interested, I can pretty much tune out the whole world. I initially went to school for engineering and loved it, but loved my neuroscience department more, so I transferred. My graduate work was in brain–machine interface development, investigating technology that could aid individuals who suffered a stroke or had amputations. Then, during my postdoc, I would end up taking a systems neuroscience approach to genetic and molecular research in NDD, specifically autism spectrum disorder (ASD). My initial interest in ASD came from repetitive behaviors and the impact on motor skills, which connected ASD to my graduate research. I also learned techniques in pediatric neuromodulation in children and adolescents with perinatal stroke and Tourette’s, respectively. I then went on to the National Institutes of Health as a research fellow, developing new projects and skills in pediatric neuromodulation, which is at the center of my research today.

What brought you to the University of Rochester?

I was aware of the University, given that my mother started her PhD in education there during my graduate years. Later, I would meet Nathan Smith, PhD, through a professional

development group. He introduced me to Ben SuarezJimenez, PhD, John Foxe, PhD, and M. Kerry O’Banion, MD, PhD, who won me over when he shared that he had grants from NASA. Eventually, I was selected as a speaker for the Del Monte Institute’s postdoctoral speaking series called NEUROYES.

I have always worried about finding a place that would appreciate my diverse scientific background, many different interests, and my use of varied tools to answer questions. URochester seemed to embrace that; they were not scared by it. I got the feeling they were open to someone who wants to bring different expertise and viewpoints together to answer really cool questions. That seemed unique.

What is your favorite piece of advice?

I guess the best piece of advice anyone has ever told me is just: “Do what you enjoy, do what you love.”

VISIT THE GALLERY TODAY

Neuroscientists in Color

Benjamin SuarezJimenez, PhD

Emma Strawderman

Emma Strawderman, an MD/PhD candidate in the Medical Science Training Program, works in the Translational Brain Mapping Program. She completed her undergraduate studies at the University of Rochester, majoring in Brain and Cognitive Sciences with minors in Biology and Statistics. Her research investigates how the most common type of brain tumor in adults can rewire the brain as it grows and integrates into neural circuits.

“Some gliomas grow slowly, so the brain often has time to shift important functions from the affected area to healthier tissue, helping patients maintain abilities like speech or movement,” Strawderman said. “This “functional reorganization” is a striking example of how adaptable the brain can be. Interestingly, the opposite hemisphere is often recruited to help, though we don’t fully understand how or when this happens.”

Recently, Strawderman’s research showed that tumors on the left side of the brain caused rewiring in the right hemisphere. The study, published in Brain Communications, used MRI scans from patients and healthy participants and found that neural connectivity in the right hemisphere could distinguish patients from the healthy controls with 90 percent accuracy. And the patterns of the right hemisphere connectivity directly correlated with where the tumor was located and the type of cancer. “This study advanced our knowledge on how gliomas drive functional reorganization distal to the tumor site, which has implications for understanding variability across patients in cognitive outcomes, disease progression, and survival. I am now building

on this research in my PhD project, linking changes in neural connectivity to specific outcomes (like language recovery after surgery) and investigating the biological mechanisms driving this reorganization using patient tissue samples.”

She was the 2023 recipient of the Rusyniak Memorial Medical Student Research Fellowship, which helped support this research project. George Rusyniak, MD, spent his career dedicated to education. This fellowship allows medical students to engage in clinical and translational research with Neurosurgery faculty at the Medical Center.

Strawderman first became interested in neuroscience in high school when she participated in the University’s Mini Medical School. “I had the opportunity to hold a human brain specimen and was completely awestruck that this gray lump of tissue once held every thought, memory, and emotion of a person’s life. I became very curious about how neurons worked together during cognition, and how that can go awry in pathology.” This sent her on the trajectory to find ways to preserve cognitive function in brain tumor patients.