Global ALS Leaders Convene to Transform Neuroscience Discovery into New Treatments

By: Staff Writer | October 28, 2026 | 15 min. read |

At the inaugural University of Miami ALS Symposium, scientists examined how biomarkers, shared disease mechanisms and cross-disciplinary partnerships could advance the prevention, diagnosis and treatment of ALS and related neurodegenerative disorders.

ALS Symposium participants gather for a group photo in a University of Miami lecture hall. — (All photos by Isabella Frias)

Key Takeaways

ALS research is helping scientists investigate biological mechanisms that may also contribute to frontotemporal dementia, Alzheimer’s disease and other neurodegenerative disorders.
University of Miami researchers are studying biomarkers that could help identify disease activity earlier and support prevention-focused clinical research.
The inaugural University of Miami ALS Symposium connected scientists, clinicians, trainees and funding organizations to encourage new research partnerships.
Biomarker and prevention studies remain active areas of research and do not yet establish that ALS can be prevented or diagnosed with a new clinical test.

The future of understanding and treating neurodegenerative disorders may depend less on studying diseases in isolation than on pinpointing what mechanisms they share.

That idea surfaced repeatedly during the University of Miami Miller School of Medicine’s inaugural ALS Symposium, hosted by the school’s new Neuroscience Institute and the University of Miami ALS Center. Discussions of genetics, biomarkers, protein biology and disease prevention pointed toward a common conclusion. Discoveries in amyotrophic lateral sclerosis (ALS) are increasingly informing research in Alzheimer’s disease, frontotemporal dementia (FTD) and other neurological disorders.

The converse is equally true, highlighting a need for collaboration across disciplines and an infrastructure to bring top scientific minds together, mobilizing the field’s combined expertise towards advancing patient care.

The Oct. 20 symposium sought to meet this goal by convening distinguished investigators whose discoveries have helped shape the field, including:

Johnathan Fryer, Ph.D., of the Translational Genomics Research Institute
Aaron Gitler, Ph.D., of Stanford University
Jeffrey Rothstein, M.D., Ph.D., of Johns Hopkins University
Nicholas Seyfried, Ph.D., of Emory University
Michael Ward, M.D., Ph.D., of the University of California, Berkeley
Benjamin Wolozin, M.D., Ph.D., of Boston University

The symposium connected these researchers with scientific talent from the University of Miami.

A Broader Vision for ALS Research

For Leonard Petrucelli, Ph.D., founding director of the Neuroscience Institute and professor of neurology at the Miller School, and Michael Benatar, M.D., Ph.D., executive director of the University of Miami ALS Center and professor of neurology at the Miller School, the gathering reflected a broader vision for neuroscience and ALS specifically at the University of Miami that connects expertise in human disease research, basic science, clinical investigation, artificial intelligence and computational biology.

“The University of Miami is uniquely poised to pioneer a new approach to disease research. By uniting our strengths, we have an unprecedented opportunity to uncover new disease mechanisms and identify promising therapeutic targets,” Dr. Petrucelli said.

Dr. Leonard Petrucelli speaks at the front of a lecture hall during the University of Miami ALS Symposium. — Dr. Leonard Petrucelli (right) believes the University of Miami is charting a new course in ALS research.

The symposium offered a glimpse of how the Neuroscience Institute plans to accelerate discovery. Researchers are bringing together experts across disciplines to facilitate open discussion and the sharing of ideas aimed at bettering our understanding of the biological processes that drive neurodegeneration. Through this collaborative environment, UM hopes to pave new avenues towards translating scientific insights into diagnostic and therapeutic strategies.

From Treating Disease to Preventing It

ALS research illustrates a broader shift underway across neuroscience. For decades, researchers were forced to focus on treating neurodegenerative diseases after symptoms appeared. But advances in genetics, biomarkers and molecular biology are now creating opportunities to identify disease-associated changes years earlier, opening the door to interventions before irreversible damage occurs.

Miller School and UM researchers are leading that change. Dr. Benatar and his colleagues at the University of Miami ALS Center are conducting some of the field’s most ambitious prevention-focused studies, including the first ALS prevention trial (targeting individuals with genetic mutations in the SOD1 gene) and a recently launched NIH-funded initiative aimed at laying the groundwork for ALS and FTD prevention trials among C9orf72 repeat expansion carriers.

Dr. Michael Benatar addresses attendees from the front of a lecture hall during the University of Miami ALS Symposium. — At the ALS Symposium, Dr. Michael Benatar said ALS research is shifting toward prevention.

“One of the major focuses of our research is whether it’s possible for us to prevent the emergence of ALS,” said Dr. Benatar, the Walter Bradley Chair in ALS Research, who provided an overview of investigative work ongoing at the ALS Center during his opening remarks. “In addition to providing comprehensive care for patients with ALS, the Center’s work is heavily focused on the development of biomarkers that can accelerate therapy development.”

How Could Biomarkers Improve ALS Research?

More specifically, the center’s work reflects growing confidence that biomarkers can help reveal disease activity long before symptoms develop, providing opportunities to better predict risk, monitor progression and evaluate whether emerging therapies are reaching their intended targets.

Researchers at the Miller School are directly spearheading efforts that could help bring these new biomarkers to patients, founding innovations like the ALS FastTrack Clinic, which connects potential ALS patients with specialists on a timeline of weeks instead of months. This streamlined pathway reduces diagnostic delays and facilitates earlier access to comprehensive care. Inspired by this model, investigators and clinicians are exploring similar rapid-access approaches for Alzheimer’s disease and other neurodegenerative conditions, recognizing that timely diagnosis is increasingly important as new diagnostic tools and therapeutic options become available.

By fostering a spirit of innovation and collaboration amongst diverse scientific researchers, the Neuroscience Institute strives to help build the open science bedrock needed to buoy the impressive translational work already underway at Miami.

The goal is to use the power of science to bring bench to bedside in the form of tangible benefits to patients. And evidence suggests that this philosophy works.

Dr. Petrucelli and his team recently developed tools to detect HDGFL2-CE, a protein that only exists when TDP-43 function is compromised in diseases like ALS, in patient biospecimens. Proteins like HDGFL2-CE could represent a way of detecting TDP-43 defects in living patients, a feat unattainable with current biomarkers. Coupled with innovations like the FastTrack Clinic, Miami could soon become a hotbed for the practical application of translational breakthroughs, capitalizing on key partnerships fostered amongst members of the Neuroscience Institute, the ALS Center and beyond.

What ALS Can Teach Us About Other Diseases

Understanding and preventing ALS is only part of the story. Researchers also increasingly view the disease as a powerful window into the biological processes that drive multiple forms of neurodegeneration. Throughout the symposium, investigators presented findings on TDP-43, a protein linked not only to ALS, but also to FTD, cognitive decline and an increasing repertoire of neurodegenerative disorders.

Their work demonstrated how TDP-43 dysfunction disrupts essential cellular processes and contributes to neuronal degeneration. It also reinforced mounting evidence that diseases traditionally studied as distinct conditions may share common molecular pathways.

Dr. Mercedes Prudencio speaks at the front of a lecture hall during the University of Miami ALS Symposium. — ALS research is having a discovery impact for other diseases, says Dr. Mercedes Prudencio.

“What is very intriguing is that all the research we’re doing in ALS is showing promise to help individuals with other diseases such as Alzheimer’s and frontotemporal dementia,” said Mercedes Prudencio, Ph.D., associate professor of biochemistry and molecular biology at the Miller School, who presented her research at the symposium. “TDP-43 has become one of the clearest examples of how discoveries in one neurological condition can reshape understanding across multiple disorders.”

The emerging picture is changing how researchers think about neurodegenerative disease, shifting the focus from isolated diagnoses toward the underlying biological mechanisms they have in common. Indeed, this was the focus of a recent workshop organized and hosted by the University of Miami ALS Center that led to the development of TDP-43 Associated Neurological Disease (TAND) as a biological construct associated with a range of phenotypic manifestations of disease.

Why Do Research Partnerships Matter?

A major objective of the symposium was to create new connections among academic researchers, clinicians, translational scientists and funding partners who share a common goal of accelerating therapeutic development.

“Breakthroughs in ALS require more than outstanding science. They require bringing together academic researchers, clinicians, industry partners and funders around a shared mission,” said Manish Raisinghani, M.B.B.S., Ph.D., chief executive officer of Target ALS. “This symposium creates an opportunity to build those connections and accelerate the path from scientific discovery to meaningful treatments.”

Dr. Raisinghani presented his unique insight into the current landscape of ALS research at the symposium alongside academic researchers, highlighting how to build connections between basic scientists, philanthropic organizations and the patients they hope to serve.

Dr. Manish Raisinghani speaks from a podium in a lecture hall at the 2026 University of Miami ALS Symposium. — Dr. Manish Raisinghani emphasized academic collaboration during his remarks at the ALS Symposium.

The event also provided opportunities for trainees and early-career scientists to develop new collaborations and identify opportunities for future partnerships. Students and trainees at the University of Miami were invited to participate in a casual luncheon with the speakers, providing them with the direct opportunity to pick the brains of some of the leading scientists in the field. Jordan Marks, M.D., Ph.D., a former trainee in the Petrucelli Lab, was also invited to speak on his research and relay his insight into navigating graduate and medical school.

For Dr. Petrucelli, those connections may prove among the most important outcomes of the inaugural gathering.

“If this meeting sparks even just one new collaboration, connects a trainee to a potential postdoctoral mentor, or inspires a student to pursue a career in ALS research, the symposium will have been a resounding success,” he said.

A Broader Vision for Neuroscience and ALS Research in Miami

The ALS Symposium is the first in a planned series of scientific gatherings hosted by the Neuroscience Institute designed to showcase major advances across neurological disease research, including Alzheimer’s disease, FTD, Parkinson’s disease and stroke. In each instance, the Neuroscience Institute plans to partner with relevant stakeholders on campus.

“This is about much more than bringing scientists together for a single day,” Dr. Petrucelli said. “We are building a collaborative research enterprise that connects exceptional people, human disease biology and emerging technologies to accelerate discoveries that can change patients’ lives.”