Research brings the cure closer every day.
Research is the only path towards finding an effective treatment or even a cure for ALS, which for now, remains an incurable and invariably fatal disease. For this reason, many patients report that they find hope through participation in research, even if they do not derive a direct therapeutic benefit from doing so.
When contemplating research, it is easy to think only about clinical trials in which an experimental treatment is evaluated, often in comparison to a placebo. Clinical trials are often labeled as phase I (early), phase II (mid) or phase III (late) stage studies. Earlier studies are typically focused more on safety and tolerability, while the primary goals of later phase studies are to find out whether or not a treatment is effective.
Clinical trials, however, are just one example of a much broader spectrum of research studies, many of which are essential to ensuring the success of subsequent trials in discovering effective treatments for ALS. For example, a cure is not possible without knowing the cause of disease. As such, studies that aim to identify the cause of disease (whether it be something in our genes, something we’re exposed to in the environment, or some combination of the two), are urgently needed since the cause of disease is unknown in most ALS patients. Similarly, the development of biomarkers is widely believed to be critical to the conduct of clinical trials, and to maximizing what we can learn from each trial. Biomarkers, for example, can help investigators decide which experimental treatments hold the most promise for advancing from early to late phase clinical trials. Biomarkers can also help investigators decide which drugs that work in animal models of ALS, are worthy of further study in people with ALS.
At the ALS Center at the University of Miami, we are actively engaged in a broad array of research studies. Research is also an integral part of the multi-disciplinary care that we provide as we want all our patients to have the opportunity to learn about and participate in research if they so choose. We encourage you to ask us about research opportunities, either at the University of Miami or elsewhere. We are here to guide and help you.
Research brings the cure closer every day.
Research is the only path towards finding an effective treatment or even a cure for ALS, which for now, remains an incurable and invariably fatal disease. For this reason, many patients report that they find hope through participation in research, even if they do not derive a direct therapeutic benefit from doing so.
When contemplating research, it is easy to think only about clinical trials in which an experimental treatment is evaluated, often in comparison to a placebo. Clinical trials are often labeled as phase I (early), phase II (mid) or phase III (late) stage studies. Earlier studies are typically focused more on safety and tolerability, while the primary goals of later phase studies are to find out whether or not a treatment is effective.
Clinical trials, however, are just one example of a much broader spectrum of research studies, many of which are essential to ensuring the success of subsequent trials in discovering effective treatments for ALS. For example, a cure is not possible without knowing the cause of disease. As such, studies that aim to identify the cause of disease (whether it be something in our genes, something we’re exposed to in the environment, or some combination of the two), are urgently needed since the cause of disease is unknown in most ALS patients. Similarly, the development of biomarkers is widely believed to be critical to the conduct of clinical trials, and to maximizing what we can learn from each trial. Biomarkers, for example, can help investigators decide which experimental treatments hold the most promise for advancing from early to late phase clinical trials. Biomarkers can also help investigators decide which drugs that work in animal models of ALS, are worthy of further study in people with ALS.
At the ALS Center at the University of Miami, we are actively engaged in a broad array of research studies. Research is also an integral part of the multi-disciplinary care that we provide. The reason is that we want all our patients to have the opportunity to learn about, and to participate in research if they so choose. We encourage you to ask us about research opportunities, either at the University of Miami or elsewhere. We are here to guide and help you.
Learn about our Research Collaboration at The ALS Center at the University of Miami
Active Studies
Consider joining our research. You can play an important part in finding the cure.
Help us to understand when and how ALS begins
Recruiting unaffected family members of genetic/familial ALS
- Unaffected individuals with a family history of ALS and who may be at genetic risk for developing ALS
- Individuals living in the United States or Canada
Help us determine the optimal timing of an experimental treatment for SOD1 ALS, and whether it can delay clinical onset
Recruiting participants
Part A (Natural History Run-In)
- Age 18+
- Carrier of a protocol-defined SOD1 mutation
- Clinically pre-symptomatic for ALS
- Plasma neurofilament light (NfL) level below protocol-defined threshold at time of screening
Help us determine the safety and efficacy of multiple experimental drugs in a single trial (one drug per participant)
Active - Not Recruiting
Individuals affected with ALS, onset of weakness in the past 36 months, and breathing function (vital capacity) at least 50% of normal. (Other, study drug-specific, eligibility criteria may also apply.)
Help us use the electronic health record for research and quality improvement
Study name:
CReATe Consortium: CAPTURE-ALS (Clinical Procedures To Support Research in ALS) Study
Recruiting clinic patients
Individuals with a diagnosis of ALS or a related disorder (e.g. PLS, PMA) receiving care at a clinical center that uses Epic as its Electronic Health Record (EHR) system
Help us validate biomarkers that may help ALS therapy development
Closed for enrollment
- Individuals affected with ALS or a related neurodegenerative disorder, including ALS-FTD, FTD, PLS, and PMA.
- Healthy controls
Help us better understand the role genes play in ALS
Study name:
CReATe Consortium: Phenotype, Genotype, & Biomarkers 2 (PGB2) Study
Recruiting patients
- Individuals with an established or suspected diagnosis of amyotrophic lateral sclerosis (ALS) or a related disorder
- Family member of an enrolled affected individual.
Help us to develop and validate biomarkers of ALS
Recruiting patients and controls
- Participants affected with ALS or a related neurodegenerative disease
- Healthy controls
Other Resources
UM ALS Research Collaboration
The ALS Research Collaboration (ARC) at the University of Miami represents a coordinated effort to understand the reasons why we lack effective therapies for patients with ALS, and to advance scientific progress towards a meaningful treatment for this disorder.
CReATe Consortium
The goal of the CReATe Consortium is to advance therapeutic development for ALS and related disorders through study of the relationship between clinical phenotype and underlying genotype, and also through the discovery and development of biomarkers.
Nationwide Clinical Trials
ClinicalTrials.gov is a database of privately and publicly funded clinical studies conducted around the world. Before participating in a study, talk to your health care provider and learn about the risks and potential benefits.
National ALS Registry
The National ALS Registry is a congressionally mandated registry for persons in the U.S. with ALS. It is the only population-based registry in the U.S. that collects information to help scientists learn more about who gets ALS and its causes.
Research Highlights
The latest findings by our team of leading researchers
Our Team of Leading Researchers
The University of Miami ALS Center is home to a team of leading researchers whose work has been internationally recognized.
Benatar/Wuu Research Group
Michael Benatar, MBChB, MS, DPhil
Professor of Neurology
Walter Bradley Chair in ALS Research
Executive Director, ALS Center
Joanne Wuu, ScM
Research Associate Professor of Neurology
Associate Director of Research
ALS Center
Nathan Carberry, MD
Assistant Professor of Neurology
Julie Steele, RN
Neuromuscular Clinical Trials Manager
Anne-Laure Grignon, MSc, MD
CRiALS and Pre-fALS Project Manager
Anne Cooley, BSc, MPH, CCRP
CReATe Project Manager
Alondra Andino-Flores, BS
Research Support Administrator
Maria Catalina Fernandez, MD
Clinical Research Coordinator
Jessica P Hernandez, MD, CCRP
Clinical Research Coordinator
Regla Saldana, MPH
Clinical Research Coordinator
Alexandra Orr, MPH, CCRC
Clinical Research Coordinator
Kyra Johnson, BA
Clinical Research Coordinator
Katrina Nguyen, BS
Clinical Research Coordinator
Enjolie Vadella, MPH
Clinical Research Coordinator
Melanie Hechavarria, MPH
Clinical Research Coordinator
Alejandra Casas, MPH
Clinical Research Coordinator
Max Galarce, BS
Clinical Research Coordinator
Ana Benacerraf, BS
Clinical Research Associate
Lauren Flamenbaum, MS
Clinical Research Associate
Yindi Li, MSPH
Research Analyst / Statistical Programmer
Qingchen Lyu, MSc
Research Analyst / Statistical Programmer
Volkan Granit, MSc, MD
Assistant Professor of Neurology
Medical Director
Kessenich Family Clinic, ALS Center
Caroline McHutchison, PhD
Post-Doc Fellow
Joaquin del Cueto, BA, PMP
Project Manager
Research Support Administrator
Christine Stanislaw, MS, CGC
Genetic Counselor
Julia McDevitt
Student
Ross Bunch
Student
Jake Falbo
Student
Emirose Thatthil
Student
Bradley Research Group
Walter Bradley, DM, FRCP
Emeritus Professor of Neurology
Founding Director
Kessenich Family Clinic, ALS Center
Sharma Research Group
Khema Sharma, MD
Professor of Neurology
Patricia Gonzalez Figueroa
Clinical Research Coordinator
Zeier Research Group
Zane Zeier, PhD
Associate Professor
Melina Ramic, B.S.
PhD Candidate
Matthew Rybin, B.S.
PhD Candidate
Join our family - together, we can change the course of this disease.
Join our family
Together, we can change the course of this disease.
ALS Treatment
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