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.

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

Study name:
Pre-fALS (Pre-symptomatic Familial ALS)

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
  • Anywhere in the United States or Canada

Help us determine the safety and efficacy of an ALS drug

Study name:
Orphazyme-Arimoclomol Phase III Clinical Trial

Fully enrolled

Individuals affected with ALS, with symptoms of weakness appearing less than 18 months prior to enrollment and preserved respiratory function

Help us to develop and validate biomarkers of ALS

Study name:
CRiALS Biomarkers

Recruiting patients and controls

  • Participants affected with ALS or a related neurodegenerative disease
  • Healthy controls

Help us validate biomarkers that may help ALS therapy development

Study name:
CReATe Consortium: TRIAL READY Study

Recruiting patients and controls

  • 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 (PGB) Study

Recruiting patients

  • Individuals with a clinical diagnosis of ALS or a related disorder
  • Family member of an enrolled affected individual.

Help us use the electronic health record for research and quality improvement

Study name:
CReATe Consortium: CAPTURE (Clinical Procedures To Support Research) 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 to develop and prepare MRI as an ALS biomarker

Study name:
CALSNIC-2 (Canadian ALS Neuroimaging Consortium)

Recruiting patients and controls

  • Affected:  Individuals affected with ALS or a related neurodegenerative disorder, including ALS-FTD, PLS, and PMA.
  • Controls:  Healthy controls 40 – 80 years old.

Help us determine the safety and tolerability of an ALS gene therapy

Study name:
Biogen-C9 Phase I Clinical Trial

Recruiting patients with C9ORF72 ALS

Individuals affected with ALS, and positive for the C9ORF72 gene mutation

Help us to determine the safety and efficacy of an experimental drug

Study name:
Biogen SOD1 Clinical Trial

Recruiting patients with SOD1 ALS

Individuals affected with ALS, and positive for an SOD1 gene mutation that is associated with rapidly progressive disease

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.

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

Volkan Granit, MSc, MD

Assistant Professor of Neurology


Medical Director

Kessenich Family Clinic, ALS Center

Julie Steele, RN

Neuromuscular Clinical Trials Manager

Sumaira Hussain, BSc, CCRP

CReATe Consortium Project Manager

Anne Cooley, BSc, MPH, CCRP

CReATe Project Manager

Anne-Laure Grignon, MSc, MD

CRiALS and Pre-fALS Project Manager

Joaquin del Cueto, BA

Project Manager

Research Support Administrator

Yindi Li, MSPH

Research Analyst / Statistical Programmer

Danielle Sheldon, BSc, CCRC

Clinical Research Coordinator

Danielle Dauphin, BA

Clinical Research Coordinator

Maria Catalina Fernandez, MD

Clinical Research Coordinator

Maria Elena Paredes, MD, CCRC

Clinical Research Coordinator

Wendy Levy, MD

Clinical Research Coordinator

Kristina Reyes, BSc

Research Assistant

Alexa Gonzalez, BA

Research Assistant

Anna Thompson, BS

Research Assistant

Katja McBane, BA

Research Assistant

Margaret Vieira, BS

Graduate Student Assistant

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

Monica Quesada

Clinical Research Coordinator

Zeier Research Group

Zane Zeier, PhD

Associate Professor

Melina Ramic, B.S.

Graduate Student

Matthew Rybin, B.S.

Graduate Student

Join our family - together, we can change the course of this disease.

Join our family

Together, we can change the course of this disease.

Copyright © 2019. ALS Center at the University of Miami. All Rights Reserved. Branding by Mok2