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ALS Research

The Emory ALS Center is actively engaged in research ranging from basic questions about the causes of ALS to clinical trials of new drugs in people with ALS. Please read below to learn more about our research program and projects.  We need your help and participation in the fight against ALS.                                                                                                                     

Oshields In our laboratories: Dr. Glass' laboratory in the Center for Neurodegenerative Disease is working on the basic mechanisms of motor neuron disease as well as potential new treatments for ALS.  Your contributions, whether it be monetary or personal participation in one of our studies, are essential for our continued productivity.  Thank you!

In the clinic: There are also non-therapeutic clinical research opportunities that we hope will help us better understand the causes of ALS, and the factors that may be important in providing the best care possible. We encourage you to participate in any trials that you may be eligible for. WE NEED YOUR HELP to better understand this disease and to find new treatments that will slow or (better yet) stop this disease! Please contact us if you are interested in participation at any level.

ACTIVE STUDIES/TRIALS:

The current clinical trial sponsored by Genentech, Inc. has expanded to include a multiple-ascending-dose segment. This will involve up to 4 cohorts of subjects who will be given daily doses of the Genentech experimental drug GDC-0134, based upon the safety of prior dosing regimens. Participants will be asked to complete two overnight inpatient stays in the clinical research unit where they will receive the first doses of drug. Drug will then be sent home with the subject, and they will return several times over the next 6-7 weeks for safety checks. Later cohorts may also be asked to take a dose of either caffeine or midazolam (a sedative medication), to assess processing of GDC-0134 by the liver. Subjects are allowed to take riluzole and edaravone during the trial.  Eligibility requirements are similar to those for the single-ascending-dose portion. 

THIS IS A COMPETITIVE-ENROLLMENT STUDY, SO PLEASE ACT FAST IF YOU ARE INTERESTED!

Contact Jane Bordeau at 404-727-1679 or jbord@emory.edu

CLICK HERE for more information.

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Single and Multiple Dose Study of BIIB067 (Isis-SOD1Rx) in Adults with ALS. This is a Phase 1 placebo-controlled study to test the safety, tolerability, and pharmacokinetics of an investigational drug that is injected into the spinal fluid.

Currently Recruiting - Eligibility requirements:

  • Patients with familial ALS due to SOD1 gene mutation (Part B)
  • Vital capacity >50% predicted

Contact Anna Partlow at 404-727-3181 or apartlo@emory.edu

CLICK HERE for more information.

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Evaluation of the Safety, Tolerability, Efficacy, and Activity of AMX0035, a fixed combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for the treatment of ALS (CENTAUR). This phase 2 placebo-controlled trial will investigate the safety and tolerability of a new combination of drugs (already FDA approved for other diseases) in ALS, called AMX0035. The study is anticipated to take place over 6 months and will include 10 study visits.

Currently Recruiting - Eligibility requirements:

  • Patients with a diagnosis of ALs
  • Onset of ALS symptoms <18 months
  • Slow vital capacity >60%

Contact Janet Brantley at 404-727-1673 or janet.m.brantley@emory.edu

CLICK HERE for more information.

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Multicenter Double Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of H.P. Acthar Gel in treatment of subjects with ALS. This phase 2 placebo-controlled trial will test the safety, tolerability, and pharmacokinetics of an investigational injectable drug in patients with ALS. Participants will have approximately 12 study visits (participation in the open label extension will add an additional 5 visits) and a site phone call.

Currently Recruiting – Eligibility requirements:

  • Patients with a diagnosis of ALS
  • Onset of ALS symptoms <2 years prior to the screening visit
  • Forced vital capacity >60%
  • Systolic blood pressure <140 mmHg, and Diastolic blood pressure <90 mmHg
  • Do not have Type 1 or Type 2 Diabetes

Contact Janet Brantley at 404-727-1673 or janet.m.brantley@emory.edu

CLICK HERE for more information.

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COMING SOON: Cytokinetics is sponsoring a study to assess safety and tolerability of an investigation drug, CK-2127107, in patients with ALS.  It is being studied as a potential new treatment to slow the decline of skeletal muscle strength, especially breathing strength, in patients with ALS.  The pill has been tried in humans before but this is the first study in patients with ALS.  The experimental drug is a pill and will be given for 12 weeks.  During this time blood will be tested for safety measures and levels of the drug in the blood.  Also, various measures of function will be measured including breathing strength, muscle strength, testing of voice and find motor skills as well as other measures such as the ALS functional rating scale. There will be 7 visits over about 16 weeks.   One quarter of participants will be randomly assigned to a placebo and three quarters will be randomly assigned to take the active drug. Please note that we are not yet recruiting but hope to before the end of Febuary.

Contact Meraida Polak RN at 404-778-3807 or mpolak@emory.edu.

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Answer ALS: Individualized Initiative for ALS Discovery. Participants donate blood (and optional spinal fluid) and undergo testing of strength and cognition. Includes visits every 3 months for a year, then phone assessments. Stem cells will be created from each patient and the cells will be studied to identify causes and subtypes for targeted treatments.

Currently Recruiting - Eligibility requirements:

  • Patients with ALS or PLS

Contact Arish Jamil at 404-727-1273 or arish.jamil@emory.edu

CLICK HERE for more information.

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CRiALS: Clinical Research in ALS: A genetic research program where participants give a blood sample for DNA and (optionally) a small skin sample to create stem cells for further research.

  • Patients with ALS OR Family members of patients with ALS OR Healthy Volunteers

Contact Arish Jamil at 404-727-1273 or arish.jamil@emory.edu

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Project MinE: is a genetic research program that aims to map the full DNA profiles of at least 15,000 people with ALS and compare them with 7,500 control subjects (people without ALS or a family history of ALS). The program originated in the Netherlands and includes researchers from 15 countries, including The United Kingdom, Portugal, Ireland, Spain and Belgium. Our center is the US site and Dr. Glass is the lead investigator in the U.S. All Emory ALS patients are invited to participate by providing a blood sample for DNA, and (optionally) a small skin sample to create stem cells for further research.  We will also be looking for “controls” who want to participate in this research.

CLICK HERE for more information.

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