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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.

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 monetary or participation in one of our studies, are essential for our continued productivity.  Thank you!

Dr. Glass is working on several projects and has been actively collaborating with other ALS investigators around the world on issues of ALS genetics, immunology, neuropathology, and animal and cellular models of ALS.  There are several ongoing and planned clinical trials outlined below.  Dr. Glass also continues his work on discovering “biomarkers” of ALS, which requires the participation of PALS and CALS.  We are collecting blood and spinal fluid samples from patients for our research.  Family members and non-related adults are also important participants in our studies, since we need to compare our results between those with and without ALS.

Dr. Christina Fournier was recently awarded a 5-year research grant from the Veterans Administration.  Her project is to develop a new questionnaire to measure the progression of ALS.  The questions will be related to function, similar to the current ALS Functional Rating Scale (ALSFRS) that is performed at each clinic visit.  The hope is that Dr. Fournier’s new scale will be more sensitive to changes in function in PALS, making it useful both for clinical care and for research trials. Please agree to participate in this important effort by filling out the questionnaires if you are asked.

We at the Emory ALS Center understand the dire need for effective treatments for ALS.  The problems we face in developing new therapies for ALS will only be solved by rigorous scientific investigation, which takes team efforts of clinicians and scientists around the world, adequate research funding, and of course time.  Most importantly, we need to partner with our patients and their families to study and better understand YOUR disease!  Your participation in research is essential!

Watch our website for upcoming clinical research and trials.  In order to understand ALS better, why it happens and what is important for progression, we need YOUR participation in research studies and clinical trials: neurology.emory.edu/ALS

 

Clinical Trials Opportunities: (treatment with an experimental drug)

Acthar: ENROLLING--This phase 2 placebo-controlled trial sponsored by Mallinckrodt 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.  For information about this study, contact Janet Brantley at 404-727-1673  or janet.m.brantley@emory.edu .

Centaur: ENROLLING-- Amylyx Pharmaceuticals is offering a phase II trial investigating the safety and tolerability of a new combination of drugs (already FDA approved for other diseases) in ALS.  AMX0035 may help preserve certain nerve cell components and reduce inflammation and oxidative damage to neurons. The study will have both a treatment arm and placebo arm and is anticipated to take place over 8 months and include 7 study visits to the clinic and 4 phone calls. Testing includes muscle strength, EKG, blood draws and breathing tests.  For information about this study, contact Janet Brantley at 404-727-1673 or janet.m.brantley@emory.edu .

Single and Multiple Dose Study of BIIB067: ENROLLING--This is a Phase 1 placebo-controlled study from Biogen to test the safety, tolerability, and pharmacokinetics of an investigational drug that is injected into the spinal fluid. Eligibility requirements include familial ALS due to SOD1 gene mutation and vital capacity >50% predicted.  For information, contact Anna Partlow at 404-778-3181 or apartlo@emory.edu.

FORTITUDE Study: ENROLLING —Cytokinetics is sponsoring a study to assess safety and tolerability of CK-2127107 in patients with ALS.  It is a potential new treatment to slow the decline of skeletal muscle strength, especially breathing strength, in people with ALS.  This drug is a pill and will be given for 12 weeks.  Blood will be tested for safety and levels of the drug in the blood.  Various measures of function will also be measured. There will be 7 visits over about 16 weeks. Contact Meraida Polak, RN at 404-778-3807 or mpolak@emory.edu.

FLX-787-203 (FLEX): COMING SOON—FlexPharma, Inc. is sponsoring a phase 1 clinical trial of an experimental drug, FLX-787-203, a chemical derivative of ginger. This drug is being explored as a therapy for muscle cramps in people who have ALS.  There will be a 28-day screening stage, a 28-day “run in” period and a treatment period for a total participation time of approximately 3 months.  For more information about participation at the Emory ALS Center, please contact study coordinator Zenisha Bain at 404-712-8578 or zenisha.bain@emory.edu

Genentech GDC-0134:  ENROLLING--This is a first-in-human study to examine whether the drug GDC-0134 is safe and tolerable in people with ALS.  Subject groups will start at a low dose of drug and subsequent groups will be given higher doses of drug if safety of the prior dose is established.  There is a placebo group.  Participants may only participate in a single dosing period.  Subjects may be required to stay as an inpatient in the hospital for 2 days while initiating dosing, and come in for six follow-up visits over the next 6 weeks after discharge.  For additional information contact Jane Bordeau, RN at 404-727-1679 or jrbord@emory.edu .

Genentech GDC-0134 Open-Label Extension—COMING SOON—Genentech will be offering an open-label extension of the GDC-0134 study detailed above. This study will be dosing with the same drug, but there will be no placebo group: all participants will receive active drug. The study will require screening and will employ the same eligibility criteria as the main study.  The study period will be approximately 12 months, including a 4-week follow-up period. Participants from previous phases of the study are eligible as well as new participants.  Please check the website or contact the study RN to find out when the study will open recruitment.

HAVE YOU ENROLLED IN THE NATIONAL ALS REGISTRY??

www.alsa.org/als-care/als-registry  

 

Clinical Research Opportunities: (no experimental drug treatment)

Research Study      

Study Details

Contact Person

Clinical Research in ALS (CRiALS)

·    To learn more about neurological disorders

·    To contribute to Project MinE

·    For ALS patients, blood relatives and healthy unrelated volunteers

·    Procedures include donation of a blood sample, skin sample and/or spinal fluid and an information questionnaire

Arish Jamil

404-727-1273

Arish.jamil@emory.edu

 

www.projectmine.com 

 

Answer ALS

·    Create a comprehensive registry of over 1000 pALS

·    Create stem cells from each

·    Use the cells to identify causes and subtypes for targeted treatments

·    For all ALS and PLS patients

·    Procedures include donation of blood and (optional) spinal fluid, assessments of strength and cognition

·    Visits every 3 months for 1 year, phone assessments thereafter

Arish Jamil

404-727-1273

Arish.jamil@emory.edu

 

www.AnswerALS.org 

 

MORE COMING SOON—STAY INFORMED AT als.emory.edu

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