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

Oshields
We are gratefyl to Bill O'Shields for his commitment to research. All the money in the world, and the brightest scientists cannot find the answers to ALS without patients like him graciously helping with the cause. In the table below are many opportunities for pALS to participate in projects.

COMING SOON

Neuraltus Pharmaceuticals, Inc. is continuing their study of NP001 in people with ALS.  This is an injectable experimental drug designed to minimize the contribution of inflammation to the development or progression of the disease by changing the action of the blood cells responsible for inflammation in the body.  Analysis of data from the phase I study of NP001 showed that people with elevated levels of the inflammation biomarker CRP may have experienced enhanced respiratory function due to action of NP001.  Subjects must be able to attend up to 5 consecutive dosing days per month over a 6-month period, and must be tested for elevated inflammation at baseline in order to participate.  For additional information, contact Jane Bordeau, RN at 404-727-1679 (jrbord@emory.edu).

The current clinical trial sponsored by Genentech, Inc. is expanding to include a multiple-ascending-dose segment. This will involve 3 cohorts of subjects who will be given increasing doses of the Genentech experimental drug GDC-0134, based upon the safety of prior dosing regimens explored in the single-ascending-dose portion currently underway. (The current trial information is in the table below) Participants will be asked to complete a 4-5 day inpatient stay in the clinical research unit where they will receive the first doses of drug. Drug wll then be sent home with the subject, and they will return several times over the next 6-7 weeks for safety checks. Some 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. Eligibility requirements are similar to those for the single-ascending-dose portion. If you are interested, please contact Jane Bordeau (jrbord@emory.edu

ALSO COMING SOON - probably in early 2017, is a study with Amylyx pharmaceuticals.  More info can be found HERE.

The table below will gives you information about all the trials and studies currently happening at Emory ALS Center.

Research Study Name Currently Recruiting Treatment Trial? Who Is Eligible to Participate? Brief Summary Contact Information Links for More Information
A Single-Ascending Dose Study of GDC-0134 to Determine Initial Safety, Tolerability, and Pharmacokinetic Parameters in Patients with ALS NO YES
  • Patients with a diagnosis of ALS
  • Vital capacity >60%
  • Able to swallow pills; no feeding tube
  • Not taking nutritional/herbal supplements
  • Non-smoker
  • No history of eye disease (ex. glaucoma)
This Phase 1 placebo-controlled study will test the safety, tolerability, and pharmacokinetics of an investigational oral agent in patients with ALS. Participants will spend 4 days in the hospital and will have several other study visits over a 15-day period.

Jane Bordeau

(404) 727-1679

jrbord@emory.edu

GDC-0134
ANSWER ALS YES NO
  • Patients with ALS or PLS
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.

Arish Jamil

(404) 727-1273

arish.jamil@emory.edu

ANSWER ALS
CRiALS: Clinical research in ALS YES NO
  • Patients with ALS
  • OR
  • Family members of ALS patients
  • OR
  • Healthy volunteers
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. 

Arish Jamil

(404) 727-1273

arish.jamil@emory.edu

PROJECT MINE
Single and Multiple Dose Study of BllB067 (Isis-SOD1Rx) in Adults with ALS YES YES
  • Patients with a diagnosis of sporadic ALS (Part A)
  • Patients with familial ALS due to an SOD1 gene mutation (Part B)
  • Vital capacity >50% predicted
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 

Anna Partlow

(404) 778-3181

apartlo@emory.edu

BllB067
LABB: A Multicenter Study for the Discovery and Validation of ALS Biomarkers NO NO
  • Patients with a diagnosis of ALS
  • Vital capacity >50%predicted
  • Able and willing to undergo a spinal tap
The purpose of this study is to discover blood and spinal fluid markers of ALS disease progression. 

Meraida Polak

(404) 778-3807

mpolak@emory.edu

LABB
Methodology Study of Novel Outcome Measures to Assess Progression of ALS YES NO
  • Patients with a diagnosis of ALS
  • Onset of ALS symptoms <2 year prior
To identify outcome measures and biomarkers for use in ALS research studies.

Anna Partlow

(404) 778-3181

apartlo@emory.edu

METHODOLOGY

 

Project MinE: 

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. 

July 25, 2016 publication.

Stem Cell Trial: 

ARTICLE: The Promise and the Reality of Stem-Cell Therapies for Neurodegenerative Diseases

We are in between the completion of Phase II and the next phase of the trial. The next phase will be directed at evaluating the ability of stem cell transplantation to slow the course of ALS. Trial updates will be posted to the Emory ALS Center website.  We are not currently recruiting, nor do we know all of the eligibility criteria.

Two new papers have been published from the Emory ALS Center research group:
1) Comparative analysis of C9orf72 and sporadic disease in an ALS clinic population
Umoh ME, Fournier C, Li Y, et al.
Mfon Umoh, a graduate student in Dr. Glass' laboratory, used our ALS clinical database to compare patients carrying the C9ORF72 mutation (the most common mutation found in families with ALS) with those who do not carry the mutation.  She found that the groups are quite similar in their age of onset and progression of disease, though patients with the mutation were more likely to have a family history of ALS and/or frontotemporal dementia. Read full article here.

2) NEK1 variants confer susceptibility to amyotrophic lateral sclerosis
Kenna KP, van Doormaal PT, Dekker AM, et al.
 
This new finding was supported by the funding generated by the Ice Bucket Challenge, and by Emory's leadership in the international consortium looking for genetic factors that affect risk in ALS, Project MinE (add website here please).  Mutations in NEK1 confer an increased risk of getting ALS in those with family histories of ALS, but also in people without a family history, i.e. "sporadic" ALS.  It is not clear whether these mutations, on their own, can cause ALS.  Discovery of the NEK1 new gene provides new targets for research into causes of ALS, and also for new ideas about therapeutic intervention. Read full abstract here.

Click Here for Recent Publications by Dr. Glass

Christina Fournier, MD completed her Neuromuscular Fellowship at Emory, and is now the associate director of the Emory ALS Clinic.  Dr. Fournier’s research interests are in clinical research, and she is actively pursuing studies in ALS biomarkers, including a project comparing the clinical features and biomarkers between patients with ALS and PLS (primary lateral sclerosis).  She was the recipient of the inaugural Virginia Freer-Sweeny fellowship in PLS, jointly funded by the Northeast ALS Consortium and the Spastic Paraplegia Foundation. She has also recently completed a study showing no meaningful differences in ALS onset or progression in patients with a history of head injury as compared to patients without a history of head injury.  Dr. Fournier is also the co-principal investigator with Dr. Glass for the ongoing immunosuppression trial for ALS.

Mfon Umoh is a student currently pursuing combined MD and PhD degrees in the Emory Medical Scientist Training Program.  Mfon came to Emory from the University of Pennsylvania, and is now working with Dr. Glass on her PhD thesis focused on the role of autophagy in ALS.  Autophagy is one of the cellular systems that handles abnormal proteins, removing them from the cell to allow for normal cellular functioning.  Abnormal proteins, such at those clumped or “aggregated” are found in the brain and spinal cord tissues of ALS patients, and are thought to be involved in the disease process.  Mfon is particularly interested in studying autophagy in the brains of patients carrying the C9ORF72 genetic mutation, which is now known to be the most common genetic abnormality found in families with ALS, and also in those with Frontotemporal Dementia.  Mfon will be using a combination of proteomics and cultures of motor neurons from ALS patients and from animals to address her questions about whether abnormalities in autophagy underlie the causes and/or progression of ALS.

Tizeta Tadesse, PhD comes to the Glass laboratory after receiving her PhD from Georgia State University, where she worked on understanding the basic function if neurons in the lab of Dr. Charles Derby.  Dr. Tadesse is focusing on one of the major themes of the Glass lab, that of how and why the connection between nerve and muscle (neuromuscular junction) fails early in the course of ALS.  Previous work in the Glass lab by Dr. Lindsey Fischer demonstrated that the one of the earliest pathological features of ALS is the disconnection of the motor nerve from the muscle, which is the underlying cause of muscle weakness in ALS.  Subsequent work showed that the function of mitochondria is important for maintaining the connection between nerve and muscle, and Dr. Tadesse is following up on that observation by developing models of mitochondrial dysfunction and asking how this might cause nerve degeneration and loss of connection with muscle.  She is also working with Dr. Glass on new biomarkers for ALS progression, and on identifying the stem cells injected into patients during the stem cell transplantation trial for ALS ongoing at Emory. (Tadesse, T., et. al. (2014), Analysis of graft survival in a trial of stem cell transplant in ALS. Annals of Clinical and Translational Neurology, 1: 900–908. doi: 10.1002/acn3.134)

Dr. Glass, in addition to his mentorship and participation in the research ongoing in the laboratory, continues his work on discovering “biomarkers” of ALS. Biomarkers can be defined as characteristics in patients that either separate them from patients with other diseases (termed “Diagnostic Biomarkers), or can be markers of disease progression than serve as quantitative measures of how rapidly the disease is progressing (“Disease activity biomarkers”).  In addition, Dr. Glass is actively participating in the ongoing clinical trials at Emory, but is also now involved in the executive leadership of the Northeast ALS Consortium (NEALS), an organization that focuses on getting new drug therapies to patients with ALS. A new project is called Project MinE, an effort started in the Netherlands with the goal of collecting DNA samples from 15,000 ALS patients and 7500 “controls” (people without ALS) in order to look for genes that do not necessarily cause ALS, but influence the course of the disease.  This is a major international effort, and Dr. Glass along with Dr. John Landers at the University of Massachusetts, are leading the US contribution.  The ALS Association has contributed an initial $1 million toward the US effort, and we are seeking further donations through the Project MinE website to extend the number of DNA samples that we can contribute. 

 

IN THE CLINIC: 

The therapeutic clinical trials for Ceftriaxone, Dexpramipexole, and NP001, are all completed.  The results were disappointing, with both Ceftriaxone and Dexpramapexole showing no benefit as compared to placebo.  NP001 showed some promise, though there has been no decision from the company on whether to pursue a new, more definitive trial. 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.  These include the Biomarkers study, and the donation of DNA and tissue samples for research. 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. 

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