We are excited about the trial results from Amylyx. However, at this time the drug is not available and thus we are unable to provide it for our patients.
ALS research in the time of COVID has been different – but we are continuing to work toward understanding the drivers of ALS. We at the Emory ALS Center continue to be actively engaged in research projects that range from basic questions about the causes of ALS to clinical trials of new drugs to treat people with ALS. Please read below to learn more about our research program and projects.
Though much of our “wet lab” research has been on hold since March, our extensive ALS database and biobank allows us to continue to ask questions about the clinical features of disease (i.e. age of onset, rapidity of progression) and how that relates to protein markers in the blood and the brain, and what relationship these measures have to a person’s genetic makeup.
Some New Projects:
- In collaboration with investigators at the University of Massachusetts and the NIH, and funded by ALSA, Dr. Glass is working on generating a repository of whole genome sequences collected from ALS patients around the world. This will be a valuable collection of genetic information unprecedented in size and will be made available to any and all investigators who are studying any aspect of ALS.
- The Emory ALS Center has just been funded by the National Institutes of Health to investigate “Pathobiology Neurodegeneration in C9ORF72 Repeat Expansion”. This project, in collaboration with investigators at the Mayo Clinic, Johns Hopkins, and the Massachusetts General Hospital will use animal models and human tissues to identify causative factors in patients with this form of genetic ALS.
- We are well into the clinical trials of gene therapies for ALS patients carrying either the SOD1 or C9ORF72 genetic mutation. Promising results have just been reported [link to NEJM press release] and we continue to test these extraordinary new therapies. New trials are being designed and will soon be initiated for other genetic targets, and even for the possibility of preventing disease.
Our Investigators:
Dr. Jonathan Glass, Director. Professor of Neurology and Pathology. Dr. Glass actively collaborates with other ALS investigators around the world on multiple projects examining ALS genetics, immunology, neuropathology, and animal and cellular models of ALS. Dr. Glass also continues his work on discovering biomarkers of ALS, which necessitates 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, serving as “controls” that allow us to compare results between people living with and without ALS.
Dr. Christina Fournier, Associate Professor of Neurology. Dr. Fournier is well into her 5-year research grant from the Veterans Administration to develop a new questionnaire to measure the progression of ALS. The first phase of the project is complete with creation and validation of the Rasch-Built Overall ALS Disability Scale (ROADS), a new and improved tool for measuring the degree of disability in PALS. The ROADS is an improvement over the ALS Functional Rating Scale (ALSFRS-R), as it is shown to be more reliable and is expected to be more responsive in capturing smaller changes in disability. It is hoped that this new scale will improve the efficiency of future ALS clinical trials. You can read about ROADS and be directed to the ROADS webinar here. Many thanks to all the patients who helped by completing the questionnaire and then completing it again from home. We will now be using the ROADS along with the ALSFRS-R in our clinics to help us get a clear picture on how our patients are progressing. Additional opportunities to participate in this important research may be available at your clinic visit. Please consider participating in this important effort, as these efforts help us to design better clinical trials in the future.
Meraida Polak, RN After her many years of devotion to ALS research and patient care at Emory, Meraida has begun a phased retirement plan. She will continue to work on our team part-time. It was Meraida who urged Dr. Glass to start the Emory ALS Clinic in 1997 and we want to thank her for her unflagging commitment to making the lives of people living with ALS better.
We are thrilled to announce Jane Bordeau, RN as our new Research Director. Jane is a familiar face among our team, having been with us since 2010. She has been a nurse coordinator on our research team on many ALS clinical and research trials.
Ezana Assefa, PhD Student. Ezana is a new graduate student working with Dr. Glass on the question of why ALS patients are so different in their clinical characteristics. This is even true for those carrying a genetic form of the disease. Ezana has embarked on a project looking at genetic factors (i.e. genetic modifiers) that may underlie this disease heterogeneity using DNA and cells generated from skin biopsy samples from patients with genetic ALS, but with varying clinical features. Ezana is just getting started, but this promises to be an exciting project.
We at the Emory ALS Center understand the dire need for effective treatments for ALS. The only way to help develop new therapies for ALS is through rigorous scientific investigation. This type of investigation requires the efforts of teams of clinicians and scientists around the world, adequate research funding, and time.
Most importantly, we need the partnership of our patients and their families to study and better understand this disease!
Visit our website to learn about upcoming clinical research and trials we are offering: www.als.emory.edu
HAVE YOU ENROLLED IN THE NATIONAL ALS REGISTRY??
NEALS Educational Webinars: In partnership with The ALS Association, NEALS offers live webinars for people with ALS, caregivers and the broader ALS community. Topics include updates on clinical trials, best practices for clinical care, and current research efforts in ALS causes and treatments. Our webinars also offer participants the opportunity to ask questions directly of the experts running clinical trials and conducting research activities.
View the full NEALS listing of webinars for people with ALS and caregivers.