Annual Meeting Highlights Clinical Trials Progress
NEALS 2013 Annual Meeting Highlights
Clinical Trials Progress
January 7, 2014
The 12th Annual Meeting of NEALS was the largest and most information-packed meeting yet. Over 300 clinicians, nurses, therapists, and researchers, from over 80 of 109 NEALS sites across North America, came together for three days to take stock of the many efforts being coordinated by NEALS, and to accelerate the pace of clinical research in ALS.
The meeting, held in October 2013 in Clearwater Beach, Florida, included training sessions for new techniques, introductions for new investigators, and intense working group meetings for researchers working together on biomarkers, tissue banking, and upcoming clinical trials. The group also heard cutting-edge updates about advances in ALS genes, theories of ALS pathogenesis, and important similarities between ALS and other neurodegenerative diseases, including Alzheimer’s disease and Parkinson’s disease. Understanding and exploiting these similarities may speed the discovery of new treatments.
“The variety and intensity of activities at this meeting is a testimony to our investigators, and is an indication of our growth and our success in attracting people to become part of NEALS,” said Merit Cudkowicz, MD, Co-Chair of NEALS.
New initiatives this year include:
~ enhancing the biorepository for tissues from ALS patients, an invaluable resource for researchers investigating new disease mechanisms and potential new therapies
~ development of NeuroBANK, a central repository for ALS clinical trial data
~ creation of a Global ALS Patient Identifier, which will allow investigators to mine data from multiple clinical trials while protecting patient confidentiality
~ a push to speed commencement of new trials through centralizing initial trial review and oversight, and using master contracts between multiple sites and research sponsors
~ expansion of the Clinical Research Learning Institute, a program dedicated to educating attendees on clinical research and therapy development and empowering this group to be advocates for ALS clinical research
Progress on more than a dozen ongoing and upcoming clinical trials coordinated by NEALS was presented. Highlights included:
Tirasemtiv: Tirasemtiv is a symptomatic treatment that improves muscle strength. After promising results from a single-dose trial, NEALS is now completing a multiple-dose trial to test the ability of the drug to provide benefit over several months. Results are expected to be announced in spring of 2014.
Diaphragm pacing system: This system implants electrodes onto the diaphragm muscle, the major breathing muscle, to replace the stimulation lost as motor neurons die. The trial is comparing use of the DPS with the standard of care at an ALS multidisciplinary clinic. Enrolling patients are randomized to one or the other group, but the study is not blinded, because of the surgery required. “There are some enormous challenges in this study,” said Jeremy Shefner, who is leading the trial, but it is vital to do, because it is important to rigorously test the therapy to determine if it offers patients benefits commensurate with the risks. The trial is expected to be completed in 2015.
Nuedexta: This drug was approved in 2011 for pseudobulbar affect, characterized by sudden and uncontrollable episodes of laughing or crying. Pseudobulbar affect is not uncommon in ALS, but the drug has never been tested specifically in ALS. “This is the first trial to be directed to palliative treatment for bulbar ALS,” noted lead researcher Richard Smith, MD.
Mexiletine: This trial will test the safety and tolerability of mexiletine, thought to have potential neuroprotective effects, based on lab studies. It is being led by Michael Weiss, MD.
TDI-132/Gilenya: This is an immune system-modifying drug, used in multiple sclerosis. While previous widespread immune-suppressing agents have failed in ALS, “we know there is inflammation in ALS,” said James Berry, MD, principal investigator for the study. TDI-132 is a more specific immunomodulator. It will first be tested for safety and tolerability in ALS. If results are positive, a larger efficacy trial is possible.
Other trials are studying the effects of exercise, nutrition, and stem cells, and examining biomarkers to speed future trials. Full details of all NEALS trials can be found here: http://alsconsortium.org/trials.php
“It has been a really remarkable year,” Dr. Cudkowicz said. The breadth and quality of the multiple active trials indicate not only the commitment of investigators, but the entire ALS community, especially patients and their families.