Members of the Northeast Amyotrophic Lateral Sclerosis Consortium assembled at the Opal Sands Resort, in Clearwater, Florida on October 2nd through October 4th for their 18th Annual Scientific Symposium with a record-breaking attendance of over 500 participants. The key word resounding in the Opal Ballroom was “excitement” – excitement about the upcoming first ALS Platform Trial that will accelerate therapy development and increase access for people with ALS. The Platform Trial was highlighted by three key NEALS meetings.
At the first meeting, the Platform Trial Coordinator Session, experienced and new research coordinators from 54 NEALS sites gathered into a well-packed room. The focus of the session was on the details of how to run one trial with three drugs and what it will look like at a site level. Ideas bounced around the room from all the dedicated, experienced ALS nurses and coordinators on how to ensure success. Everyone was eager to help their patients, each other and to actively participate!
The excitement about the HEALEY ALS Platform Trial continued with a Platform Trial Study Meeting where representatives from the three companies, Simon Read, PhD, Chief Scientific Officer of Ra Pharmaceuticals, Inc., Mary Donohue, Senior Director of Clinical Operations of Biohaven and Robert Glanzman, MD, FAAN, Chief Medical Officer of Clene Nanomedicine spoke about their companies’ investigational therapies for ALS and participation in the first platform trial in ALS. All were excited and honored to be involved in this amazing breakthrough in how therapies are developed for people with ALS.
Dr. Merit Cudkowicz, the Chief of Neurology at Massachusetts General Hospital, followed suit introducing the HEALEY ALS Platform Trial to the full NEALS audience during the General Session Meeting. “We are so excited to bring this new concept to the ALS field. The HEALEY ALS Platform trial will cut down time to developing an effective treatment by 50% and decrease costs by a third. Most importantly, many more people receive active treatment. The first three companies all agreed to provide open label access to the participants at the end of the main trial. The adaptive nature of the HEALEY Platform trial is crucial to speeding up therapy development. The inclusion of novel biomarkers and the ability to share learnings will further improve treatments are developed.”