Work at the Healey Center for ALS is gaining attention around the globe. Our outstanding faculty were invited to speak at fourteen conferences across the globe about our research, our organizational approach and on our efforts to bring patients, families, communities and scientists together to focus on solving one problem – ALS.
Dr. Cudkowicz was awarded the American Neurological Association’s 2019 Raymond D. Adams Lectureship for her presentation entitled “Accelerating ALS Therapeutics: Master Platform Trials and Other Approaches.”
Established in 2000 to honor Dr. Raymond D. Adams, emeritus Bullard Professor of Neuropathy at Harvard Medical School and emeritus Chief of the Neurology Service at Massachusetts General Hospital, Dr. Cudkowicz’s lecture was presented at the 144th annual meeting of the American Neurological Association during the Optimizing Clinical Trials Symposium on Oct. 15.
“A Powerful Promise” Keynote Speech at Press/Ganey Conference
Dr. Cudkowicz was also selected as a keynote speaker at Press/Ganey’s National Client and Executive Leadership Conference Nov. 18-20 in Orlando, Florida. Press/Ganey is a nationally respected consulting company that focuses on designing tools to improve health care performance, including safety, clinical excellence, patient experience and workforce engagement. Dr. Cudkowicz will talk about the importance of the physician-patient relationship when experiencing a diagnosis of ALS. After her speech, Dr. Cudkowicz will join Thomas H. Lee, MD, Press Ganey chief medical officer, an internist/cardiologist at Brigham and Women's Hospital, for a question-and-answer session about what it takes to be a highly motivated physician and how organizations can support them. Dr. Lee is the author of the new book The Good Doctor that explores resiliency among highly effective mid-career physicians, including a chapter that shares Dr. Cudkowicz’s story.
Connecting in Australia
Sabrina Paganoni, MD, PhD, who works closely with Dr. Cudkowicz to lead the HEALEY ALS Platform Trial, will present a plenary talk with statistical expert Ben Saville, PhD, from Berry Consultants. The talk entitled “The ALS Platform Trial: Design Considerations and Statistical Efficiencies,” will take place on Dec. 4 in Perth, Australia, at the 30th International Symposium on ALS/MND, hosted by the Motor Neuron Disease Association.
At that symposium, the first annual Healey Award for Innovation will be announced. This award will be the first to recognize exceptional achievements for an individual or team in scientific advances in ALS from around the world.
Scientific Impact Award
Dr. Paganoni has also been awarded the American Association of Neuromuscular & Electrodiagnostic Medicine Scientific Impact Award, which celebrates a midcareer member of the AANEM for serving as a first author, second author or senior author on a published paper in a national or international journal within the last two years. Dr. Paganoni received this year’s award for her second authorship on a December 2017 article in Muscle & nerve titled “Phase IIa Trial of Fingolimod for Amyotrophic Lateral Sclerosis Demonstrates Acceptable Acute Safety and Tolerability.” The title of the article only tells part of the story. Dr. Paganoni’s drive to study ALS began with her mentor, physician scientist Dr. Lisa Krivickas, who worked with Dr. Paganoni even while she was afflicted with ALS. She inspired a mentee with a strong desire to help others conquer ALS.
Networking with Other Disease Groups
The success and speed of launch of the HEALEY ALS Platform Trial has not gone unnoticed! Members of the Healey Center have been invited to share their experience with several other disease groups who want to also start platform trials.
Alzheimer’s Disease Meeting: Dr. Paganoni was invited to give a talk about “The use of platform trials in Central Nervous System disorders” at the 20th International Conference on Alzheimer’s Drug Discovery on September 17, in Jersey City, NJ. Dr. Paganoni shared the scientific, operational and statistical efficiencies of the platform approach and how platform trials could accelerate the development of effective treatments for other neurologic diseases, including Alzheimer’s.
Duchenne Platform Trial Stakeholder Meeting: Marianne Chase, Director of Research Operations at the Healey Center, participated in a discussion of governance of the Platform Trial approach hosted by the Parent Project Muscular Dystrophy on September 9, in Silver Spring, Maryland. Ms. Chase joined a conversation with parents of patients, pediatricians and others within the Duchenne Muscular Dystrophy community. As the Duchenne scientific community prepares a Master Trial Platform for that disease, they are eager to include the voices of parents of children with this disease. Ms. Chase was able to share the process the Healey Center undertook in preparation for the Healey ALS Platform Trial.
Make a Difference for the Healey Center
Join our quest to discover life-saving therapies for individuals who are affected by ALS. The key to our success is our dedication to collaboration. At the Sean M. Healey and AMG Center for ALS at Mass General, we partner with the best clinician-scientists, our patients and friends like you to turn laboratory successes into promising therapies.