How the Healey ALS Platform Trial is Helping to Accelerate the Pathway to New ALS Treatments
Sabrina Paganoni, MD, PhD, speaks about the HEALEY ALS Platform Trial, a novel approach to testing new therapeutics for ALS patients.
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Dr. Merit Cudkowicz is the Director of the Sean M. Healey & AMG Center for ALS, Chief of Neurology at Mass General, Director and the Julieanne Dorn Professor of Neurology at Harvard Medical School. Dr. Cudkowicz’s research and clinical activities are dedicated to the study and treatment of people with Amyotrophic Lateral Sclerosis (ALS). Dr. Cudkowicz is one of the founders and past Co-Chairs of the Northeast ALS Consortium (NEALS), a group of over 134 clinical sites in the United States, Canada, Europe and the Middle East dedicated to performing collaborative clinical trials and research in ALS. She has brought innovations to accelerate the development of treatments for people with ALS, including senior role in first antisense oligonucleotide treatment for a neurological disorder (SOD1 ALS), adaptive trial designs, central IRB - all with goal to bring the best treatments rapidly. Dr. Cudkowicz is Principal Investigator of the Clinical Coordination Center for the National Institute of Neurological Disorders and Stroke’s Neurology Network of Excellence in Clinical Trials (NeuroNEXT). Dr. Cudkowicz is launching the first platform trial initiative in ALS, the HEALEY ALS Platform Trial, a program that will greatly accelerate therapy development in ALS.
Dr. Cudkowicz received the American Academy of Neurology 2009 Sheila Essay ALS award, the 2017 Forbes Norris Award from the International MND Alliance, the 2017 Pinnacle Award from the Boston Chamber of Commerce and the 2019 Ray Adams American Neurological Association Award. A dedicated educator, Dr. Cudkowicz mentors many young neurologists in clinical investigation of ALS and related neurodegenerative disorders. Dr. Cudkowicz completed her undergraduate degree in chemical engineering at Massachusetts Institute of Technology and obtained a medical degree in the Health Science and Technology program of Harvard Medical School. She served her internship at Beth Israel Hospital in New York and her neurology residency and fellowship at MGH. She also obtained a master’s degree in Clinical Epidemiology from the Harvard School of Public Health.
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Mass General Neurology
55 Fruit St.
Boston, MA 02114
Phone: 855-644-6387
Sean M. Healey and AMG Center for ALS
55 Fruit Street
Wang Ambulatory Care Center
8th Floor
Boston, MA 02114
Phone: 617-724-3914
Mass General Neurology: Neurological Clinical Research Institute
165 Cambridge St.
6th Floor
Boston, MA 02114
Phone: 617-726-5732
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My research activities are dedicated to the study and treatment of patients with Amyotrophic Lateral Sclerosis (ALS) and other neurological disorders. As Director of the MGH Neurological Clinical Research Institute and the Principal Investigator for the NINDS supported NeuroNEXT Clinical Coordinating center (www.neuronext.org), I design clinical trials to evaluate the safety and effectiveness of therapies that have shown promise in preclinical studies. In ALS, I co-founded an international consortium to test novel therapies in ALS, the Northeast ALS (NEALS) consortium. The NEALS consortium consists of over 128 clinical sites in the United States and Canada dedicated to performing joint academic led clinical trials in ALS.. We are leading several new trials in ALS. There is a critical need in neurology for highly trained clinical investigators who work collaboratively with basic scientists to develop new therapeutic strategies.
In 2018 we launched the Sean M. Healey & AMG Center for ALS at Mass General. This new Center is dedicated to accelerating therapy development and access to treatments for people with ALS. We are launching the first Platform Trial initiative in ALS where several treatments will be tested in one platform approach. This groundbreaking initiative will cut in half the time to develop treatments, increase by 67% ability of people to receive active treatment and cut costs of drug development by 33%. This approach, already successful in oncology, will revolutionize how treatments are developed in ALS. The Healey Center bring collaborators from around the world to work together to find treatments for people with ALS.
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Mass General is recognized as a top hospital on the U.S. News Best Hospitals Honor Roll for 2024-2025.
Listen to an interview with Dr. Cudkowicz on Charged, a podcast devoted to stories of the relentless daily pursuit of excellence at Mass General.
Sabrina Paganoni, MD, PhD, speaks about the HEALEY ALS Platform Trial, a novel approach to testing new therapeutics for ALS patients.
The Healey & AMG Center has announced the latest updates regarding Regimen E of the HEALEY ALS Platform Trial evaluating Trehalose.
Episode #14 of the Charged Podcast.
The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital awarded the inaugural annual Sean M. Healey International Prize for Innovation in ALS to the team that brought to trial the first antisense oligonucleotide (ASO) therapy for ALS.
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The HEALEY ALS Platform Trial is designed to evaluate multiple investigational products simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS.