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“The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) is proud to announce completion of enrollment for the first Healey ALS MyMatch trial, the ACACIA Trial(ClinicalTrials.gov identifier: NCT07047209).”
Based on translational research from the Wainger Lab, the ACACIA Trial is evaluating digoxin as a potential treatment for amyotrophic lateral sclerosis (ALS). In preclinical studies, digoxin, an established medication used to treat several heart conditions, reduced disease-related features in induced pluripotent stem cell (iPSC)-derived neurons and demonstrated beneficial effects in mouse models of ALS. The ACACIA Trial will determine whether these promising preclinical findings translate into clinical benefit for people living with ALS.
The Wainger Lab is excited to work with support of the LifeArc organization, a non-profit working to advance therapeutics for rare diseases. Together, we are investigating whether the well-known heart medication, digoxin, can be used to protect neurons and slow progression of ALS.
Congratulations to Dr. Wainger and the Wainger Lab on being the recipient of a 2025 Spark Award from the Gene and Cell Therapy Institute at Mass General Brigham. Dr. Wainger presented “Potassium Channel Gene Therapy for Pain” at the GCTI Symposium in December 2025.
Dr. Aaron Held presented at the International Symposium on ALS/MND in San Diego, December 2025: AP21A and the autophagy pathway modify TDP-43 toxicity. Congratulations Aaron!
Congratulations to Instructor Christine Marques, Ph.D. on her recent presentation at the 2025 NEALS meeting in Clearwater FL: Phenotypic Drug Screening in Human iPSC-Derived Neurons Identifies Modifiers of Dipeptide Repeat-Induced Toxicity and TDP-43 Pathology in Amyotrophic Lateral Sclerosis/Frontotemporal Dementia
Happy Holidays from the Wainger Lab! We showed off our artistic side at the Clay Room in Brookline where we painted our own pottery while eating sushi. Cheers to a happy, healthy, and successful 2025!
The Wainger Lab is warmly welcomes a new group of research assistants to the lab this Fall. Advika, Abegale, and Laura are working on clinical research projects and Isabel, Chris, Sam, Victoria, and Sophia are in lab working on preclinical projects.
Congratulations to the research assistants who moved on this year to med school and grad school and have joined the ranks of lab alumni. We will miss you Lauren, Adina, Sid, Martha, and Sharon!
Congratulations to Aaron and Christine who both received the 2024 Mussallem Transformative Scholars Award from the Sean M. Healey & AMG Center for ALS at MGH for their work in the field of ALS research.
Congratulations to Aaron, Christine, and Grace for their first-author papers this past year!
Held A, Adler M, Marques C, Reyes CJ, Kavuturu AS, Quadros ARAA, Ndayambaje IS, Lara E, Ward M, Lagier-Tourenne C, Wainger BJ. iPSC motor neurons, but not other derived cell types, capture gene expression changes in postmortem sporadic ALS motor neurons. Cell Rep. 2023 Sep 26;42(9):113046. PMID: 37651231; PMCID: PMC10622181.
Marques C, Held A, Dorfman K, Sung J, Song C, Kavuturu AS, Aguilar C, Russo T, Oakley DH, Albers MW, Hyman BT, Petrucelli L, Lagier-Tourenne C, Wainger BJ. Neuronal STING activation in amyotrophic lateral sclerosis and frontotemporal dementia. Acta Neuropathol. 2024 Mar 13;147(1):56. PMID: 38478117; PMCID: PMC10937762.
Chahyadinata G, Nam JH, Battenberg A, Wainger BJ. Physiological profiling of cannabidiol reveals profound inhibition of sensory neurons. Pain. 2024 May 29. PMID: 38815194.
We are sending our best wishes to Dan as he begins his new position in the Genomic Medicine Unit at Sanofi. He is working on developing new viral and non-viral gene therapies for peripheral neuropathies. We will miss him greatly!
We’re adding two more to the list of Wainger Lab alumni! Katherine is off to begin her journey of becoming a physician-scientist in the MSTP program at the Perelman School of Medicine in Philadelphia, while Joao has moved on to investigate Alzheimer’s Disease at Tufts Medical School. We will miss you both greatly and will forever appreciate the incredible contributions you have made to the group!
We’ve had to say goodbye to two wonderful lab members over the last few weeks. We wish the best to Xiaofan as she returns to her position as an anesthesiologist in China and to Patrick as he begins with Eli Lilly in the pain and neurodegenerative disease sectors. We appreciate everything you have done for the lab and look forward to seeing where you go!
Congratulations to Brian on receiving a Blavatnik Sensory Disorders Research Award for the project entitled A Neuronal Electrical Excitability Recording System for Improved Diagnosis and Biomarker Assessment of the Therapeutic Impact of Drugs in Peripheral Sensory and Pain Disorders. Check out what the foundation does here.
Congratulations to Brenda for beginning the UC Berkeley-UCSF Joint Medical Program (MS/MD). We’re excited to see where you go!
Brian gives the John McNeish Memorial Lecture: Human Stem Cell Modeling to Clinical Trial at the virtual International Society for Stem Cell Research’s annual meeting. Check it out here.
Congratulations to Aaron for receiving the Ruth L. Kirschtein Postdoctoral Individual National Research Service Award from the NIH!