Sean M. Healey & AMG Center for ALS Announces Completion of Enrollment for First Healey ALS MyMatch Trial

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). The ACACIA Trial is a biomarker-driven, Phase 2a open label trial of oral digoxin, a repurposed experimental medication in ALS. Over a four month enrollment period, 41 participants started study medication at three clinical trial sites.

ALS MyMatch is an ongoing series of early phase (1b/2a) clinical trials that are designed to address a critical gap in ALS research: the need for early-phase studies that identify and define target populations, determine best dose and demonstrate target engagement. This method personalizes treatment. The program utilizes genetic, biofluid, imaging and other biomarkers to match participants to the right experimental treatment trials for them. The program includes measuring cerebrospinal fluid (CSF) levels and determining central nervous system (CNS) penetration. Trials are designed to generate clear data under efficient trial processes and rapid enrollment conditions. Supported by strong preclinical science for digoxin and future candidates, the trials also de-risk and guide decisions for later-phase trials (2b/3), including determining optimal dosage and identifying target populations.

“ALS MyMatch is designed to increase efficiency by accelerating trial startup and shortening the time for participants’ to screen and enter a trial,” says Merit Cudkowicz, MD, Director of the Healey & AMG Center and Executive Director of the Mass General Brigham Neuroscience Institute. “All the innovative initiatives to accelerate treatment development from the Network of Excellence for ALS (NEALS) are part of ALS MyMatch, including use of a single Institutional Review Board (sIRB), highly trained research sites, and a patient advisory committee, leading to rapid enrollment and high-quality data and operations.”

Each trial in ALS MyMatch is named after a tree to symbolize our striving for hope and the deep scientific roots from which the trial stems. The ACACIA Trial specifically evaluates digoxin. The trial is guided by results from several bench-to-bedside translational experiments using TDP-43 and C9ORF72 disease-relevant ALS models, led by Brian Wainger, MD, PhD, Associate Professor of Neurology and Anesthesiology at Harvard Medical School, Alexander Healey Endowed Chair in ALS, and Co-Principal Investigator of the ACACIA Trial, and his research group.

Digoxin is a medication traditionally used to treat heart conditions including heart failure and irregular heartbeats. Recent research from the Wainger Lab suggests that among thousands of drugs screened in preclinical ALS models using neurons made from induced pluripotent stem cell (iPSCs) lines, digoxin reduced ALS features in the iPSC-derived neurons as well as in mouse ALS models. The benefit of digoxin in people living with ALS remains unknown and is being evaluated as a next step in the ACACIA trial.

“The initiation of Healey ALS MyMatch – ACACIA Trial evaluating digoxin is an important step forward in our efforts to deepen our understanding of the mechanisms that drive ALS and how digoxin may influence these disease processes,” says Dr. Brian Wainger.

“Enrolling the first participant in ACACIA Trial in three months from the launch of the ALS MyMatch program is a testament to the dedication of the team and our urgency to bring trials to the ALS community,” says Suma Babu, MBBS, MPH, Principal Investigator of the ALS MyMatch program and the ACACIA trial and Co-Director of the Neurological Clinical Research Institute (NCRI) at MGH. “We are thankful for all participants and their families, donors, staff at study centers and collaborators who have dedicated their time to this trial.”

ALS MyMatch is a multi-site initiative that brings together high enrolling ALS research centers and is a NEALS Consortium affiliated program. Research centers for the ACACIA trial include MGH in Boston; Northwestern University in Evanston, Ill.; and Nova Southeastern University in Fort Lauderdale, Fla. ALS MyMatch has partnered with the Acceleration Centers of Enrollment (ACE) program, a community-driven philanthropic partnership program focused on expediting start up and recruitment at study centers. Additional ACE sites will be activated as new ALS MyMatch trials launch.

For more information about the Healey ALS MyMatch program and the ACACIA Trial, please visit: massgeneral.org/neurology/als/research/healey-mymatch

About the Sean M. Healey & AMG Center for ALS at Mass General

At the Sean M. Healey & AMG Center for ALS at Mass General, we are committed to bringing together a global network of scientists, physicians, nurses, foundations, federal agencies, and people living with ALS, their loved ones, and caregivers to accelerate the pace of ALS therapy discovery and development.

Launched in November 2018, the Healey & AMG Center, under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, is reimagining how to develop and test the most promising therapies to treat the disease, identify cures and ultimately prevent it.

With many clinical trials and lab-based research studies in progress right now, we are ushering in a new phase of ALS treatment and care. Together, we will find the cures.

About the Neurological Clinical Research Institute

The Neurological Clinical Research Institute (NCRI) at Mass General is an academic research organization composed of innovative researchers experienced and passionate about designing, developing, facilitating, and conducting multicenter clinical trials in neurological diseases. Our mission is to accelerate translational research in neurological disorders by initiating clinical development of novel therapies and leading trials of these compounds. We strive to be at the cutting edge of innovative trial design to accelerate therapy development by creating new trial methodology, discovering novel biomarkers and refining outcome measures.