Young investigators receive MGH awards for ALS research
Scholars study genetic abnormalities, nerve cells
Three researchers at Massachusetts General Hospital (MGH) have received 2025 MGH ALS Young Investigator Awards for their research on genetic abnormalities, faulty energy systems, and why some nerve cells are more susceptible to damage than others in amyotrophic lateral sclerosis (ALS).
The awards announced by the Sean M. Healey & AMG Center for ALS at MGH in Boston recognize early-career investigators making major contributions to understanding how ALS develops and how it can be better diagnosed and treated.
Two new scholars — Yuyu Song, MD, PhD and Seung Hun Park, PhD — join four others as 2025–2027 Mussallem Transformative Scholars in ALS. Each receives a two-year, $150,000 scholarship from the Linda and Mike Mussallem Foundation.
Song, an assistant professor in MGH’s neurology department, is focused on the concept of selective vulnerability, the question of why certain types of nerve cells are more affected than others in ALS and frontotemporal dementia (FTD), a related disease that often occurs alongside ALS.
ALS results from the loss of motor neurons, the nerve cells that control voluntary muscle movements, in the brain and spinal cord. In FTD, neurons are lost mainly in two regions of the brain called the frontal and temporal lobes, leading to problems with behavior and language.
ALS research focuses on cells, molecules
Song will compare the molecular and cellular mechanisms involved in the loss of motor versus frontotemporal neurons using both postmortem tissue and lab-grown induced pluripotent stem cells derived from patients. She’ll test medications called kinase inhibitors to see if they can repair damaged nerve cells.
In addition to exploring new treatment strategies based on the mechanisms underlying ALS and FTD, Song will develop a screening platform to test medications in the lab before they’re prescribed. The goal is to identify which treatments may work best for each patient.
Park, an instructor at MGH’s Gordon Center for Medical Imaging, is developing new imaging tools to monitor mitochondria, energy-producing structures that often malfunction in ALS. When mitochondria are faulty, they produce harmful molecules that damage nerve cells.
ALS04, a small molecule designed to target mitochondria, lights up under infrared imaging, enabling real-time monitoring of the early changes thought to drive disease. Park will also study whether ALS04 can reduce stress and keep mitochondria functioning, which could slow the progression of ALS.
Continuing their work as second-year Mussallem Scholars are Jin Cai, PhD; Aaron Held, PhD; Christine Marques, PhD; and Tiziana Petrozziello, PhD. Cai is studying how nerve cells communicate, while Held is looking in detail into TDP-43, a protein that forms toxic clumps in ALS. Marques is testing a group of compounds, including one that’s already approved in the U.S., in mouse models of ALS. Petrozziello is exploring how to improve connections between nerve and muscle cells by targeting another protein called tau.
Another MGH ALS Young Investigator Award, the 2025 Byrne Family and Judith & Jean Pape Adams Endowed Fellowship, went to Adel Boudi, PhD, who’s exploring the role of gene fusions — when two genes combine inappropriately — in ALS.
His work will focus on a specific gene fusion called FAM69A–EVI5, which has been found in postmortem tissue from the brains and spinal cords of ALS patients. Boudi will use lab-grown motor neurons to find out whether FAM69A–EVI5 drives ALS. The findings could lay the groundwork for future studies on disease mechanisms and treatment strategies.
The Healey & AMG Center has a global network of researchers, doctors, patients, and advocates working to speed treatment development. Among the center’s clinical efforts is HEALEY ALS (NCT04297683), a trial testing multiple candidate treatments simultaneously against a shared placebo group to shorten development timelines.
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