Sabrina Paganoni, MD, PhDSabrina Paganoni, MD, PhD

Peer-reviewed results of a trial led by the Healey Center’s Dr. Sabrina Paganoni, will be published soon. The CENTAUR trial, highlighted in a Wall Street Journal article, tested a drug that showed promise in slowing disease progression in individuals with ALS.

The CENTAUR trial, led by Amylyx Pharmaceuticals began as an undergraduate project in 2013 by two students from Brown University: Josh Cohen, who studied biomedical engineering, and Justin Klee, who studied neuroscience. After college and post-graduate research, the pair launched Amylyx Pharmaceuticals to study novel approaches to neurodegenerative diseases and turned to Healey Center Director Dr. Cudkowicz and Mass General’s expertise in ALS research and patient care for advice.

“These two scientists took a novel approach to the problem of nerve cell death,” Dr. Cudkowicz said, “and with guidance from our team, and under the direction of Principal Investigator Dr. Sabrina Paganoni, moved the clinical trial forward quickly and carefully.”

AMX0035 is an investigational product  designed to tackle the loss of neurons in the motor cortex and spinal cord (which results in the loss of movement control) and the brain’s immune system overreaction of killing more neurons, causing a toxic cycle that speeds the course of the disease. AMX0035 also improves cellular metabolism, helping them continue to function normally in the face of disease.

“The results from this clinical trial are very encouraging,” said Dr. Paganoni. “Patients on AMX0035 demonstrated a statistically significant slowing of ALS disease progression, as measured by the ALS Functional Rating Scale.

Thanks to the tireless efforts of researchers, clinicians and clinical trial participants, the Healey Center and our partners are moving ever closer to the cure.