Immune system dysregulation and elevated inflammation are key factors in the development of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, a fatal neurodegenerative condition.
In a recent study published in The FASEB Journal, researchers discovered that repeated infusions of specific immune cells have proven to delay the onset of ALS, extend survival in mice, and reduce inflammation markers in a patient with the disease. The study was conducted by researchers at Massachusetts General Hospital, part of the Mass General Brigham healthcare network.
“This marks the initial step towards a phase I clinical trial for our novel cell therapy for ALS, which is currently in the planning phase,” explained Dr. Mark C. Poznansky, the senior author of the study. Dr. Poznansky is the Director of the Vaccine and Immunotherapy Center, an attending physician in General and Transplant Infectious Diseases Medicine at Massachusetts General Hospital, and a professor of Medicine at Harvard Medical School.
Prior work by Dr. Ruxandra F. Sîrbulescu, an assistant professor of Neurology and co-corresponding author of the article, demonstrated that the direct application of purified B cells, a type of immune cell responsible for producing antibodies, can reduce inflammation and aid in recovery in mice with skin or brain injuries.
This beneficial effect is achieved through a process known as pligodraxis, in which the B cells acquire immunoregulatory and neuroprotective properties to facilitate tissue repair in an injured setting.
“Our initial studies revealed the remarkable impact of B cells on brain lesions – these cells not only protected brain structure and function but also prompted us to consider their application as a therapy for neurodegenerative disorders,” stated Dr. Sîrbulescu.
In the latest research, the team evaluated the impact of B cell infusions in ALS-prone mice and in a human ALS patient.
In mice: Repeated intravenous administration of B cells from donor mice significantly delayed disease onset, prolonged survival, reduced cell death, and lowered a neurodegeneration marker.
In the human patient: Repeated infusions of donor B cells were deemed safe and resulted in decreased levels of various inflammatory markers.
“Our findings demonstrate that B cells, easily obtainable from blood, can effectively treat ALS in a well-established mouse model. With approval from the FDA and our hospital, we proceeded to test this treatment on an ALS patient,” said Dr. Poznansky.
“Our research confirmed the safety and efficacy of B cell therapy in mice, and we successfully translated this approach to humans, marking a significant breakthrough in the treatment of ALS, a disease that currently lacks a cure,” added Dr. Poznansky.
