Monoclonal Antibodies As A Future Therapeutic Against Neurodegenerative Diseases

Monoclonal antibodies developed by a team of scientists at the NYU Langone Medical Center’s Center for Cognitive Neurology may be a stepping-stone towards potential new treatments against neurodegenerative diseases, including Alzheimer’s and Parkinson’s disease.

The majority of neurodegenerative diseases, such as Alzheimer’s, Lewy Body and other dementias, Parkinson’s and prion diseases share a triggering disease mechanism – a change in a particular protein shape and loss of function, leading to protein aggregates in the brain and the formation of highly toxic plaques of misfolded proteins.

The team of scientists led by Fernando Goni, PhD, an adjunct associate professor of Neurology, and Thomas Wisniewski MD, director of the Center for Cognitive Neurology at NYU Langone, developed a novel class of monoclonal antibodies that can effectively target the misfolded proteins (which are prone to bind other misfolded proteins and form toxic protein aggregates) underlying Alzheimer’s disease, i.e., abnormal beta-amyloid and tau proteins. The team was also successful in targeting abnormal proteins associated with Parkinson’s development.

These findings suggest that monoclonal antibodies targeting misfolded proteins in soluble, aggregated states are a potential new treatment for neurodegenerative diseases. (Of note, monoclonal antibodies are produced by a single type of cell and all share the same activity).

Fernando Goni, PhD, study co-lead author commented, “There is a commonality underlying the misfolding in many neurodegenerative diseases and we are targeting it. We are confident this is the right strategy and our monoclonals are showing they are up to the task. There is potential for specific therapeutic agents for neurodegenerative diseases.”

Dr. Wisniewski, the Lulu P. and David J. Levidow Professor of Neurology and a professor of Pathology and Psychiatry and also study co-lead author added, “We have been developing this strategy for many years, and now we have results. Other labs are trying similar strategies. The importance of this concept is being increasingly recognized.”

These promising results were presented during this years’ Alzheimer’s Association International Conference, held in Washington, D.C. They were built upon previous studies where the team hypothesized that an early targeting of these misfolded proteins could prevent protein aggregates’ formation, blocking the progression of neurodegenerative diseases, such as Alzheimer’s and Parkinson’s. The team is continuing their studies in animal models where they will combine monoclonal antibodies with other therapeutic strategies, in the hopes to initiate future clinical trials to test their findings.