Scientists at Northwestern and Case Western Reserve universities have introduced a polymer-based therapeutic for Huntington’s disease, marking a significant development in the treatment of this debilitating condition. Huntington's disease, an incurable illness, causes nerve cells to deteriorate in the brain due to a genetic mutation that leads proteins to misfold and aggregate.
The newly developed treatment utilizes peptide-brush polymers to prevent these protein aggregates. In animal studies, the treatment reversed symptoms by rescuing neurons and showed no significant side effects, indicating its non-toxic nature.
The researchers propose that with further testing, this therapy could potentially be administered as a weekly injection to delay or reduce symptoms in patients carrying the genetic mutation. The findings were published on November 1st in the journal Science Advances.
“Huntington’s is a horrific, insidious disease,” said Nathan Gianneschi from Northwestern University, who led the development of the polymer therapeutic. He emphasized that there is currently no cure or effective treatment for stopping or reversing the disease.
Gianneschi holds multiple positions at Northwestern University and co-led the study with Xin Qi from Case Western Reserve University. Their work builds on previous research by Qi’s team which identified a peptide capable of disrupting harmful protein interactions in Huntington's disease.
Despite initial challenges with peptides being easily broken down by enzymes and struggling to enter cells effectively, Gianneschi's team created a biocompatible polymer structure that overcomes these issues. This structure not only protects peptides but also aids their entry into cells across the blood-brain barrier.
In laboratory experiments using mice models of Huntington’s disease, this new polymer remained active significantly longer than traditional peptides. It successfully prevented mitochondrial damage and improved brain cell health, resulting in extended lifespans for treated mice who exhibited behavior similar to healthy mice.
Gianneschi plans to optimize this polymer further and explore its potential application in other neurodegenerative diseases. He remains motivated both personally and scientifically due to his connection with a childhood friend diagnosed with Huntington’s at age 18.
The study received support from various institutions including the International Institute of Nanotechnology Convergence Science Medicine Institute grant and several National Institutes of Health awards.