There’s new hope for patients.
Huntington’s disease is a devastating, genetic neurological disorder that leads to the progressive loss of motor skills and cognitive functions. As the disease advances, it causes patients to lose the ability to speak, walk, swallow, and focus, and it often proves fatal within two decades of symptom onset. Currently, there is no cure for Huntington’s disease, but a promising new study conducted in mice offers a potential breakthrough.
The disease is caused by mutations in specific genes that result in harmful protein clumping within brain cells. These clumps disrupt brain function and lead to neuronal death. In an effort to develop a treatment, researchers are exploring the use of “peptide-brush polymers.” These peptides are naturally occurring proteins that can potentially prevent the damaging clumping of proteins associated with Huntington’s disease.
In mouse studies, this polymer-based treatment showed promising results by “rescued” damaged brain cells and even reversing some of the symptoms of Huntington’s disease. The research team, including scientists from Northwestern University and Case Western Reserve University, observed significant improvements in brain health and behavior in the treated mice, though they cautioned that results in mice do not always translate to humans.
Nathan Gianneschi, a professor of chemistry at Northwestern University and one of the study’s co-lead authors, expressed his personal investment in the research, noting that a childhood friend was diagnosed with Huntington’s at a young age. The study, published on November 1 in Science Advances, builds on earlier work by Xin Qi’s team at Case Western, who identified a key protein that causes protein clumping in Huntington’s disease. Qi’s team also discovered a peptide that can potentially stop this harmful clumping by interfering with the protein-protein interactions that lead to cellular damage.
The polymer developed by Gianneschi’s lab allowed the peptide to cross the blood-brain barrier and target the affected brain cells. In mouse experiments, the polymer successfully entered the brain and prevented mitochondrial damage—protecting the brain cells. The treated mice exhibited more normal behavior, such as exploring their environment, which they had previously avoided due to the effects of the disease. The polymer treatment also showed no signs of toxicity, an encouraging sign for future human trials.
The next phase of this research will focus on refining the polymer for potential human testing, with hopes that it could not only treat Huntington’s disease but also provide a therapeutic approach for other neurodegenerative conditions.
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