Huntington’s Disease Discovery Opens Door to a New Class of Treatments

A new study by researchers at Lawrence Berkeley National Laboratory has discovered a novel factor driving neurodegeneration in Huntington's disease (HD), a severe inherited condition. Huntington's disease is caused by a mutated gene that gains more repeats of a specific nucleotide sequence as the disease progresses, leading to the death of brain cells in adulthood.

While prior research has shown that the excess repeats contribute to cell death, the study published in Nature Communications reveals that increased DNA breaks throughout the genome also play a significant role.

The researchers found that individuals with HD exhibit a marked increase in breaks in DNA strands across the genome. They demonstrated that administering an antioxidant treatment can suppress these breaks and rescue mice from neuron damage and disease symptoms. This finding suggests that targeting DNA damage with antioxidants, rather than focusing on altering the mutated gene or stopping its expansion, could open a new avenue for treating Huntington's disease.

The study's lead author, Aris Polyzos, emphasized that while there is no cure for Huntington's disease, this discovery provides a promising new direction for potential treatments. Cynthia McMurray, a co-author and senior researcher, highlighted that this approach could be simpler and more accessible than previous methods, which have struggled to produce effective therapies.

The team plans to further investigate whether these findings apply to human cells and explore the possibility of translating this discovery into clinical trials.