An experimental drug has been shown to alleviate symptoms of Huntington's disease and extend lifespan in mouse models, according to a Jun. 29 study. Further studies are required to determine whether these results may also apply to humans.
Huntington's disease is a hereditary disorder that has so far been considered incurable, with clinical manifestations including impaired motor control and psychiatric symptoms. The new study indicates that the drug candidate anle138b can significantly reduce the toxic protein clumps in the brain characteristic of the disease.
Mice treated with anle138b retained their mobility for longer periods, experienced less brain shrinkage, and lived longer than untreated mice. The compound not only alleviated symptoms but also addressed the underlying cause by preventing harmful protein clumps from destroying nerve cells and their connections. These findings were confirmed through experiments using human stem cells derived from Huntington's patients.
The research was led by Professor Irina Dudanova at the University of Würzburg, along with doctoral student Miguel da Silva Padilha. The substance was developed by teams at the Max Planck Institute of Multidisciplinary Sciences in Göttingen and Ludwig-Maximilians University in Munich, now at MODAG GmbH. Additional participants included researchers from the Max Planck Institute of Biological Intelligence and the University of Cologne.
"Our data show that specifically targeting toxic protein aggregates with the compound anle138b is a promising approach for stabilizing neuronal health in the long term," said Irina Dudanova regarding the study's findings.
A key milestone highlighted by researchers was confirmation of these effects in human cells: "In our experiments with induced pluripotent stem cells — that is, precursor cells derived from Huntington patients' cells — we also observed that the addition of anle138b reduced the amount of huntingtin aggregates," said Dudanova.
