There appears to be a ray of hope for patients with Huntington’s disease, which is a rare inherited genetic disorder, characterized by un-coordinated body movements and decreased mental ability. Researchers at Mass General Institute for Neuro-degererative Disorders (MIND) claim to have developed a molecule, which has been proven to slow down disease progression.
According to the scientists, the molecule C2-8 was tested on mice and appeared to delay the degenerative process of Huntington’s disease. Researcher Steven Hersch has stated that although the course of action and the bio-chemical interaction of the molecule with body cells still remain unknown, it is speculated that the molecule is likely to have similar effects in humans as well. The molecule or its derivatives still remain to be converted into a drug form.
Aleksey Kazantsev, was the first scientist to propose C2-8 as a potential therapeutic drug considering its unique ability to ‘ability to block the aggregation of the mutant huntingtin protein in yeast and animal tissue and to improve function in a fruit fly model’. Subsequent research also established that this molecule did not have any toxic side effects on the mouse-model and possessed the ability to penetrate the blood-brain barrier.
Although mice treated with C2-8 molecule seemed to fare better in terms of strength, endurance and stability, the molecule did not appear to prolong their life spans as compared to untreated mice.
This animal study is an important milestone in the treatment of HD and taking the research further would definitely yield promising results.
via biologynews.net
Image source: meduni-graz