A novel ‘gene-silencing’ drug could be the key to treating two devastating neurological disorders, spinocerebellar ataxia type 2 (SCA2) and amyotrophic lateral sclerosis (ALS).
SCA2 is an inherited disorder that inflicts damage on the brain’s cerebellum, causing patients to have issues with balance, coordination, walking and similar movements. ALS induces degeneration of nerve cells in the brain and spinal cord making patients gradually lose their ability to perform basic functions like move, speak, eat, or breathe.
There are different factors that can initiate the onset of these diseases, but two new studies indicate the first signs of a possible treatment approach for both SCA2 and ALS.
Researchers from the University of Utah and Stanford University performed two separate preclinical trials with a therapy that targets the ataxin-2 gene, both yielding novel results in mouse models.
First, the Utah scientists engineered mice to carry the human disease gene for SCA2 so the rodents exhibited many of the same symptoms as humans with the disease, according to the University of Utah.
After the rodents received injections of oligonucleotide, which targeted the mutated gene, results showed that the animal’s cerebellum displayed signs of restoration and they were able to perform significantly better on a balance and coordination tests compared to their counterparts on placebo. The mice did not exhibit any harmful side effects.
Similar results were seen in the Stanford study, which focused on ALS. The researchers genetically engineered a group of mice to produce high levels of TDP-43. A buildup of this toxic protein in the brain can be an indicator of ALS. These modified mice had difficulty walking and have life spans of no more than 30 days.
However, injecting this drug into the nervous system of these animals staved off early death and other neurological problems associated with ALS.
Both labs plan to continue to conduct further preclinical experiments to verify the safety of this regimen before progressing into human studies.