14 DAY TRIAL // A group of researchers from the Loyola University Chicago (LUC) announces the development of a new cure for SCA1, a fatal, progressive disorder affecting the central nervous system. The team reports that the treatment shows great promise.
Spinocerebellar ataxia (SCA) in fact represents a wider array of disorders, in the sense that it has many distinct types, the majority of which can be classified as individual diseases. In the new study, the team addressed SCA type 1 exclusively.
According to the background information the team published in the latest issue of the top scientific journal Nature Medicine, the condition is genetic in nature, which makes it especially difficult to treat from the start.
For the new investigation, the research team focused its efforts on studying the multipurpose protein called the vascular endothelial growth factor (VEGF). Past studies had linked low levels of this molecule to the development of SCA1.
Some of the symptoms associated with the development of this condition include the loss on muscle coordination, which can be seen in losing balance while walking and poor hand control. Difficulty swallowing, indistinct speech, neuropathy, weakness and memory problems are common later on.
In the new study – which was carried out on unsuspecting lab mice – researchers found that boosting VEGF levels in the rodents lead to significant improvements in their condition. The progress was observable in both balance and muscle coordination.
According to the research team, boosting VEGF levels represents a potentially useful treatment for patients with SCA1, and could possibly be of help in other neurodegenerative syndromes as well.
Funding for the research came from the US National Institutes of Health (NIH), the National Organization for Rare Disorders, the Brain Research Foundation and the National Ataxia Foundation.
LUC Stritch School of Medicine Department of Pathology and Cardinal Bernardin Cancer Center associate professor Ameet R. Kini, MD, PhD, was a coauthor of the study. The first author of the paper was LUC investigator Marija Cvetanovic, PhD, Eurekalert reports.
The research team says that they now plan to conduct a series of clinical trials, which are meant to assess whether the discoveries apply to humans as well. The results are expected to be made available within a few years, after which time a new cure for SCA1 could make its way into the market.