TAMPA, Fla. (WFLA) — The University of Florida’s Scripps Biomedical Research team said they may have found a solution to treating Amyotrophic lateral sclerosis, or Lou Gehrig’s disease, and dementia. In lab tests, the team said they were able to restore neurons in mice using the new treatments.
According to the UF Scripps researchers, treatment involved eliminating the parts of patient RNA, or ribonucleic acid, that can cause the diseases. Doing so helped them “restore the health of neurons in the lab and rescued mice,” that were infected.
Publishing their research in the Proceedings of the National Academy of Sciences, study authors said options for the treatment included both pill or injection.
Dr. Matthew Disney, chair of the UF Scripps chemistry department said that the team’s experiment showed “the compound is small enough to cross the blood-brain barrier, a hurdle other approaches have failed to clear.”
As a disease, ALS “destroys neurons that control muscles, leading to worsening muscle loss and eventually death,” according to a publication by UF about the experimental treatment. Disney said the mutation involved can also lead to a form of frontotemporal dementia, which causes parts of the brain to shrink, affecting personality, behavior, and how well a patient can speak. Ultimately, the disease also leads to patient deaths.
“The compound works by binding to and using natural cellular processes to eliminate that disease-causing RNA by alerting the cell’s degradation machinery to dispose of it as waste,” Disney said. Now that the research team is seeing results in using the treatment, Disney said it could also be applied to healing other “untreatable neurological diseases” where “toxic RNA” is involved.
Using the compound in mice bred to have ALS, the UF research team treated their test subjects every day for two weeks. Afterward, the mice who received the treatment showed “significantly reduced markers for disease and improved health.”
Now, the study will move forward, examining how the chemical compound affects cellular health of another form of ALS. Disney said their evidence and results so far are a “notable advance in the field of RNA drug discovery.”
“We show for the first time that you can make brain-penetrant molecules that eliminate toxic gene products,” Disney said. “The fact that we have highlighted this in ALS shows that this can be a general approach for other neurological diseases, including Huntington’s, forms of muscular dystrophy and others.”
