New studies point the way to broadly effective treatments for ALS

In the quest to find a cure for ALS patients, a team of researchers led by USC Stem Cell scientist Justin Ichida has identified two promising avenues for developing new treatments for diverse forms of this devastating disease. The researchers collected skin or blood samples from patients with both familial and sporadic ALS and reprogrammed the skin and blood cells into motor neurons. These patient-derived motor neurons provided an opportunity to screen thousands of FDA-approved drugs and drug-like molecules to find ones that might be effective against multiple forms of ALS. By leveraging a public bioinformatics database to identify how drugs affect the genetic landscape underpinning ALS, the scientists found that suppressing the SYF2 gene would increase the survival of motor neurons derived from patients with diverse forms of ALS. They also found that inhibiting a protein, the PIKFYVE kinase, could represent another effective strategy for treating many different forms of ALS.

Source: ScienceDaily.com