Cheap drug already available may improve autism symptoms
Key Points:
- Yale University researchers have identified levocarnitine, a low-cost prescription drug currently used to treat low carnitine levels, as a potential treatment to ease autism spectrum disorder (ASD) symptoms in a subset of individuals with specific gene mutations.
- The discovery was made by testing 774 FDA-approved drugs on genetically modified zebrafish exhibiting autism-like traits, with levocarnitine showing the ability to reverse disruptive behaviors and promote more typical brain activity in fish with mutations in the SCN2A and DYRK1A genes.
- These gene mutations are relatively rare in the broader ASD population, suggesting levocarnitine may only benefit a small group of people with autism who carry these specific genetic variants.
- The researchers emphasize the importance of studying known autism risk genes and have created an open-source database of drugs to facilitate future research, but they caution that levocarnitine still requires human trials before it can be recommended as a treatment.
- ASD diagnoses have increased significantly in recent years due to expanded diagnostic criteria and greater awareness, but currently, no medication cures autism or universally alleviates its symptoms.