Autism symptoms: cheap drug levocarnitine may ease them for some people

Yale researchers screened 774 FDA-approved drugs in zebrafish models and found that levocarnitine appeared to reverse autism-like behaviors in fish carrying SCN2A or DYRK1A mutations, but those mutations are rare and human testing has not yet been done.

Researchers at Yale analyzed a large set of existing FDA-approved drugs in zebrafish engineered to show autism-like traits to look for candidates that might affect behavior. The team screened 774 drugs, narrowed that group to 520 non‑toxic compounds with significant behavioral effects, and identified levocarnitine as a standout in their analysis. Levocarnitine is already approved for treating low carnitine levels and is inexpensive per dose. The researchers emphasize that the findings come from animal models and that human testing is still required. Key findings: - The study screened 774 FDA-approved drugs and retained 520 that were non-toxic and showed significant effects on zebrafish behavior. - Levocarnitine (sold as Carnitor) was associated with restored or more typical behavior in zebrafish carrying mutations in the SCN2A and DYRK1A genes. - In the zebrafish models, levocarnitine also correlated with more balanced metabolism and more typical patterns of brain activity. - SCN2A mutations are estimated to account for about 1 in 333 autism cases, and DYRK1A mutations are estimated in fewer than 1% of diagnosed people, suggesting any benefit would be limited to a small subset. - The researchers created an open-source, searchable drug database and note that levocarnitine still needs testing in humans; they caution against treating the drug as an established therapy. Summary: The findings point to a possible drug-gene interaction that could be relevant for people with specific autism risk gene mutations, but the mutations involved are uncommon in the broader autism population. Undetermined at this time.