Researchers at Yale University have revealed the results of a new study that may open a potential door to understanding some aspects of autism spectrum disorder treatment, by testing a common drug originally used for other medical purposes
In the study, the team examined 774 FDA-approved drugs to determine their effects on the behavior of zebrafish genetically modified to exhibit traits associated with autism. During these experiments, the researchers observed that levocarnitine, commercially known as Carnitor, was the most effective in improving the fish's responsiveness to their environment and their ability to interact with it.
Levocarnitine is primarily used to treat a rare genetic disorder known as carnitine deficiency, a condition in which the body is unable to produce or transport this essential substance for energy production within cells. It is also sometimes prescribed for other conditions, with hundreds of thousands of prescriptions dispensed annually in the United States.
Researchers believe that this drug may affect brain functions by boosting energy production in areas where reduced activity is observed in some people with autism, especially areas related to language and emotion, although its precise mechanism of action is still not fully understood.
As part of the study, researchers modified the genes of zebrafish to introduce human genes associated with autism, such as SCN2A and DYRK1A, which are relatively rare in those affected, as it is estimated that only a small percentage of them carry mutations in them.
The team also tested the effects of the drugs on unmodified fish, and later on human stem cells, to ensure safety and monitor various biological effects, before concluding that levocarnitine was the most effective at influencing responses associated with these mutations.
The study's lead author, Dr. Elaine Hoffman, explains that the wide variety of symptoms and genes associated with autism makes finding a single treatment that is effective for all cases extremely difficult, adding that studying the genes associated with the disorder may help identify potential treatment options.
However, the researchers stressed that these results are preliminary and do not mean that the drug can be used to treat autism at present, stressing the need to avoid taking it for this purpose outside of medical supervision, and that the matter still requires clinical trials on humans that may take years.
The researchers hope their data will contribute to the development of future research that may lead to more precise treatments targeting specific types of autism, rather than searching for a single, all-encompassing treatment for all cases.
The study was published in the journal "Proceedings of the National Academy of Sciences".
