Reacción a "A single dose of rapamycin in mice reduces behaviours similar to those seen in the autism spectrum for a few hours"

Is the study of good quality?

‘The study is of high scientific quality; it is rigorous, both in its design and in its execution. It is a fairly comprehensive study that uses cutting-edge and diverse methodology to analyse whether acute treatment with rapamycin is capable of improving any of the symptoms associated with the autism spectrum and epilepsy.’

Are there any limitations that need to be taken into account?

“The study has several limitations. The first is that, although the authors observe that some of the symptoms of autism spectrum disorder improve or disappear, this effect is very short-lived. Previous studies have shown that chronic treatment with rapamycin leads to tolerance (it ceases to be effective) and, furthermore, has serious side effects.

Although a rapid effect of rapamycin is observed, highlighting the involvement of the mTOR pathway in autism, the mechanism has not yet been demonstrated. When microglia are depleted in the brain, this replicates the effect of rapamycin, but the same does not occur in older mice.”

Does the press release accurately reflect the study, including whether it could be applied to humans?

“In my opinion, the press release is fine; it states that there is still a long, long way to go before a treatment can be applied to humans. The fact that the reversibility of some of the symptoms has been demonstrated, even in adults, is encouraging. Now we need to find that treatment.”

What are the implications and how does this fit with existing evidence?

“This work is consistent with a wealth of previous evidence demonstrating the link between the maternal inflammatory response and autism, as well as the fact that neuronal hyperexcitability exists. Perhaps one of the most important implications is that the symptoms of autism can be treated, even in an adult brain. Neuronal hyperexcitability had previously been demonstrated, and treatments have also been proposed to reduce this hyperexcitability – treatments that may be more specific and have fewer side effects than rapamycin.”

What is really important about this study: the use of rapamycin as a treatment, or the maternal inflammatory response and its link to autism?

“In my humble opinion, what is really important about this study is that it demonstrates that the symptoms of autism can be treated, even in an adult brain; the priority now is to find that treatment. There is a genuine paradigm shift regarding the reversibility of autism, or rather, of the symptoms of autism.

As for the link between the maternal inflammatory response and autism, this had already been demonstrated previously in experimental models that replicate many of the structural and behavioural changes seen in the autism spectrum disorder (ASD) in humans. This study confirms it once again.”