Researchers have reversed autism-like brain changes in adult mice within hours using a single dose of the drug rapamycin, according to a study by UCLA Health. The findings, published in Nature Communications, suggest that some autism-related brain changes may still be treatable in adulthood.
Study Details
The study subjected pregnant mice to inflammation triggers, resulting in offspring that developed lasting autism-like changes to brain function and behaviour. A single dose of rapamycin to the adult offspring rapidly improved several changes, including brain overactivity, seizure risk, sensory sensitivity, repetitive behaviours, and abnormal brain functional network organisation.
UCLA Health stated: "The findings suggest that some autism-related brain changes may still be treatable in adulthood, pointing to possible therapeutic approaches that target the underlying pathway rather than only symptoms." It added that the effects of rapamycin are temporary and repeated dosing loses efficacy, making rapamycin itself an unlikely candidate as a broadly-used treatment.
Dr Harley Kornblum, the study's senior author, said: "It suggests the adult brain may be more adaptable than we assumed, even when the underlying structural changes from early development are still there. This points us toward the brain's functional circuitry, not just its physical structure, as a target for future treatment approaches."
Dr Janel Le Belle, an associate professor in the UCLA Department of Neurosurgery, stated: "These results reframe how autism-associated symptoms might be treated. If the adult brain remains capable of functional normalisation, then some features of autism may be successfully addressed without needing to correct underlying structural differences."
What is Rapamycin?
Rapamycin, also known by its generic name sirolimus, is licensed and used in the UK on prescription. It is primarily used to prevent organ rejection in kidney transplant patients and to treat a rare chronic lung disease. Related medications are also used for certain targeted cancer treatments. Beyond established uses, rapamycin is being studied for extending healthy lifespan and for chronic illnesses including myalgic encephalomyelitis.
Causes of Autism
The National Autistic Society says evidence suggests that autism may be genetic and likely has multiple genes responsible. It stresses that autism is not caused by emotional deprivation or the way a person has been brought up, and that there is no link between autism and vaccines.
The National Autistic Society also stresses that "the concept of a 'cure' is offensive for many autistic people and their families" and that an intervention should never aim to treat autism, but there are interventions that can support autistic people in their daily lives.
