A cheap drug touted by longevity enthusiasts could help to reverse the symptoms of autism, a study has claimed.
Rapamycin is an antibiotic approved by the FDA in 1999 to prevent organ transplant rejection. In recent years, however, biohackers have also latched onto the drug amid claims it could slow down aging.
Some studies showed it could extend the lifespan of mice by 14 percent, but others in humans have highlighted drawbacks, including blunting the effects of exercise.
Now, researchers from the University of California, Los Angeles say they have found that administering just one dose of the drug may temporarily reverse symptoms of autism.
Administering the drug to adult mice with autism, they found that the rodents’ symptoms disappeared in just two hours.
Dr Harley Kornblum, a neuroscientist who led the research, said: ‘The level of functional normalization achieved over this short time suggests new mechanisms by which possible treatments may act.
‘It suggests adult brains may be more adaptable than we assumed, even when the underlying structural changes from early development are still there.’
Autism was once rigidly defined as a serious developmental disorder characterized by intellectual disability and the struggle to learn or understand language.
Scientists have found that rapamycin can ease autism symptoms in adult mice
But in recent years, experts have loosened the definition. Symptoms include repetitive words or behaviors, struggling to vary routine and difficulties in social situations.
An estimated five million American adults, or one in 20, suffer from the condition, now called autism spectrum disorder, while one in 31 children also have it.
Doctors typically treat patients using speech and language therapy to help them understand and manage atypical behaviors.
Many are also prescribed medications, such as those used for anxiety and depression, which scientists say can help to ease social difficulties.
There is no cure for the condition.
In the study, published in Nature Communications, scientists injected autistic adult mice with a solution containing five milligrams of rapamycin.
Within two hours, they found the mice displayed fewer repetitive behaviors, such as persistently grooming or running in a circle in an open space.
This reduction lasted about 72 hours, they said, before the mice reverted to their previous behavior.
After five weeks of injecting the mice with 5mg of rapamycin daily, the researchers also found that the effect of the drug subsided.
Because the response was so quick, researchers said rapamycin had not rewired the brain. Instead, they suggested it had likely calmed dysfunctional or misfiring neurons which can drive autism.
Tests showed mice that took rapamycin had less abnormal brain activity.
The study was not carried out in humans, meaning it is not clear what impact the drug may have in people with autism.
Researchers said further tests were needed before it could be recommended as a treatment for autism.
But they suggested that the results opened the door to potential new treatments for the condition.
The researchers added in the release: ‘The study did not identify rapamycin as a viable treatment for [autism] symptoms in humans given its temporary effects and potential for toxicity from repeated doses.
‘Instead, researchers said the drug’s effects revealed new therapeutic targets for the development of future treatments.’
Rapamycin, found in the 1970s, is an antibiotic prescribed to prevent organ transplant rejection
Dr Janel Le Belle, a neurologist also involved in the study, added: ‘These results reframe how autism-associated symptoms might be treated.
‘If the adult brain remains capable of functional normalization, then some features of autism may be successfully addressed without needing to correct underlying structural differences.’
Researchers warned that taking rapamycin raises the risk of numerous side effects.
Common side effects of the medication include high blood pressure, kidney injury, infections and unusual bruising or swelling on the ankles, hands or feet.
In serious cases, it has also been linked to blood clotting problems, lung problems and slow wound healing.
Biohacker Bryan Johnson, 48, stopped using the drug last year, after five years of taking the medication in an attempt to expand his lifespan.
He said the risks outweighed the benefits, adding it had likely caused him to suffer from skin infections, a higher resting heart rate and reduced blood sugar sensitivity.
There is no cure for autism, but researchers are researching numerous drugs that they believe may help to ease the condition.
Scientists are also testing leucovorin, a form of vitamin B, as a potential treatment after it was touted by Donald Trump and Robert F Kennedy Junior.
Most commonly used to counteract the toxic effects of certain chemotherapies, some experts say it may treat some forms of autism that have been linked to low folate levels, a type of vitamin B needed for healthy cell growth and function.
A study published in May this year warned that use of the vitamin had surged amid the publication of high-profile cases suggesting it could benefit autism.
















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