New drug that may help reverse autism
New drug that may help reverse autism is to be tested on children for the first time after successful clinical trials on mice
- Drug called suramin is already used to treat sleeping sickness in Africa
- Scientists in the U.S. have found it corrects autism-like symptoms in mice
- A small clinical trial on children with the condition will be started this year
A drug that may reverse autism is to be tested on children with the condition for the first time, scientists have revealed.
results show that the drug called suramin, which is already used to
treat sleeping sickness in Africa, corrects autism-like symptoms in
At the molecular level, it normalises faulty brain connections, cell-to-cell signalling, and metabolic effects thought to underlie the disorder.
Groundbreaking: Testing of the drug suramin on mice in America has found it can help to correct autism-like symptoms (stock image)
The drug targets a cell messaging system that produces a metabolic response to stress.
According to a new theory, autism is strongly linked to this pathway, known as purinergic signalling.
in the U.S. found that the drug corrected 17 types of abnormality
linked to autism in genetically modified mice, including social
Autism is a wide ranging
condition, mostly seen in boys, that affects a person's ability to
socialise and communicate and can have a devastating lifelong impact.
Around 600,000 children and adults in the UK are thought to have the
Robert Naviaux, co-director of the Mitochondrial and Metabolic Disease
Centre at the University of California in San Diego, said: 'Our theory
suggests that autism happens because cells get stuck in a defensive
metabolic mode and fail to talk to each other normally, which can
interfere with brain development and function.
'We used a class of drugs that has been around for almost a century to treat other diseases to block the 'danger' signal in a mouse model, allowing cells to return to normal metabolism and restore cell communication.'
New hope: A small clinical trial on children could be carried out within the year at the Mitochondrial and Metabolic Disease Centre at the University of California in San Diego, pictured
added: 'Of course, correcting abnormalities in a mouse is a long way
from a cure for humans. But we are encouraged enough to test this
approach in a small clinical trial of children with autism spectrum
disorder in the coming year.
trial is still in the early stages of development. We think this
approach, called antipurinergic therapy or APT, offers a fresh and
exciting new path that could lead to development of a new class of drugs
to treat autism.'
The findings are published in the online journal Public Library of Science ONE.
Naviaux and his team believe both genetic and environmental causes of
autism can be traced to a sustained cell danger response linked to
immunity and inflammation.
cells are exposed to classical forms of dangers such as a virus,
infection or toxic environmental substance, a defence mechanism is
activated,' said the professor.
results in changes to metabolism and gene expression (activity) and
reduces the communication between neighbouring cells. Simply put, when
cells stop talking to each other, children stop talking.'
is an inhibitor of purinergic signalling that has been used to treat
African sleeping sickness since shortly after it was first synthesised
The 'striking effectiveness' of the drug in mice could pave the way to a 'completely new class of anti-inflammatory drugs to treat autism and other disorders', said Professor Naviaux.
Read more: http://www.dailymail.co.uk/health/article-2293113/New-drug-help-reverse-autism-tested-children-time-successful-clinical-trials-mice.html#ixzz2NWtIPQiJ
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