It is no secret that autism is an incredibly enigmatic disease. This is what makes it frustrating for researchers, and especially for those affected by the Autism Spectrum Disorder (ASD). But there is hope. French researchers have had success with a new drug in experiments on rodents. Although the new autism drug is not yet a solution for loved ones, it does open many doors.
Today, the researchers responsible for the breakthrough published their findings in the journal, Science where they detail that a strong diuretic or “water pill” called bumetanide helped autism symptoms in mice when given before birth.
Bumetanide is currently used as a treatment to reduce swelling and for fluid retention in patients with heart or liver disease and for those with high blood pressure. When used in the rodents prenatally, there seemed to be a biochemical transformation in the brain that mitigates the neurotransmitter GABA from producing an excess of electrical action in the brain.
The team of researchers from the French Institute of Health and Medical Research (INSERM) administered the drug to pregnant rodents before they gave birth. Specifically, the bumetanide was given to mice that carried a gene defect that results in Fragile X syndrome in humans and rats that were prenatally exposed to a drug called valproate. Both of these types of rodents exhibit symptoms similar to that of autism.
The offspring of the rodents that were given the drug prenatally did not seem to acquire any of the autism-like symptoms. When compared with a control group of healthy rodent young there was not a significant behavioral difference.
Rob Ring, chief science officer at Autism Speaks, said that this gives medicine an interesting, new course of action to bear in mind, but warns that this autism new drug is not yet a solution for loved ones.
The research team is testing the drug on 30 autistic children in a supervised trial with the hopes that it will be the first cure for the main underlying ASD symptoms.
However, there are many unknowns regarding effect and dosage and the drug should not be used outside of clinical trials yet. The president and CEO of the Kennedy Krieger Institute – a research facility and clinic – Gary Goldstein said that cures that work in rodents often do not work in humans and could even result in harmful effects.
Regardless of the success the research team had with rodents, the drug will not be administered to pregnant women for a variety of reasons. First of all, at this point it is impossible to tell before birth whether a child will have autism or not. Often, it is not known until the child is around four years old and the drug should not be given to a healthy baby.
Experts do believe that the earlier the drug can be given to a child with ASD, the better the chance of alleviating the symptoms. Yehezkel Ben-Ari, lead researcher on the INSERM team, stresses the importance of early treatment and is testing 2-year-old children as researchers work to diagnose children at an earlier age and even in utero.
Ben-Ari said that he is optimistic about the drug’s benefits, but also acknowledges that it will most likely not be a cure-all drug and ASD patients may need other treatments because of the complexity of the disorder.
While the new autism drug, bumetanide, offers a deeper look into the causes of autism and a chance for a cure, it is important to remember that the autism new drug is not yet a solution for loved ones. Still, the hope is that this is just the beginning of the breakthrough.
By Rebecca Hofland