A strong diuretic drug – used for decades to treat high blood pressure and increase urination rate – has improved the behavioral symptoms of autism in children.
Several lines of evidence suggest that autism interferes with the neurotransmitter GABA, which typically puts a damper on neural activity, Nature News explains. And this disruption of GABA is due to increased levels of chloride ions in the brain cells, ScienceNOW explains. Reducing these chloride ion levels might help to treat the condition.
Turns out, bumetanide is known to reduce intracellular chloride and to reinforce the actions of GABA.
Also known as a "water pill," bumetanide is used to reduce the swelling and fluid retention caused by various medical problems, such as heart or liver disease. It also treats high blood pressure by causing the kidneys to get rid of unneeded water and salt from the body into the urine.
A team led by Yehezkel Ben-Ari at the Mediterranean Institute of Neurobiology performed a double-blind randomized clinical trial using bumetanide with 60 autistic and asperger children (ages 3 to 11).
- The team randomly assigned the participants to receive either 1 milligram daily for three months, or a placebo daily for the same period.
- The drug reduced symptoms associated with autism in less severely affected children: those who got bumetanide improved by 5.6 points on a 60-point scale that's often used to assess behaviors related to autism.
- Parents described the bumetanide-treated children as more “present” with enhanced communication with their environment.
Earlier this year, Ben-Ari cofounded Neurochlore to develop and commercialize bumetanide as a treatment. The team is now preparing for a larger, multicenter trial in Europe.
The work was published in Translational Psychiatry a couple weeks ago.