There was an Open-Label clinical trial in Sweden that explored the use of Diuretic drug in treating the core symptoms of autism. There were clinical trials in France that showed promising results. Let me teach you the science.
Let’s get into understanding a Diuretic drug and autism in a clinical trial setting. Bumetanide is a diuretic drug, and it’s prescribed by doctors to help reduce fluid retention and swelling. Typically, it’s used for those who have congestive heart failure, liver disease, or kidney disease. And it works by acting on the kidneys to increase the flow of urine. This diuretic drug influences neurotransmitters. Seems kind of a bit out there, but let’s get into the science and I’ll explain. Bumetanide reduces intracellular chloride, thereby reinforcing GABAergic inhibition. Gaba is a neurotransmitter. Several researchers are focused on the excitatory neurotransmitter glutamate. You might have heard that before, and its role in autism. There’s a lot of research that’s been done on Glutamate being an excitatory transmitter, and there are genetic studies that support the involvement of glutamate with autism. Neurotransmitters can be complex. There is a delicate balance in the body with glutamate, which is excitatory, and the inhibitory transmitter GABA in the brain. Some researchers are focused on GABA dysfunction in children with autism. So let’s focus on GABA a bit more.
GABA complexity
In utero, there is a high intracellular chloride concentration. And when that is the case, GABA operates mainly as an excitatory transmitter. During maturation, meaning the baby is growing and then it’s born and aging occurs, the intracellular chloride concentration decreases, and therefore, GABA switches from being an excitatory neurotransmitter to being an inhibitory neurotransmitter after birth. It’s pretty amazing how neurotransmitters can switch their function. In our body, there are two chloride co-transporters that control the intracellular level of chloride. The diuretic drug, Bumetanide, reduces intracellular chloride. And here this diuretic drug can actually restore GABA inhibition in patients with neurodevelopmental disorders by decreasing the neuronal chloride concentration. So basically, you’re taking the intracellular chloride concentration and returning it back to the levels when the child was in utero before being born. So what the science is doing here is it’s taking this diuretic drug and changing the chloride concentrations to mimic those when in utero.
So GABA, in utero is excitatory. After birth, it’s inhibitory.
And so this drug allows GABA to be an excitatory neurotransmitter rather than an inhibitory. That’s the whole scientific premise of this research. Let’s get to the research.
The Clinical Trial
There were six children with autism aged 3 to 14 years and they were treated with Bumetanide, the diuretic drug in an open-label three-month trial. Dosing was very specific to weight. All participants had their serum electrolytes checked so that’s sodium, potassium, chloride. They also had an ECG before they started treatment and the electrolytes were monitored throughout the course of this clinical trial. Obviously, this is very important to do because if you’re increasing urination and you’re changing the concentration of chloride – You’ve got to make sure that the body is still well supported and that there are still electrolytes that the body can use for typical functioning. Due to the risk of potassium deficit during treatment with a diuretic all of the children received extra potassium as a prophylactic and again this is not something to just try at home by yourself. This is a prescription medication. You really want to be working with a knowledgeable practitioner so that your child is supported in all these different areas and that some really good decisions can be made because there are side effects!
Side Effects
So four of the children had side effects within a few days after starting the treatment and the side effects were severe hyperactivity, depressive mood, aggressiveness, large urinary volumes at night, and the doses were decreased for those individuals.
Results
Here are the results Bumetanide showed positive results for all six participants according to the parental satisfaction survey (the results were quantified based upon a survey from the parents). Did the parents see any change? That’s how the results were quantified. Changes were seen concentrated in the communication and cognitive abilities. After the three month treatment period, all the parents wanted their children to continue the treatment, and here are the researcher’s conclusions directly from the research paper: