Wednesday, May 15, 2013

Experiment on disabled children first, then maybe on animals, if you wish

I am up to my ears in work and I really shouldn't be blogging now, but...

In early January, I wrote a post titled I am skeptical about bumetanide treatment of autism. It addressed a 2012 clinical trial using the diuretic bumetanide to improve the condition of autistic children, published with much fanfare in a peer-reviewed journal.

The idea behind this trial contradicted my gut feeling that you cannot rearrange the unusually wired brain of autistics by giving them some pill. However, in that post, I put more serious objections than a gut feeling: that the study had been done on children and that it had not been preceded by a similar study on an animal model. I wrote: "Explaining their hypothesis in the Introduction chapter, the authors wrote, "GABAergic signals are altered in autism as evidenced by the following: The excitation/inhibition ratio is modified in experimental models..." (5 references cited). However, to my best knowledge, the researchers never tried to test their working hypothesis on these models that so wonderfully provided argumentation to start an experiment on human beings." I had earlier complained about the worrying tendency of modern scientists to test all sorts of treatments on vulnerable human patients without trying them on animals first.

Today, I googled bumetanide autism just so, to see any sequel to the story. And what do you think came out? An article by Virginia Hughes titled Controversial study touts blood pressure drug for autism published on the Simons Foundation Autism Research Initiative site. Quoting from this article: "Several experts are puzzled because the study used diagnostic measures of autism in unusual ways. They also say that the sample size is too small to know whether the effects are real" (27 children in the test group and 27 controls - M.M.)... "Bumetanide treatment was associated with statistically significant improvements on total ADOS scores only when the researchers removed the nine most severely affected children from the group. Seven of the nine were in the placebo group, suggesting that the placebo group was skewed toward the more severe end of the spectrum." What then remains of the claim "placebo-controlled", eh?

But, for me, the most intriguing bit comes last: "Ben-Ari’s team is trying to confirm his theory by looking at intracellular chloride levels in two animal models of autism" (emphasis mine - M.M.). In other words, we first experiment on defenceless disabled children whose parents are dying to see them normalized, and then, when the scientific community starts to ask inconvenient questions, we resort to an animal study! Bravo, bravo, bravo.

An commenter wrote on my previous post: "I too am a huge skeptic, but a double-blind, placebo-controlled trial was good enough for me. So I have tried bumetanide for 3 weeks with my 9 year old autistic son. The results are great..." This is what I feared - that parents would immediately start administering the drug to their children. At that time, I didn't know what to answer to that father. But now, if he is still here, I would ask him (and everybody else in the same situation) to reconsider. Contrary to the initial claims of researchers that bumetanide produces "no side effects" on autistic children, Hughes reports, "Some researchers say they worry that the drug’s side effects may have effectively unblinded some of the participants. Of the 27 children treated with bumetanide, 6 developed a potassium deficiency that had to be treated with a potassium syrup" (emphasis mine - M.M.).

My guess: Newer studies on bumetanide and autism are likely to find less (down to zero) improvement and more side effects.