potential new medicine for autism in young children availabl

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cathylynn
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26 Mar 2013, 2:47 pm

"a manufacturer-funded study"... makes me immediately suspicious. however if the results are as they claim this could be a wonderful breakthrough for the treatment of ASD.

i would assume, however, that it wouldn't be a panacea... it would be beneficial for those whose autism results from, or is due in part to, metabolic errors involving the pathways that utilize BH4 -- which certainly isn't everyone with autism

nonetheless, it's a promising development. i'd like to see more research in this direction.



AardvarkGoodSwimmer
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26 Mar 2013, 3:29 pm

Quote:
http://psychiatry.jwatch.org/cgi/conten ... oc_jwpsych

Repurposing a PKU Drug for Autism

"In several double-blind, placebo-controlled studies and numerous open trials, tetrahydrobiopterin (BH4) has shown benefit for core symptoms of autism spectrum disorders (ASD) in young children (<5 . . [less than or equal to five years of age] . . . "

" . . . In an investigator-designed, manufacturer-funded study, . . "

" . . . Sapropterin was associated with significant improvement in core symptoms, both the primary outcome of language skills and secondary measures of communication and relationships. . . "

" . . . Because this medication is already safety-tested and FDA-approved for phenylketonuria, repurposing the drug for ASD is clinically reasonable (see JW Psychiatry Mar 4 2013). Physicians treating very young autistic patients can discuss use of BH4 with parents, emphasizing the lack of longitudinal data on possible long-term adverse effects, but noting that it makes sense for a drug to be primarily useful during neurodevelopment."



AardvarkGoodSwimmer
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26 Mar 2013, 3:53 pm

"investigator-designed, manufacturer-funded study" Okay, I'm still not going to be real crazy about it. The investigators are likely to be quick on the uptake regarding what types of studies drug manufacturers are likely to fund. And from co-equal partners and stakeholders (cough, cough), the drug companies might end up being the dominant ones.

"core symptoms" I hope they're not talking about stimming which I think is functional and largely a good thing. I mean, if there is a core symptom of autism it's more likely to be sensory issues, which stimming can help relieve. (and sure I'm practical. I'm in favor of redirecting stimming, the public vs. private distinction, that kind of thing)