The first gene therapy drug, Glybera, was approved by the EU in 2014.
The new drug, Strimvelis, is for 'bubble boy disorder'.
Gene Therapy will likely be a cure for genetic systemic malodor disorders.
Gene Therapy is of interest to those with metabolic genetic malodor disorders because gene therapies will likely be 'cures' for genetic disorders. So if someone has a sub-par FMO3 enzyme that causes systemic body odor and/or halitosis, gene therapy could be considered the 'holy grail' cure.
Gene therapy has been suggested for about 30 years but a main obstacle was that it often involves viruses and these could cause a severe/fatal immune reaction. Also there may be some resistance from big pharmas as a one-treatment cure may mean loss of revenue. And investment costs and cost of treatment were seen as too costly (Glybera cost around $1.2 million per treatment.
But finally the obstacles seem to be clearing, and the technology has caught up.
FMO3 protein
Oxidizes/reduces many sulfides and amine (and phosphates)
Gene produces a 532 amino acid code (and a stop at 533)
How would gene therapy be relevant to metabolic/systemic body odor/halitosis ?Oxidizes/reduces many sulfides and amine (and phosphates)
Gene produces a 532 amino acid code (and a stop at 533)
My view is that most causes of systemic B.O/halitosis are probably to do with a sub-par FMO3 enzyme issue. In these cases gene therapy should make the FMO3 enzyme work normally. Gene therapy has taken 30 years to get to this stage but over the next 10 years could quickly become cures for genetic disorders.
How do Gene Therapies work ?
My impression is that they are usually manufactured viruses that are put in the target gene to make the gene work correctly. It will probably be to do with what type of fault you have in the gene. For FMO3, Most people have 'missense' faults, where an incorrect amino acid has been put in the 532 amino acid sequence. So this would probably require a 'missense' drug. Another severe type is a 'nonsense' fault, where the 532 long FMO3 code is told incorrectly to stop early on (a false stop). One drug, Ataluren, is being targeted to ignore this 'false stop' so in the case of FMO3 the 532 code will ignore the false stop. But 'nonsense' mutations are much rarer than missense and I presume a nonsense targetting drug would not help with a missense mutation.
Links to story on Strimvelis drug approval :
BBC news site
News18.com
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5 comments:
Is this available to patients yet?
in theory it is in Europe (the EU). I guess it is then up to each country in the EU to decide if they will approve it in their own healthcare system. But I guess in the EU private Drs will be able to prescribe it to private patients who pay for it.
I assume it won't be cheap.
the 1st drug said $1 mill for a course (you only need 1 course ?), and the 2nd drug said 'considerably less' but I guess still $000's.
But I suspect these type of drugs will quickly become much cheaper.
When it becomes free on the nhs i'll consider it :)
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