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First UK child to receive gene therapy for fatal genetic disorder is now healthy

livescience.com

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Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#11

Post by elil17 »

xxpor wrote: post #8
jiggawatts wrote: post #5

https://en.m.wikipedia.org/wiki/Atidarsagene_autotemcel > The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment." Wow… instead of a lifesaving cure they recommend the treatment of the symptoms until the kid dies because it’s cheaper .

The world doesn't have unlimited resources. You have to make a call somewhere.

Governments and non-profits already funded the development of gene therapy. Letting private companies charge money for it and then blocking people who need it from getting it is a policy decision.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#12

Post by cameronh90 »

jiggawatts wrote: post #5

https://en.m.wikipedia.org/wiki/Atidarsagene_autotemcel > The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment." Wow… instead of a lifesaving cure they recommend the treatment of the symptoms until the kid dies because it’s cheaper .

What if the cost was $100 trillion?

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#13

Post by whiddershins »

elil17 wrote: post #11
xxpor wrote: post #8

Earlier quoted context omitted.

The world doesn't have unlimited resources. You have to make a call somewhere.

Governments and non-profits already funded the development of gene therapy. Letting private companies charge money for it and then blocking people who need it from getting it is a policy decision.

Doing the procedure has a cost. There must be some pressure to reduce the cost, else it will never reduce.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#14

Post by hummus_bae »

jiggawatts wrote: post #5

https://en.m.wikipedia.org/wiki/Atidarsagene_autotemcel > The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment." Wow… instead of a lifesaving cure they recommend the treatment of the symptoms until the kid dies because it’s cheaper .

Another country where nationalized healthcare is a bad idea.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#15

Post by ls612 »

jiggawatts wrote: post #5

https://en.m.wikipedia.org/wiki/Atidarsagene_autotemcel > The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment." Wow… instead of a lifesaving cure they recommend the treatment of the symptoms until the kid dies because it’s cheaper .

[flagged]

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#16

Post by herlitzj »

SamBam wrote: post #4

The article was skimpy on the details. Can someone explain how this part works? > The new gene therapy [...] works by inserting into the body working copies of the genes that are faulty in MLD, thus restoring the ability to break down sulfatides. How does the new copy of the gene get into every existing cell that needs it? A virus?

https://www.thelancet.com/journals/lancet/article/PIIS0140-6...

This paper seems to have more details on the actual procedure

Patients were treated and monitored according to the schedule described in the appendix (p 17) and as previously reported.16, 17 HSPCs harvested from bone marrow or mobilised peripheral blood (MPB) were transduced with clinical-grade lentiviral vector encoding human ARSA cDNA under the control of the human phosphoglycerate kinase gene promoter.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#17

Post by MaryBall »

SamBam wrote: post #4

The article was skimpy on the details. Can someone explain how this part works? > The new gene therapy [...] works by inserting into the body working copies of the genes that are faulty in MLD, thus restoring the ability to break down sulfatides. How does the new copy of the gene get into every existing cell that needs it? A virus?

Yes, you’re right! A lentiviral vector is used to insert the working gene into the patient’s stem cells. Obviously this is a non-pathogenic version of the virus, so it’s ability to make you ill has been essentially “switched off”. However the ability for the virus to insert genetic information into the DNA of cells remains.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#18

Post by blibble »

hummus_bae wrote: post #14
jiggawatts wrote: post #5

https://en.m.wikipedia.org/wiki/Atidarsagene_autotemcel > The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment." Wow… instead of a lifesaving cure they recommend the treatment of the symptoms until the kid dies because it’s cheaper .

Another country where nationalized healthcare is a bad idea.

I think you'll find most insurance policies have a maximum

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#19

Post by nivenkos »

hummus_bae wrote: post #14
jiggawatts wrote: post #5

https://en.m.wikipedia.org/wiki/Atidarsagene_autotemcel > The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment." Wow… instead of a lifesaving cure they recommend the treatment of the symptoms until the kid dies because it’s cheaper .

Another country where nationalized healthcare is a bad idea.

But healthcare isn't nationalised in Ireland. Was that your point?

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#20

Post by shadowgovt »

xxpor wrote: post #8
jiggawatts wrote: post #5

https://en.m.wikipedia.org/wiki/Atidarsagene_autotemcel > The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment." Wow… instead of a lifesaving cure they recommend the treatment of the symptoms until the kid dies because it’s cheaper .

The world doesn't have unlimited resources. You have to make a call somewhere.

There are, on the high end of the estimate, 1,600 kids in the UK that might have this disease. It's estimated about 5 are born per year.

I suspect we can somehow find enough pennies in the couch cushions to get those kids a therapy, especially if it's curative.

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