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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

#21

Post by bglazer »

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, it's a virus, specifically a lentivirus, which inserts its own genome into the cells DNA, including the therapeutic gene. Looks like they first extract bone marrow from a patient, apply the virus which adds the gene, then they put the "fixed" bone marrow cells back into the patient (probably after killing most existing bone marrow through some really unpleasant procedures). The re-inserted, treated cells then expand and begin making new cells that have inherited the fixed gene.

In general, gene therapy is probably most useful currently for diseases that affect the bone marrow (and the eyes), because these are two tissues where you can precisely deliver the virus. It's still challenging to control where the virus goes and which cells it infects if you just inject it straight into the bloodstream.

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

#22

Post by MaryBall »

amelius wrote: post #7
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?

Also, when cells replicate, will the new ones automatically be of the edited type? Or is it not that simple?

Because this therapy edits the stem cells that are found in the bone marrow, it essentially means that the disease is being fixed at the source. Bone marrow stem cells eventually turn into blood cells and immune cells. So if 100% of the cells have been edited, then the resulting cells they turn into will be the edited type.

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

#23

Post by themaninthedark »

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 .

And this in turn leads people to be against national health care as they are worried that the government will be the one making the decision that it is cheaper to let people die rather then treat them.

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

#24

Post by elil17 »

whiddershins wrote: post #13
elil17 wrote: post #11

Earlier quoted context omitted.

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.

I agree there needs to be pressure. Why not regulatory pressure?

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

#25

Post by krona »

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.

In quality-adjusted life years (QALYs), there is an upper limit to the cost of any intervention because the most you can save from one intervention is one life. If the same money can be used to substantially improve the lives of 100 people with other interventions, then the cost-utility analysis may say a particular intervention is not effective.

You might not like the utilitarian approach but this is how the UK measures effectiveness.

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

#26

Post by Traubenfuchs »

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?

In this specific case CRISPR-CAS9 was used to edit "hematopoietic stem and progenitor cells". This means all modified stem cells (sadly not 100% of them) will from now on produce cells with the gene bugfix applied.

This does not imply that germ line cells were changed and COULD mean the person could still have a high risk of making children with the original gene defect.

https://pubmed.ncbi.nlm.nih.gov/34882002/

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

#27

Post by shadowgovt »

cameronh90 wrote: post #12
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?

Its sticker price is actually $3.8 million. At that price, it could be administered to every child in the UK with the disease at about the cost of 1 year of the UK's defense budget.

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

#28

Post by shadowgovt »

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.

Not sure I follow. It's not like a therapy with a sticker price of $3.8 million is more available in countries without nationalized healthcare.

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

#29

Post by gambiting »

shadowgovt wrote: post #20
xxpor wrote: post #8

Earlier quoted context omitted.

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.

The problem is, as always, with allocation of resources. If you are running NHS budgets and these treatments cost £1M each(we don't know what price was agreed in the end, but let's say it's £1M per treatment), that's £1.6 billion to treat 1600 kids. £1.6 billion is a lot of money that can save a lot of more than 1600 people if used for other therapies. It's a horrible choice to make of course, but it's the reality of it. Yes of course the government and the country has enough money to afford it - but NHS is given a fixed budget.

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

#30

Post by mrtksn »

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.

In Europe they have these things called planes, it's like a tube you enter and sit for a few hours and once exit you are in a driving distance to American hospitals where you can pay and receive the same treatment as everyone else without nationalised healthcare.

Best of the both worlds.

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