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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

#61

Post by mmaunder »

I wonder if a similar approach can be used for Von Hippel Landau.

“ Mutations in the VHL gene cause von Hippel-Lindau syndrome. The VHL gene is a tumor suppressor gene, which means it keeps cells from growing and dividing too rapidly or in an uncontrolled way. Mutations in this gene prevent production of the VHL protein or lead to the production of an abnormal version of the protein. An altered or missing VHL protein cannot effectively regulate cell survival and division. As a result, cells grow and divide uncontrollably to form the tumors and cysts that are characteristic of von Hippel-Lindau syndrome.”

https://medlineplus.gov/genetics/condition/von-hippel-lindau...

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

#62

Post by theGnuMe »

meese712 wrote: post #59
bglazer wrote: post #21

Earlier quoted context omitted.

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 ex…

I'm confused how this would reduce disease progression compared to a much cheaper allo bone marrow transplant then if they are only modifying hematopoietic stem cells since a BMT is just doing the exact same thing except with someone else's non affected cells. BMTs are a horrific procedure though so this definitely has an advantage in that regard. I would also have to imagine they would have to do myeloablative chemo…

Well it removes graft vs host complications which is a win and the requirement to take immunosuppressants for life.

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

#63

Post by Waterluvian »

CraigJPerry wrote: post #58
Waterluvian wrote: post #55

Earlier quoted context omitted.

The thing that tires me about this old chestnut is that it’s just a shell game and not actually a substantive idea. “Just take money from somewhere else.” It’s akin to saying that we will use the latest AI technology and machine learning to make our value proposition work. It sounds satisfying but it says nothing.

Why would you need to take money from somewhere else? This is misunderstanding how money works for a currency issuer.

Lol exactly. “Just print more money!”

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

#64

Post by theGnuMe »

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's certainly enough resources for this and any medical treatment.

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

#65

Post by robocat »

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 .

> because it’s cheaper.

Compromises always have to be made because the world does not have infinite resources. Unfortunate, but true.

One way to allocate resources between all our competing needs is by using money.

You can come up with other systems of allocation, however all systems will be unfair and arbitrary in surprising ways. There is always a need to choose between ugly choice A and ugly choice B, because our resources are constrained.

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

#66

Post by gambiting »

linuxftw wrote: post #57
gambiting wrote: post #29

Earlier quoted context omitted.

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…

That would imply that people are dying right now due to NHS rationing resources, which NHS will never admit to.

Well no, but it could be used to(for example) shorten the ambulance waiting time, and that alone would save more than 1600 people.

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

#67

Post by fnord77 »

bglazer wrote: post #21
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 ex…

> probably after killing most existing bone marrow through some really unpleasant procedures

usually a massive dose of radiation, right?

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

#68

Post by kiba »

gambiting wrote: post #29
shadowgovt wrote: post #20

Earlier quoted context omitted.

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…

That assume the technology never gets cheaper over time. I would hope it gets better over time as we cure more and more rare diseases.

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

#69

Post by meese712 »

theGnuMe wrote: post #62
meese712 wrote: post #59

Earlier quoted context omitted.

I'm confused how this would reduce disease progression compared to a much cheaper allo bone marrow transplant then if they are only modifying hematopoietic stem cells since a BMT is just doing the exact same thing except with someone else's non affected cells. BMTs are a horrific procedure though so this definitely has an advantage in that regard. I would also have to imagine they would have to do myeloablative chemo…

Well it removes graft vs host complications which is a win and the requirement to take immunosuppressants for life.

Definitely true GVHD sucks. The article gave me the impression that they were saying it was somehow superior at stopping the disease because it said this "stem cell transplants, have sometimes been used to slow the disorder's progression in infants,"

(fyi a decent amount of stem cell transplant survivors do not have to take immunosuppressants for life)

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

#70

Post by [deleted] »

theGnuMe wrote: post #62
meese712 wrote: post #59

Earlier quoted context omitted.

I'm confused how this would reduce disease progression compared to a much cheaper allo bone marrow transplant then if they are only modifying hematopoietic stem cells since a BMT is just doing the exact same thing except with someone else's non affected cells. BMTs are a horrific procedure though so this definitely has an advantage in that regard. I would also have to imagine they would have to do myeloablative chemo…

Well it removes graft vs host complications which is a win and the requirement to take immunosuppressants for life.

[deleted]
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