First UK child to receive gene therapy for fatal genetic disorder is now healthy
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Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#2 > Libmeldy is made using stem cells that are derived from a patient's blood or bone marrow and can give rise to different types of blood cells, according to the European Medicines Agency(opens in new tab) (EMA). These stem cells carry the new, functional genes into the body, where they give rise to white blood cells that travel through the bloodstream.
> In clinical trials, Libmeldy offered clear benefits to infantile and juvenile patients who hadn't yet developed MLD symptoms; these patients were able to break down sulfatides at normal rates and showed typical patterns of motor development, for example. The benefit of the therapy seemed to last several years, but at this point, "it is not yet clear whether it will persist life-long, and extended follow-up is needed," the EMA noted.
> Libmeldy is approved for use in the European Union and U.K., although the U.K.'s drug price watchdog initially rejected the therapy due to its hefty list price of £2.8 million ($3.4 million at today's exchange rates), BBC News(opens in new tab) reported in 2022. The therapy's manufacturer, Orchard Therapeutics, then offered Libmeldy to the NHS at a significant discount.
> The gene therapy has not yet been approved by the U.S. Food and Drug Administration(opens in new tab).Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#3Related (somewhat):
First gene therapy for Tay-Sachs disease successfully given to two children - https://news.ycombinator.com/item?id=30408104 - Feb 2022 (77 comments)
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#4The 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?
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#5https://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.
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#6https://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 .
People are often surprised by this, but healthcare and insurance systems assign an explicit numerical value to human lives and run with it all the time.
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#7The 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?
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#8https://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.
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#9The 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?
In general it depends. If you modified existing blood cells genotype then it wont persist indefinitely. You'd need to modify the bone marrow. But then some cell lines work like you posed in your question.
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#10https://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 .