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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

#161

Post by refurb »

wheelerof4te wrote: post #81

How exactly do you edit a gene? Like, it is very, very small thing. How do you even measure it, let alone replace it?

Its similar to chemistry. You cant see molecules.

We have a set of tools that let you determine biological structures (sometimes entirely, sometimes only partially).

We also have a set of tools to modify genes. Some viruses already do this, so we use them. Were also using CRISPR which is based on the machinery of bacteria.

So you use the modification tools to change genes and the analytical tools to prove you did it correctly.

Of course the level of complexity is way higher since your dealing with a living organism and the modification tools (and analytical tools) aren't perfect, so there is a ton of trial and error to do to correctly.

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

#162

Post by refurb »

arpanetus wrote: post #74

you can't just simply state that he's healthy without actually seeing his lifespan

You cant but you can place a very high probability on it.

With genetic diseases often the difference between healthy and not is a single enzyme is missing.

If you can prove the enzyme level is restored and all the downstream effects match healthy individuals, youve basically restored them to what healthy people are.

Durability of the change is the big issue. Some gene therapies wane over time.

And of course if damage had occurred before the gene therapy, that may never go away.

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

#163

Post by refurb »

concordDance wrote: post #151
refurb wrote: post #130

Earlier quoted context omitted.

Sure it is. Look at the Blue Cross Blue Shield plan for Michigan. It covers a number of gene therapies at a cost of $2.2M for one of them. Not all European countries pay for it. https://www.bcbsm.com/amslibs/content/dam/public/employers/d...

You can get private insurance in Europe too.

For gene therapy?

The economics would never work out unless premiums were sky high or the insurance is mandatory or across most of your population.

It only works if premiums are spread across a large population so a $2.5M therapy becomes a $1 increase in premium for 2.5M people.

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

#164

Post by gambiting »

mckravchyk wrote: post #158
gambiting wrote: post #42

Earlier quoted context omitted.

What's more, Europe also has these private hospitals which aren't connected to the national healthcare system and which will provide you with top level care if you are willing to pay, no travel to US necessary. Private health insurance ( gasp! ) is also a thing, should you want to partake in that system.

Europe consists of many countries, and one of them is more than happy to take 9% / 5% of your income in exchange for one quite crappy public health care system - think whenever you need to see a specialist, you have to pay with your own money anyway (or wait months, years to get an appointment). If you want private health insurance, you have to fund the super expensive good for nothing public one anyway.

I...don't see how that's relevant to what I said, but.....ok? I don't really disagree.

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

#165

Post by tialaramex »

eternauta3k wrote: post #133
bcrosby95 wrote: post #106

Earlier quoted context omitted.

Anything dealing with human lives needs to assign a value to it. You don't want to spend $100 billion on something that's just gonna save 1 person. But spending $100 on something that's gonna save 1 person is something pretty much everyone is fine with. Somewhere in the middle is where most people disagree.

Saying yes to one person's expensive treatment is saying no to 4 people's cheap treatment.

It's not that simple. Economically it's an investment.

Twenty years ago I had Hodgkins Lymphoma. Obviously the NHS fixed that. This was doubtless quite expensive, it needed a team of experts, expensive chemicals and radiotherapy machinery. But, if they don't they're out a young productive software engineer because Hodgkins kills people, even assuming you're so callous your response is to just immediately put me down like a dog so as to reduce the cost of palliative care or diverting resources from relatives - Whereas since they fixed it that's maybe 30-40 years of economic productivity and ensuing tax income they're getting.

Now, obviously not every such intervention is literally profitable in a financial sense, but I chose my case because even boiled down to simply money this actually can make sense anyway. If you agree it made sense to fix me (and I don't see why you wouldn't) then beyond that it's only about value judgements.

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

#166

Post by stavros »

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…

My 3-year-old niece got her eyes treated for some degenerative disease in the same way. It's amazing what science can do now, a few months earlier and she would have gone blind before her next birthday.

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

#168

Post by gus_massa »

CraigJPerry wrote: post #157
concordDance wrote: post #150

Earlier quoted context omitted.

The real constraint is indeed what that money buys, e.g the aforementioned person hours. And if you print money and inflate then the price of goods rises. So sure, you might only pay a bit more for the treatment but now fruits are more expensive and your population gets slightly more unhealthy.

>> and inflate >> >> Inflation caused by excess money supply (NB: not inflation caused by excess money velocity) cannot survive taxation. Taxation destroys money supply.

So you are actually proposing to raise taxes. The problems is that everyone hate that, and will avoid paying taxes and/or vote for the opposition party.

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

#169

Post by whiddershins »

elil17 wrote: post #24
whiddershins wrote: post #13

Earlier quoted context omitted.

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?

because that is Dirigisme, which we reject for good reasons

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

#170

Post by elil17 »

pc86 wrote: post #54
elil17 wrote: post #46

Earlier quoted context omitted.

I’d just change the “or we won’t use it” to “or your patents go away.”

And then instead of reading "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment" today, we would have read something like "current regulatory environment precludes research and development expenditures into atidarsagene autotemcel" in some biopharm prospectus a decade ago. You can't have it both ways and preventing people from maki…

But you can have it both ways because most rare disease research is already funded by governments and nonprofits
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