First UK Child To Receive Gene Therapy For Fatal Genetic Disorder Is Now Healthy 11
A 19-month-old girl named Teddi recently became the first child in the U.K. outside a clinical trial to receive a new gene therapy for metachromatic leukodystrophy (MLD), a fatal genetic disorder, the National Health Service (NHS) announced. Roughly six months out from treatment, "Teddi is a happy and healthy toddler showing no signs of the devastating disease she was born with," the NHS statement reads. Live Science reports: The genetic disorder MLD disrupts cells' ability to break down sulfatides, a fatty material used to insulate the wiring that runs through the white matter of the brain and much of the nervous system beyond the brain. Sulfatide buildup destroys brain and nerve cells, resulting in cognitive problems, a loss of motor control and sensation, seizures, paralysis and blindness, according to the Genetic and Rare Diseases Information Center. Eventually, the disorder leads to death. [...]
The new gene therapy, called Libmeldy (generic name atidarsagene autotemcel), was only recently cleared for use by the NHS and works by inserting into the body working copies of the genes that are faulty in MLD, thus restoring the ability to break down sulfatides. 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 (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.
The new gene therapy, called Libmeldy (generic name atidarsagene autotemcel), was only recently cleared for use by the NHS and works by inserting into the body working copies of the genes that are faulty in MLD, thus restoring the ability to break down sulfatides. 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 (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.
Good (Score:5, Interesting)
Gene editing is here to stay, and that's a good thing. The already existent laws against assault and violence make it illegal to do something harmful with it. As for idiots who think gene editing can be banned, they are really stupid. You cannot hope to enforce any ban on gene editing any more than you can prevent say fentanyl synthesis .. in fact the latter is harder. Gene editing can be done cheap, like it or not. All you need is a decently equipped lab, in fact many DIY Biologists have labs that are adequate for it. None of the equipment needed is particularly hard to build or purchase. Biohackers have been doing gene editing for many years now, google it.
Re:Good (Score:5, Insightful)
The rare diseases are much easier to cure because the body is more likely to have most of its defenses doing the heavy lifting. For example, curing a genetic condition involves simply fixing the gene. Diabetes on the other hand is much harder. The whole immune system is configured against it, and it is not just a matter of replacing the insulin producing cells but you have to decide how it can be protected from the immune system mounting an attack. That said there are many companies working on viable approaches to curing diabetes, everything from encapsulated beta cells to immune system modification or a combination of that. I believe Sernova and Viacyte (PEC-Encap) are the closest to market on a functional cure but many other companies are showing almost the same amount of progress.
Re: (Score:3)
The rare diseases are much easier to cure because the body is more likely to have most of its defenses doing the heavy lifting. For example, curing a genetic condition involves simply fixing the gene.
There's no such thing as "simply" when you're talking about an entire person. What made this relatively simple is that they can replace the bone marrow and the resulting immune cells will break down the excess sulfatide on their own, without having to update the genes of every cell in your body, or even a single solid organ.
Diabetes on the other hand is much harder.
Interestingly enough, the immune system attacking sulfatide could actually be responsible both type 1 and type 2 diabetes, so this could actually be really, really close to doing just t
Re:Good (Score:5, Insightful)
This is all fine and good for rare diseases that the pharma industry had no intention of curing anyway.
You said rare disease, but what about non-rare afflictions such as smallpox? Think of the billions of dollars the pharma industry has lost over the decades since they wiped it off the face of the planet.
Or did you mean polio which, except for a few small isolated pockets in third world countries and the U.S., has also been cleared from the planet? Rinderpest ring a bell?
Tell us, oh sage of the medical industry, how are pharmaceutical companies supposed to rid people of diseases which are genetically based, such as this girl? You want them to come up with a pill or shot? Explain how that is supposed to work, because I can guarantee people like you wouldn't take such medication even if it were possible because you'd be whining about being tracked or having your DNA altered.
Ever wondered why researchers always work on cures for totally anectodal diseases that almost nobody has and never on stuff that a lot of people suffer from?
Ever wonder how discoveriess in one area can lead to discoveries in other areas? Viagra for example? It's almost as if when you learn something about one disease or affliction you then look to see if that knowledge can be used elsewhere. Who would have thought such a thing exists?
Re:Good (Score:4, Interesting)
Okay I'll bite:
- Smallpox vaccine: invented by one dude in the 18th century and developed with help from public money (a grant from the King of England). Then vaccination programs mostly driven by publicly funded institutions - WHO and CDC - for the past 70 years.
- Polio vaccine: developed by a researcher from the U. of Michigan with public money. Vaccination programs mostly driven by WHO for the past 70 years.
- Rinderpest vaccine: developed by a vet working for the Filipino government (public money) in the 1920's. Vaccination efforts funded by the WOIE, IAEA and FAO (public money).
- Viagra: developed by Pfizer as a for-profit heart medication. Then Pfizer realized there's much more profit to be had in giving boners to wealthy elderly men, so they rebranded it.
Your attempt are cynicism would be more convincing if your examples didn't support the exact opposite of what you're trying to say.
Re: (Score:3)
The already existent laws against assault and violence make it illegal to do something harmful with it.
Not really. It hasn't been tested in court yet.
Think outside the USA (Score:2)
'The already existent laws against assault and violence make it illegal to do something harmful with it.'
Whilst such laws may constrain behaviour within Western countries, it won't prevent abuses in countries with weaker or non-existence legal frameworks; the fact that the mad scientist with a secret lab trope is mainstay of many bad SF books doesn't mean it's not valid...
https://www.dailymail.co.uk/ne... [dailymail.co.uk]
Re: (Score:2)
Yes I compared it to fentanyl production, which is illegal but rogue people do it, regardless of how badly we don't want them to.