15 months ago, they removed his bone marrow, modified its genetic makeup to produce healthy red blood cells, and replaced it. He has been symptom free for 15 months. It's only one case, but if it can be replicated, as seems likely, it is an early huge step in the use of genomics to cure disease. There will be vastly more.
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Science is amazing
Awesome sauce, sucks it was the French and not us that developed it but still good news.
It's not a new technology. Lentivirus have been around since the 80s, but there are many hazards associated with the technology. Cancer and severe-immunological events are very possible side-effects.
It is my speculation that the French gov't is just allowing these Doctors to retry this technology with enhanced safe-guards, whereas the US FDA, which allowed previous gene therapy trials that resulted in death of several children, are going to be way more cautious and allow other countries prove the safety.
This technique would also be insanely expensive, especially with the constant monitoring safeguards.
If interested on more information, history, and complications of gene therapy, check this Wikipedia link.
Why? In the scientific community progress is progress and benefits the world.
Its about time LOLUVa accomplished something
Whoah whoah whoah. Let's just keep in mind there's French and then there's people who wish they were French, but are even more useless than the actual French.
Credit to the first group for this amazing scientific discover. Nothing but disdain for LOLUVa who is squarely in the second.
True... I guess its good they finally found some good use out of those white flags. Scientific formulas don't write themselves, you know!
As someone who has lived in France for the last 3+ years, I have to defend them slightly. It is very difficult to have Germany as your next-door neighbor. France set-up the Maginot Line after the 1st World War to help their defenses, but unfortunately Belgium stood as a nice detour.
The lesson learned there was basic Sun Tzu - Where your enemy is weak, strike. Where your enemy is strong, fade.
just...WOW!
Damn, I didn't even know French had a son with sickle cell.
All the legs!!!
Holy shit.
They fucking rewrote the programming of his bone marrow.
What a time to be alive.
Today, curing sickle cell anemia. In a few years
I keed I keed.
This is awesome. I can't even imagine how his parents must be feeling; like having a totally new kid, I'm sure. If I were that father, I'd be taking every staff member that helped my son with that therapy for drinks, even if they want to drink at the place that marks up its prices because it's next door to the hospital (looking at you, Baptist Health Applebee's!).
Gotta love science, technology, and medicine man. That is fucking awesome.
Now let's find a way to make it cheaper and available for the masses.
Thanks for sharing! My wife is a hematologist/oncologist and I really enjoyed this OT link. Amazing to read, curious to get her take on it. I also appreciated the follow up link/comment on DNA modification. Incredible...sickle cell affects so many more folks than we can imagine. Any breakthrough would be welcome news.
Please share your wife's opinion when you have a chance. I would love to hear it.
Spoke to my wife, in short:
Great research is being done, all across the globe. Unfortunately, until this science is picked up in the research triangles of America, and has been through long-term randomized studies, no single person or hospital will be willing to eat the high costs. The NCCN for cancer, for example, has to approve certain treatments as part of their guidelines for insurance companies to cover. Cancer, blood disorder treatments are very expensive. For example, my wife's initial co-pay is $400 for the uninsured. Many patients will come to her, without insurance, saying they cannot pay even that amount. She will still see them. Can you imagine if someone, insurance or not, wanted this medicine? Costs can reach $40K.
So, to answer your question: Dr's here are all for research and new drug trials. But until a drug company or medical group, with deep pockets can afford to prove it's worth on US soil, we won't see this become a mainstay. Treatments that are showing promise today are usually 5-10 years away from mainstream use.
I hope this helps!
I like science because science does incredible stuff like this! Too bad we will no longer financially support science in this country.
I may be reading this wrong, but this definitely sounds political, so
It also directly effects every major University in the US, VT included. The US is entering a new era where higher education itself is made into a political issue.
Politics may not be appropriate for this blog, and I agree. It'll be interesting to see how apolitical this site will remain as VT continues to take certain stances on political matters.