Medical breakthrough uses HIV for good
Posted 11/6/09 , edited 11/6/09

New gene therapy halts 2 boys' rare brain disease

By LAURAN NEERGAARD, AP Medical Writer Lauran Neergaard, Ap Medical Writer – Thu Nov 5, 5:12 pm ET

WASHINGTON – French scientists mixed gene therapy and bone marrow transplants in two boys to seemingly halt a brain disease that can kill by adolescence. The surprise ingredient: They disabled the HIV virus so it couldn't cause AIDS, and then used it to carry in the healthy new gene.

The experiment marks the first time researchers have tried that long-contemplated step in people — and the first effective gene therapy against a severe brain disease, said lead researcher Dr. Patrick Aubourg of the University Paris-Descartes.

Although it's a small, first-step study, it has "exciting implications" for other blood and immune disorders that had been feared beyond gene therapy's reach, said Dr. Kenneth Cornetta, president of the American Society of Gene and Cell Therapy.

"This study shows the power of combining gene therapy and cell therapy," added Cornetta, whose own lab at Indiana University has long researched how to safely develop gene delivery using lentiviruses, HIV's family.

The research was published in Friday's edition of the journal Science.

In 20 years of gene therapy research, there have been few home runs and some headline-making setbacks — including a risk of leukemia caused by otherwise successful gene therapy for another rare disorder, "bubble boy disease." That's a risk that specialists hope a lentivirus-based gene therapy will eliminate.

Best known from the movie "Lorenzo's Oil", adrenoleukodystrophy, or ALD, is a rare genetic disease that, in its most devastating form, destroys the coating of nerve fibers in boys' brains. Without that coating, called myelin, the neurological system breaks down. The disease typically strikes between the ages of four and 10, leading to blindness, deafness, dementia and loss of muscle control, and killing them within a few years.

Bone marrow transplants can halt ALD by letting new myelin-forming stem cells take root. But it's difficult to find a matching marrow donor, and the transplant itself is very risky.

So what if stem cells from the boys' own bone marrow could be genetically corrected, eliminating the ALD mutation? To do that, Aubourg's team had to overcome a technical hurdle: Gene therapy works when scientists harness deliver a healthy new gene by attaching to a virus that can harmlessly infect cells. But none of today's so-called gene therapy "vectors" could penetrate enough of the stem cells needed for an ALD treatment to work.

Unlike most viruses, HIV can penetrate stem cells, and it sticks permanently. So Aubourg's team removed the genetic parts of HIV that make it dangerous, leaving basically a scaffolding to carry the new therapeutic gene.

Then they culled stem cells from two 7-year-old boys in the early stages of ALD, and mixed in the healthy gene. The boys underwent bone marrow-destroying chemotherapy and then had their genetically corrected stem cells reinserted.

Two years later, the boys have shown no sign of worsening brain damage and are functioning well with 15 percent of their blood cells producing the healthy protein, said Aubourg, who plans to test the experimental procedure in more patients. An advocacy group, the Stop ALD Foundation, is working to raise money for a similar U.S. study.

http://news.yahoo.com/s/ap/us_med_gene_therapy
Yay xD
Think of the possibilities it may lead to! XD
Share your thoughts
If this isn't worth being a thread, lock it up (was bored)~ XD
49717 cr points
Send Message: Send PM GB Post
76 / Unreachable Dream...
Offline
Posted 11/6/09
A breakthrough indeed. Hope tis really safe.
Posted 11/6/09

mikomiko123 wrote:

A breakthrough indeed. Hope tis really safe.


lets hope. that would suck to have HIV in you and it "somehow" was still active and turn into AIDS. that would suck.
~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~
for the negative part (since i'm a negative asshole) why don't they TRY to find a cure for HIV/AIDS instead of finding a cure for something else. if they spent all their research into findthing a cure for HIV/AIDS instead of that i think we could have found a cure by now
11039 cr points
Send Message: Send PM GB Post
29 / M
Offline
Posted 11/6/09
We've actually been trying lentiviruses as vectors for a while now - this is a good step but there has been A LOT of failure leading up to it. Most therapies that seem promising in test-tubes dont work well in humans - plus think about having to get a bone marrow transplant to administer a treatment like the above article - not a very pleasant treatment.

Gene therapy works best with rare disease like above cause they can be traced to one mutation or at least very few. Due to complexities of the more well know disease like cancer, it will be a long time before we see gene therapy there. That said the AIDS virus has a lot of potential due to its natural mechanism - very interesting approach.
49717 cr points
Send Message: Send PM GB Post
76 / Unreachable Dream...
Offline
Posted 11/6/09

Your_Typical_Friend wrote:


mikomiko123 wrote:

A breakthrough indeed. Hope tis really safe.


lets hope. that would suck to have HIV in you and it "somehow" was still active and turn into AIDS. that would suck.
~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~
for the negative part (since i'm a negative asshole) why don't they TRY to find a cure for HIV/AIDS instead of finding a cure for something else. if they spent all their research into findthing a cure for HIV/AIDS instead of that i think we could have found a cure by now


How was that negative? Lol

92114 cr points
Send Message: Send PM GB Post
27
Offline
Posted 11/6/09
my only thought on this is are the boy technically infected with the virus, and if so what about their possible future children.
15149 cr points
Send Message: Send PM GB Post
F / Where you are?
Offline
Posted 11/6/09
Don't be nasty you won't get and you wouldn't need a cure.
You must be logged in to post.