Both the boys were aged seven years when the therapy was done two years back.
Dr. Katherine A. High of the Children’s Hospital of Philadelphia was quoted as saying, “This is a disease that never, ever stabilizes” on its own.
She further added, “The fact that they were able to achieve that means they are getting a therapeutic effect.”
This is the third successful gene therapy treatment done in recent weeks and the fifth disease for which gene therapy has proved to be helpful.
Dr. Theodore Friedmann of UC San Diego said, “That’s a major achievement for a field that has been in the clinic for only 18 or 19 years. This is a new form of medicine and deserves to be seen as such.”
Apart from the two boys mentioned above, the French team has already successfully treated a third boy who has the disease. At present, the details of this case are unavailable.
The research team is planning to extend the trial to other age groups too, including older men suffering from a milder form of the same disease.
The research procedure
The latest research was carried out by a team headed by Dr. Nathalie Cartier and Dr. Patrick Aubourg of Paris Descartes University.
A healthy form of the ALD gene was inserted into an HIV virus that had been “defanged” so that it became incapable of causing the disease.
The research team segregated bone-marrow stem cells from the two boys. The HIV virus was then used to introduce the healthy ALD gene into both of them.
The researchers then performed a surgery that was similar to that of a bone-marrow transplant. They wiped out the marrow of the boys and introduced the new modified cells, which multiply to form a new marrow.
It was found that almost 15 percent of the cells started the production of the desired protein, and from there on the production has continued for the last two years in both the boys.
This effect was similar to the one that can be seen after a successful bone-marrow transplant.
“That’s good news because many patients don’t have access to bone-marrow transplants (that are good matches), and it is not an innocuous procedure,” Friedmann said.
The research team stressed that the new procedure is safe. Explaining the utility of gene therapy, Friedmann said that gene therapy “has crossed a threshold, scientifically and medically, and also in credibility.”
Adrenoleukodystrophy-- one of the worst neurological disorders
About 120 young boys are diagnosed with adrenoleukodystrophy (ALD) in the U.S. each year. Dr. Florian Eichler of Massachusetts General Hospital, explained that children born with the faulty ALD gene appear to be normal till up to about five years of age.
“When a really catastrophic process of progressive, relentless demyelination (of the brain) sets in that leaves them vegetative or dead within one to two years,” Dr. Eichler said.
The progression of the deadly disease can be slowed down only if it is diagnosed before brain deterioration starts. For stopping the progression, Lorenzo’s oil-- a blend of fats from olive and rapeseed oils-- is utilized. This oil supposedly brings down unusually huge levels of harmful long-chain fatty acids in the brain.
However, if the brain deterioration begins, the only option left is a bone marrow transplant. If a child has a closely matched sibling, the transplant can delay the progression of the disease.
Although transplant from a less closely matched donor can also prove to be helpful, it can have major side-effects too. For instance, some patients might have to use a wheelchair after the transplant.
The latest study findings have been reported in the journal Science.
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