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.
