Without risky bone marrow surgery before the first symptoms appear, children suffering from a rare brain disease - adenoleukodystrophy (ALD), which mostly affects boys - previously could not live longer than five years.
This vicious disease leads to the death of nerve cells in the brain and the sufferer loses the ability to move, speak and think. Even successful transplants led to permanent disabilities.
That is exactly why the new study, the results of which were published in the "New England Journal of Medicine", is so special. For the first time, doctors managed to cure ALD with the help of gene therapy.
After two years of gene therapy, a treatment expected to cost hundreds of thousands of dollars, 15 of 17 boys with the gene mutation that causes ALD did not develop symptoms. One of the remaining two, who died, was in an advanced stage of the disease when he started receiving treatment and there was no way to save him, while the other was withdrawn from research.
Gene therapy effectively halted the progression of the disease, explains study leader David A. Williams.
Therapy has its limitations, since it is necessary to start as early as possible.
Genes are delivered to the patient's cell by a deactivated form of HIV, and it takes a year for healthy cells to multiply in the bone marrow and reach the brain, where they play a role in protecting neurons.
The advantages are that patients do not have to wait for a donor, nor do they have to take immunosuppressants, which can have severe side effects, reports B92.
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