Study: Gene therapy stabilizes fatal brain-wasting disease, ALD
gene therapy

Marking the third success for gene therapy in recent weeks, an experimental treatment by French researchers has led to the stabilization of the deadly degenerative disorder, adrenoleukodystrophy (ALD), in two young boys.

The breakthrough treatment, to be reported in the journal Science on Friday, comprised an effective blend of bone marrow stem cells and gene therapy to halt the rare and fatal brain-wasting illness that is inherited and affects only men.

The therapy, which was administered to the two seven-year old boys two years back, stabilized ALD in the both the boys.

Prior to the therapy, performed in France, both the boys were plagued with X-linked ALD, which was gradually stripping away layers of fatty acids protecting nerve fibres in their brains.

ALD is so deadly a disease that it slowly brings about a downward spiral of physical and mental disability, and ultimately death. It is so devastating that ALD disorder patients hardly reach adolescence. The appalling toll of the disease was depicted in the 1992 film “Lorenzo’s Oil”, based on the true story of the untiring struggle of an American couple, looking to find a cure for their ALD-affected son.

Hailing the experimental success of the therapy, Dr. Katherine High of the Children's Hospital of Philadelphia, who was not involved in the research, said: “This is a disease that never, ever stabilizes. The fact that they were able to achieve that means they are getting a therapeutic effect.”

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