Youths afflicted with adrenoleukodystropy – ALD – have a measure of hope for overcoming their condition and living a full and normal life, thanks to gene therapy as developed on mice.

It took more than a decade to refine the therapy, in which stem cells taken from the boys’ bone marrow were hacked with healthy copies of the gene, then returned to their bodies. Without them, the boys would soon be dead.

“They would now be unable to speak, to walk, to communicate, to sit, to eat. They would be in an advanced stage of the disease, in a vegetative state,” said Patrick Aubourg, a pediatric neurologist at France’s National Institute for Health and Medical Research who led the treatment’s development. “Instead they go to school. They live a normal life.”
[…]
One of the children — their identities remain confidential — received the treatment two and a half years ago. The other received it three years ago. Their story is described in a paper published Thursday in Science. In both, the disease has stopped progressing. Their brain scans show myelin damage that has stopped, and their new genes are active as ever.

The results are as striking as any previously delivered by gene therapy, a biotechnological technique that after nearly two decades of anticipation has largely failed to deliver on its lab-bench promise — though that may be changing.

“There is reason to think that this will last for the rest of their lives,” said gene therapist Nathalie Cartier of NIHMR, the study’s lead author.

I certainly hope so. These children deserve a chance to live as much as any human being. Even despite the cost of some mice, so long as they are dealt with humanely. And I am sure they are not tortured, nor are they expended in any way but judiciously and sparingly.

I am convinced that the anti-testing crowd are so privileged with a life devoid of any evil that they see saving children at the expense of lab mice as evil simply out of lack of anything to compare against. Honestly, had these people lived through, say, a war, or famine, or any sort of hardship at all, I’m sure they would not see saving infinite children’s lives at the expense of a few mice is such an evil thing.