An experimental gene therapy treatment appears to have helped eight children with a rare and incurable neurological disorder, although it may have been responsible for the death of one, researchers reported on Tuesday.
They said the treatment appeared safe and effective enough to try in more children with late infantile neuronal ceroidlipofuscinosis, or LINCL, a form of deadly Batten disease.
The treatment, in which a virus carrying the corrective gene was infused directly into the brain, appeared to slow the decline of eight out of 10 children treated, Dr. Ron Crystal of New York-Presbyterian Hospital/Weill Cornell Medical Center and colleagues reported.
“We are encouraged by this. It’s not a cure,” Crystal said in a telephone interview.
Like all forms of gene therapy, the hope is that the mutant cells will take up the new gene and start working normally.
Children with LINCL start showing symptoms at about age 4. They lose coordination, vision and speech and usually die unable to breathe on their own, between 10 and 12.
One child suffered an epileptic seizure weeks after treatment and died and another child died of unknown causes two years after treatment.
Eight of the children showed a measurable slowing of the inevitable decline usually seen in the condition.
Only about 200 children are alive with the disease globally at a given time.
“The disease is caused by mutations in the CLN2 (ceroid lipofuscinosis, neuronal 2) gene,” Crystal and colleagues wrote in their report, which was published in the journal Human Gene Therapy.
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