Parkinsons disease patients and stroke victims have received transplants of fully formed brain cells before, but the malleable brain cells involved here have never before been implanted.
Batten disease is caused by a defective gene that fails to create an enzyme needed in the brain to help dispose of brain cellular waste. The waste piles up and kills healthy cells until the patient dies. Most victims die before they reach their teens.
The idea is to inject the sick kids with healthy, immature neural stem cells that will engraft in a brain that will direct them to turn into cells able to produce the missing enzyme.
Such an experiment showed promise in Batten-afflicted mice, but such an ethically charged test has never been tried before in humans.