Dendrimer-Based Therapy Shows Promise For Cerebral Palsy

Newborn children with cerebral palsy might in the future be spared from living with some of the cognitive and motor-skill impairments caused by the disorder. That’s because Sujatha Kannan and Rangaramanujam M. Kannan, now at Johns Hopkins University School of Medicine; Roberto Romero of the National Institutes of Health; and coworkers have developed a dendrimer-drug nanomedicine to treat brain inflammation associated with cerebral palsy (Sci. Transl. Med., DOI: 10.1126/scitranslmed.3003162). The developmental disorder is caused by injury to a baby’s brain either in the womb or soon after birth. The researchers tested their dendrimer-based therapy, which has a branched polyamidoamine core decorated with an outer shell of N-acetyl-l-cysteine (NAC) groups, on baby rabbits with cerebral palsy. The rabbits were given a dose of the nanomedicine within six hours of birth, and after five days, the animals displayed almost normal motor skills, as well as improvement to the damaged white matter in their brains. The researchers think the dendrimer ferries the drug NAC into brain cells and releases it. There, NAC, a building block of glutathione, helps replenish the antioxidant, which usually protects cells from oxidative damage but is depleted in cerebral palsy patients.