Invention:
Researchers at the University of Arizona have developed a cell based assay system that can be utilized to develop a drug screen for compounds that normalize the phenotype of mutant neurons. This makes possible the development of a pharmaceutical that, when administered at a crucial stage, can effectively normalize brain development. Additionally, the system can also be used to determine which neuronal defects have occurred in patients afflicted with various developmental brain disorders. This provides physicians with the essential information utilized in deciding how to proceed with treatment.
Background:
The prevalence of mental retardation in today's society is a staggering 1-3% of the population. Unfortunately, no pharmaceutical exists today that functions to normalize brain development in children with mental retardation or other developmental brain disorders. Current therapies are limited to dietary manipulations in a subset of metabolic disorders to minimize accumulation of toxic chemicals in the brain, psychoactive compounds developed to treat adults with various forms of mental illness, anticonvulsants for seizure control, and intensive educational intervention programs. These therapy strategies necessitate the development of a therapeutic that can effectively treat various developmental brain disorders.
Advantages:
- Currently there are no therapeutics for the normalizing brain development
- Development of drugs that can be administered in a period of peak developmental brain plasticity to reduce neuron damage
Applications:
- Development of a drug screen for normalization of the phenotype of mutant neurons
- Identification of neuronal defects underlying developmental brain disorders
- Drug discovery for anti-cancer agents that reduce tumor metastasis
