Researcher Explores Potential Treatment for Lou Gehrig’s Disease

Professor Li Niu in the Department of Chemistry is making strides in the fight against Lou Gehrig’s disease or Amyotrophic Lateral Sclerosis (ALS).

The Human BrainNiu holds four patents and has two pending patent applications for RNA aptamers, which is a novel class of drug candidates for the potential treatment of ALS.

ALS is a progressive neurodegenerative disease characterized by selective degeneration of lower motor neurons in the spinal cord and brainstem and upper motor neurons in the motor cortex. The disease is characterized, as well, by the loss of motor neurons which eliminates the brain’s ability to initiate and control muscle movement. The life expectancy of an ALS patient averages two to five years; currently there is no cure and few effective treatments.

Niu’s research involves a family of proteins called glutamate ion channel receptors, which are indispensable to brain functions such as memory and learning. When the receptors are excessively activated, extra calcium ions get into the cell, inducing cell death and making these channels potential targets for drugs against ALS.

The challenge remains that most inhibitors of glutamate ion channel receptors synthesized as potential ALS drugs are poorly water soluble and are generally non-specific organic compounds. Niu believes RNA molecules can be identified and used to inhibit glutamate ion channel receptors with good water solubility, higher potency and better selectivity. His research involves chemical modifications to improve stability or resistance to RNA-degrading enzymes so the aptamers have reasonable lifetime in vivo. His research group plans to test the effectiveness of these RNA aptamers in ALS animal models. The aptamers could also be used for potential treatment of epilepsy, stroke and other neurological diseases.

Niu’s research is supported by grants from the National Institutes of Health, the Department of Defense, and the Muscular Dystrophy Association.