CNS Disease - The Scripps Research Institute/Opko Health

The lab of Phil LoGrasso, Scripps Florida's senior director of discovery biology and a professor of molecular therapeutics, is one that stands out in the area of central nervous system disease.

LoGrasso and his team's work revolves around a family of enzymes called kinases. The researchers have identified and validated a series of compounds that inhibit an enzyme called c-Jun N-terminal kinase, or JNK (pronounced "junk"), that have shown neuroprotective effects in a variety of experimental models of human diseases, particularly Parkinson's disease.

In previous studies, Scripps researchers found that JNK plays a central role in neuronal survival. During oxidative stress, JNK migrates to the mitochondria. Coupled with JNK activation, that migration is associated with numerous serious health issues, such as neuronal cell death, stroke and heart attack. Oxidative stress is implicated in a number of diseases that range from cancer and heart failure to Alzheimer's disease, amyotrophic lateral sclerosis (ALS) and Parkinson's disease.

In March 2012, Scripps inked a development and commercialization deal with Miami-based Opko Health ($OPK) for its novel compound SR-3306, which blocks the destruction of brain cells in animal models of Parkinson's disease. The compound has shown greater than 90% protection against induced cell death of primary dopaminergic neurons, while in mouse models of induced neuron death, protective levels were 72%.

"This is one of the best opportunities we have for the development of an effective neuroprotective treatment for Parkinson's patients," LoGrasso said in a previous statement.

Parkinson's disease is of huge interest to drug developers, as the dopamine-destroying disease affects about 1 million Americans. Currently available drugs for Parkinson's disease--including levodopa and MAO-B inhibitors--can keep symptoms of the disease at bay but not stop its progression.

LoGrasso's lab is hopeful that SR-3306 could potentially treat other diseases, too. In October 2012, the LoGrasso team won a $2.1 million grant from the U.S. Department of Defense to study its JNK-inhibiting compounds for treatment of ALS (Lou Gehrig's disease), a motor neuron disease.

In February, LoGrasso and his colleagues published new data on SR-3306, showing that the compound also protects tissue in animals after major ischemic events, such as heart attack and stroke.

If scientists are successful, SR-3306 could be the first compound of its kind to protect the brain from the devastating consequences of Parkinson's disease.

For more:
Compound preserves heart cells, tissue after heart attack or stroke damage

CNS Disease - The Scripps Research Institute/Opko Health
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