Joined together by JNK
Scripps Institute and OPKO Health announce deal for promising Parkinson’s therapy
JUPITER, Fla.—The Scripps Research Institute has announced aglobal license agreement with OPKO Health granting OPKO exclusive rights todevelop, manufacture and commercialize a novel compound that could potentiallyabate the devastating effects of Parkinson's disease. The compound, SR 3306,inhibits a class of enzymes called c-jun-N-terminal kinases (JNK) that areessential to neuron survival. If successful, SR 3306 could be the firsttreatment to prevent, slow or halt the progression of Parkinson's disease.
Parkinson's is a neurodegenerative disease caused by theloss of dopaminergic neurons in the substantia nigra, a part of the braininvolved in motor control. Thedopamine-transmitting neurons of the substantia nigra are responsible forinhibiting motor behaviors. As a result, the loss of these neurons rendersthose with Parkinson's disease unable to control their motor movements.
JNKs have been implicated in cell death through inhibitionof the JNK pathways. SR 3306 can potentially protect the brain against celldeath that is characteristic of Parkinson's and other neurodegenerativedisorders.
Lead researcher Dr. Philip LoGrasso, a professor in the Departmentof Molecular Therapeutics and senior director of drug discovery at Scripps'Jupiter, Fla., campus, has been studying the effects of SR 3306 for severalyears and recently published his findings in the American Chemical Societyjournal Chemical Neuroscience. Hisresults show that small doses of the drug produced a marked increase in neuronsurvival in cell cultures and animal models.
"It was a surprise that that level of neuroprotectionreduced the behavioral impact so strongly," LoGrasso says, "but it's indicativeof how it might perform in human patients. While SR-3306 doesn't represent acure, it does appear to have the potential of stopping the progression of thedisease."
Increasing the drug's market potential is its ability to beadministered orally, as well as the success it has had in preventing neurondeath in a rat model that mimics the physical symptoms of Parkinson's disease.
"These studies present compelling data on the first oral,brain-penetrating inhibitor to show significant efficacy in preventingneurodegeneration in both mouse and rat models of Parkinson's disease," saysLoGrasso.
Executives at OPKO are equally enthusiastic about thepotential SR 3306 has as a therapeutic for Parkinson's.
"We are excited to be working with Dr. LoGrasso and theScripps Research Institute to develop this important compound which couldprevent the progression of Parkinson's disease, and not just treat the symptomsof the disease," says Dr. Phillip Frost, chairman and CEO of OPKO.
There are currently no other drugs on the market that targetthe cause of Parkinson's disease, Frost notes. Current treatments such aslevodopa and MAO-B inhibitors are aimed at controlling symptoms. These drugswork by creating more dopamine in the brain to compensate for the loss ofdopaminergic neurons, thus reducing symptoms, but their effects only extend sofar.
"A drug like SR-3306 that prevents neurodegeneration wouldbe a quantum leap in the clinical treatment of Parkinson's disease because allcurrent therapies treat only the symptoms of the disease, not the underlyingpathologies," adds LoGrasso.
Though development of SR 3306 at OPKO is just beginning, thediscovery presents a promising avenue for treating Parkinson's disease, acondition that, according to the Parkinson's Disease Foundation, affects morethan 60,000 Americans and as many as 10 million people worldwide. According toLoGrasso, "This licensing agreement will help ensure that the development ofthis promising compound keeps moving forward. This is one of the bestopportunities we have for the development of an effective neuroprotectivetreatment for Parkinson's patients."
Financial details of the agreement were not disclosed.