Empowered Patient Podcast Crossing the Blood-Brain Barrier to Treat ALS and Other Neurodegenerative Diseases with Stan Abel ProJenX
Stan Abel is the Chief Executive Officer of ProJenX, working on developing therapeutics to get into the brain to access the motor neurons that are dying in ALS and other brain and neurological diseases. Their drug prosetin is optimized to be bioavailable and administered in a small amount of liquid which is an advantage to someone living with ALS. Research is showing promise in increasing motor neuron survival in other neurodegenerative diseases like Parkinson's and perhaps Alzheimer's.
Stan explains, "In ALS, we need the therapeutics to get into the brain to access the motor neurons where the pathology is taking place and where those motor neurons are dying. The team at Columbia actually optimized a family of compounds. Not only were they screening these drugs to see that they were neuroprotective, and in these ALS models, they could see that we were actually really improving the survival of motor neurons in vitro, which means basically in a Petri dish. They optimized the compound so that it would easily cross the blood-brain barrier."
"These are two really important aspects. I think the advantage, in fact, that ProJenX has and prosetin has versus other programs that have been advanced in ALS is that this drug, all along the way, was based on ALS disease pathology. It was then was optimized to actually get into the brain and access the motor neurons that need the help."
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