Stem Cell Therapy Aims to Rebuild Brain Circuits in Parkinson's Patients with Dr. Howard Federoff Kenai Therapeutics

Dr. Howard Federoff, Scientific Co-Founder, Chief Medical Officer, and Executive Vice President of Corporate Medicine and Science at Kenai Therapeutics, is developing a cell therapy for Parkinson's disease that involves transplanting IPSCs into the brain to replace lost dopamine neurons. The goal is not to cure the underlying cause of the disease, but to restore motor function and reverse the disease's progression. Initial clinical focus was on patients with moderate to moderate-severe idiopathic Parkinson's, showing efficacy and safety, leading to expanding the study to include earlier-stage and familial forms of the disease.

Howard explains, "Kenai was formed about three and a half years ago, and the intention is to develop a cellular product, which means that what is manufactured will be eligible in the right patient groups who have a diagnosis of Parkinson's to be placed into the brain. And consequent to its placement, the cells will then form new circuits, dopamine circuits that are lost owing to the disease diagnosis that will have occurred years earlier."

"The induced pluripotent stem cell approach is one we favor for several reasons. The nature of what then becomes the IPSC, as we like to call it, starts with a normal human volunteer whose medical history is very detailed and does not contain any familial personal history of neurodegeneration. That cell then undergoes a process called reprogramming. And in our case, it's done with a slightly different approach than many others. And the reprogramming effectively creates a cell that is pluripotent, meaning it can become any cell of the body. That's when it is designated as an induced pluripotent stem cell."

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