C-Abl tyrosine kinase is a protein present in most of the cells in our bodies, including brain cells. Research has suggested that in some cases of Parkinson’s c-Abl is abnormally over-active and this contributes to nerve cell inflammation and eventual cell death. The biotech company, Inhibikase, has been developing a drug which dampens down abnormal c-Abl protein function and recently, they provided an update on its development.

Research shows that in Parkinson’s, abnormal c-Abl function contributes to poor energy regulation in nerve cells, inflammation stress, and accumulation of the toxic protein alpha-synuclein in models of Parkinson’s. Scientists have been trying to identify drugs that can inhibit the over-active c-Abl in the hope that this will slow the progression of Parkinson’s. Inhibikase’s drug, IkT-148009, is one such treatment being tested in phase 1 clinical studies.

Some years ago, Cure Parkinson’s supported a large clinical trial investigating the disease-modifying effects of a c-Abl drug called nilotinib. Nilotinib is already used to treat cancer, and we were hoping to repurpose this drug for Parkinson’s. When the trial results indicated that insufficient nilotinib had reached the brain, Inhibikase stepped in and developed a new drug, similar to nilotinib but better at accessing the brain and central nervous system.

This first-in-human phase 1 investigation involved 88 healthy elderly volunteers and 13 people with mild to moderate Parkinson’s. The drug was found to be safe and well tolerated, and caused no serious side-effects. Inhibikase is now setting up a second longer term study in people recently diagnosed with Parkinson’s (who are not taking medication) which they hope to start later this year.

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