Oral GT-02287 seen to safely reach adult brain cells in Phase 1 trial

A new experimental treatment for Parkinson’s disease, called GT-02287, showed promising results in an early clinical trial. Healthy adults who received the treatment had about a 53% increase in the activity of an important enzyme called GCase, compared to those given a placebo. GCase helps cells clean out harmful waste, and problems with this enzyme are linked to Parkinson’s, especially in people with mutations in the GBA gene. GT-02287, taken as a pill, is designed to restore GCase function, prevent toxic protein buildup, and protect brain cells from damage. In mouse studies, the treatment improved movement, thinking skills, and nerve cell health. It even slowed or stopped Parkinson’s progression in mice, regardless of whether they had GBA mutations. The first human trial found GT-02287 to be safe and well-tolerated. It was able to reach the brain, where it could potentially slow disease progression. The next trial, starting soon, will test the drug in Parkinson’s patients over three months to further evaluate its safety and effects. Gain Therapeutics, the company developing GT-02287, plans to work with the U.S. Food and Drug Administration to advance the drug’s development. If successful, this treatment could offer hope to people with and without genetic causes of Parkinson’s.