Study Rationale: TRPML1 is a promising drug target that offers the potential to slow the course of Parkinson’s disease. This study utilizes Bexorg’s revolutionary postmortem human brain platform to identify biomarkers that will be essential for successfully bringing TRPML1-targeted medicines to patients.
Hypothesis: By administering a TRPML1 activator to cellularly active, postmortem human Parkinson’s disease brain, this study aims to identify molecules that can be measured in clinical trials to detect the effect of the drug, increasing the probability of conducting successful trials.
Study Design: Brains are donated by patients for research following stringent guidelines that adhere to the highest ethical standards. Bexorg technology restores cellular function in donated postmortem brains, but prevents any activity associated with consciousness. A candidate therapeutic drug targeting TRPML1 will be administered to these brains, and the molecular response will be quantified to identify biomarkers for use in clinical trials.
Impact on Diagnosis/Treatment of Parkinson’s disease: A biomarker to measure the desired effect of a drug in clinical trials is essential for a successful outcome. Identifying such a biomarker for TRPML1 activators will increase the likelihood of bringing medicines that can slow the progression of Parkinson’s disease to patients. Currently, there are no approved drugs that slow the progression of Parkinson’s disease.
Next Steps for Development: A biomarker for TRPML1 activity will enable the initiation of clinical trials to test whether TRPML1 activators are safe and effective in treating Parkinson’s disease and related disorders.