Study Rationale: Parkinson’s disease (PD) damages brain cells that control movement, largely due to problems in the cells’ energy factories, called mitochondria. A protein called VDAC1 helps regulate mitochondria function, but in PD it can form harmful clusters (oligomers), triggering inflammation, stress, and brain cell death. X-tosis has developed VBIT-4, a drug designed to prevent VDAC1 from forming these clusters, helping protect brain cells and slow disease progression. Laboratory studies in Parkinson’s model have shown that VBIT-4 can reduce PD-associated brain damage and improve motor function. This project will advance VBIT-4 closer to clinical trials by testing the drug and its effect on related biomarkers.
Hypothesis: We believe that blocking VDAC1 oligomerization will protect mitochondria, prevent brain cell death, and slow Parkinson’s progression. We also believe that measuring VDAC1 and related disease-specific biomarkers in blood and spinal fluid will help identify which patients may benefit most from this treatment.
Study Design: We will test VBIT-4 in a laboratory model of PD that mimics early brain changes linked to toxic alpha-synuclein protein buildup. We will assess how VBIT-4 affects brain degeneration and disease-relevant biomarkers, as well as VDAC1 levels. In parallel, we will analyze blood and spinal fluid samples from people with Parkinson’s and healthy individuals to measure VDAC1 and related biomarkers. This combined approach will help guide future clinical studies and patient selection. Finally, we will advance development tasks that are required for early discussions with regulatory agencies.
Impact on Diagnosis/Treatment of PD: This project may lead to a new treatment that slows or stops Parkinson’s progression, rather than just treating symptoms. It may also help develop simple tests using blood or spinal fluid to select patients for treatment and track how well the drug is working.
Next Steps for Development: If successful, the next steps will include safety studies and preparation for human clinical trials. Biomarker results will guide patient selection and help design early studies to test whether the drug is safe, reaches its target, and shows signs of benefit for people with PD.