While significant strides have been made toward therapies that can prevent, slow or stop Parkinson's disease (PD) progression, no currently approved Parkinson's treatment can do this. Therapeutics that target alpha-synuclein, the sticky protein that clumps in the brains of people with PD, may modify the course of disease. We are developing a new therapy that aims to prevent alpha-synuclein clumps from forming by engineering additional genetic material into immune cells (T cells) in a patient.
We expect the treatment to reduce clumping of alpha-synuclein in the brains of people with PD and to improve Parkinson's symptoms.
We created pre-clinical models of Parkinson's with alpha-synuclein clumps in the brain. In this study, we will treat the models with genetically modified immune cells, which can recognize and destroy alpha-synuclein clumps. We then will evaluate the amount of alpha-synuclein clumps and the extent of cell death in the models' brains. We also will monitor the movement of treated models. The results could allow us to estimate how effective the treatment would be in slowing disease progression and improving motor symptoms for people living with PD. A small test study we recently conducted demonstrated that the treatment can slow alpha-synuclein clumping and brain cell death.
Impact on Diagnosis/Treatment of Parkinson's disease:
Patients' own T cells have never been used to slow alpha-synuclein clumping in PD. This new treatment strategy is potentially more efficient when compared to other approaches, such as those that use antibodies. Cells can freely travel into the brain where alpha-synuclein clumps are located while antibodies -- proteins that immune cells make and release into the blood stream -- cannot. Therefore, using immune cells to remove alpha-synuclein clumps may be a more efficient approach.
Next Steps for Development:
Once pre-clinical studies have been completed, we will test this therapy in a clinical trial, which will be the first of its kind.