Study Rationale: Parkinson’s disease (PD) is estimated to affect as many as 1 million Americans and up to 10 million people worldwide. The exact cause of PD is not yet fully understood, and currently available treatments can help manage symptoms but do not slow or stop disease progression. Some people with abnormal changes in a gene called Leucine-rich repeat kinase 2, known as LRRK2 have higher risk of developing PD. Overactive LRRK2 is thought to be one of the reasons for developing the disease. SNP614 is a drug called an antisense oligonucleotide (ASO) that can lower the amount of LRRK2. SNP614 has been shown in laboratory animals to be very effective and safe.
Hypothesis: SNP614 is safe and effective at lowering LRRK2 in the laboratory and in animal models, and by completing the set of studies, it can be given to patients with PD.
Study Design: In this project, we will conduct a set of studies that need to be completed before a new drug can be tested in humans. These tests are done in the laboratory and in animals to learn how the drug behaves in the body, whether it causes any harmful effects, and what amount is safe to give to patients. The goal is to make sure the drug is likely to be safe enough for a small number of PD patients to try it in a first clinical trial. All the results are included in a report sent to the FDA to get permission to begin human testing.
Impact on Diagnosis/Treatment of Parkinson’s disease: SNP614 can provide PD patients with a treatment option that can slow or even stop disease progression. SNP614 will be given to PD patients by injection into the fluid that surrounds and protects the brain and spinal cord. This drug delivery method is commonly used to treat disorders of the brain and is considered safe.
Next Steps for Development: Once we finish this study and get permission from FDA, we will start the human clinical trials. The first clinical trial will focus on making sure that SNP614 is safe in adults with PD.