Study Rationale:
We are testing ANAVEX2-73 (also known as blarcamesine), which previous research has shown helps improve behaviors as well as normalizes biochemical changes in a Parkinson’s disease animal model (6OHDA, which was supported by MJFF). In 132 patients with Parkinson’s disease dementia, the drug significantly improved cognitive function and memory as well as REM sleep. This includes complex cognitive tasks that impact quality of life such as making a choice between similar objects and remembering daily personal experiences, which could be impaired in Parkinson’s disease. In patients with Alzheimer’s disease a Phase 2a trial demonstrated a concentration dependent response in both cognition (MMSE) and function (ADCS-ADL) over 148 weeks (longer than 3 years).
The drug works by activating the Sigma-1 receptor protein in the brain. This protein helps brain cells stay healthy by reducing the effects of certain kinds of stress, preventing toxic proteins from building up in brain cells, and possibly protecting brain cells in other ways. This study is an important step in discovering if activating this protein will slow or reverse damage to brain cells and help them work normally again, thereby slowing or stopping Parkinson's progression.
Hypothesis:
We want to know if this new drug, ANAVEX2-73 (blarcamesine) can safely travel through the body to the final destination in parts of the brain most affected by Parkinson’s disease, which will help us determine if this new drug can help people with Parkinson’s disease.
Study Design:
In this study, researchers will give ANAVEX2-73 (blarcamesine) to up to 24 patients with Parkinson’s disease and healthy volunteers. The drug will be bound to a special tag or marker which will allow us to visualize the drug as it moves through the body to brain, using a medical imaging tool called Positron Emission Tomography (PET).
The researchers will also take blood samples from patients to learn about how the drug breaks down in the body to make sure it is safe and has minimal side effects.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
This project will help us understand if this drug can safely make its way to the brain cells that are affected by Parkinson’s disease. This study may lead to a new treatment for Parkinson’s disease that will reduce or even reverse symptoms with improvements in the ability to walk, talk, eat, and smile, especially when combined with other medications.
Next Steps for Development:
If this study is successful, the drug will be tested in more patients and at different doses. This “efficacy testing” would determine the best dose of the drug to help people with Parkinson’s disease and whether it performs better than existing medications for Parkinson’s disease (or if a combination treatment is better).