Shake It Up is pleased to announce co-funding of two new research projects this month with our partners at The Michael J. Fox Foundation for Parkinson’s Research.
INVESTIGATING THE ROLE OF REPEAT EXPANSIONS AND MITOCHONDRIAL DYSFUNCTION IN PARKINSON’S DISEASE.
Researcher: Professor Melanie Bahlo PhD FAHMS
Institution: The Walter and Eliza Hall Institute of Medical Research
Study Rationale: Both genetic mutations and exposure to environmental risk factors contribute to causing Parkinson’s disease (PD). Genetic risk for a PD patient could be due to multiple genes, different types of mutations or, simply, one genetic mutation, in one gene. Our team have developed novel computational tools that enable us to discover several new types of mutations and other genetic signals of PD in a person’s DNA. We will leverage these unique tools to investigate a new source of mutations, called repeat expansions, which we have already proved to be the genetic cause of diseases that are similar to PD.
VALIDATION OF RRX-001 AS A NOVEL DISEASE-MODIFYING THERAPEUTIC FOR PARKINSON’S DISEASE
Project Title: Validation of RRx-001 as a novel disease-modifying therapeutic for Parkinson’s disease
Study Rationale:
Our project will test the effectiveness of a new treatment for Parkinson’s disease (PD), called RRx-001. We believe this drug could be beneficial in PD by blocking inflammasome activation and other mechanisms which drives persistent inflammation that is linked to PD progression.
Hypothesis:
We propose that RRx-001 could be a safe and effective treatment for Parkinson’s based on our results in animal models and our ongoing human clinical trials with this drug in other conditions.
Study Design:
This project will test the effectiveness of RRx-001 in multiple animal models of Parkinson’s disease and also in cell culture studies. We will determine if RRx-001 can prevent or rescue key aspects of Parkinson’s pathology in disease models, which would indicate that this drug could have potential to work in human studies.
Impact on Diagnosis/Treatment of Parkinson’s disease:
There are currently no treatments for PD. Our drug has been shown to be safe and well-tolerated in human clinical trials date with no major adverse events reported. Therefore, if our testing of RRx-001 in PD models is successful in this project, it can directly enter clinical trials for PD as a new treatment.
Next Steps for Development:
If results of our testing in animal models are positive, we will seek additional funding to progress this drug into human clinical trials.
