The Cure Parkinson's Trust (CPT) is delighted to announce that a novel anti-diabetic drug (MSCD-0160) has been approved by our research committee and trustees for further investigation in Parkinson's (PD) following previously funded work.

MSDC-0160 has some functional similarities to an earlier drug tested in PD models (pioglitazone) and targets the mitochondrial protein MPC (mitochondrial pyruvate carrier), which is a key controller of cellular metabolism. MSDC-0160 is a promising potential treatment for PD because it can be given in much higher doses than pioglitazone due to less side-effects. Consequently, MSDC-0160 most likely engages the target MPC more effectively in the brain.

The goal of this ongoing work is to determine both the level of interaction of MSDC-0160 with MPC and provide new evidence to continue the development of MSDC-0160 as a disease-modifying treatment for PD. This research is the next key step to bringing this drug into a clinical trial.

Pre-Clinical Testing of Bydureon/Exendin-4 with MSDC-0160

Also approved for funding is the testing of the two diabetic drugs, both individually and in combination, in a model of PD and a model of prodromal PD (a condition that includes the loss of the sense of smell and precedes onset of motor symptoms).

Scientists at Van Andel Research Institute in Michigan, USA will test the effects of Bydureon/Exendin-4 and MSDC-0160 on inflammation, protein clumping and neuronal death—key features of PD. A paper published in The Lancet this week reported on a clinical trial co-funded by The Cure Parkinson’s Trust that showed potential slowing of disease progression in PD by treatment with Exendin-4. Because the precise mechanisms of action likely differ between the two anti-diabetic drugs, MSDC-0160 and Exendin-4, the newly funded project will explore if the effects of the two drugs are additive in PD models, and suggest that combination therapies could be tested clinically in the future. Testing in a model of prodromal PD is particularly exciting because the long term goal is to develop therapies that can prevent the onset of PD motor symptoms in the future.

We are thrilled and grateful that The Cure Parkinson’s Trust will continue funding this important preclinical work. Our goal is to continue to build a strong foundation for our planned clinical trial, which will evaluate MSDC-0160’s safety and potential to slow the progression of Parkinson’s disease in humans.

Patrik Brundin - VARI Associate Director of Research Professor & Director of the Centre for Neurodegenerative Science

Exendin-4 has already shown some clinical promise as a disease-modifying drug and MSDC-0160 also has demonstrated pre-clinical evidence of positive outcomes.  It is hoped the results of this research will further support additional clinical trials involving diabetes treatments. This study will investigate whether the combination of these drugs has better potential disease-modifying effects than each drug tested independently. It will be the first time that two complementary agents have been studied in this way in Parkinson’s to understand if the combination is more potent and if it can change the course of the disease. It is our hope that the outcome of this research will provide evidence of the potential benefit of combining the two drugs clinically.

**A recent meeting hosted by CPT discussed the groundbreaking results of the recently published Bydureon trial and related research on GLP-1 agonists which hold promise for repurposing as potential Parkinson’s therapies. Read more...