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Supplemental Biomarkers Study to Clinical Trial for CoQ10

Objective/Rationale
There is a critical need for biomarkers of Parkinson’s disease (PD), for accurate and earlier diagnosis, and for monitoring treatment effects. We are investigating two potential blood tests as biomarkers for PD, based upon metabolomic and transcriptomic analysis, in patients entering a clinical trial of high dose CoQ10. We will determine whether the tests accurately diagnose PD, whether they reflect predicted effects of CoQ10 on metabolism and gene expression, and how this associates with CoQ10’s potential neuroprotective action.

Project Description
The QE3 clinical trial is being carried out in almost 70 centers across the US and Canada to test whether CoQ10 can slow down PD progression. We will ask 60 people with PD entering the study to provide blood samples before and after starting their assigned study drug (CoQ10 or placebo), and will compare their blood samples with those from people without PD. Samples will be analysed using metabolomic or transcriptomic analysis, to determine differences in patterns of metabolism and gene expression respectively. By comparing people with PD versus without PD, we will test whether our analyses can accurately diagnose PD. By comparing PD before versus PD during the CoQ10 trial, we will determine whether the tests can measure changes induced by the study drug, that could potentially be related to a neuroprotective effect.

Relevance to Diagnosis/Treatment of Parkinson’s Disease:  
This project may identify a blood test that could be used in early PD for accurate diagnosis, that would advance clinical treatment and to potentially allow preventive measures. These methods also may identify individual molecules that differ in PD, that could advance our knowledge of PD itself and possibly identify new targets for treatment. Finally, this project will set up a bank of well-defined blood samples that can be used to research future diagnostic tests.

Anticipated Outcome
We expect to learn whether sophisticated metabolomic or transcriptomic analysis can accurately diagnose early PD. We also expect to find whether these tests will be useful in detecting differences in metabolism or gene expression due to high dose CoQ10, that could relate to its potential neuroprotective effect. Importantly, we will establish a blood sample bank from a  group of individuals with PD who will be carefully followed over time. This will be invaluable for more advanced tests as they become available.

Progress Report

There is a critical need to discover biomarkers of Parkinson’s disease (PD), for better diagnosis, and to track treatment. We are investigating two blood tests, named metabolomics and transcriptomics, in people entering a clinical trial of high dose CoQ10. The QE3 clinical trial is currently being carried out in almost 70 centers across the US and Canada, and is testing whether CoQ10 can slow down PD progression. We are now in the process of collecting samples in 60 people with PD entering the trial. By comparing people with PD versus people without PD we will test whether our methods will accurately diagnose PD. By comparing blood samples taken before CoQ10 versus during the CoQ10 trial, we will see whether the tests can measure changes induced by the study drug potentially be related to a neuroprotective effect. Aside from their clinical use, these methods also have the potential to identify individual molecules that differ in PD, or after treatment, and that could advance our knowledge of PD itself. This project will also set up a repository of well-defined blood samples that can be used by researchers in the future.

Final Outcome

The NINDS-funded multi-center phase III clinical trial nicknamed “QE3” aimed to investigate possible protective effects of high doses of the antioxidant Coenzyme Q10 upon Parkinson’s disease progression. We used this opportunity to collect blood samples from a subset of people who enrolled into the QE3 study. All of these individuals had early PD, were not taking medications, and the pattern and severity of their symptoms were carefully measured. For comparison, we also collected blood samples from healthy individuals who did not have PD. These samples may now be used to develop and test new “biomarker” technologies to provide a means to better understand molecular changes in PD, and to help early and accurate diagnosis.


Researchers

  • Claire Henchcliffe, MD, DPhil

    New York, NY United States


  • Myron Flint Beal, MD

    New York, NY United States


  • Timothy R. Mhyre, PhD

    Washington United States


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