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Validation of a Novel Mitochondrial Drug Target for Parkinson’s Disease

Study Rationale: Mitochondria provide cells with energy, but they also help regulate the flow of large amounts of calcium ions that flood neurons when they are active. In Parkinson’s disease (PD), mitochondria become overloaded with calcium ions. This excess calcium triggers the opening of a large pore in the mitochondrial membrane, which leads to the death of dopamine-producing brain cells. NRG Therapeutics is developing drugs that can inhibit the opening of this pore and thus prevent the loss of brain cells, and we have recently identified the mitochondrial protein with which these drugs interact.

Hypothesis: We hypothesize that learning more about the mitochondrial protein to which our drugs bind, and how this protein may become dysfunctional in PD, will facilitate progression of treatments that inhibit the opening of this pore into early clinical trials.

Study Design: Using cells grown in the laboratory that show biochemical changes related to PD, we will genetically delete the mitochondrial protein with which our drugs interact and assess whether this treatment improves cell health. We will also delete this protein in a preclinical animal model of PD that expresses human alpha-synuclein, a protein known to be a major toxic factor in PD. We will then determine whether deletion of this mitochondrial target protein improves PD symptoms.

Impact on Diagnosis/Treatment of Parkinson’s disease: NRG Therapeutics is developing inhibitors that enter the brain and prevent the opening of mitochondrial pores in dopamine-producing cells. These inhibitors hold significant potential to be developed as drugs that can protect the brain cells that are lost in PD and thereby slow or halt progression of the disease.

Next Steps for Development: The next step in development is to test the effect of these novel drugs in preclinical models of PD. If shown to be protective in these models, they would then be progressed into studies to assess their safety and tolerability in animals.


  • Neil Derek Miller, PhD

    Cambridge United Kingdom

  • Anthony Richard Rutter, PhD

    Cambridge United Kingdom

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