What’s percolating in the field of Parkinson’s disease research? Scientists from Charles River and the Michael J. Fox Foundation discuss therapeutic advances, animal models and reproducibility.
The US National Institutes of Health estimates about 500,000 people are living with Parkinson’s disease, a neurodegenerative disorder that is perhaps most associated with tremors and movement problems.
The disease is triggered by a disruption in normal production of dopamine, a brain chemical critical to a slew of functions including movement, mood and attention. It’s not entirely clear what event or events throw off dopamine’s natural cycle, which is impeding our efforts to try and cure, prevent or even slow the progression of PD.
But there is hope. Research in the development of disease-modifying drugs are progressing well, with several candidates in late-stage clinical trials. Among the candidates are three drugs that target alpha-synuclein (α-syn), a protein found abundantly in the brain. In PD patients, fibril clumps of abnormal α-syn gradually accumulate and eventually, scientists believe, causes normal dopamine-producing cells from working properly. Other candidates include repurposed drugs that could be beneficial for PD.
“We have made significant progress in the last few years getting some good candidates into clinical trials,” says Terina Martinez, a Senior Associate of Research Programs at the Michael J. Fox Foundation for Parkinson’s Research. “And it is very important having these modifying therapies for Parkinson’s because the mainstay therapy is system support and that has not changed in decades.”
Martinez, who manages the Foundation’s emerging targets portfolio, oversees their preclinical and animal models program, and leads the Industry Tools Consortium, recently talked with Robert “Sandy” Hodgson, Director of Science R&D and CNS Pharmacology at Charles River Laboratories, to discuss the Parkinson’s research landscape. Along with some of the promising candidates coming down the pipeline, the discussion also dealt with the persistent problems in reproducing findings in vivo, choosing the right animal models, and the value of partnerships.
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