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Candidate Parkinson’s Drug Advances to Phase 3 Trial

The Michael J Fox Foundation has reported today, 1st September 2015, that researchers testing the compound inosine for its effect on Parkinson’s progression have funding from the National Institutes of Health for a Phase III trial, the last step before regulatory approval. Inosine is a precursor to the antioxidant of urate.

The Michael J. Fox Foundation’s largest grant to a single investigator thus far awarded $5.6M in 2008 to Michael Schwarzschild, PhD, of Massachusetts General Hospital for a Phase II trial of inosine. The MJFF-funded Phase II study showed that inosine is safe, tolerable and does raise urate levels in people with early-stage PD.

Because inosine is already commercially available, Foundation and government funding is essential for testing its efficacy as a Parkinson’s treatment. Pharmaceutical companies are not incentivized to invest in testing of a compound already on the market.

A new paper from Dr. Schwarzschild’s team (from non-MJFF funded research), however, points to a urate-specific role in neuroprotection. This finding grows the evidence for likely disease modification with inosine.

The investigators report that urate stimulates brain cells called astrocytes, the first step in a chain reaction releasing another antioxidant and activating a protein pathway. Both may protect brain cells from degeneration.

Journal Reference:
Rachit Bakshi, Hong Zhang, Robert Logan, Ila Joshi, Yuehang Xu, Xiqun Chen, Michael A. Schwarzschild. Neuroprotective effects of urate are mediated by augmenting astrocytic glutathione synthesis and release. Neurobiology of Disease, 2015; DOI: 10.1016/j.nbd.2015.08.022

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