Parkinson's in mice halted by blocking single enzyme

What's the news: Blocking a single enzyme prevents neuron loss and Parkinson's symptoms in mice.

Why should we believe it: This news is based on a new study published last week by scientists at Harvard Medical School and University of Cambridge. The scientists looked at the effects of blocking a single enzyme, called USP30.

USP30 depletion improves mitophagy, the process of clearing out old and nonfunctioning mitochondria in cells, including neurons. The result of blocking this enzyme in this study:

• Protected neurons, and reduced neuron loss in a mouse model of Parkinson's

• Significantly reduced symptoms of the disease

Why this is a big deal: The scientists first blocked this enzyme via a "knockout" model, where they genetically engineered mice that lack the gene to produce this enzyme.

But the second way was via a drug-like molecule, MTX115325, that was fed to mice and effectively blocked USP30. This points to the possibility that a similar treatment might work in humans.

So what's next: Human trials of course. The good news is, Mission Therapeutics, a Cambridge-based biotech firm, already has a pipeline of several projects to explore USP30 inhibitors in humans. Let's hope their trials soon show positive results.