LRRK2 back on track

The LRRK2 Safety Initiative was put together by The Michael J. Fox Foundation for Parkinson's Research (MJFF) after the Genentech Inc. unit of Roche raised red flags about the target's safety. While the results have sufficiently allayed concerns to keep inhibitor programs alive in at least three pharmas, they still leave open questions regarding lysosomal biology and the impact this class of compounds could have on the brain.

In February 2015, researchers from Genentech published data in Science Translational Medicineshowing two of its LRRK2 inhibitors caused lesions in the lungs of cynomolgus monkeys. Rather than see the target abandoned, MJFF thought it was worth exploring the basis of the toxicity. ...