Strong results announced for new LRRK2 inhibitor clinical trial for Parkinson’s

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Author: Parkinson's Life editorsPublished: 10 January 2018

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The pharmaceutical company Denali Therapeutics has announced strong positive results from the first in-human LRRK2 inhibitor clinical trial.

The treatment lowers LRRK2 protein activity in body cells, which could ultimately have the potential to slow – or stop – Parkinson’s progression.

The small molecule inhibitor of LRRK2, called DNL201, achieved an average of more than 90% inhibition of LRRK2 kinase activity observed at peak.

Dr Ryan Watts, PhD, CEO of Denali Therapeutics, said: “Mutations in LRRK2 are a major risk factor for Parkinson’s disease. Targeting this degenogene represents a promising approach to develop disease modifying medicines for patients suffering from this terrible disease.”

He continued: “By restoring LRRK2 activity to normal levels, we believe we can reverse lysosomal dysfunction, which could potentially benefit both patients with LRRK2 mutations, as well as idiopathic Parkinson’s disease patients who exhibit lysosomal dysfunction.”

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