{"id":438637,"date":"2026-05-21T17:05:09","date_gmt":"2026-05-21T17:05:09","guid":{"rendered":"https:\/\/www.newsbeep.com\/nz\/438637\/"},"modified":"2026-05-21T17:05:09","modified_gmt":"2026-05-21T17:05:09","slug":"breakthrough-parkinsons-drug-targets-disease-at-its-genetic-roots","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/nz\/438637\/","title":{"rendered":"Breakthrough Parkinson\u2019s Drug Targets Disease at Its Genetic Roots"},"content":{"rendered":"

\"Synapse<\/a>Scientists have reported promising early results from a novel Parkinson\u2019s treatment designed to suppress the activity of a key disease-associated gene. Shutterstock<\/p>\n

An experimental gene-silencing therapy safely lowered LRRK2 protein levels in an early Parkinson\u2019s trial, but its effect on symptoms remains untested.<\/p>\n

An experimental medicine built to silence a gene closely associated with Parkinson\u2019s disease has produced encouraging results in its first clinical test in people, according to a study published in Nature Medicine.<\/p>\n

The treatment, called BIIB094, is designed to target LRRK2, the most common genetic contributor to Parkinson\u2019s disease. Variants in LRRK2 are known to raise the risk of developing the neurodegenerative condition, which affects nearly 10 million people around the world.<\/p>\n

Researchers have long thought that reducing the activity of the LRRK2 protein might help slow or alter the course of Parkinson\u2019s disease. However, turning that concept into a practical therapy has been difficult, said study coauthor Danielle Larson, MD, \u201915, \u201918 GME, assistant professor in the Ken & Ruth Davee Department of Neurology\u2019s Division of Movement Disorders.<\/p>\n

\u201cThis was a multi-center clinical trial looking at an antisense oligonucleotide therapy for LRRK2-specific Parkinson\u2019s disease,\u201d Larson said. \u201cThe main goal was to examine the safety of delivering this therapy to patients, with the hope that if it proved safe, future studies could evaluate whether it might slow disease progression.\u201d<\/p>\n

Safety came before efficacy<\/p>\n

The trial examined whether BIIB094 could lower LRRK2 levels safely in people with Parkinson\u2019s disease. Larson said the results indicate that this can be achieved without major safety concerns.<\/p>\n

The randomized, placebo-controlled trial included 82 people with Parkinson\u2019s disease divided across two parts. In the first part, 40 participants received one dose of BIIB094 or placebo. In the second part, 42 participants received four doses of BIIB094 or placebo, given every four weeks. The therapy was administered intrathecally, meaning it was delivered directly into the cerebrospinal fluid through a lumbar puncture.<\/p>\n

In the second part of the study, participants were grouped according to whether they carried a known LRRK2 genetic variant.<\/p>\n

The treatment was generally well tolerated across both parts of the trial. Adverse events were common, but most were mild to moderate and did not prevent dosing. According to the study, no serious adverse events related to BIIB094 were reported.<\/p>\n

LRRK2 levels fell sharply<\/p>\n

In addition to its safety findings, the study showed strong evidence that BIIB094 affected the biological target it was designed to reach. Cerebrospinal fluid analysis found that LRRK2 protein levels dropped by as much as 59 percent in treated participants.<\/p>\n

Those reductions occurred whether or not patients carried a known LRRK2 mutation, Larson said, suggesting the therapy might eventually be relevant to a wider Parkinson\u2019s population, not only people with a confirmed genetic diagnosis.<\/p>\n

\u201cThe antisense oligonucleotide was designed specifically to reduce LRRK2 expression,\u201d Larson said. \u201cBecause overactivity of this protein kinase is thought to be part of the problem in Parkinson\u2019s disease, reducing LRRK2 levels could be protective and potentially modify the disease.\u201d<\/p>\n

Larson said the results point to a possible way LRRK2-targeted therapies might act on the disease process itself rather than only treating symptoms.<\/p>\n

Clinical benefits remain unproven<\/p>\n

Larson emphasized, however, that the trial was not intended to test whether BIIB094 improved symptoms, cognition, movement, or disease progression. Larger studies that follow patients for longer will be needed to learn whether lowering LRRK2 levels leads to meaningful benefits.<\/p>\n

\u201cThe next step would be a phase 2 study with a larger group of patients,\u201d Larson said. \u201cInstead of focusing only on safety, those trials would look at efficacy, whether the therapy can slow disease progression using motor assessments and standard Parkinson\u2019s rating scales.\u201d<\/p>\n

Precision therapies move closer<\/p>\n

Larson said the findings represent an important move toward targeted treatments that address the underlying biology of Parkinson\u2019s disease, and they highlight the promise of BIIB094 and other precision medicine approaches.<\/p>\n

\u201cThis is one of the first antisense oligonucleotide, or ASO, therapies in Parkinson\u2019s disease to have safety and tolerability data,\u201d Larson said. \u201cIt really paves the way for other ASO-based treatments to be developed, potentially targeting different biological pathways.\u201d<\/p>\n

Reference: \u201cLRRK2-targeting antisense oligonucleotide in Parkinson\u2019s disease: a phase 1 randomized controlled trial\u201d by Omar S. Mabrouk, Ben Tichler, H. Moore Arnold, Eva C. Thoma, Sara M. Alexanian, Jingxian Chen, Tzu-Ying Liu, Beth Hirschhorn, Roy Llorens Arenas, John W. Annand, Unnati Kapadnis, Alan A. Shomo, Kelly E. Glajch, Kyle Ferber, Yuka Moroishi, Julie Czerkowicz, Jennifer Inra, Ronald B. Postuma, Tanya Gurevich, Pablo Mir, Huw R. Morris, Jason Aldred, Matthew A. Brodsky, Aaron Ellenbogen, Danielle Larson, Christopher M. Tolleson, Andrew Siderowf, Charalampos Tzoulis, Ernest Balaguer, Maria J. Marti, Hien T. Zhao, Holly B. Kordasiewicz, Roger Lane, Warren D. Hirst, Stephanie Fradette and Danielle L. Graham, 24 March 2026, Nature Medicine.
DOI: 10.1038\/s41591-026-04262-4<\/a><\/p>\n

The study was funded by Biogen.<\/p>\n

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