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International Parkinson and Movement Disorder Society
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News Release: Safety and Biomarker Effects of Novel Therapeutic for Parkinson's Disease with GBA Mutations

International Congress of Parkinson's Disease and Movement Disorders, Nice, France

EMBARGOED until Monday, September 25, 2019
Media Contact:
Elizabeth Clausen, +1 414-276-2145,


Safety and Biomarker Effects of Novel Therapeutic for Parkinson’s Disease with GBA Mutations

NICE, FRANCE – A novel clinical therapeutic, LTI-291, is found to be safe and tolerable among Parkinson’s disease (PD) patients with GBA mutation, according to a study released today at the International Congress of Parkinson’s Disease and Movement Disorders®.

Dana C. Hilt and a team of researchers in Cambridge, MA, USA, investigated the safety and biomarker effects of LPI-291, an allosteric CNS penetrant GCase activator, among PD patients with mutations in the GBA gene. Forty GBA-PD patients were enrolled and administered LTI-291 or placebo for 28 days. Peripheral blood mononuclear cells (PBMCs) and plasma were analyzed for glycosphingolipid (GSL) changes at days 6, 14 and 28. There were no serious adverse events reported and any adverse events were the same in the LTI-291 dose groups versus the placebo. Significant changes in PDMC GSLs were observed and were consistent with the drug having its intended biologic effect on GCase. Furthermore, the GSL effects were larger in the GBA-PD patients carrying GD mutations versus mild mutations.

Jean-Christophe Corvol, Assistant Professor in Neuropharmacology at Pitié-Salpêtrière Hospital in Paris, France states, “Mutations in the lysosomial enzyme Glucocerebrosidase (GCase) gene (GBA) cause the Gaucher disease when present at the homozygous state, but has been shown to increase the risk of Parkinson’s disease by 6-fold in heterozygous carriers leading to the hypothesis that this pathway is involved in the pathophysiology of the disease. Hilt and collaborators reports a first phase 2a randomized placebo-controlled clinical trial on the safety and biomarker effects of LTI-291, an allosteric CNS penetrant GCase activator in PD patients with GBA mutations. The product was well tolerated, and glycosphingolipid markers in peripheral blood cells were significantly changed in patients treated with LTI-291 as compared to placebo. Further studies are needed to confirm the clinical relevance of these findings in PD patients with GBA mutations.”

About the International Congress of Parkinson's Disease and Movement Disorders®: Meeting attendees gather to learn the latest research findings and state-of-the-art treatment options in Movement Disorders, including Parkinson's disease. Over 5,100 physicians and medical professionals from more than 95 countries will be in attendance to view 2,200 scientific abstracts submitted by clinicians from around the world.

About the International Parkinson and Movement Disorder Society:

The International Parkinson and Movement Disorder Society (MDS), an international society of over 8,500 clinicians, scientists, and other healthcare professionals, is dedicated to improving patient care through education and research. For more information about MDS, visit

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