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Lopid (gemfibrozil), an FDA-approved lipid-lowering drug, has been shown to improve life expectancy and motor function in mice with late infantile neuronal ceroid lipofuscinosis (late infantile LNC, or LINCL), which belongs to a a group of disorders referred to collectively as Batten’s disease.

The study, “Food and Drug Administration Approved Lipid-Lowering Drug Gemfibrozil Increases Longevity in Late Infantile Neuronal Ceroid Lipofuscinosis Mouse Model, »Appeared in the Journal of Neurochemistry.

LINCL is a rare neurodegenerative disease caused by mutations in the Cln2 gene, resulting in deficiency or loss of function of the enzyme TPP1. Deficiency of TPP1 then causes a build-up of lipid (fatty) -protein pigments in the brain, which is a common feature of lysosomal overload disease.

These genetic metabolic diseases result from abnormal functioning of lysosomes, the cell organelles responsible for breaking down large molecules.

Similar to other neurodegenerative disorders, neuroinflammation and neuronal death are seen in LINCL. Symptoms onset occur in children 2 to 4 years old, and despite continued research effort, no effective treatment is currently available for LINCL patients, who usually die young. Therefore, new therapies capable of significantly extending life expectancy and improving quality of life are badly needed.

The research team was led by Kalipada Pahan, PhD, professor in the Department of Neurological Sciences at Rush University Medical Center in Chicago. Scientists used a mouse model of LINCL, deficient in TPP1, to study the therapeutic potential of Lopid, a drug that helps lower cholesterol and triglyceride levels in the blood. Lopid has also been shown to stimulate neuroprotection and anti-inflammatory responses.

The study found that an eight week oral treatment with Lopid resulted in an increase in longevity by 10 weeks and improved motor activity. Additionally, Lopid partially decreased the load on storage materials, increased the levels of anti-inflammatory molecules, and prevented neuronal death in different parts of the brain.

Interestingly, given the TPP1 deficiency in these mice, the results indicate that the beneficial effects of Lopid are independent of TPP1. This is a key finding, as it shows potential for the development of a treatment to improve the overall accumulation in the brains of LINCL patients.

Compared to other potential neuroprotective drugs, Lopid has significant advantages because it can be administered orally, is fairly non-toxic, and has been well tolerated in human and animal studies.

Overall, “our results identify [Lopid] as a possible therapeutic agent to prolong the lifespan of LINCL patients, ”the researchers wrote.