New drug candidate shows promise for treating ALS in animal studies

A promising drug candidate for treating amyotrophic lateral sclerosis (ALS) has emerged from a collaborative effort by researchers at the University of Helsinki and their partners. The candidate, known as cerebral dopamine neurotrophic factor (CDNF), has shown encouraging results in animal studies, prolonging lifespan and alleviating disease symptoms in rats and mice.

ALS is a rapidly progressing, fatal neurodegenerative disease that targets nerve cells in the brain and spinal cord, leading to muscle atrophy and paralysis. Currently, there is no cure for ALS, and available treatments provide only modest benefits.

Led by Assistant Professor Merja Voutilainen, the Regenerative Neuroscience Group at the University of Helsinki, in collaboration with national and international scientists, explored the therapeutic potential of CDNF in various cellular and animal models of ALS. CDNF, initially discovered in 2007 by Professor Mart Saarma’s laboratory, is primarily located in the endoplasmic reticulum (ER) within cells, an organelle essential for protein synthesis.

Published in the journal Brain, the study utilized genetically modified animal models with human ALS-related mutations (TDP43-M337V and SOD1-G93A) to investigate CDNF’s impact on disease progression and elucidate its mechanism of action.

One key focus was on examining ER stress, a cellular response involved in protecting cell proteins. Chronic ER stress is common in many neurological diseases and can lead to cell death.

The findings were promising. Administering CDNF to ALS-afflicted rodents significantly improved their motor function and halted the progression of paralysis. This improvement correlated with an increased number of surviving motoneurons in the spinal cord. The researchers propose that CDNF may rescue motoneurons by reducing the ER stress response and preventing cell death. Notably, ER stress was observed in all animal models, irrespective of specific genetic mutations.

Professor Michael Sendtner from the University of W├╝rzburg in Germany, a renowned ALS researcher and co-author of the study, remarked, “This study paves the way for a rational therapy to counteract one of the most severe cellular abnormalities in ALS: ER stress.”

Dr. Merja Voutilainen, an Assistant Professor at the University of Helsinki and the study’s director and senior author, expressed optimism, stating, “CDNF shows tremendous potential for developing new, science-based treatments for ALS.”

Source: University of Helsinki

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