Home Medizin Studie schlägt neuartigen Ansatz zur Verlangsamung der neuromuskulären Degeneration bei ALS-Patienten vor

Studie schlägt neuartigen Ansatz zur Verlangsamung der neuromuskulären Degeneration bei ALS-Patienten vor

von NFI Redaktion

A new study, led by the Center for Biomaterials and Tissue Engineering (CBIT) at the Universitat Politècnica de València (UPV), in collaboration with Instituto de Investigación Sanitaria La Fe (IIS La Fe) and the University of Zaragoza (Unizar), proposes a novel approach to slow down the neuromuscular degeneration in patients with Amyotrophic Lateral Sclerosis (ALS). This condition leads to loss of motor neurons and paralysis of skeletal muscles, currently having no cure or effective treatment.

The research team at UPV, CIBER BBN, IIS La Fe, and Unizar hypothesized that skeletal muscles could actively contribute to the pathology of the disease and identified skeletal muscle tissue as a therapeutic target for ALS treatment. Previous studies demonstrated that the Boron transporter NaBC1, when activated, synergistically enhances and accelerates muscle repair. Its effectiveness in an ALS mouse model representative of the familial (genetic) SOD1 variant model of the disease has been confirmed. „This model is the most studied for conducting preclinical studies prior to human research. It mimics the degeneration of lower motor neurons and exhibits muscle denervation atrophy before obvious signs of neurodegeneration,“ explained Patricia Rico, a researcher at CBIT-UPV.

The study involved injecting boron-loaded alginate hydrogel developed in CBIT laboratories into the quadriceps muscles of ALS mouse models. „We found that only four injections of the treatment administered to the symptomatic mice’s quadriceps significantly improved motor function. Additionally, the hydrogels effectively reduced muscle atrophy, delayed the onset of symptoms, and prolonged the mice’s survival,“ emphasized Patricia Rico.

Furthermore, the UPV, CIBER BBN, and IIS La Fe team found that the restoration of muscle pathologies had a retrograde neuroprotective effect, modulated neuroinflammation, and mitigated the loss of motor neurons.

These findings suggest that targeting muscle tissue could be a therapeutic approach to slow down neuromuscular degeneration in ALS. A potential combination therapy targeting motor neuron inflammation and muscle regeneration could have a greater chance of success in extending and improving the quality of life for those suffering from ALS,“ emphasized Juan Francisco Vázquez, a researcher at IIS La Fe and coordinator of the ALS Unit at Hospital la Fe.

The research team is currently evaluating the repositioning of a Boron compound drug, SOD1, in the same ALS mouse model, suggesting a new therapeutic indication for skeletal muscle regeneration. „If the results with this drug are positive, the new therapeutic indication could be the regeneration of skeletal muscles. Repositioning this drug would represent a significant regulatory and clinical study advantage,“ highlighted Dr. Vázquez.

Donation from the StopELA Association

The work led by UPV has caught the interest of the StopELA patient association, which has made a donation to contribute to the progress of this research. The donation will go towards recruiting a student to write a doctoral thesis under the coordination of Patricia Rico and Juan Francisco Vázquez, neurological coordinator of the Department of Motor Neuron Diseases at the Hospital Universitari i Politècnica La Fe de València.

StopELA was established in 2019 as an educational project at the Sant Cristófor Mártir School in Picassent. The association has raised €70,000 through the sale of bracelets and the organization of sports and cultural events to finance the neuromuscular pathology and ataxia research project at IIS La Fe. The association continues its efforts to raise awareness and support for research by selling solidarity keychains and voluntary contributions from its members.


Universitat Politècnica de València

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