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Juan González Valdivieso, CIBER BBN researcher at the Centre for Biomaterials and Tissue Engineering (CBIT) of the Universitat Politècnica de València (UPV), has received a Marie Curie postdoctoral grant (MSCA) for the development of a new project that aims to contribute to improving treatments for patients with Amyotrophic Lateral Sclerosis (ALS).

Funded by the Aristos programme, the project focuses on muscle tissue as a therapeutic target and seeks to preclinically validate the efficacy in ALS therapies of a boron compound drug that has already been approved as a chemotherapy treatment for some types of cancer.

Thus, the project proposes a new strategy to stimulate muscle repair using ultra-low drug doses.

Previous studies

Juan González Valdivieso's research group, led by Dr Patricia Rico, discovered in recent studies that the boron transporter, NaBC1, enhances muscle regeneration.

'We showed that boron activates this transporter, which leads to the recovery of damaged muscle activity, whether due to injury or disease. To be able to transfer the results of our research to the clinic as quickly as possible, we are exploring whether other boron compounds that are already drugs approved by regulatory agencies have the same effect as boron in terms of activating its NaBC1 transporter and can therefore contribute to the regeneration and recovery of damaged muscle,' stresses Juan González Valdivieso.

3D model

In this project, which will run until 2028, the CBIT-UPV and CIBER-BBN team will work with muscle cells from a biopsy of an ALS patient as a biological tool for research into the disease and will study the mode of action of the boron compound drug in ultra-low doses in ALS muscle.

In addition, it will develop a new 3D model of human ALS muscle tissue as a tool to identify the mechanisms of ALS muscle pathogenesis and develop new treatments.

‘We hope that this tool will help to understand the role of skeletal muscle in ALS, as well as to determine effective drug doses and test other drugs for new therapies against the disease,’ says Patricia Rico.

Key developments

Traditionally, ALS research has focused on the nervous system. However, this project will allow the study of a new approach to the disease, focusing on muscle tissue.

The research proposed in this project will allow the development of new biological tools for ALS research by creating a human-immortalised muscle cell line from a biopsy of an ALS patient, which would be the first of its kind in the world. In addition, the in vitro 3D model of human ALS muscle tissue would be the first screening tool to identify the mechanisms of ALS muscle pathogenesis for drug discovery and testing.

To date, all available 3D models are derived from iPSCs as organoids or cocultures of muscle cells and motor neurons. Thus, this proposal aims to develop the first 3D human model using immortalised skeletal muscle cells from an ALS patient, achieving a more representative model of muscle pathology in ALS than current models.

‘Thus, both the immortalised cell line and the 3D muscle model will allow, for the first time, the in-depth study of muscle pathology related to the disease and serve for the further development of combined neuro-muscular environments,’ adds Juan González Valdivieso.

In addition, the project will provide sufficient preclinical results to advance clinical trials of a systemic application of ultra-low doses of this boron-based drug as a therapeutic strategy to slow and halt muscle atrophy in ALS. 'This would allow faster progress in applying a drug already approved by regulatory agencies as a new therapy for ALS,' concludes Patricia Rico.