New experimental drug improves motor function and prolongs life in ALS model
A collaborative study involving the UAB has shown the ability of the molecule MP-010 to improve motor function and prolong life expectancy in a mouse model of Amyotrophic Lateral Sclerosis (ALS), a severe neurodegenerative disease with no effective treatments.
A collaborative study recently published in the British Journal of Pharmacology has shown the ability of the molecule MP-010, developed by the spin-off Miramoon Pharma, to improve motor function and prolong life expectancy in a mouse model of Amyotrophic Lateral Sclerosis (ALS). This severe neurodegenerative disease currently has no effective treatments.
The study was led by researchers from the Neurodegenerative Diseases area of the CIBER (CIBERNED): Adolfo López de Munain (University of the Basque Country and IIS BioGipuzkoa), Francisco Gil-Bea (Ikerbasque, University of Navarra and IIS BioGipuzkoa), co-founders of Miramoon Pharma, and Rosario Osta (University of Zaragoza and IIS Aragón) and Xavier Navarro (Institute of Neurosciences of the Universitat Autònoma de Barcelona).
The research focuses on the modulation of ryanodine receptors (RyRs), a group of proteins key in the regulation of calcium levels within neurons, a crucial mechanism for ALS progression. The research group verified that MP-010 stabilises the function of RyRs in a murine model of ALS, and evaluated its efficacy by testing nerve function, muscle function, histological analysis and survival rate.
“The results showed that MP-010 preserved nerve function, delayed the onset of motor problems, improved muscle coordination, maintained neuromuscular connections, and protected motor neurons in the spinal cord”, says Laura Moreno-Martinez, first author of the study and CIBERNED researcher at the University of Zaragoza and the Biomedical Research Centre of Aragon (CIBA).
Mice treated with MP-010 lived longer compared to those that received a placebo. This study suggests that drugs targeting RyRs, such as MP-010, may have significant therapeutic potential in the treatment of ALS. However, further research is needed to understand how they work at the molecular level and to assess their feasibility in human patients.
The collaboration between the five CIBERNED groups, within a cooperative project funded by the consortium, has been key to advance this project, highlighting the importance of multidisciplinary cooperation in biomedical research.
About ALS
ALS is a progressive neurodegenerative disease affecting the motor neurons responsible for voluntary muscle control. It causes a progressive loss of global strength and mobility leading to paralysis, with a devastating impact on the quality of life of patients and their families. The prevalence of the disease is around 3 to 5 cases per 100,000 inhabitants, which means a total of approximately 4,000 people affected in Spain.
Reference article:
Moreno-Martinez L, Gaja-Capdevila N, Mosqueira-Martín L, Herrando-Grabulosa M, Rodriguez-Gomez L, et al. Novel FKBP prolyl isomerase 1A (FKBP12) ligand promotes functional improvement in SOD1G93A amyotrophic lateral sclerosis (ALS) mice. Br J Pharmacol. 2025; doi: 10.1111/bph.17448.