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The MP-010 molecule, developed by Miramoon Pharma, acts on ryanodine receptors, opening a new therapeutic avenue to address ALS, according to a study led by researchers from CIBERNED and several research institutes, including the IIS Aragón.
A collaborative study recently published in the journal British Journal of Pharmacology, has shown the The ability of the MP-010 molecule, developed by the spinoff Miramoon Pharma, 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.
The study has been Led by research staff from the Neurodegenerative Diseases Department of 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 (Autonomous University of Barcelona).
The research focuses on the modulation of ryanodine receptors (RyRs), a group of proteins key to regulating calcium levels within neurons, a crucial mechanism for the progression of ALS. The research group demonstrated that MP-010 stabilizes RyR function in a mouse model of ALS and evaluated its efficacy using nerve function tests, muscle function tests, histological analyses, and survival rates.
“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." ensures Laura Moreno-Martinez, first author of the work and CIBERNED researcher at the University of Zaragoza and the Biomedical Research Center of Aragon (CIBA).
"What's more -concludes the research group– Mice treated with MP-010 lived longer compared to those receiving a placebo. This study suggests that Drugs targeting RyRs, such as MP-010, could 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 evaluate their viability in human patients.”
Collaboration among the five CIBERNED groups, within a cooperative project funded by CIBERNED, has been key to advancing this project, highlighting the importance of multidisciplinary cooperation in biomedical research. "Involvement Miramoon Pharma has been fundamental, not only in the development of MP-01, but also to accelerate the drug discovery process, highlighting the value of partnering with innovative startups to drive the advancement of new therapies." the research team highlights.
About ALS
ALS is a progressive neurodegenerative disease that affects the motor neurons responsible for voluntary muscle control. It causes a progressive loss of strength and overall mobility, leading to paralysis, with a devastating impact on the quality of life of patients and their families. The prevalence of the disease is approximately 3 to 5 cases per 100.000 inhabitants, which represents a total of approximately 4.000 people affected in Spain.
Reference: 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.
Source: Ciberned
