An experimental drug improves motor function and extends lifespan in an ALS model
A collaborative study involving the Universitat Autònoma de Barcelona (UAB) has shown that the molecule MP-010 can enhance motor function and extend lifespan in a mouse model of Amyotrophic Lateral Sclerosis (ALS), a severe neurodegenerative disease for which there are currently no effective treatments.
The study, recently published in the British Journal of Pharmacology, demonstrated the ability of MP-010 — a molecule developed by the spin-off company Miramoon Pharma — to improve motor function and prolong survival in a mouse model of ALS.
The research was led by scientists from the Neurodegenerative Diseases area 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, along with Rosario Osta (University of Zaragoza and IIS Aragón) and Xavier Navarro (INc-UAB).
The study focused on modulating ryanodine receptors (RyRs), a group of proteins that play a key role in regulating calcium levels within neurons — a mechanism crucial to the progression of ALS. The researchers found that MP-010 stabilizes RyR function in a mouse model of ALS, and assessed its efficacy through tests of nerve and muscle function, histological analyses, and survival rate monitoring.
“Our results showed that MP-010 preserved nerve function, delayed the onset of motor symptoms, improved muscle coordination, maintained neuromuscular connections, and protected motor neurons in the spinal cord,” explains Laura Moreno-Martinez, first author of the study and researcher at CIBERNED, the University of Zaragoza, and the Biomedical Research Centre of Aragón (CIBA).
Mice treated with MP-010 lived longer than those given a placebo. The study suggests that drugs targeting RyRs, such as MP-010, may hold significant therapeutic potential for ALS treatment. However, further research is needed to understand their molecular mechanisms and to assess their viability in human patients.
Collaboration among the five CIBERNED research groups, within a cooperative project funded by the consortium, was key to advancing this work, highlighting the importance of multidisciplinary cooperation in biomedical research.
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.