New preprint showcases results in an ALS mouse model

New preprint showcases results in an ALS mouse model

Miramoon Pharma’s pipeline targets many disorders and we are very happy to report the results of a research study in which one of our drug candidates, MP-010, was tested in an amyotrophic lateral sclerosis mouse model to see if it could improve motor function and extend the mice lifespan. The experiments were led by Dr Francisco Gil-Bea, Ikerbasque Research Associate and principal investigator at Biogipuzkoa Health Research Institute. The results have just been submitted to an academic journal for peer review and, as we are committed to open research, a pre-print has been already published in BioRxiv (“Novel FKBP12 ligand promotes functional improvement in SOD1G93A ALS mice”). (**A preprint is a manuscripts before certification by peer review, allowing other scientists to see, discuss, and comment on the findings immediately).

Amyotrophic lateral sclerosis (#ALS), a severe nerve degeneration condition, currently lacks effective treatments. This study explores a potential new approach targeting a cellular process involved in ALS progression. Specifically, it focuses on a group of proteins called ryanodine receptors (RyRs), which play a role in regulating calcium levels within nerve cells.

Dr Gil-Bea’s group tested in a mouse model of ALS a several drugs that interacts with RyRs. They assessed the drug’s effectiveness using various measures including nerve function tests, tissue examinations, muscle function assessments, and survival rates.

One of the tested drugs, Miramoon Pharma´s MP-010, showed promise by preserving nerve function, delaying the onset of motor problems, improving muscle coordination, maintaining nerve connections, and protecting nerve cells in the spinal cord. Importantly, mice treated with MP-010 lived longer compared to those given a placebo.

These findings suggest that drugs targeting RyRs, like MP-010, could be beneficial in treating ALS. However, more research is needed to understand how these drugs work at a molecular level and their potential for use in ALS patients.

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