The team led by Dr. Rubén Artero, from the genetics department of the University of Valencia, is one of the research groups seeking to achieve a treatment and / or cure for our Limb-girdle Muscular Dystrophy 1F. In coordination with the rest, it is one of the first groups that formed the medical alliance that works on our disease and those that could find a palliative to help us improve the quality of life until the definitive cure arrives.
We have just renewed the agreement by making a payment of € 21,361 + VAT (€ 25,846), which will cover the coming months of work that is vital that it not stop. As always and from the beginning, its main objective has been to develop and characterize a model of our disease in Drosophila (vinegar flies) and, subsequently, to test the model for several libraries of more than 1,400 drugs and natural compounds already approved to treat other diseases. The latter is to be done in order to obtain candidate compounds to help improve our disease.
They had already managed, among many other things, to characterize the disease in the animal model by expressing our mutated protein, and they had achieved effects such as reduced locomotor capacity. In recent months they have managed to develop a more approximate model of our disease in the fly, with atrophy both in the abdominal muscles and in the thorax muscles of the fly, with the locomotive capacity being more affected still. Thanks to this better approximation of the model of our disease in Drosophila, compound screening will be more effective and make the results obtained more reliable and faster to apply in humans.
This screening of compounds was already underway but, due to the coronavirus crisis, universities have closed and research has had to stop. When the alarm state is lifted, work will resume. The objective for the rest of 2020 is to have very advanced testing and to have compounds that give improvement in flies, as well as to ratify the results of compounds that already have the possibility of being candidates for experiments prior to tracing. Subsequently, candidate compounds after screening should be corroborated in human cells. At the same time, in collaboration with other groups of researchers involved in our disease, we work on cell samples from patients. With all this, it is intended to continue tightening the fence to have in the shortest possible time possible drugs that, ultimately, those affected can take.
Along with tracking, the laboratory has other objectives to progress in the knowledge of our disease. In the model of our fly disease, it is possible to study in depth the specific molecular processes or cellular pathways involved in our disease, as well as the role of the mutated protein in the cell or the involvement of other proteins in the process. All this will help to better understand the behavior of the disease and shed light on proposing other approaches and / or targets for a more effective treatment for the disease.
As always, we emphasize that the times of science are not as fast as we all would like. But they have some necessary steps so that the work done is not in vain and that it can be applied to humans with confidence in the results and without danger to them.
The payment of the aforementioned amount serves to cover, mainly, the salary of the next months of the pre-doctoral student Águeda Blázquez, who is in charge of working exclusively on our disease, as well as the expenses of the material and reagents necessary for the experiments that will be carried out in the Translational Genomics laboratory of the genetics department of the University of Valencia led by Dr. Rubén Artero. We want to thank them and the rest of the department for all their work and dedication inside and outside the laboratory. As well as his personal involvement beyond professional and all his efforts towards research, even in times of crisis that we are experiencing because of the global pandemic caused by COVID-19. Thanks also to all Conquistando Escalones partners, donors and collaborators for helping us raise funds and pay for research. In these times when, among other things, it will be difficult to carry out events in the short term, it is more important than ever.