Previous evidence that certain natural products, such as caffeine, increase the MBNL1 protein in vitro in myoblasts from patients with DM1, comes from the project titled “Development of new treatments for Myotonic Dystrophy, in vivo drug discovery”[1], a public-private consortium project led by Dr. Artero and funded by the Fundació La Marató de TV3 in 2009. These preliminary results were published in the patent applications “Caffeine for the treatment of Myotonic Dystrophy type 1 and type 2″[2] and “Compounds for the treatment of Myotonic Dystrophy type 1 and type 2″[3], the latter licensed to Myogem Health Company in 2015.
Since caffeine belongs to a large family of compounds known as xanthines, the continuation of preliminary research focused on thoroughly exploring the behavior of these molecules in DM1 models. It was also anticipated that combinations of xanthines, and particularly combinations of caffeine with at least one other xanthine, could present synergistic effects in in vitro and/or in vivo tests in DM1 models.
From the results obtained through this research, the product MYODM has been developed and is presented below:
SUMMARY OF RESULTS
1. The study of the effect of MYODM on movement capacity, measured through the indirect muscles associated with flight, allows tripling the functionality of DM1 model flies, achieving recovery of between 60-70% of the flight capacity compared to healthy control flies.
2. The study of the effect of MYODM on muscle area achieves approximately 40% recovery of the muscle area of the indirect flight muscles compared to diseased flies, while reaching almost 85% of the muscle area of healthy flies. In other words, MYODM significantly reduces muscle atrophy in the DM1 model.
3. The study of the effect of MYODM on cardiac dysfunction in the dystrophic fly animal model confirms that the product is safe in vivo and that supplementing the diet of these diseased individuals can significantly improve key heart parameters, such as heart rate and FS fraction.
4. MYODM allows a 96% increase in lifespan and a 65% increase in average survival of dystrophic flies compared to healthy flies.
5. Feeding DM1 model flies with MYODM allows for a reduction of Redox levels (measure of ROS) by approximately 30% compared to diseased flies fed exclusively with the standard nutritive medium.
6. Autophagy (the catabolism of cellular components to obtain energy and eliminate toxic proteins and damaged organelles) is activated in DM1. Feeding DM1 model flies with MYODM allows a reduction of autophagy levels by more than 50%.
