The fight against amyotrophic lateral sclerosis (ALS) is expanding into the realm of scientific research in the Canary Islands. Researchers from the Spanish National Research Council (CSIC) and the startup Molefy Pharma, located in the Science Park of La Laguna, are developing a new molecule aimed at slowing the progression of this complex disease.
Currently, therapeutic options for ALS are limited. Dr. Ana Martínez, a CSIC researcher and co-founder of Molefy Pharma, points out that the only available drug, riluzole, only offers a temporary extension of life, and there are highly specific advanced therapies for a small group of patients with particular genetic mutations.
The main challenge focuses on sporadic ALS, which accounts for the majority of diagnosed cases. This issue was addressed during the 'ALS in the Canary Islands: Knowing to Advance. Science, Care, and Sociosanitary Commitment' meeting, recently held in Tenerife.
The molecule under development, named AP2, stems from CSIC research and is now being advanced by Molefy Pharma. The project has initiated its Phase 1 clinical trials, focused on evaluating safety in healthy volunteers. If results are favorable, it is anticipated that in later phases, the drug could be tested on ALS patients in hospitals across both the Canary Islands and the rest of Spain.
AP2's mechanism of action targets the modulation of a protein that exhibits alterations in the disease, with the goal of halting the progressive degeneration of motor neurons. Researchers are hopeful that if the results obtained in cellular and animal models are replicated in humans, AP2 could become a disease-modifying treatment.
Drug development is an inherently long, costly process with a high failure rate, where only a minimal fraction of studied molecules ultimately reach patients. However, the AP2 team hopes to expedite timelines thanks to its designation as an 'orphan medicinal product' by the European Medicines Agency. Nevertheless, the exact development timelines remain uncertain and depend on trial outcomes, as acknowledged by Martínez herself.
