ALS is a disease that does not have any well-validated in vitro
and in vivo
models due to a high level of missing heritability. The majority of these models are developed based on a limited number of genes that are signatures of familial ALS, which accounts for less than 10% of the total ALS cases. Insilico Medicine aims to develop personalized therapies for ALS patients directly by utilizing our AI drug discovery platform. After identifying novel targets in ALS and the available drugs for repurposing, we comprehensively review these targets and collaborate with the hospitals and experts of ALS to discuss the possibilities to bring these therapeutics into clinical use.
In 2013, Zhavoronkov and Cantor proposed a model for personalized science, with a team of physicians and academic experts in a research center collaborating with a patient to generate a patient-specific research project for disease treatment (Rejuvenation Res, 16(5):414-8
). This paradigm aims to determine the best course of action in just one patient as a N-of-1
study, and eventually identify alternative options for disease treatment.