I am a graduate student and resident in the field of neurosurgery and would like to share an unusual and very personal story of developing a drug. Developmental biologist Dr. Matthew Scott and I went from purely basic biological research in our lab at Stanford University to discovering a target for drug development to identifying a drug for a pediatric brain cancer called medulloblastoma to a clinical trial – all within five years and for just US$500,000, with the money raised through philanthropic donations.
That’s surprising for two reasons. First, the odds of successfully getting a drug to clinic are very low. For every 10,000 candidate drugs, only five are ever tested in clinical trials. The few that do make it into pharmacies have on average a $1 billion development cost for research, testing and clinical trials. For this reason, the gap between scientific discoveries and clinical trials is commonly referred to as the “valley of death.” The other reason my experience is atypical is because most new drugs are developed for a disease of interest by pharmaceutical companies. In contrast, we identified existing drugs that were developed for other diseases, but could be repurposed.
Our basic science approach shaved a decade off the usual development time. The clinical trial will begin this winter, so whether we succeed remains to be seen. But our story is worth sharing as a promising path for finding new drug candidates.
This article highlights the scope for developing therapeutics with new innovative, alternative approaches. The current trillion dollars pharmaceutical industry is in peril where the current drug development pipeline is slow, inefficient and incapable of being extended to multi-combination drug therapies as well as minimally focused on prevention. The complex modeling of diseases and biological functions has been limited because of the inability to integrate large scale molecular pathways.
Watch this Video to understand how recent advances provide breakthrough technology for doing scalable modeling of complex molecular systems to dramatically accelerate drug discovery and development.