ACCRF’s Research Agenda represents the best efforts of the best minds at the best cancer institutions in the world to find a cure for adenoid cystic carcinoma (ACC). As with many rare diseases, ACC poses some peculiar challenges for researchers. The lack of ACC specimens and models – the building blocks of research – has been accompanied by the lack of a logically-sequenced plan to access the most promising technological platforms of scientific discovery. These challenges must be met with ingenuity and determination in order to gain more widespread focus, greater resources and new insights.
The task of developing improved therapies for a rare disease may appear daunting, but some powerful forces bode well for the future. Tectonic shifts in the way biomedical research is conducted are making it more realistic to expect major advances for ACC and other rare cancers.
Rare Disease Research: Shifting Paradigms
Traditional View New Paradigm Public health improvements require a focus on primarily common diseases and cancers All cancers are rare cancers; personalized medicine is necessary to deal with the diversity of genomic profiles Innovation spills over from common diseases to rare diseases Innovation spills over from rare diseases to common diseases Patients and researchers are too dispersed to reach a critical mass for fundraising or research The internet has lowered the barriers to aggregating interested parties Research costs are too high High-throughput technologies are permitting genome-wide studies at tractable costs, outpacing Moore’s Law Profit opportunities are minimal in rare diseases Rare disease therapies have become blockbusters, aided by orphan drug designations Download Rare Disease Research — Shifting Paradigms