Drug Development in the 21st century – are we going to cure all diseases

(Bild: Israel Sun)

Prof. MD. DSc. Aaron Ciechanover
Technion – Israel Institute of Technology Haifa, Israel

Many important drugs such as penicillin, aspirin, or digitalis, were discovered by serendipity — some by curious researchers who noted an accidental phenomenon, some by isolation of active ingredients from plants known for centuries to have a specific therapeutic effect. Other major drugs like statins were discovered using more advanced technologies, such as targeted screening, yet, the discoverers were looking for a different effect. In all these cases, the mechanisms of action of the drug were largely unknown at the time of their discovery, and were discovered only later. With the realization that not all patients with diseases that physically and histopathologically appear to be the same — different malignancies for example — respond similarly to treatment, and their clinical behavior is different, we have begun to understand that their molecular basis is distinct. Accordingly, we are exiting the era where our approach to treatment is “one size fits all”, and enter a new one of “personalized medicine” where we shall tailor the treatment according to the patient’s molecular/mutational profile. Here, unlike the previous era, the understanding of the mechanism will drive the development of the new drugs. This era will be characterized the development of technologies where sequencing and processing of individual genomes will be cheap (US$ <1,000) and fast (a few minutes), by identification and characterization of new disease-specific molecular markers and drug targets, and by design of novel, mechanism-based, drugs to modulate the activities of these targets. It will require a change in our approach to scientific research and development and to education, where interdisciplinarity will domineer and replace in many ways the traditional, discipline-oriented approach.Aaron Ciechanover wurde 2004 mit dem Nobelpreis für Chemie ausgezeichnet.