Old Methods for New Drugs
PALO ALTO – Despite high expectations, productivity is frustratingly low in biopharmaceutical research and development. Although expenditures have increased, the number of new medicines resulting from human-genome sequencing has not. This means that a broad variety of diseases are not being treated effectively, in the developed or the developing worlds.
Advances in molecular sciences, corresponding to the sequencing of the human genome in the 1980’s, led to the identification of all the human proteins – large, complex molecules necessary for many of the body’s functions. Understanding the proteins’ roles then led to greater knowledge of the underlying causes of diseases. For example, mutations, or defects, at specific molecular locations in human DNA were found to be responsible for some cancers, raising the hope of developing successful therapies tailored to individual patients.
Scientists believed that the ability to visualize and understand human biology at a more detailed level would lead to many new medicines. Identifying the defective molecular parts, known as the drug targets, should have made addressing the causes of disease easier, and would revolutionize the pharmaceutical sciences. And so it has – but without increasing the number of new medicines.