Author Affiliation: Howard Hughes Medical Institute,
Chevy Chase, Md.
In today’s scientific era, molecular research has paved the way
for the development of pharmaceuticals that are improving the lives of millions
of patients. The discovery of the human immunodeficiency retrovirus as the
causative agent of AIDS led to the development of reverse transcriptase inhibitors
such as zidovudine. Monoclonal antibodies directed against tumor necrosis
factor α have changed the lives of many patients with rheumatoid arthritis.
Imatinib mesylate, a specific inhibitor of the bcr-abl protein kinase, has
transformed the prognosis for patients with chronic myeloid leukemia; and
as drug-resistant variants arise, DNA sequencing can identify the mutations
responsible and provide a rational basis for further drug development.1,2 In 2004 the first angiogenesis inhibitors
were approved by the US Food and Drug Administration, in one case for treatment
of metastatic colorectal cancer and in another for age-related macular degeneration.3,4 There is reason for optimism that this
is just the beginning, and that the huge investments in molecular biology
and genomics research made 10 to 30 years ago will provide an increasingly
robust flow of new and effective medicines.
Cech TR. Fostering Innovation and Discovery in Biomedical Research. JAMA. 2005;294(11):1390-1393. doi:10.1001/jama.294.11.1390